Research News

Iacobas to serve on vascular anomalies committee (Baylor College of Medicine - June 18, 2021)
Dr. Ionela Iacobas, assistant professor of pediatric hematology oncology and medical director of the Texas Children's Hospital Vascular Anomalies Center, was invited to join the Scientific Committee of the International Society for the Study of Vascular Anomalies. The committee, which consists of 10 international experts in the field, drives the organization's scientific initiatives and oversees the scientific program at ISSVA's World Congress.

Pautler selected 2021 Fellow of the ISMRM Society (May 21, 2021)
Dr. Robia Pautler, associate professor in the departments of Molecular Physiology and Biophysics, Neuroscience, Radiology at the Baylor College of Medicine and Co-Director Small Animal Imaging Facility (MRI), Texas Children's Hospital, is selected as one of the 2021 Fellows of the International Society for Magnetic Resonance in Medicine (ISMRM), a multi-disciplinary nonprofit association that promotes innovation, development, and application of magnetic resonance techniques in medicine and biology throughout the world. Paulter was selected as a Fellow for her contributions to the pre-clinical manganese-enhanced MRI for neuronal tract tracing and for outstanding service to the ISMRM.

Athenex, Texas Children’s Cancer Center, and the Center for Cell and Gene Therapy at Baylor College of Medicine present new clinical data on GD2 CAR-NKT cells in neuroblastoma at ASGCT Annual Meeting (May 18, 2021)

Athenex, Inc. (NASDAQ: ATNX), a global biopharmaceutical company dedicated to the discovery, development, and commercialization of novel therapies for the treatment of cancer and related conditions, led by its Orascovery platform, today announced that investigators from Texas Children’s Cancer Center and the Center for Cell and Gene Therapy at Baylor College of Medicine presented new clinical data from the ongoing GINAKIT2 phase 1 study of Athenex’s cell therapy candidate KUR-501 targeting GD2 in neuroblastoma at the American Society of Gene & Cell Therapy (ASGCT) 24 ^th Annual Meeting on Friday, May 14, at 11 am ET.

Biological E Ltd gets approval to start Phase III clinical trial of its COVID vaccine candidate developed at Texas Children's Hospital's Center for Vaccine Development (April 27, 2021)
Biological E. Limited (BE), a Hyderabad-based vaccine and pharmaceutical company, today announced that it has successfully completed the Phase I/II clinical trial of its COVID-19 subunit vaccine candidate in India and received the approval to start the Phase III clinical trial from the Central Drugs Standard Control Organization (CDSCO) - Subject Expert Committee (SEC). BE started the Phase I/II Clinical Trial of its COVID-19 Vaccine Candidate in the second week of November 2020. Its candidate includes an antigen developed by Texas Children’s Hospital Center for Vaccine Development and in-licensed from BCM Ventures, Baylor College of Medicine’s integrated commercialization team, along with Dynavax Technologies Corporation’s (Nasdaq: DVAX) advanced adjuvant CpG 1018TM.

Dr. Hyunlok Chung anounced as a winner of the 2021 Warren Alpert Distinguished Scholars Award (April 13, 2021)
The Warren Alpert Foundation postdoctoral program was established 3 years ago to advanced neuroscience training as a bridge toward developing a career trajectory in academia, Industry, or governmental science. This highly competitive national program has been awarded to 5-7 individuals each year providing two years of support in a setting that permits independent scholarship to advance career development. Chung received this award for his proposal titled, "Role of very long-chain fatty acids in neuroinflammation".

AACR to recognize the St. Baldrick’s Foundation-Stand Up To Cancer Pediatric Cancer Dream Team with 2021 Team Science Award (April 13, 2021)
Texas Children's and Baylor's, Drs. Nabil Ahmed, Meenakshi Hegde, Will Parsons and Sujith Joseph along with Bambi Grilley and Melanie Frost Moll are among those involved in this team effort to develop new immunotherapy approaches to treat childhood cancers.

Study reveals how long-term infection and inflammation impairs immune response as we age (Mar 25, 2021)
A study led by Dr. Katherine King, associate professor at Baylor College of Medicine and Texas Children’s Hospital, shows for the first time that long-term infection and chronic inflammation drive CH mediated by the loss of Dnmt3a function. In addition, the study offers key insights into the mechanism by which chronic inflammation leads to CH and demonstrates the critical role of DNMT3a in regulating normal HSC responses to infections. The study was published in the journal Cell Stem Cell.

Early training delays symptom onset in mouse model of Rett syndrome (Mar 24, 2021)
New scientific findings bring hope that early training during the presymptomatic phase could help individuals with Rett syndrome, a neurodevelopmental disorder, retain specific motor and memory skills and delay the onset of the condition. Researchers at Baylor College of Medicine and the Jan and Dan Duncan Neurological Research Institute at Texas Children’s Hospital reported in the journal Nature that, in a mouse model of Rett syndrome, intensive training beginning before symptoms appear dramatically improved the performance of specific motor and memory tasks and substantially delayed the appearance of symptoms.

Maletic-Savatic lab discovers a novel marker of adult human neural stem cells (Mar 24, 2021)
Researchers have found a novel biomarker, BASP-1, that is restricted to NSCs in neurogenic niches in the mammalian brains, including humans, during development in utero and after birth. With this newly discovered biomarker, scientists can better understand the relevance and intricate mechanisms of neurogenesis, which may lead to new future therapeutic approaches to treat and manage neurological and neuropsychiatric disorders associated with diminished neurogenesis.

Researchers identify DNA elements that affect MECP2 expression (Mar 18, 2021)
Researchers at Baylor College of Medicine and the Jan and Dan Duncan Neurological Research Institute at Texas Children's Hospital (NRI) have identified and characterized two regions of DNA required for the proper expression of Mecp2/MECP2 in mice and humans.

Texas Children's teams up with Children's Hospital of Philadelphia to study sepsis (Mar 12, 2021)
PROMPT BOLUS (PRagMatic Pediatric Trial of Balanced vs. nOrmaL Saline FlUid in Sepsis) is a clinical trial to compare two commonly used treatments for pediatric sepsis to see if one is more effective and safer than the other. The treatments in this study are two different intravenous (IV) fluid types. One fluid is 0.9%“normal” saline and the other is balanced fluids (including lactated Ringer’s [LR] or Plasma-Lyte [PL]).

A preclinical study validates antisense oligonucleotides as a feasible approach to treat MECP2 duplication disorder (Mar 4, 2021)
A preclinical study published from the laboratory of Dr. Huda Zoghbi, professor at Baylor College of Medicine and director of the Jan and Dan Duncan Neurological Research Institute at Texas Children’s Hospital, provides experimental evidence that supports the use of antisense oligonucleotides as a feasible strategy to treat MDS. The study also offers crucial insights into the pharmacodynamics of this approach, which will serve as an important guide for the design and implementation of future clinical trials for this disorder. The study appears in the journal Science Translational Medicine.

Texas Children's receives a major grant to study pediatric brain tumors (Mar 3, 2021)
Dr. Lisa Kahalley, Associate Professor and Associate Chief of Research for the Psychology Section and Director of Neurobehavioral Oncology for the Texas Children’s Cancer & Hematology Centers, was awarded a $6.7M R01 grant from the National Cancer Institute for a large, multi-national, multi-disciplinary study comparing symptom burden/toxicity, neurocognitive change, and functional outcomes in pediatric brain tumor patients treated with proton vs. photon radiotherapy. Dr. Kahalley is leading this study with Dr. Donald Mabbott at The Hospital for Sick Children (Toronto) and Dr. Pamela Hinds at Children’s Hospital (DC).

An FDA-supported consortium anchored at Texas Children’s Hospital supports innovation and development of new pediatric devices (Mar 1, 2021)
The Southwest National Pediatric Device Innovation Consortium (SWPDC), anchored by Texas Children’s Hospital and Baylor College of Medicine, is a virtual accelerator for innovators focused on developing novel devices for pediatric care nationwide. They are one of five Food and Drug Administration (FDA)-supported national consortia that are addressing the national shortage of much-needed innovations in pediatric devices, a public health problem acknowledged by the FDA.

Updated spina bifida guidelines cover care across the entire lifespan (Feb 9, 2021)
The spina bifida team at Texas Children's Hospital and Baylor College of Medicine led by Dr. Jonathan Porter along with experts at the Spina Bifida Association and Collaborative Care Network, National Center on Birth Defects & Developmental Disabilities at the CDC authored updated care recommendations.

How Texas Children’s Hospital successfully pivoted to eHealth amidst a global pandemic (Jan 28, 2021)
Amidst the global COVID-19 pandemic, Texas Children’s Hospital rapidly innovated and expanded system-wide eHealth services to ensure the hospital could continue providing the best possible care to its patients. In this article, Dr. Robert Ball, medical director of eHealth at Texas Children’s and professor at Baylor College of Medicine, and Dr. Elizabeth Onugha, a pediatric nephrologist at Texas Children’s and assistant professor at Baylor College of Medicine, recount how they and their colleagues quickly and effectively pivoted to eHealth amid COVID-19.

Dr. Benjamin Arenkiel recognized by TAMEST for breakthrough findings (Jan 13, 2021)
Dr. Benjamin Arenkiel, assistant professor of molecular and human genetics and neuroscience at Baylor College of Medicine and an investigator at the Jan and Dan Duncan Neurological Institute at Texas Children's Hospital, has been awarded the prestigious 2021 Edith and Peter O’Donnell Award in Medicine from the Academy of Medicine, Engineering and Science of Texas (TAMEST). He is being recognized for his breakthrough identification of the brain’s neural pathways that are connected to eating disorders, addiction and other neuropsychiatric disorders, leading to a better understanding of the makeup of neural circuits and how they talk to each other.

The American Diabetes Association revises guidelines for hemoglobin A1C levels in children with type 1 diabetes (Jan 11, 2021)
Diabetes is characterized by elevated levels of sugar or glucose (hyperglycemia) in the blood. This occurs due to the lack of the hormone insulin in type 1 diabetes, and to reduced insulin levels in combination with insulin resistance in type 2 diabetes. A recent review of data supports stricter control of hemoglobin A1C levels (HbA1C) among pediatric patients with T1D. This review was led by Dr. Maria J. Redondo, pediatric endocrinologist at Texas Children’s Hospital and professor at Baylor College of Medicine, in collaboration with Dr. Sarah Lyons, pediatric endocrinologist at Texas Children’s and assistant professor at Baylor College of Medicine, along with other leading endocrinologists and diabetes experts across the U.S.

Texas Children's Hospital celebrates a decade of neurological research with the 10th anniversary of the Jan and Dan Duncan Neurological Research Institute (Jan 11, 2021)
Last month, the Jan and Dan Duncan Neurological Research Institute (Duncan NRI) at Texas Children's Hospital and Baylor College of Medicine celebrated 10 years of innovation, world-renowned research and scientific breakthroughs.

EIF2AK2 variants cause symptoms akin to Pelizaeus-Merzbacher disease (Dec 21, 2020)
A comprehensive analysis of two unrelated individuals now reveals that variants in Eukaryotic Initiation Factor 2 Alpha Kinase 2 (EIF2AK2) gene can result in clinical symptoms that are remarkably similar to Pelizaeus-Merzbacher disease (PMD). The lead authors of this study, who are child neurologists and neuroscientists at Texas Children’s Hospital and Baylor College of Medicine, suggest that “EIF2AK2-related Leukoencephalopathy, Developmental Delay, and Episodic Neurologic Regression (LEUDEN) syndrome,” a brand-new disorder that they discovered in March 2020 should be considered as a differential diagnosis for PMD and other hypomyelinating dystrophies.

Novel gene variants that modify the risk of late-onset Alzheimer’s disease discovered (Dec 7, 2020)
Researchers at Baylor College of Medicine and Texas Children’s Hospital have found 216 new genetic modifiers of late-onset Alzheimer's disease, many previously not suspected to play a role in this disease. This is an exciting first step toward understanding this paradox. The newly-identified biomarkers could potentially be used in the future to refine risk assessment and patient prognosis in APOEɛ2 and APOEɛ4 carrier populations and act as therapeutic targets for this untreatable condition.

A role for the slow oscillations of the neocortex in epileptic spasm generation (Dec 7, 2020)
Epileptic spasms are a type of brief seizures that are the hallmark of catastrophic seizure disorders, which are characterized by severe cognitive and motor deficits. While common in West syndrome (or infantile spasms), these spasms can occur in people of any age group. Very little is known about the underlying neurophysiological mechanisms and the neuronal circuit(s) responsible for the generation of these spasms. Researchers from Baylor College of Medicine and Texas Children’s Hospital now provide evidence that epileptic spasms originate from the pyramidal cells in the deep layers of the neocortex. Further, they uncovered a novel neurophysiological phenomenon that explains how cortical neurons generate spasms. This is also the first study to reveal a hitherto unknown relationship between a normal brain state, like sleep, and spasms. The study was recently published in the Annals of Neurology.

Dr. Rayne Rouce featured highlighted by American Society for Transplantion and Cellular Therapy (Dec 7, 2020)
In an interview in Nucleus, an online magazine of ASTCT, Dr. Rayne Rouce discusses how she is leveraging the lessons she learned through the ASTCT Leadership Course to help guide her decisions during COVID-19.

ing receives a highly competitive federal award (Dec 1, 2020)
Dr. Katherine King, associate professor of the Infectious Disease Division in the Department of Pediatrics at Baylor College of Medicine and Texas Children’s Hospital, received a highly competitive Emerging Investigator Award. This award of $600,000 per year for seven years is from the National Heart, Lung Blood Institute (NHLBI) at the National Institutes of Health (NIH).

BICRA gene provides answers to patients, doctors and scientists (Nov 23, 2020)
Physicians and scientists are constantly on the lookout for new disease genes that can help them understand why patients have undiagnosed medical problems. Often the first clues come from genetic testing that reveals a change or mutation in a gene that they see in a child but not their parents. This is exactly what led to a new study published today in the American Journal of Human Genetics.

Maraxilibat reduces debilitating itching in children with Alagille Syndrome (Nov 18, 2020)
On behalf of Childhood Liver Disease Research Network (ChiLDReN), Texas Children’s Hospital and Baylor College of Medicine researchers report that prolonged treatment with Maraxilibat resulted in clinically meaningful improvements in debilitating itching (pruritus) and related quality of life outcomes in children with Alagille syndrome. This syndrome is a rare genetic systemic disorder in which problems with bile flow can cause significant liver injury and potential liver failure necessitating liver transplantation. The novel pharmacological approach addresses a major unfulfilled therapeutic need to control severe itching in pediatric patients with Alagille syndrome.

An epidemic outbreak of Mesoamerican Nephropathy in Nicaragua linked to nickel toxicity (Nov 12, 2020)
For more than 20 years, an epidemic of chronic kidney disease (CKD) of unknown origin has severely affected specific coastal communities along South America’s Pacific coastline from Mexico to Panama leading to more than 50,000 deaths. The condition, known as Mesoamerican Nephropathy (MeN), has a perplexing clinical presentation. Unlike traditional forms of CKD, it affects healthy young working-age individuals who do not have other traditional risk factors for kidney disease, such as diabetes or hypertension. The underlying cause of this devastating public health crisis has remained a mystery.

NIH funds a new Fragile X Research Center at Baylor College of Medicine and Duncan NRI at Texas Children’s Hospital (Nov 11, 2020)
The National Institutes of Health (NIH) chose Baylor College of Medicine as a site for one of the three Centers for Collaborative Research on Fragile X syndrome (FXS), the most common inherited form of intellectual and developmental disability (IDD). The new Fragile X Center will receive about $9 million over the next five years and will be led by co-directors, Dr. David Nelson, professor of Molecular and Human Genetics at Baylor College of Medicine and investigator at the Jan and Dan Duncan Neurological Research Institute (Duncan NRI) at Texas Children’s Hospital, and Dr. Peter Todd, associate professor of Neurology at the University of Michigan School of Medicine.

The Intellectual and Developmental Disabilities Center at Baylor College Receives Competitive Federal Grant to Advance Research and Therapies (Nov 11, 2020)
The Eunice Kennedy Shriver Intellectual and Developmental Disabilities Research Center (IDDRC) at Baylor College of Medicine recently received a highly-competitive grant from the National Institutes of Health (NIH). The $6.4 million five-year competitive grant was awarded to the IDDRC’s director, Dr. Huda Zoghbi, professor at Baylor College, director of the Jan and Dan Duncan Neurological Research Institute (Duncan NRI) at Texas Children’s Hospital and Howard Hughes Medical Institute investigator; as well as Dr. David Nelson, professor at Baylor College and Dr. Rodney Samaco, assistant professor at Baylor College, who are the Center’s co-directors and investigators at the Duncan NRI.

Abstract submissions and registrations open for the 2021 Pediatric Research and Fellows Symposium (Nov 2, 2020)
Texas Children’s Hospital and Baylor College of Medicine’s Department of Pediatrics are looking forward to celebrating the research accomplishments of our pediatric research trainees and pediatric fellows on April 20, 2021. The Pediatric Research and Fellows’ Symposium will feature oral presentations from the top scoring abstract authors, a keynote scientific address, a moderated poster session, a keynote academic presentation, and a workshop focused on “For the Greater Good”.

NIH-funded study no difference in safety of efficacy between three common anticonvuslant medicines (Oct 27, 2020)
There are three treatment options commonly used by doctors in the emergency room to treat patients with refractory status epilepticus, severe seizures that continue even after benzodiazepine medications, which are effective in controlling seizures in more than two-thirds of patients. New findings published in the New England Journal of Medicine reveal that the three drugs, levetiracetam, fosphenytoin, and valproate, are equally safe and effective in treating patients with this condition. The study was supported by the National Institute of Neurological Disorders and Stroke (NINDS), part of the National Institutes of Health.

Texas Children's Awarded Inaugural Survivorship Champion's Prize (Oct 15, 2020)
Children's Cancer Cause names Texas Children's Hematology and Cancer Center's Long-term Survival Program as the 2020 recipient of the inaugural Survivorship Champion's Prize in recognition of the Program's innovative work to provide comprehensive, integrated care for childhood cancer survivors.

Researchers find CAR-NKT could be promising immunotherapy for solid tumors (Oct 14, 2020)
Researchers at Baylor College of Medicine, Texas Children's Hospital and the University of North Carolina at Chapel Hill have genetically modified human NKT cells with a chimeric antigen receptor (CAR) that enables them to specifically recognize and attack neuroblastoma, a form of childhood cancer. Expressed with the CAR is interleukin-15 (IL-15), a natural protein that supports NKT cell survival.

Kirk earns Fasser Visionary Award (Baylor College of Medicine - October 9, 2020)
Susan Kirk, MSPAS, PA-C, assistant professor of hematology/oncology at Baylor and Texas Children's Cancer Center, is the 2020 recipient of the Carl E. Fasser Visionary Leadership Award. Named for the longtime director of the Physician Assistant Program at Baylor College of Medicine, the award recognizes excellence by a physician assistant in education, research, clinical practice and service. The award was announced during National PA week, Oct. 6

First reversal of Type 1 Diabetes using precision medicine (Oct 8, 2020)
In a letter published today in the New England Journal of Medicine, a team of physicians from Baylor College of Medicine, Texas Children’s Hospital, and the University of California, San Francisco, describe a remarkable case of a Type 1 diabetes (T1D) patient, who no longer needs insulin to maintain optimal blood sugar levels. The physicians employed a precision/personalized medicine approach to specifically correct the underlying genetic mutation, which was the primary driver of this patient’s diabetes.

ASPHO spotlights Dr. Lubega's career path (Oct 6, 2020)
The career path of Dr. Joseph Lubega, assistant professor, Baylor College of Medicine, associate director of Pediatric Hematology-Oncology Fellowship Program and Vice Chair of Global Health SIG at ASPHO was spotlighted in an article by ASPHO.

Texas Children’s, Baylor College and other area institutions receive $12 million NIH grant to study multidrug resistance (Oct 2, 2020)
The National Institute of Allergy and Infectious Diseases (NIAID) awarded a five-year, $12 million grant to the Dynamics of Colonization and Infection by Multidrug-Resistant Pathogens in Immunocompromised and Critically Ill Patients (DYNAMITE) program, which includes researchers from the Microbiome Center at Texas Children’s Hospital and Baylor College of Medicine, as well as several other institutions in the Texas Medical Center, to study factors that increase the risk of multidrug-resistant bacterial infections in severely ill and immunocompromised patients.

Global HOPE provides infrastructure for improving pediatric cancer care in Africa (Oct 2, 2020)
Since Burkitt lymphoma was initially described in Uganda in the 1960s, it has emerged as the most common pediatric cancer in equatorial Sub-Saharan Africa and the most common pediatric non-Hodgkin lymphoma worldwide. In the early years, survival rates were slim. After decades of improvements in treatment options, today, the cure rate for Burkitt lymphoma exceeds 90% in high-income countries like the U.S. and those in Western Europe. But the prognosis is dramatically worse in low-income countries in Africa, with survival rates estimated at less than 30%. Doctors at Baylor College of Medicine, Texas Children’s Cancer and Hematology Centers, and Texas Children’s Global HOPE (Hematology-Oncology Pediatric Excellence) Program are working to improve those odds. They have been caring for children with endemic Burkitt lymphoma in Malawi and Uganda, striving to improve survival rates.

NIH grant supports national study of atypical diabetes causes (Oct 1, 2020)
Diabetes mellitus is the most common metabolic disease affecting hundreds of millions around the world. Although patients are typically classified as having type 1 or type 2 diabetes, increasingly many persons do not fall under these categories. They may have atypical or new forms of diabetes and need a more precise diagnosis and specific treatment. The National Institutes of Health has selected Baylor College of Medicine and the University of Chicago to lead a national, multicenter study to discover atypical and rare forms of diabetes, the Rare and Atypical Diabetes Network (RADIANT).

Low doses of insecticide trigger neurodegeneration in insects (Sep 28, 2020)
Insect populations are consistently declining around the world and intense use of insecticides is suspected to play a role. This week, a study published in the Proceedings of the National Academy of Science USA by researchers at the University of Melbourne, Baylor College of Medicine, Texas Children’s Hospital and other institutions shows that very low doses of imidacloprid, the world’s most used insecticide, trigger neurodegeneration and disrupt body-wide functions, including energy production, vision, movement and the immune response, in the laboratory fruit fly, Drosophila melanogaster.

Dr. Huda Zoghbi named Citation Laureate by Clarivate Web of Science (Sep 23, 2020)
Dr. Huda Zoghbi, director of the Jan and Dan Duncan Neurological Institute at Texas Children’s Hospital and a Howard Hughes Medical Institute investigator at Baylor College of Medicine, has been named a Citation Laureate for 2020 by Clarivate Web of Science.

Dr. Taylor Receives Pediatric Infectious Disease Society Antimicrobial Stewardship Fellowship Award (Sep 17, 2020)
Margaret “Maggie” Taylor, M.D., a second-year pediatric infectious disease fellow at Baylor College of Medicine, was one of three candidates awarded the Pediatric Infectious Disease Society (PIDS) Antimicrobial Stewardship Fellowship Award on Aug 26, 2020. This award is given annually to fellows pursuing research in antimicrobial stewardship and provides mentorship from leaders within the Pediatric Committee on Antimicrobial Stewardship.

Baylor College of Medicine selected as a new Center of Excellence for research on environmental health disparities (Sep 17, 2020)
Baylor College of Medicine has received a grant from the National Institutes of Health to fund a new Maternal and Infant Environmental Health Riskscape (MIEHR) Research Center. Baylor will receive $7.1 million over five years and is one of only three sites in the country selected as a Center of Excellence for environmental health disparities research by the National Institute of Minority and Health Disparities. The goal of this new center is to identify key drivers of racial disparities in pregnancy outcomes, such as preterm birth and hypertensive disorders during pregnancy.

Three research teams earn Dunn Awards (Sep 4, 2020)
Three teams of Rice University and Baylor College of Medicine researchers have been named winners of the 2020 John S. Dunn Collaborative Research Awards. The teams include Baylor College and Texas Children's researchers: Drs. Swathi Balaji, Andrew DiNardo and Mingshan Xue.

Fighting cancer with rejection-resistant, ‘off-the-shelf’ therapeutic T cells (Aug 20, 2020)
Personalized cancer treatments are no longer just options of the future. In the past few years, researchers have made significant progress in ‘teaching’ the body’s immune T cells to recognize and kill specific cancer cells, and human clinical trials have shown that this approach can successfully eliminate tumors.

Novel regulators of myelin repair and regeneration discovered (Aug 13, 2020)
Myelin, a type of glial cell, forms an insulating sheath that surrounds the nerves of the brain and the spinal cord and is critical for rapid and accurate transmission of nerve signals. Damage or loss of myelin sheath is a hallmark of several neurological diseases in adults (e.g. multiple sclerosis) and in infants (e.g. cerebral palsy) and is common after injury. However, little is known about the signaling mechanisms involved in the repair and regeneration of myelin. A recent study from Baylor College of Medicine and Texas Children’s Hospital has identified a new regulator (Nedd4) and genetic pathway (Daam2-VHL-Nedd4) that controls myelin formation during development and after injury. The study appears in the journal Genes and Development.

Dissecting the genetics of neurodevelopmental disorders – one cell at a time (July 20, 2020)
In recent years, a large number of genes have been implicated in neurodevelopmental disorders (NDD) such as autism, developmental delays, intellectual disabilities and epilepsy. However, without a thorough understanding of how a gene functions in different brain cells during various stages of development and later in post-natal life, it is difficult to determine how they individually – and as a group – contribute to disease pathologies.

Shulman receives Derek Denny-Brown Young Neurological Scholar Award (July 17, 2020)
The American Neurological Association (ANA), a leading and among the oldest associations for academic neurology, selected Dr. Joshua Shulman for this year’s Derek Denny-Brown Young Neurological Scholar Award in the Physician-Scientist - Basic Sciences category.

CAR T cell immunotherapy results in patient recovery (July 16, 2020)
n a clinical trial evaluating a novel immunotherapy option for cancer treatment, a child with rhabdomyosarcoma, a form of muscle cancer, that had spread to the bone marrow, showed no detectable cancer following treatment with chimeric antigen receptor (CAR) T cells that were engineered to target the HER2 protein on the surface of the cancer cells.

COVID-19 study, recruitment underway to assess emotional impact on children and their families (July 16, 2020)
For many of us, 2020 has been an unprecedented year filled with numerous challenges. Since March, the COVID-19 global pandemic has disrupted our normal way of life. Many of us are working from home. We’re social distancing ourselves from each other, and wearing protective face masks wherever we go – both at work and in public - to protect ourselves and curb the spread of this virus.

Meta-Analysis of the Alzheimer’s Disease Human Brain Transcriptome and Functional Dissection in Mouse Models (July 15, 2020)
A collaborative study in Cell Reports led by Drs. Zhandong Liu and Joshua Shulman presents a meta-analysis of the #Alzheimer #disease human #brain #transcriptome & functional dissection in mice.

Nano-radiomics unveils treatment effect on tumor microenvironment (July 13, 2020)
Drs. Robin Parihar & Ketan Ghaghada at Texas Children's Hospital and Baylor College of Medicine developed Nao-Radiomics, a novel method to assess the effect of celular immunotherapy against the inhibitory effects of tumor microenvironment to improve treatment of cancers.

Grant money available for COVID-19 related, other critical need projects (July 7, 2020)
Calling all innovators! The Southwest National Pediatric Device Innovation Consortium (SWPDC) needs your help in identifying pediatric device projects that support COVID-19 related and other critical need projects.

Solving the CLN6 mystery in Batten Disease (June 30, 2020)
Batten disease is a family of 13 rare, genetically distinct conditions. Collectively, they are the most prevalent cause of neurodegenerative disease in children, affecting 1 in 12,500 live births in the U.S. One of the Batten disease genes is CLN6. How mutations in this gene lead to the disease has been a mystery, but a study led by researchers at Baylor College of Medicine and Texas Children’s Hospital and published in the Journal of Clinical Investigation reveals how defective CLN6 can result in Batten disease.

Chao receives the Philip R. Dodge Young Investigator Award (June 29, 2020)
Dr. Hsiao-Tuan Chao, pediatric neurologist, assistant professor at Baylor College of Medicine and an investigator at the Jan and Dan Duncan Neurological Research Institute at Texas Children’s Hospital, received the prestigious 2020 Philip R. Dodge Young Investigator Award from the Child Neurology Society.

Texas Children's publishes a paper detailing the clinical course of 57 children with COVID19 (June 23, 2020)
Interim Physician-in-Chief Dr. James Versalovic and several other Texas Children’s physicians recently published a paper in the Oxford Academic: The Journal of the Pediatric Infectious Diseases Society detailing the clinical course of 57 children with COVID-19 cared for at Texas Children’s at the beginning of the pandemic. Dr. Catherine Foster with Infection Control and Prevention is the lead author of the paper and Dr. Judith Campbell with Infection Control and Prevention is the senior author.

Lipid emulsions can prevent liver disease in preterm babies (June 22, 2020)
Study by Drs. Doug Burrin, Lee Call & Muralidhar Premkumar provides clues on how lipid emulsions prevent liver disease in preterm babies.

Size of fetal neural tube defect can predict adverse neurological outcomes after prenatal NTD repair (June 18, 2020)
Study led by Dr. Magdalena Sanz Cortes finds evaluation of the size of fetal neural tube defect can predict adverse neurological outcomes after prenatal NTD repair.

Baylor and Texas Children's researchers launch a landmark online pediatric cancer study (June 18, 2020)
Researchers at Baylor College of Medicine and Texas Children’s Hospital have launched a landmark online study, called “The Reasons Why Us,” to investigate the environmental and associated genetic risk factors for pediatric cancer. This epidemiological study is being conducted in partnership with The Oliver Foundation in Florida, which so far has engaged nearly 400 families who have been affected by pediatric cancer and want to participate in the research.

High-molecular weight hyaluron reduces tissue scarring in kidney injury (June 18, 2020)
Research team led by Dr. Sundeep Keswani finds interleukin10-induced production of large-molecular weight hyaluron reduces tissue scarring after kidney injury, revealing a potential strategy for chronic kidney diseases.

Unraveling a novel role for TFEB in liver development, regeneration and biliary cancer (June 17, 2020)
Transcription factor EB (TFEB) is a master regulator protein that allows cells to adapt and respond appropriately to ever-changing environmental cues, such as changes in nutrition, physical exercise and infections. The protein senses changes in the cell’s immediate environment and alters fundamental metabolic processes to ensure that cellular processes remain at equilibrium. A recent study by the researchers at the Jan and Dan Duncan Neurological Research Institute at Texas Children’s Hospital, Baylor College of Medicine and Telethon Institute of Genetics and Medicine (TIGEM) in Italy, reports a novel role for TFEB in liver development and regeneration.

Cancer Center faculty and trainees recognized with awards (June 15, 2020)

Following faculty received 2020 BCM Awards -

Flores, Ricky, Clark Faculty Service Award
Sasa, Ghadir and Slone, Jeremy, Norton Rose Fulbright Faculty Excellence Award for Teaching and Evaluation
Venkatramani, Raj, Norton Rose Fulbright Faculty Excellence Award for Enduring Educational Materials
Shapiro, Mary, Early Career Faculty Award for Excellence in Patient Care
Erana, Rodrigo, Kamdar, Kala, Martinez, Caridad, Rednam, Surya, Yates, Amber, STAR Faculty Award for Excellence in Patient Care
Bernini, Juan Carlos, Chintagumpala, Murali, Dreyer, ZoAnn, Mahoney, Donald, McClain, Kenneth, Master Clinician Faculty Award for Excellence in Patient Care.

Others in the Center who were also recently recognised are -

El-Mallawany, Nader, received the teaching award from graduating pediatric residents at TCH/BCM.
Smith, Valeria, selected as a BCM Critical Thinking and Problem Solving (CTAPS) facilitator for academic year 2020-2021.
Khaled Sanber, BCM resident in Internal Medicine MeRIT program, mentored by Meena Hegde, received Early Stage Professional Abstract Award by the International Society of Cell and Gene Therapy (ISCT), Modulating negative receptor signals to improve anti-glioblastoma CAR T-cell therapy. Dr. Sanber is one of the three recipients of this award announced at the 2020 ISCT Annual Meeting.

Hematology and Oncology faculty selected as leaders of national professional associations (June 15, 2020)

Michelle Fritsch named president-elect for the Association of Pediatric Oncology Social Workers, the national social work association.
Quinn Franklin named president-elect for the Association of Child Life Professionals, the national child life specialist association.
Rayne Rouce selected as Chair of Pediatric Hematological Malignancies for 2020 ASH Annual Meeting.
Donald Parsons invited to join the National Cancer Institute's PDQ Pediatric Treatment Editorial Board to provide guidance related to pediatric cancer genomics and neuro-oncology.
Philip Lupo selected to serve on the Genetics of Health and Disease Study Section, part of the NIH Center for Scientific Review.
Ghadir Sasa invited to join the Bone Marrow Transplant Clinical Trials Network Non-Malignant Disorders State of the Science Committee preceding the symposium in Honolulu in February of 2021.

Dr. Mahoney receives George R. Buchanan Lecturership Award (June 15, 2020)
Dr. Donald Mahoney received the George R. Buchanan Lectureship Award from the American Society of Pediatric Hematology/Oncology. This honor is in recognition of his scientific contributions to the field and his outstanding mentorship. The award will be presented at the 2021 ASPHO Conference in Portland, Oregon.

Dr. Geoffrey Preidis leads the AGA technical review on the use of probiotics for specific gastrointestinal conditions (June 9, 2020)
American Gastroenterological Association (AGA), a leading society of medical professionals, has published the first-ever clinical guidelines for the use of probiotics in the prevention and treatment of common gastrointestinal (GI) conditions. These guidelines are a culmination of a comprehensive two-year technical review conducted by a team of researchers and gastroenterologists. The review team, led by Dr. Geoffrey Preidis, pediatric gastroenterologist at Texas Children’s Hospital and assistant professor at Baylor College of Medicine, performed a rigorous analysis of published clinical trials to find evidence supporting the use of probiotics in preventing and treating specific GI disorders. The technical review and guidelines were published in Gastroenterology, the official journal of AGA.

Assessing cancer diagnosis in children with birth defects (May 29, 2020)
Scientific studies suggest that children with birth defects are at increased risk of cancer. However, it has not been assessed whether the type of cancer, the age at which they are diagnosed or the extent of cancer spread at the time of diagnosis, is different for children with birth defects compared to children without birth defects. In this study led by Baylor College of Medicine and Texas Children's Hospital, researchers compared about 13,000 children with cancer but no birth defects with nearly 1,600 children with cancer and one or more birth defects. The results were published in the journal Cancer.

Study finds excess monitoring of oxygen levels in certain pediatric populations (May 26, 2020)
Monitoring blood oxygen levels with continuous pulse oximetry is overused in infants with bronchiolitis who do not require supplemental oxygen, according to a multi-institutional study published recently in JAMA. The researchers found frequent use of continuous pulse oximetry and a wide variation in its usage among hospitals in their sample, despite national recommendations advising against the practice.

Research reveals new way to predict genetic diseases (May 18, 2020)
Most genetic diseases are caused by spontaneously arising mutations in the genetic code. Point mutations that cause miscoding of the amino acid, arginine, occur more often than mutations in any other amino acids. A comprehensive genome-wide study from the laboratory of human geneticist Dr. Michael Wangler, assistant professor at Baylor College of Medicine and investigator at the Jan and Dan Duncan Neurological Research Institute at Texas Children’s Hospital, has led to a method to predict human diseases based on arginine codon usage pattern. The study appeared in the journal Genetics in Medicine.

Dr. Cohen receives the HTRS 2020 Mentored Research Award (May 11, 2020)
Dr. Clay Cohen, pediatric hematologist at Texas Children’s Hospital Cancer and Hematology Center/Baylor College of Medicine, received his Pediatric Hematology and Oncology fellowship training at Texas Children’s Hospital. Dr. Cohen joined Dr. Joel Moake’s laboratory during his fellowship at Rice University and developed his research project, investigating the role of the vascular wall in coagulation. Following the completion of his fellowship in 2018, Dr. Cohen has remained at Texas Children’s as a faculty member, where he is an active member of the Hemostasis and Thrombosis clinical team and is continuing his basic research with Dr. Moake. To date, his research project has demonstrated that human endothelial cells and fibroblasts produce the coagulation proteins necessary for factor X activation, and that coagulation reactions occur on human endothelial cell surfaces without the addition of external coagulation proteins or phospholipids.

Dr. Abhishek Seth receives grant to study urological birth defects (Apr 30, 2020)
Dr. Abhishek Seth, pediatric urologist at Texas Children’s Hospital and assistant professor in the divisions of pediatrics and urology at Baylor College of Medicine, received a federal grant from the National Institutes of Health to support his research on congenital urological anomalies.

Dr. Huda Zoghbi awarded the Brain Prize (Apr 29, 2020)
Dr. Huda Zoghbi and Sir. Adrian Bird have mapped Rett syndrome, a rare neurological disorder that primarily affects girls during their early childhood. Their research also gives a unique insight into the epigenetics and overturns previous understanding, indicating that neurological developmental disorders are not necessarily irreversible. For this, two neuroscientists are now receiving the world's most prestigious prize for brain research – the Lundbeck Foundation Brain Prize.

Bellen elected to prestigious national academies (Apr 28, 2020)
Dr. Hugo J. Bellen, professor of molecular and human genetics and neuroscience at Baylor College of Medicine, Howard Hughes Medical Institute investigator, and member of the Jan and Dan Duncan Neurological Research Institute at Texas Children's Hospital has been elected as one of the newest members of the American Academy of Arts and Sciences, one of the country’s most prestigious honorary titles. He is among 276 artists, scholars, scientists, and leaders in the public, non-profit, and private sectors elected to the Academy for the class of 2020.

Promising new treatment for recurrent pediatric brain cancer (April 27, 2020)
Two pediatric brain cancers that are challenging to treat if they recur, medulloblastoma and ependymoma, are the target of a clinical trial using a new type of therapy. A multi-institutional, international team led by researchers at Baylor College of Medicine, Texas Children’s Hospital and the Hospital for Sick Children (SickKids) has developed a novel approach that delivers appropriately-targeted chimeric antigen receptor (CAR) T cell therapy directly into the cerebrospinal fluid that surrounds the tumor.

An international collaboration finds a novel neurodevelopmental syndrome (April 23, 2020)
An international study led by researchers at Baylor College of Medicine, Texas Children’s Hospital and Hunan Provincial Maternal and Child Health Care Hospital in China identified spontaneous point mutations in a cyclin-dependent kinase 19 (CDK19) gene, which causes a novel early-onset neurodevelopmental disorder characterized by uncontrolled early-onset seizures. The study provides the first experimental evidence that links mutations in the CDK19 gene to a human disorder. It appears in American Journal of Human Genetics.

Researchers engineer better way to target leukemia cells (April 8, 2020)
Acute Lymphoblastic Leukemia (ALL) is the most common childhood cancer. While most children respond well to chemotherapy, some experience resistant or relapsed disease. Building on an immunotherapy treatment called CAR T cell therapy, researchers at Baylor College of Medicine, working with colleagues from multiple institutions, were able to engineer a new fighter T cell that more effectively targeted leukemia cells in preclinical studies.

Srivaths recognized with Baylor College of Medicine's Women of Excellence award (April 8, 2020)
A group of women from across all mission areas and roles at Baylor College of Medicine were honored as Women of Excellence for their leadership, mentorship and commitment to academic medicine, including Dr. Lakshmi Srivaths of Texas Children's Cancer and Hematology Centers. In addition, allies who support women in their roles at Baylor also were recognized. See the award video and list of winners.

Dr. Ivan Chinn’s research recognized by international disease association (Apr 7, 2020)
Dr. Ivan Kingyue Chinn, a physician-scientist and director of the Immunogenetics Program at Texas Children’s Hospital, and assistant professor in the Pediatric Immunology, Allergy and Retrovirology section at Baylor College of Medicine, was honored by the Associazione Italiana Linfoistiocitosi Emofagocitica. This Italian nonprofit organization supports the families of children affected by hemophagocytic lymphohistiocytosis (HLH), a group of immune disorders that affect 1 in 50,000 children each year.

A new screening approach may improve outcomes for newborns with biliary atresia, a serious liver disease (Apr 6, 2020)
A study led by physicians and scientists at Texas Children’s Hospital and Baylor College of Medicine has developed a new screening approach for early diagnosis of biliary atresia, a serious pediatric liver disease that is the leading cause of liver transplantation among children. The study published in the Journal of American Medical Association reports elevated serum levels of direct or conjugated bilirubin can quickly and accurately identify newborns with this condition within two weeks of birth.

Inappropriate cellular response to stress linked to a new neurological disorder (Mar 24, 2020)
An international team led by researchers at the Baylor College of Medicine and Texas Children’s Hospital in collaboration with the Undiagnosed Diseases Network have identified gene variants in the EIF2AK family of kinases leads to previously unidentified neurodevelopmental disorders - in which inappropriate response to stress (such as fever triggered by viral infections) triggers degeneration of the nerve fibers connecting major brain regions, collectively known as the white matter.

Researchers learn to control brain cell that triggers tremor (Mar 18, 2020)
Researchers at Baylor College of Medicine and the Jan and Dan Duncan Neurological Research Institute at Texas Children’s Hospital have improved our understanding of how tremor — the most common movement disorder — happens, opening the possibility of novel therapies for this condition.

Surgery research team renews grant for wound repair (Mar 13, 2020)
Dr. Sundeep Keswani and his collaborator, Dr. Swathi Balaji, assistant professor at the Baylor College of Medicine, in the Laboratory for Regenerative Tissue Repair and Dr. Paul Bollyky, associate professor at Stanford University were awarded a renewal on one of their current R01s to continue investigating new ways to achieve regenerative wound repair after dermal injury.

New insight on ACOX1-related neurodegenerative disorders (Mar 12, 2020)
A recent study led by researchers at Baylor College of Medicine and Texas Children's Hospital in Houston reports that a hyperactive variant of enzyme ACOX1 produces elevated levels of toxic reactive oxygen species (ROS) and causes a previously unidentified late-onset neurodegenerative disorder. The team named this new syndrome “Mitchell disease” in reference to the first patient to be diagnosed with this disorder.

Zhandong Liu and team win a highly competitive award from Allen Institute (Mar 12, 2020)
Drs. Zhandong Liu and Jinyuan Hu are one of the three teams that were announced as winners of the Allen Institute Cell Lineage Reconstruction DREAM Challenge conducted by the Allen Institute and Sage Bionetworks, an open science competition for computational approaches to building accurate cell lineages in developmental biology.

The Neurosciences Program at Texas Children's hosts an inaugural retreat (Mar 4, 2020)
Texas Children’s Neurosciences Program is all about improving patient outcomes. Every day, our team of neurologists, neurosurgeons, geneticists, physician-scientists and researchers are working together tirelessly to pioneer innovative therapies to improve the lives of children with neurological disorders. On February 21, neuroscience leadership at Texas Children’s together with faculty and staff convened for the inaugural Neurosciences Retreat at the Jan and Dan Duncan Neurological Research Institute (NRI). This informative and engaging event provided our multidisciplinary team of neurologists, neurosurgeons, clinicians, researchers, behavioral health experts, and more the opportunity to reflect on past successes, discuss areas of possible improvement, identify transformative goals and actively plan for the future.

Srivaths honored by blood disorder foundation (Mar 2,2020)
Dr. Lakshmi Srivaths, professor of pediatrics – hematology, was selected as an Honorary Member of the Foundation for Women and Girls with Blood Disorders. In this role, she will help advance the foundation's efforts in improving clinical care, education and research in the field of adolescent females with bleeding and thrombophilia/thrombosis. She has been involved with the foundation for 10 years in various roles, including as a member of the Medical Advisory Council and chair of the Education and Advocacy Sub-committee.

Rooney elected microbiology fellow (Mar 2, 2020)
Dr. Cliona Rooney, professor of pediatrics and part of the Center for Cell and Gene Therapy, was one of 68 new fellows elected to American Academy of Microbiology for 2020. Fellows are an honorific leadership group within the academy, elected annually through a selective, peer-review process based on their records of scientific achievement and original contributions that have advanced microbiology.

Xue lab develops animal models for STXBP1 encephalopathy (Feb 19, 2020)
A study from the laboratory of Dr. Mingshan Xue, assistant professor at Baylor College of Medicine and investigator at the Jan and Dan Duncan Neurological Research Institute at Texas Children’s Hospital, reports the generation of novel mouse models that mimic the key features of STXBP1 encephalopathy – a rare neurological condition that encompasses a broad range of cognitive, motor and psychiatric dysfunction, as well as recurrent seizures.

Study highlights pediatrics medical overuse (Feb 5, 2020)
A literature review in JAMA Pediatrics by Dr. Nathan Money & others at Texas Children's Hospital and Baylor College of Medicine highlights established and emerging practices that represent medical overuse in pediatrics setting.

Rooney honored as leader in science (Baylor College of Medicine - January 31, 2020)
Dr. Cliona Rooney, professor of pediatrics and in the Center for Cell and Gene Therapy, was honored as one of four 2020 Women Leaders in Science by BioHouston, a local organization seeking to position the city as a leader in life science and biotechnology commercialization. Rooney was honored with her fellow awardees at the 11th annual WISE Luncheon last week at the River Oaks Country Club.

Dr. Katherine King weighs in on coronovirus outbreak (Feb 3, 2020)
In an interview with Chinese American news station, Sinovision, infectious disease expert, Dr. Katherine King, weighs in on the coronirus outbreak.

Brain tumors remodel neuronal synapses to promote growth (Jan 30, 2020)
Scientists in Dr. Benjamin Deneen's lab at Baylor College of Medicine and Texas Children's Hospital have found new evidence that glioma, a lethal form of brain cancer, alters the activity of neighboring neurons, accelerating a vicious cycle that drives tumor-associated epilepsy and tumor progression.

Dr. King receives the St. John’s School’s Distinguished Alumna award (Jan 29, 2020)
Dr. Katherine King, Associate Professor of Pediatric Infectious Diseases, Baylor College of Medicine, Co-Founder of Doctors for Change. This award is given to an alumnus/a in recognition of outstanding achievements or contributions to St. John's, the community, the state, or the nation.

TAMEST protégés selected (BCM Newslink - January 34, 2020)
Drs. Mirjana Maletic-Savatic , Maria Elena Bottazzi and Dr. Maksim Mamonkin from Texas Children's Hospital were selected as the 2020 Academy of Medicine, Engineering and Science of Texas (TAMEST) Protégé for 2020.

Study led by Dr. Maria Redondo finds sustaned increase in BMI among older children and teens raises their risk for type 1 diabetes (Jan 22, 2020)
The study results support that elevated BMI may exacerbate islet autoimmunity prior to clinical T1D, particularly in children with lower risk based on age and HLA. Interventions to maintain normal BMI may prevent or delay the progression of islet autoimmunity.

Heczey lab finds an effective way to curb liver cancers (Jan 21, 2020)
The study published in Cancer Immunology Research finds glypican-3-specific CAR T cells co-expressing IL15 and IL21 have superior expansion and antitumor activity against hepatocellular carcinoma

Fruit fly screen uncovers genes regulating peroxisomal disorders (Jan 16, 2020)
A recent study in the laboratory of Dr. Michael Wangler, assistant professor at Baylor College of Medicine and investigator at the Jan and Dan Duncan Neurological Research Institute at Texas Children’s Hospital, uncovered 18 genes that regulate peroxisomal biology – many of which may be potential therapeutic candidates for PBD-ZSD and other disorders affecting the peroxisomes. The study was published this week in G3: Genes/ Genomes/Genetics journal.

Dr. Hugo Bellen elected 2020 Vice-President/2021 President of the Genetics Society of America (Jan 8, 2020)
Dr. Hugo Bellen, professor at Baylor College of Medicine and investigator at the Howard Hughes Medical Institute and the Jan and Dan Duncan Neurological Research Institute, has been elected the 2020 Vice-President/2021 President of the Genetics Society of America.

ACTG2 variants discovered as a reliable diagnostic marker and prognostic tool for visceral myopathy (Jan 7, 2020)
Visceral myopathy is a rare disorder of smooth muscle dysfunction that manifests in a broad range of symptoms ranging from severe gastrointestinal and genitourinary dysfunction requiring surgical and nutritional interventions in newborns to mild gastrointestinal issues in adults. An international research team led by Dr. Michael Wangler, assistant professor at Baylor College of Medicine, clinical geneticist and investigator at the Jan and Dan Duncan Neurological Research Institute at Texas Children’s Hospital, performed an in-depth clinical and genomic analysis which revealed ACTG2 gene as the primary determinant of severe symptoms and worse long-term prognosis in majority of the patients.

Link between herpes virus and Alzheimer's disease refuted (Dec 18, 2019)
Researchers at Baylor College of Medicine and Texas Children's Hospital report today in the journal Neuron evidence that refutes the link between increased levels of herpes virus and Alzheimer’s disease. In addition, the researchers provide a new statistical and computational framework for the analysis of large-scale sequencing data.

Loss of OXR1 causes lysosomal dysfunction and a novel neurodevelopmental disorder (Nov 29, 2019)
An international collaborative study has recently discovered a new neurodevelopmental disorder characterized by brain atrophy, speech and motor delays, hypotonia (low muscle tone), seizures and intellectual disability in five affected individuals with pathogenic variants in the Oxidative Resistance 1 (OXR1) gene. Studies in fruit flies unearthed a novel role for OXR1 in regulating the acidic environment of the lysosomes (“the cellular garbage recyclers”), disruption of which is likely the underlying cause of this disorder.

Children's Cancer Cause Presents Rosen Award to Dr. David Poplack (Nov 19, 2019)
The Leonard M. Rosen Memorial Research Award is presented annually at the Children’s Cancer Cause Cocktail Reception in New York City. This year, Children’s Cancer Cause was proud to present the 4th Annual Rosen Award to David Poplack, MD, Director of Global HOPE.

Inaugural Rett syndrome symposium (Nov 19, 2019)
During the final week of October, Texas Children’s Hospital hosted visitors from around the world for a very special Rett Syndrome Symposium and Workshop. The two-day event was momentous for many reasons. It was the inaugural meeting held in the stunning new Auditorium and Conference Center at the Jan and Dan Duncan Neurological Research Institute (Duncan NRI) at Texas Children’s Hospital. It fortuitously coincided with Rett Syndrome Awareness Month. And the symposium also marked the 20th anniversary of the discovery of the underlying cause of Rett syndrome. In 2000, NRI director Dr. Huda Zoghbi’s research team made the pioneering discovery that loss-of-function mutations in methyl-CpG binding protein, MECP2, were the underlying cause of Rett syndrome.

Study reveals how maternal Zika virus infection results in microcephaly among newborns (Nov 14, 2019)
Microcephaly is a birth defect in which a newborn has a significantly smaller and under-developed brain. Although several inherited genetic mutations and environmental factors such as maternal malnutrition, in utero infections (e.g. Zika virus), exposure to alcohol or toxic drugs and fetal injury are known to cause microcephaly, very little is known about the underlying causative mechanisms. A new study from the laboratory of Dr. Hugo J. Bellen, professor at Baylor College of Medicine, investigator at the Howard Hughes Medical Institute and Jan and Dan Duncan Neurological Research at Texas Children’s Hospital has uncovered a novel genetic pathway that causes microcephaly in infants and have identified promising therapeutic targets. The study was published today in the journal Developmental Cell.

Dr. Heather Born receives the 2019 Duncan NRI Zoghbi Scholar Award (Nov 11, 2019)
Heather Born, Ph.D., postdoctoral associate in the laboratory of Dr. Anne Anderson, at Baylor College of Medicine, received the 2019 Duncan NRI Zoghbi Scholar Award. This is a special fund established by Dr. Huda Zoghbi, director of the Jan and Dan Duncan Neurological Research Institute (Duncan NRI) at Texas Children's Hospital, to honor the many generations of trainees who have worked in her laboratory and foster the next generation of successful scientists.

Learning to stop cancer in its roots (Nov 8, 2019)
A study published in Blood journal from laboratory of Dr. Daniel Lacorazza, associate professor of pathology & immunology at Baylor College of Medicine and principal investigator in the Experimental Immunology & Hematology Laboratory at Texas Children’s Hospital finds a way to eliminate leukemia stem cells in chronic myeloid leukemia.

Research collaboration focuses on CDKL5 Deficiency Disorder (Oct 30, 2019)
CDKL5 Deficiency Disorder (CDD) is a devastating neurodevelopmental disorder caused by mutations in the CDKL5 gene. The Loulou Foundation, a private non-profit foundation dedicated to the development of novel therapeutics for CDD, and Baylor College of Medicine and the Jan and Dan Duncan Neurological Research Institute (NRI) at Texas Children’s Hospital have launched a joint research collaboration focused on therapeutic research for this disorder.

Novel approach identifies factors linked to poor treatment outcomes in ALL (Oct 24, 2019)
The survival of patients with pediatric acute lymphoblastic leukemia (ALL) has significantly improved in the last several decades. However, novel strategies to identify cases that are likely to respond poorly to treatment are still needed. In a new study published in the journal EBioMedicine, researchers at Baylor College of Medicine and Texas Children’s Cancer Center reported that profiling the bone marrow metabolome, the constellation of small molecules produced by metabolism in patients’ bone marrow, at the time of diagnosis, enabled them to identify patients who were most likely to respond poorly to treatment.

Faculty earn AAP awards (Oct 18, 2019)
Dr. Fernando Stein, professor of pediatrics at Baylor and Texas Children's Hospital, was announced as the recipient of the Distinguished Career Award from the American Association of Pediatrics. Stein, a past president of AAP, is a critical care pediatrician and serves as medical director of the progressive care unit at Texas Children's. Dr. Susan Torrey, associate professor of pediatrics & emergency medicine, is the recipient of the Michael Shannon Humanitarian Award. She is associate director of the education division of the Global Hematology-Oncology Pediatric Excellence Program, or Global HOPE, which develops pediatric hematology and oncology services and resources in Africa.

Texas Children’s now part of national NIH-funded consortium for research in eosinophilic gastrointestinal diseases (Oct 14, 2019)
The National Institutes of Health renewed a five-year, $7.57 million grant to a multi-institutional Consortium of Eosinophilic Gastrointestinal Disease (CEGIR) to continue clinical research and train the next generation of experts in eosinophilic gastrointestinal disorders (EGID). For the first time, Texas Children’s Hospital physicians and Baylor College of Medicine pediatrics faculty are a part of this NIH-funded collaboration. Drs. Carla M. Davis and Anthony Olive are the local co-principal investigators while Drs. Sara Anvari and Eric Chiou are other contributors to this national research effort.

Researchers discover new intellectual disability syndrome (Oct 10, 2019)
Intellectual disability affects almost 200 million people around the globe. Yet, not much is understood about the underlying genes or molecular pathways involved in these conditions. An international group led by researchers at Baylor College of Medicine, Texas Children’s Hospital and the University of Geneva reports in the American Journal of Human Genetics a new neurological syndrome that appears to be especially common in countries where marriages between genetically related individuals, such as cousins, are prevalent.

Tau-mediated RNA splicing errors linked to Alzheimer’s (Oct 9, 2019)
A collaborative study published today in the journal Cell Reports provides evidence for a new molecular cause for neurodegeneration in Alzheimer’s disease. The study, led by researchers at Baylor College of Medicine and the Jan and Dan Duncan Neurological Research Institute at CrypSplice, integrates data from human brain autopsy samples and fruit flies to reveal a novel mechanistic link between alterations in RNA splicing and tau-mediated neurodegeneration in Alzheimer’s disease.

Meningioma molecular profile can predict tumor recurrence (Oct 9, 2019)
Although typically benign, about one-fifth of meningiomas, the most common primary brain tumors, recur despite complete surgical removal. The current meningioma classification does not consistently predict whether the tumor will recur, but researchers at Baylor College of Medicine and the Jan and Dan Duncan Neurological Research Institute at Texas Children's Hospital report today in the Proceedings of the National Academy of Sciences that using molecular profiles that might better predict meningioma recurrence.

Rett Syndrome Symposium and Workshop (Oct 7, 2019)
This fall marks the 20th anniversary of the discovery of the genetic basis of Rett syndrome. In honor of this important milestone, Drs. Adrian Bird and Huda Zoghbi are co-hosting a Rett Syndrome Symposium and Workshop Monday and Tuesday, October 28 and 29, at the Jan and Dan Duncan Neurological Research Institute in Houston.

Texas Children’s receives national recognition for sepsis improvement efforts (Sept 24, 2019)
Texas Children’s Hospital was recently honored with the 2019 Sepsis Heroes Award presented by the Sepsis Alliance at the organization’s eighth annual Sepsis Heroes gala on September 12. Sepsis Alliance, the nation’s leading sepsis organization, recognized Texas Children’s for its continued support and instrumental efforts in raising sepsis awareness and improving treatment for sepsis – the body’s life-threatening response to infection that affects 1.7 million children and adults each year.

Cancer Center on forefront of cutting-edge research, clinical trials (Sept 19, 2019)
As one of the best pediatric cancer centers in the nation, experts at Texas Children’s Cancer Center are on the forefront of cutting-edge basic, translational and clinical research. The center’s world-renowned faculty have pioneered many of the now standard protocols for treating and curing children with cancer.

Lubega to lead global special interest group
Dr. Joseph Lubega, assistant professor of pediatrics at Baylor and Texas Children's Cancer Center, recently was appointed vice chair of the Global Special Interest Group of the American Society of Pediatric Hematology/Oncology. His responsibilities will include organizing the annual special interest group meeting, engaging members who have an interest in global care, and working to extend the reach of ASPHO beyond the U.S. Lubega previously served as medical director of the Texas Children's Cancer Center's Global Hematology Oncology Pediatric Excellence (HOPE) Program in Uganda.

Jan and Dan Duncan Neurological Research Institute and Leica Microsystems Establish Center of Excellence in Microscopy (Sept 10, 2019)
The Jan and Dan Duncan Neurological Research Institute (NRI) at Texas Children's Hospital and Leica Microsystems, Inc. combined efforts to establish the Leica Microsystems Center of Excellence at the NRI. The new Center will support a mission to drive new discoveries and insights from scientific research performed using the imaging systems.

Kaplow earns grant for hurricane trauma response (Sept 6, 2019)
Dr. Julie Kaplow, associate professor of pediatrics – psychology at Baylor, chief of psychology service and director of the Trauma and Grief Center at Texas Children's Hospital, received a grant from the Robert Wood Johnson Foundation to partner with the Boys and Girls Club of Puerto Rico in an effort to assist children and families impacted by Hurricane Maria. The grant will support a large-scale mental health needs assessment and training of community and school clinicians in evidence-based, culturally informed treatments for childhood trauma and grief.

Texas Children’s and Baylor physicians at the forefront of nationwide research initiatives for spina bifida (Sept 3, 2019)
The Meyer Center for Developmental Pediatrics at Texas Children’s Hospital, Baylor College of Medicine and the Spina Bifida Association recently hosted the Houston Spina Bifida Education Day for patients, families and caregivers. Held at Houston Methodist Hospital, the event showcased newly established, evidence-based national guidelines for the care of individuals living with spina bifida. This is the first time the guidelines, written in part by Texas Children’s and Baylor clinicians and researchers, were shared with such a broad audience.

Researchers develop a new behavioral intervention to support family management of type 1 diabetes among African American/Black and Hispanic/Latino school-aged children (Aug 29, 2019)
Researchers are seeking families to participate in the Type 1 diabetes empowerment and management (TEAM) study led by Dr. Ashley Butler, behavioral scientist and assistant professor at Texas Children’s Hospital/ Baylor College of Medicine. The goal of this study, funded by the National Institutes of Health (NIH), is to create and test the effectiveness of delivering a family program among African-American/Black and Hispanic/Latino parents of school-aged children (5-9 years) with type 1 diabetes (T1D). Researchers believe the intervention – Family Teamwork-Peer Delivery (FT-P) – has the potential to promote positive diabetes management.

Texas Children’s hosts inaugural family conference for EBF3-HADD syndrome (Aug 26, 2019)
Texas Children’s Hospital hosted the first ever family conference for the EBF3-HADDS (Hypotonia Ataxia Developmental Delays Syndrome caused by changes in the EBF3 gene) syndrome between July 25 – 27, 2019 in Houston, TX. The conference was organized by the EBF3-HADDS Foundation, a new nonprofit organization created in 2018 by families to promote awareness, research and support for this genetic syndrome.

Bertuch named to NIH study section (Aug 23, 2019)
Dr. Alison Bertuch, associate professor of pediatrics – hematology/oncology at Baylor and Texas Children’s Cancer Center, recently was named chair of the Molecular Genetics B(MGB) Study Section of the National Institutes of Health Center for Scientific Review, for a two-year term. Applications reviewed by the MGB study section involve molecular mechanisms of genome replication, maintenance and gene expression.

Loss of ataxin-1 gene linked to Alzheimer’s disease risk (Aug 23, 2019)
A collaborative study including researchers at Baylor College of Medicine, Massachusetts General Hospital at Harvard Medical School and the Jan and Dan Duncan Neurological Research Institute at Texas Children’s Hospital reveals today in the journal Cell that the ataxin-1 gene, which is known to cause the rare neurodegenerative disease called spinocerebellar ataxia type 1 (SCA1), also can increase the risk of Alzheimer’s disease in a mouse model of the condition.

Baylor and Texas Children's faculty contribute to cancer initiative planning (BCM Newslink - August 16, 2019)
Drs. Sharon Plon, Donald W. (Will) Parsons, Michael Scheurer and Philip Lupo, of Baylor College of Medicine and Texas Children's Cancer Center, were invited to participate in the Childhood Cancer Data Initiative symposium at the National Cancer Institute July 29-31, along with other scientific stakeholders and leaders. This symposium was the scientific planning session for the proposed initiative, announced during the State of the Union address in February, to spend $500 million on childhood cancer research over the next 10 years, including efforts to collect, analyze and share data to make it easier for researchers and oncology care teams to learn from every child, adolescent and young adult with cancer.

Gene linked to Alzheimer's disease is involved in neuronal communication (Aug 14, 2019)
A study published this week in the journal Cell Reports from Dr. Joshua Shulman's lab sheds new light on how the CD2AP gene may enhance Alzheimer's disease susceptibility. Integrating experiments in fruit flies, mice and human brains, a multi-institutional team led by researchers at Baylor College of Medicine and Texas Children's Hospital found that the CD2AP gene is involved in synaptic transmission, the process by which neurons communicate. Digging deeper, the researchers discovered that CD2AP affects neuronal communication by regulating the levels of key regulatory proteins present at neuron terminals (synapses).

ASHG honors Huda Zoghbi with Victor A. McKusick Leadership Award (Aug 2, 2019)
The American Society of Human Genetics (ASHG) has named Huda Zoghbi, MD, as the 2019 recipient of the Victor A. McKusick Leadership Award. This award, named in honor of the late Victor A. McKusick, MD, recognizes individuals whose professional achievements have fostered and enriched the development of human genetics as well as its assimilation into the broader context of science, medicine, and health.

Collaborative research initiative reveals a novel neurological syndrome (July 19, 2019)
A multi-institutional study reveals the link between loss of WDR37 gene to a new neurological syndrome that is characterized by brain malformations and a range of symptoms including visual impairment, epilepsy, developmental delays and intellectual disability. The study was published in the American Journal of Human Genetics.

Scollon recognized by alma mater (July 15, 2019)
Sarah Scollon, assistant professor of pediatrics – oncology, has received the Diane Baker Alumni Award from the University of Michigan Genetic Counseling Program, where she earned a master's degree in genetic counseling. The award honors genetic counselors who display the same vision and commitment to the field as its namesake. Scollon will deliver the annual Diane Baker Alumni Lecture in September.

Lindsay to serve on AAP clinical trial committee (July 15, 2019)
Dr. Holly Lindsay, assistant professor of pediatrics – hematology/oncology, recently was appointed to serve as the American Academy of Pediatrics representative to the Institute for Advanced Clinical Trials for Children Steering Committee. In this role, she will advocate for the inclusion of pediatric and adolescent patients in adult clinical trials to increase the therapies available for these patients. Lindsay is part of the Texas Children's Cancer Center and Baylor College of Medicine's Adolescent and Young Adult Oncology Program.

Gramatges appointed to National Institutes of Health study section (July 11, 2019)
Dr. Monica Gramatges’s nomination to the National Institutes of Health (NIH) Cancer, Heart, and Sleep Epidemiology Panel A Study Section (CHSA) was recently approved and her five year term will begin July 1, 2019. The nomination process is quite extensive and requires concurrence from senior investigators outside of NIH, NIH Program staff, and official approval from the NIH Director, Dr. Francis Collins. In this role, Dr. Gramatges will join a team of scientists to serve as a peer reviewer for grants submitted to the NIH that are within a particular research field, including childhood cancer research.

Genomic technology predicts risk for children predisposed to cancer (July 3, 2019)
A review authored by Drs. Sharon E. Plon and Philip J. Lupo at Texas Children’s Cancer and Hematology Centers, surveys the evolving landscape of genes known to increase susceptibility to certain childhood cancers and supports the increasing use of genomic technology in cancer diagnosis. The article was published in the Annual Reviews of Genomics and Human Genetics.

McClain receives Lifetime Achievement Award (June 27, 2019)
Dr. Kenneth McClain received a Lifetime Achievement Award for his research and care of patients with Langerhans Cell Histiocytosis (LCH) from Mr. Apostolos Kontoyannis at the 29th Nikolas Symposium in Athens, Greece, May 16, 2019. The Kontoyannis family has organized this "think tank" symposium to advance the understanding and cure of LCH patients by bringing clinicians and scientists from various disciplines together for an intense 3 day meeting of presentations and collaborative discussions.

Study finds increased risk of certain cancers among children and adolescents with birth defects (June 20, 2019)
A multi-institutional study led by Dr. Philip Lupo, co-director of the Childhood Cancer Epidemiology and Prevention Program at Texas Children's Cancer and Hematology Center and associate professor at Baylor College of Medicine, finds children and adolescents with specific birth defects are at an increased risk of developing certain cancers. Dr. Sharon Plon, a leading medical geneticist who co-directs the Cancer Genetics and Genomics Program in the Texas Children’s Cancer and Hematology Center and a professor at Baylor College of Medicine and Dr. Jeremy Schraw, a post-doctoral fellow at Baylor College of Medicine were other key investigators involved in this study.

Researcher receives funding to develop an innovative new app for children with chronic abdominal pain (June 19, 2019)
Dr. John Hollier, pediatric gastroenterologist at Texas Children’s Hospital, recently received a Mentored Patient-Oriented Research Career Development Award (K23) from the National Institutes of Health (NIH) to support the development of a novel health care delivery initiative.

Afraid of food? The answer may be in the basal forebrain (June 17, 2019)
A recent study conducted in Dr. Benjamin Arenkiel's laboratory at the Jan and Dan Duncan Neurological Research Institute has identified novel circuits in basal forebrain that perceive and transmit odors from the food/enviroment to the hypothalamus and dramatically controls feeding behaviors.

Gastric bypass surgery offers better health outcomes to severely obese teenagers (June 14, 2019)
Severely obese adolescents derive significantly better health outcomes compared to similarly overweight adults after weight loss surgery, even though both groups of patients achieved similar weight loss, reports a recent study published in the New England Journal of Medicine.

Kline recognized for global humanitarianism (June 14, 2019)
Dr. Mark Kline, chair of pediatrics at Baylor and pediatrician-in-chief of Texas Children's Hospital, received the International Humanitarian and Global Health Leadership Award from the U.S. Cooperative for International Patient Programs in recognition of his exceptional contributions to global health and international humanitarian initiatives. He received the award on June 13 at the USCIPP Leadership Awards Dinner at the Petroleum Club of Houston, which is held in conjunction with the organization's annual meeting to discuss best practices in caring for international patients and in the delivery of healthcare abroad.

Faculty selected for leadership training (June 14, 2019)
Dr. Heather Haq, assistant professor of pediatrics hematology/oncology, was selected to participate in the highly competitive American Academy of Pediatrics Young Physicians Leadership Alliance training program, designed to develop leaders and build a leadership community amongst early career pediatricians and pediatric subspecialists.
Dr. Wendy Allen-Rhoades, also assistant professor of pediatrics hematology/oncology, was one of two physicians selected as an American Society of Clinical Oncology 2019-2020 Health Policy Leadership Development Program Fellow. She will receive training in leadership, communication and advocacy along with practical experience working with ASCO staff to shape cancer-related policies that directly affect individuals with cancer and clinical care.

Miterko receives Masao Ito award (June 12, 2019)
Lauren Miterko, graduate student in Dr. Roy Sillitoe's laboratory at the Jan and Dan Duncan Neurological Research Institute at Texas Children's Hospital and Baylor College of Medicine received the prestigious Masao Ito Award from the Society for Research on Cerebellum and Ataxias.

Chakrabarty receives career development award (June 12, 2019)
Dr. Rikhia Chakraborty, assistant professor of pediatrics - oncology, has received a Peer Reviewed Cancer Research Program Career Development Award, part of the Congressionally Directed Medical Research Programs that foster novel approaches to biomedical research. The award provides support for her research on "Cancer immunotherapy as treatment option for Langerhans cell histiocytosis."

Visualization of placental microbes reveal sparse community (June 12, 2019)
It is known that different areas of the body – the gut, mouth, vagina and even skin – are inhabited by many different species of bacteria that make up the microbiome and support key functions in the human body. Researchers at Baylor College of Medicine and Texas Children's Hospital previously found evidence that the placenta harbors a sparse but still present community of microorganisms, which they and other researchers speculate may contribute to key functions in pregnancy, including immunity.In a study in the current edition of the American Journal of Obstetrics & Gynecology, researchers used imaging techniques to visualize these sparse microbes in the placenta.

Clinical Research Center/Research Resources Office presents research award to Dr. Patricia Baxter (June 11, 2019)
The Clinical Research Center/Research Resources Office presented the Clinical Research Award for second Quarter 2019 to Dr. Patricia Baxter, Department of Pediatrics-Hematology/Oncology, Baylor College of Medicine.

BrightFocus Foundation honors Dr. Huda Zoghbi (June 11, 2019)
Dr. Huda Y. Zoghbi, the founding director of the Jan and Dan Duncan Neurological Research Institute at Texas Children’s Hospital; Ralph D. Feigin, MD, endowed chair and professor at Baylor College of Medicine; and an investigator with the Howard Hughes Medical Institute; was honored by the BrightFocus Foundation, a nonprofit organization that supports exceptional basic scientific research and provides public education on brain and eye diseases.

Obesity and certain non-autoimmune genes may contribute to type 1 diabetes (June 6, 2019)
A recent collaborative article led by Dr. Maria Redondo, a pediatric endocrinologist at Texas Children’s Hospital, provides current perspectives on the etiology of type 1 diabetes. This article presents evidence supporting the emerging theory that obesity and certain type 2-linked genes, may also contribute to the progression of type 1 diabetes. The article was published in the journal Diabetes Care.

American Pediatrics Society honors Dr. Huda Zoghbi and inducts seven other Baylor/Texas Children’s faculty (May 28, 2019)
Dr. Huda Zoghbi, M.D., has been named as the 2019 Norman J. Siegel New Member Outstanding Science Award recipient by the American Pediatrics Society (APS), a professional group whose mission is to shape the future of academic pediatrics through engagement of distinguished child health leaders.

DARPA-funded project aims to develop a magnetic headset to share visual perceptions from one brain to another at the speed of thought (May 21, 2019)
A Rice University-led team of neuroengineers is embarking on an ambitious four-year project to develop headset technology that can directly link the human brain and machines without the need for surgery. As a proof of concept, the team plans to transmit visual images perceived by one individual into the minds of blind patients. The team includes 15 co-investigators from Rice, Baylor College of Medicine, the Jan and Dan Duncan Neurological Research Institute at Texas Children’s Hospital, Duke University, Columbia University and Yale’s John B. Pierce Laboratory.

The 2019 Michael E. DeBakey Award for Excellence in Research (May 20, 2019)
The 2019 Michael E. DeBakey M.D. Award for Research Excellence ceremony honored five Baylor College of Medicine/Texas Children's Hospital faculty members for their excellence in published scientific contributions to clinical or basic science research over the past three years.

Faculty awards celebrate excellence in education, service and patient care (May 16, 2019)
Faculty members from Baylor College of Medicine and Texas Children's Hospital will be recognized for their outstanding contributions to education, community service and clinical care before their colleagues, family members and College leaders on May 16, 2019.

Wulff earns research grant (May 16, 2019)
Dr. Jade Wulff, clinical postdoctoral fellow in pediatric hematology/oncology at Baylor and Texas Children's Cancer and Hematology Centers, has received a St. Baldrick Foundation Fellow grant for her project, "Targeting integrin-mediated signaling in metastatic Ewing sarcoma." Fellow grants support early career scientists whose research is focused on new cures and treatments for childhood cancers.

Friedman named to professionalism board (May 16, 2019)
Dr. Ellen M. Friedman, professor of otolaryngology and director of the Center for Professionalism, recently was elected to the Academy for Professionalism in Health Care Board of Directors as an at-large member. She will serve a two-year term to promote and optimize patient care through professionalism education, scholarship, policy and practice in all health-related fields.

Cancer and Hematology Centers honor staff with Bravo Award (May 16, 2019)
Texas Children’s Cancer and Hematology Centers recently honored four team members with the Bravo Award for going above and beyond to ensure our patients and families receive the best possible care.

Texas Children's offers a new life-saving treatment for hepatocellular carcinoma (May 16, 2019)
A multidisciplinary team, led by Dr. Kamlesh Kukreja, expert in Interventional Radiology; is successfully performing the Trans-Arterial Radioembolization procedure – called TARE – on children who have this rare disease. “There are only three hospitals in the country performing TARE and we are one of them,” Kukreja said. “We are the only one in the state of Texas.”

Researchers develop a new bioinformatics tool to analyze CRISPR data (May 9, 2019)
A team led by researchers at Baylor College of Medicine has developed a new bioinformatics tool that analyzes CRISPR pooled screen data and identifies candidates for potentially relevant genes with greater sensitivity and accuracy than other existing methods. The new analytical web-based tool also is quicker and more user friendly as it does not require bioinformatics training to use it.

Dr. Amber Yates educates the public on sickle cell disease with TED-Ed video lesson (May 9, 2019)
Dr. Amber Yates, co-director of Texas Children's Sickle Cell program in the Center and Hematology Center explain how red blood cells get misshapen and sticky in patients with sickle cell disease in a TED-Ed video.

Dr. McClain receives the 2019 George Buchanan Lectureship Award (May 3, 2019)
Dr. Kenneth McClain received the 2019 George R. Buchanan Lectureship Award from Dr. Buchanan himself at the annual conference of the American Society for Pediatric Oncology in New Orleans. He is the fourth recipient of the award, which recognizes his outstanding clinical expertise, significant research contributions and mentorship

Dr. Sharon Plon to serve on National Council for Human Genome Research (May 3, 2019)
Dr. Sharon Plon, professor of pediatrics - oncology and molecular and human genetics at Baylor and co-director of the Texas Children's Cancer Center's Cancer Genetics and Genomics Program, has been appointed by the Secretary of the Department of Health and Human Services to the National Advisory Council for Human Genome Research until 2020. The council advises government agencies on genetics, genomic research, training and programs related to the human genome initiative. In addition, Plon received the 11th annual Niehaus Southworth Weissenbach Award in Clinical Cancer Genetics from the Memorial Sloan Kettering Cancer Center. As part of the award events, she delivered Medical Grand Rounds on May 3 titled "Genetic Predisposition to Childhood Cancer in the Genomic Era" and consulted with genetics clinicians.

American Pediatrics Society honors Dr. Huda Zoghbi and inducts seven other Baylor/Texas Children’s faculty (April 29, 2019)
Dr. Huda Zoghbi, M.D., has been named as the 2019 Norman J. Siegel New Member Outstanding Science Award recipient by the American Pediatrics Society (APS), a professional group whose mission is to shape the future of academic pediatrics through engagement of distinguished child health leaders.

McGinley receives John S. Dunn Research Collaborative Award (April 22, 2019)
Dr. Matthew McGinley, assistant professor at Baylor College and investigator at Cain Foundation Labs, receives a research seed grant to develop a platform to better understand mechanisms that integrate audio information with the brain’s broader cognitive maps and in the process improve technologies like cochlear implants.

A new and improved microbiome-based diagnostic strategy can help doctors deliver personalized treatments to children with irritable bowel syndrome (April 19, 2019)
Researchers find a new and improved diagnostic tool to help physicians pinpoint the underlying causes contributing to irritable bowel syndrome (IBS) in pediatric patients with greater accuracy and precision. The hope is that this information will allow targeted treatments for children with this chronic condition. The study, which was published in the Journal of Molecular Diagnostics, was led by Dr. James Versalovic, who serves as pathologist-in-chief and director of the Microbiome Center at Texas Children’s Hospital, as well as Professor and Vice Chair of Pathology & Immunology at Baylor College of Medicine.

Several Texas Children's faculty selected to receive top honors by the Houston Business Journal (April 15, 2019)
Texas Children's faculty members have been selected to receive 2019 Health Care Heroes Awards from the Houston Business Journal in several categories. They will be honored at ceremony May 23. They include:
Outstanding Health Care Practitioner: Dr. Ricardo Flores, clinical director, Cancer and Hematology Centers at Woodlands; Dr. Julie Kaplow, chief of psychology;
Outstanding Physician: Dr. Daniel DeSalvo, assistant professor of pediatrics and director of medical student education; Dr. Lisa Hollier, professor of obstetrics and gynecology; Dr. Peter Hotez, professor of pediatrics and dean of the National School of Tropical Medicine; and Dr. Rayne Rouce, assistant professor of pediatrics, hematology/oncology section.
Rising Star: Dr. Hsiao-Tuan Chao, child neurologist; and Dr. Arindam Sarkar, resident physician in family and community medicine.

Rayne Rouce appointed to a leadership role in the diversity office (April 15, 2019)
The Office of the Provost and the Office of Institutional Diversity, Inclusion and Equity at the Baylor College of Medicine announced the appointment of Dr. Rayne H. Rouce, assistant professor in the department of pediatrics, section of Hematology and Oncology as associate director of community engagement.

Joseph, Hegde receive Immunotherapy Scholar Award from the Faris Foundation (April 10, 2019)
Dr. Sujith Joseph, Senior Staff Scientist, and Dr. Meenakshi Hegde, Assistant Professor, both members of the Department of Pediatrics, Section of Hematology-Oncology, Baylor College of Medicine and Texas Children’s Cancer Center have been awarded the Faris D. Virani Immunotherapy Scholar Award of $300,000 over two years, by The Faris Foundation.

Several Texas Children's clinicians receive Women of Excellence Award (April 8, 2019)
More than a dozen Texas Children’s clinicians, who are also in academic roles at Baylor College of Medicine, were recently honored with the college’s Women of Excellence Award.

A one-of-a-kind high-tech maternal ICU opened at Texas Children's Hospital (Apr 1, 2019)
Located in the labor and delivery unit of Texas Children’s Pavilion for Women, the new four-bed maternal intensive care unit opened in January. Dedicated to obstetric (OB) intensive care, the unit offers a specialized, private space for high-risk expectant and postpartum mothers.

Medically Speaking: Urinary Tract Infections (Mar 26, 2019)
Urologist, Dr. Ming-Hsien Wang discusses best practices to treat pediatric urinary tract infections (UTIs) in the latest episode of this video series and shares findings from a recent double-blind study on the use of prophylactic antibiotics to treat UTIs.

Bariatric surgery program expands to The Woodlands, first surgeries performed (Mar 20, 2019)
Just months after successfully expanding our spine surgery program outside the medical center, the Department of Surgery and Texas Children’s Hospital The Woodlands are celebrating another important milestone – the expansion of our bariatric surgery services.

Texas Children’s again largest U.S. pediatric transplant program (Mar 20, 2019)
Transplant Services at Texas Children’s once again led the way as the nations’ largest pediatric transplant program, performing a remarkable 107 solid organ transplants in 2018. That figure includes the highest volume of pediatric liver, lung and kidney transplants in the United States.

Top four device startup companies named at Impact Pediatric Health Pitch Competition at SXSW (Mar 19, 2019)
On March 9, twelve finalist startup companies vied for awards and valuable grant funding at the fifth annual Impact Pediatric Health, a one-of-a-kind pitch competition held at SXSW that showcases the best in pediatric health care innovations. Out of 50 national and international startup applicants, the judges selected four companies to receive $25,000 grants in the Medical Devices category, provided by Southwest National Pediatric Device Consortium (SWPDC).

Autism study focuses on underrepresented communities (Mar 19, 2019)
A new grant to Baylor College of Medicine will build on the ongoing SPARK for Autism research study by focusing on fighting disparities and lack of diversity in autism research. In Texas, the SPARK study is led by Dr. Robin Kochel, associate professor of pediatrics – psychology at Baylor College of Medicine and associate director for research at the Texas Children’s Hospital Autism Center.

Dr. Hsiao-Tuan Chao named McNair Scholar (Mar 14, 2019)
Dr. Hsiao-Tuan Chao, assistant professor of pediatrics and molecular and human genetics, faculty at the Jan and Dan Duncan Neurological Research Institute, and associate program director of the basic neuroscience pathway in pediatric neurology at Baylor College of Medicine, has been named the newest McNair Scholar at Baylor.

Dr. Sarah Injac receives Emerging Research grant from Pediatric Cancer Research Foundation (Mar 7, 2019)
Dr. Sarah Injac, is a welcome addition to the Emerging Research Grant recipients. Her work at Baylor College of Medicine and Texas Children's Hospital offers hope to children with medulloblastoma, the most common malignant brain tumor of childhood with approximately 500 cases diagnosed in the United States each year.

SHANK3 : the good, the bad and the hopeful (Mar 7, 2019)
Some neuropsychiatric conditions may boil down to how well brain cells communicate with each other. This can be affected by a number of factors, including having too much or too little of proteins that function at the synapse – the point of communication between two brain cells. SHANK3 is one of these proteins. In their labs, Dr. Huda Zoghbi, Dr. Jimmy L. Holder Jr. and their colleagues have been extensively studying SHANK3, and they and other labs have discovered what is good, bad and hopeful about this protein.

Human stool can be used to treat a serious infection in children (Mar 5, 2019)
Human feces from healthy donors (termed ‘fecal microbiota transplantation’) can be used to treat severe diarrhea in children caused by recurrent Clostridioides or Clostridum difficile (C. difficile) infections that are unresponsive to standard antibiotic treatments, suggests a recent position statement issued by the leading professional bodies of pediatric gastroenterologists in the U.S. and Europe.

Shortages of pediatric cancer medicines threaten outcomes (Mar 4, 2019)
Shortages of essential chemotherapy drugs for children undergoing cancer treatment has been an increasingly frequent obstacle for patients and hospitals across the country. In a new position paper in JAMA Pediatrics, Baylor College of Medicine’s Drs. Stacey Berg and Brooke Bernhardt address this issue and call for the development of an essential medicines list for this group of patients to help ensure reliable access and forecast future shortages.

Texas Children's Hospital and Baylor College of Medicine researchers receive more than $9 million in grants from Cancer Prevention & Research Institute of Texas (Feb 27, 2019)
The Cancer Prevention and Research Institute of Texas (CPRIT) has awarded physicians and scientists from Baylor College of Medicine and Texas Children’s Hospital a combined $9,266,605 for seven projects in breast cancer, childhood cancers, T Cell therapy and lung cancer prevention.

Helen Heslop receives Lifetime Achievement Award (Feb 22, 2019)
Dr. Helen Heslop, director of the Center for Cell and Gene Therapy, received the Lifetime Achievement Award from the American Society for Blood and Marrow Transplantation at the Transplantation and Cellular Therapy Meetings held last week in Houston. Her key contributions to the field research on adoptive T cell immunotherapy to improve hematopoietic stem cell transplantation and cancer therapy.

Discovery improves understanding of Lou Gehrig's disease (Feb 26, 2019)
A collaborative study led by researchers at Baylor College of Medicine and the Jan and Dan Duncan Neurological Research Institute of Texas Children’s Hospital improves our understanding of how amyotrophic lateral sclerosis (ALS), also called Lou Gehrig’s disease, develops.

Dr. Donald William Parsons for being named the deputy director of Cancer and Hematology Centers (Feb 20, 2019)
Texas Children’s Cancer and Hematology Centers Director Dr. Susan Blaney recently appointed Dr. Donald Williams “Will” Parsons to the role of deputy director of the Cancer and Hematology Centers.

Researchers find a new way to automate the diagnosis of elbow fractures in children (Feb 18, 2019)
A recent study conducted at Texas Children’s Hospital describes a new, automated method to efficiently and accurately diagnose elbow injuries and abnormalities in children. Published in “Radiology: Artificial Intelligence,” this is the first study to demonstrate the efficacy of using artificial intelligence methods to analyze radiographic images of pediatric patients.

Two independent mechanisms are involved in tuberous sclerosis (Feb 7, 2019)
The current idea about how tuberous sclerosis occurs places mTORC1, a protein complex that regulates cell metabolism, as the major driving force behind the disease. But according to a new study published in the Proceedings of the National Academy of Sciences U.S.A. by researchers at Baylor College of Medicine and Texas Children’s Hospital, the development of this rare condition also involves a second mechanism that is independent of mTORC1. The findings can potentially lead to new treatments that might benefit patients who partially respond to current therapies focused on mTORC1.

A new treatment regimen for adolescents with chronic Hepatitis C virus infection identified (Jan 30, 2019)
A study led by Dr. Daniel Leung, Associate Professor and Director of the Viral Hepatitis Programat Texas Children’s Hospital and Baylor College of Medicine reports a new treatment for adolescents affected by chronic Hepatitis C infections (HCV). This study was a part of an ongoing, three-part clinical trial called ZIRCON, a multi-center international effort to find safe and effective therapies to treat HCV in children and was published in Hepatology Communications.

Certified PA earns national recognition in pediatrics (Jan 29, 2019)
Susan Kirk, PA-C, who works at Texas Children’s Hematology Center, has been nationally recognized for earning a specialty credential called a Certificate of Added Qualifications (CAQ) from the National Commission on Certification of Physician Assistants (NCCPA).

ACOG recognizes Drs. Espinoza and Vidaeff for their work on pregnancy complications (Jan 29, 2019)
Drs. Jimmy Espinoza and Alex Vidaeff were recently honored by the American College of Obstetricians and Gynecologist (ACOG) for preparing new guidelines for the management and prevention of complications of pregnancy including preeclampsia and gestational hypertension as well as chronic hypertension during pregnancy.

Dr. Julie Kaplow named Chief of Psychology (Jan 29, 2019)
Director of Texas Children’s Trauma and Grief Center Dr. Julie Kaplow was recently named Chief of Psychology.

Dr. Kjersti Aagaard awarded mentor award from Nature (Jan 22, 2019)
Dr. Kjersti Aagaard, an expert in maternal-fetal medicine at Baylor College of Medicine and Texas Children’s Hospital, has been honored with a 2018 Nature Award for Mentoring in Science.

Poplack and Weiner honored by cancer foundation (Jan 10, 2019)
Dr. David Poplack, the Elise C. Young Chair in Pediatric Oncology at Baylor, longtime director of the Texas Children's Cancer and Hematology Centers and now associate director, and director of the Global Hope program, received the Angel Award in recognition of lifetime achievement at the Children's Brain Tumor Foundation's Dream and Promise Awards Benefit in Houston last month. In addition, Dr. Howard Weiner, professor of neurosurgery at Baylor and chief of neurosurgery at Texas Children's Hospital, was the organization's medical honoree for his commitment to patient care.

Dr. Rajkumar Venkatramani named director of Solid Tumor Program (Jan 9, 2019)
On January 1, 2019, Dr. Rajkumar Venkatramani assumed the role of director of the Solid Tumor Program at Texas Children’s Cancer Center. Venkatramani, associate professor of pediatrics at Baylor College of Medicine, is a board-certified pediatric hematologist-oncologist who is internationally recognized for his expertise in the care of children with pediatric solid tumors.

The Sturge-Weber Foundation announces Thuy L. Phung, as new Director of Pathology Strategies (DPS) (Dec 19, 2018)
The Sturge-Weber Foundation (SWF) announces the addition of Thuy L. Phung, MD, PhD as Director of Pathology Strategies (DPS). The Sturge Weber Foundation (SWF) will lead a national effort across 25 clinical care centers in the US to establish a tissue biorepository for research in vascular anomalies. Texas Children's Hospital is one of the SWF clinical centers.

Phung named to dermatopathology society (Dec 14, 2018)

Dr. Thuy Phung, associate professor and associate director of global pathology in the Department of Pathology & Immunology and member of the Texas Children's Hospital Vascular Anomalies Center, recently was elected secretary-general of the Asian Society of Dermatopathology. In this role, she will work with the society to enhance the quality of the practice of skin pathology and promote the development and advancement of dermatopathology as a clinical specialty in Asia.

New insights into Zika's microcephaly link, similarity to dengue (Dec 13, 2018)
New insights into how dengue and Zika viruses cause disease reveal shared and virus-specific mechanisms. An international, multi-institutional team led by researchers of the University of California, San Francisco and Baylor College of Medicine report in the journal Cell that these viruses counteract a human and mosquito immune defense mechanism and hijack specific host proteins for virus replication. They also discovered that Zika virus causes microcephaly in fruit flies by disrupting the function of ANKLE2, a protein involved in brain development both in flies and humans. These findings open new avenues to design therapeutic strategies to combat these widespread and severe infectious diseases.

Dr. Huda Zoghbi elected as a fellow of the National Academy of Inventors (Dec 13, 2018)
Dr. Huda Zoghbi has been elected as a fellow of the National Academy of Inventors this week. Drs. Mary K. Estes, Bert O’Malley and Huda Zoghbi of Baylor College of Medicine were three of the 148 renowned academic inventors who received the distinction this week.

Understanding formation of new neurons in adult brains (Dec 10, 2018)
A team of researchers at Baylor College of Medicine, the Texas Heart Institute and Texas Children’s Hospital has developed a powerful new approach to understand the formation of new neurons in the mammalian adult brain. Published in the journal Cell Reports, this work opens exciting new pathways that can be further developed to repair malfunctioning brain circuits.

Loss of SYNGAP1 function results in abnormalities in sensory processing (Nov 19, 2018)
In addition to cognitive defects, neurodevelopmental disorders are often associated with deficits in sensory perception and processing. However, very little is known about the biological mechanisms that underlie these sensory impairments.In a study published in Nature Neuroscience, one of the co-authors, Dr. Jimmy L. Holder Jr., neurologist and director of the new SYNGAP1 Center of Excellence at Texas Children’s Hospital, investigator at the Jan and Dan Duncan Neurological Research Institute at Texas Children’s and assistant professor at Baylor College of Medicine, analyzed retrospective clinical data of SYNGAP1 patients from a registry maintained by Bridge the Gap – SYNGAP1 Education and Research Foundation.

Sudden death of a loved one is one of the common causes of reduced academic performance among youth (Nov 14, 2018)
A recent study led by Dr. Julie Kaplow, director of Trauma and Grief Center at Texas Children’s Hospital/Baylor College of Medicine finds sudden death of a loved one (e.g. parents, grandparents, close friend or relatives) is a common traumatic event experienced by teenagers and can have a profound negative impact on their academic performance and functioning.

Sheehan helps design trial network for sickle cell disease (Nov 12, 2018)
Dr. Vivien Sheehan, assistant professor of pediatrics - hematology/oncology at Baylor and Texas Children's Cancer and Hematology Centers, has assisted in the design of the American Society of Hematology Sickle Cell Disease Clinical Trial Network to advance development of new and more effective therapeutics for individuals with sickle cell disease.

Dr. Sharon Plon's team develop a novel computational tool to annotate microRNA (Nov 12, 2018)
A graduate student in Dr. Plon's lab, Ninad Oak has developed a novel computational tool called ADmiRE, which stands for Annotative Database for miRNA Elements. ADmiRE extensively annotates human microRNA variants to determine which ones are likely to contribute to or cause disease.

Evenings with Genetics offers update on rare Angelman Syndrome (Nov 7, 2018)
The next installment of Evenings with Genetics on Tuesday, Nov. 13, will focus on Angelman Syndrome, with expert speakers providing an update on treatment and research efforts for the rare neurogenetic disease. Dr. Carlos Bacino, professor of molecular and human genetics at Baylor and part of the newly-formed Angelman Syndrome Clinic at Texas Children’s Hospital, and Dr. Arthur Beaudet, the Henry and Emma Meyer Chair and Professor in Molecular and Human Genetics at Baylor, will lead the discussion. They will be joined by a special guest speaker, Dr. Deborah Sukin, a parent advocate of a child with Angelman Syndrome.

Research reveals defective transport of lysosomal enzymes causes a type of Batten disease (Nov 7, 2018)
Neuronal Ceroid Lipofuscinoses (NCLs a.k.a. Batten disease) are a group of fatal neurodegenerative disorders that result from defective lysosomal function due to mutations in different CLN genes. A surprising discovery from the laboratory of Dr. Marco Sardiello, assistant professor at Baylor College of Medicine and investigator at the Jan and Dan Duncan Neurological Research Institute at Texas Children’s Hospital shows defective lysosomal biogenesis as the underlying cause of Neuronal Ceroid Lipofuscinoses 8 (NCL8).

Researchers find a potential new approach to control cancer growth (Oct 19, 2018)
A team of researchers at Baylor College of Medicine and Texas Children’s Hospital has revealed a connection between mTORC1 and Src, two proteins known to be hyperactive in cancer. The study, published in the journal Nature Communications, shows that Src is necessary and sufficient to activate mTORC1 and offers the possibility to develop novel approaches to control cancer growth.

Hispanic children with acute lymphoblastic leukemia at higher risk for neurotoxicity (Oct 12, 2018)
Michael E. Scheurer, PhD, MPH, associate professor of pediatrics at Baylor College of Medicine and director of the Epidemiology Center at Texas Children’s Cancer Center, and colleagues analyzed medical records of 280 pediatric patients with newly diagnosed acute lymphoblastic leukemia (ALL).Hispanic children with ALL appeared more likely than non-Hispanic patients to develop neurotoxicity during methotrexate chemotherapy, according to study results.

Dr. Gaber earns mentorship award (Oct 12, 2018)
Dr. M. Waleed Gaber, associate professor of pediatrics (hematology/oncology) at Baylor College of Medicine and at TexasChildrens's Cancer and Hematology Centers, received the Scholar-in-Training Excellence in Mentorship Award from the Radiation Research Society for providing exceptional professional and personal mentoring.

Dr. Hari Krishna Yalamanchili receives the 2018 NRI Zoghbi Scholar Award (Oct 11, 2018)
Dr. Hari Krishna Yalamanchili, Ph.D., postdoctoral associate in the laboratory of Dr. Zhandong Liu, at Baylor College of Medicine, received the 2018 NRI Zoghbi Scholar Award, a special fund established by Dr. Huda Zoghbi, director of the Jan and Dan Duncan Neurological Research Institute (NRI) at Texas Children's Hospital, to honor the many generations of trainees who have worked in her laboratory and to foster the next generation of successful scientists.

Undiagnosed Diseases Network finds 31 new syndromes (Oct 11, 2018)
Identifying genes responsible for rare or unknown disorders using traditional approaches can be extremely time-consuming and typically takes several years or decades. However, the Undiagnosed Diseases Network (UDN), a consortium of researchers including Baylor College of Medicine, Texas Children’s Hospital, Stanford University and other institutions, has found 31 new syndromes and has been able to make diagnoses for 132 patients within two years of the group’s inception.

New high-dose radiation therapy available for pediatric liver cancer patients (Oct 5, 2018)
Pediatric liver cancer affects approximately 200 children each year in the United States. Treatment options are limited if the tumor cannot be completely removed with surgery. Although chemotherapy can be used, it often has limited efficacy and is associated with significant short and long term side effects. Thus, there is an urgent need to find therapeutic strategies to help children with liver tumors. A team of physician-scientists at Baylor College of Medicine and Texas Children’s Hospital are now using a new treatment option for pediatric patients with liver tumors, called Transarterial Radioembolization (TARE).

Research reveals a novel link between congenital cataracts and dynamin-binding protein (Oct 4, 2018)
Cataract, a condition in which the eye’s natural lens get clouded, is the most common cause of vision loss in older people and can be corrected by routine surgery. But congenital cataract, which occurs in infants and children, is particularly serious since it can inhibit visual development leading to permanent vision loss or impairment, which cannot be entirely reversed with cataract surgery. A new study has now found compelling evidence that links dynamin-binding protein (DNMBP) to congenital bilateral cataract and severe vision loss.

SYNGAP1 Center of Excellence at Texas Children’s Hospital receives research grant (Oct 3, 2018)
Jimmy Holder Jr., M.D./Ph.D., a pediatric neurologist and scientist at the Jan and Dan Duncan Neurological Research Institute at Texas Children’s Hospital and assistant professor at Baylor College of Medicine, was awarded a $25,000 research grant from the Bridge the Gap – SYNGAP Education and Research Foundation.

Dr. Hsiao-Tuan Chao awarded NIH Director's High-Risk, High-Reward grant (October 2, 2018)
Dr. Hsiao-Tuan Chao, instructor of pediatrics at Baylor College of Medicine and a child neurologist and postdoctoral researcher at the Jan and Dan Duncan Neurological Research Institute (NRI) at Texas Children’s Hospital, has been awarded the National Institutes of Health Director’s Early Independence Award from the High-Risk, High-Reward Research program

Texas Children's hematologist, Dr. Vivien Sheehan, develops treatment for Sickle Cell disease (September 27, 2018)
Sickle cell disease affects more than 100,000 Americans and millions more worldwide. This red blood cell disorder can be treated, but a widely available cure has yet to be found. Texas Children’s hematologist and Assistant Professor of Pediatrics Dr. Vivien Sheehan has recently developed a new sickle cell treatment based on her laboratory research. Sheehan has found that metformin, a commonly used medication for diabetes, has the ability to induce fetal hemoglobin in developing red blood cells.

Drs. Philip Lupo and Karen Rabin receive NIH grant to study the link between Down syndrome and Acute Lymphoblastic Leukemia (ALL) (September 25, 2018)
Children with Down syndrome are about 20 times more likely to develop acute lymphoblastic leukemia (ALL) than other children. To better understand this predisposition for cancer, a team of researchers from Baylor College of Medicine, Emory University and St. Jude Children’s Research Hospital have been awarded a grant from theGabriella Miller Kids First Pediatric Research Program (Kids First) to sequence the genomes of more than 2,000 individuals with Down syndrome.

In the phase II, UDN expands to add five new clinical sites, a new metabolomics core and new model organism core (September 24, 2018)
NIH has given many new grants to academic medical centers across the nation to improve and accelerate the diagnosis of rare and undiagnosed The new awards are part of the second phase of the National Institutes of Health’s Undiagnosed Diseases Network (UDN).

CPRIT awards $6 million to find new biomarkers and therapies for childhood liver cancers (September 19, 2018)
Texas Children’s Hospital and Baylor College of Medicine researchers received a $6 million research grant from the Cancer Prevention and Research Institute of Texas to find new predictive biomarkers and therapies for high-risk pediatric liver cancers.

Texas Children’s named Clinical Research Center of Excellence for CDKL5 disorders (September 10, 2018)
Texas Children’s Hospital has been recognized as a Clinical Research Center of Excellence (COE) for CDKL5 Deficiency Disorder and is one of only five pediatric institutions in the country to receive this honor.

Long genes are not preferentially altered in Rett and MeCP2 duplication syndromes (September 7, 2018)
Many recent large-scale transcriptome studies have shown that long genes (>100 kb) are preferentially dysregulated in neurodevelopmental disorders. A recent study from the laboratory of Dr. Zhandong Liu, associate professor at Baylor College of Medicine and investigator at the Jan and Dan Duncan Neurological Research Institute at Texas Children’s Hospital shows that expression of long genes is not preferentially altered in MeCP2 syndromes.

New histiocytotic disorder insights may improve therapies (August 30, 2018)
In a review published in New England Journal of Medicine, physician-scientists from Baylor College of Medicine, Texas Children’s Cancer and Hematology Centers and the Icahn School of Medicine review the history of LCH and the recent developments that will help propel the treatment for patients with LCH into the realm of personalized medicine.

Two neuronal lineages in the hindbrain act together to ‘jumpstart’ rhythmic breathing at birth (August 28, 2018)
A study published in eLife from the Zoghbi lab has found two neuronal lineages in the hindbrain coordinate and relay the changes in oxygen and carbondioxide levels to the rhythmogenic neurons in the central respiratory circuit to establish and maintain optimal breathing rhythms, which are especially critical for the survival of a newborn.

Southwest Pediatric Device Consortium at Texas Children’s Hospital and Baylor College of Medicine receives $6.75M FDA P50 grant to expand development of medical devices for children (August 24, 2018)
The Southwest National Pediatric Device Consortium (SWPDC), anchored by Texas Children’s Hospital and Baylor College of Medicine, recently received a prestigious P50 grant from the U.S. Food & Drug Administration (FDA). The five-year, $6.75M grant will begin on Sept. 1

Xue lab develops a better optogenetic tool to study neuronal function (August 20, 2018)
The scientists in the laboratory of Dr. Mingshan Xue, assistant professor at Baylor College of Medicine and an investigator at the Cain Foundation Laboratories and the Jan and Dan Duncan Neurological Research Institute atTexas Children's Hospital, have modified an existing light-sensitive tool to produce fewer undesired effects, making it a better tool to study neuronal function.

Texas Children’s leads effort to get lifesaving drug approved by FDA (August 20, 2018)
Thanks to the more than 10 years of dedication and hard work of neonatologists and neonatal nutrition experts at Texas Children’s, Boston Children’s and the University of California at Los Angeles (UCLA) hospitals, premature babies with liver disease now have better access to a potentially lifesaving drug.

Four pediatrics faculty receive the 2018 research mentorship awards (August 14, 2018)
The following Baylor College of Medicine/Texas Children’s Hospital faculty affiliated to the department of pediatrics were selected for the 2018 research mentorship awards for providing outstanding research guidance to medical, graduate students, and postdoctoral trainees - Andrea Cruz, MD MPH, associate professor of pediatrics (Emergency Medicine section), Richard Kellermayer, MD, associate professor of pediatrics (Gastroenterology section), Stacey Berg, MD, professor of pediatrics (Texas Children’s Cancer and Hematology centers) and Deborah Thompson, PhD, RD, professor of pediatrics (Children's Nutrition Research Center).

Srivaths named to blood disorders foundation (August 3, 2018)
Dr. Lakshmi Srivaths, professor in the department of pediatrics and co-director of Young Women's Bleeding Disorders Clinic, has been appointed as chair of the education/advocacy subcommittee for the national Foundation for Women and Girls with Blood Disorders. The chair will be in charge of the subcommittees organization and ongoing activities, which include creating education for clinic staff, referring providers, providers outside of primary institution and organized/informal education opportunities.

Fruit flies help UDN researchers uncover new neurological disorder in seven patients (July 27, 2018)
Spontaneously arising mutations in a single copy of the IRF2BPL gene are associated with a previously undiagnosed neurological disorder in seven unrelated individuals, reports a recent collaborative study. This project was initiated when the corresponding author of this study, Dr. Loren D.M. Pena, associate professor of pediatrics; and Dr. Vandana Shashi, professor of pediatrics, both at the Duke Clinical Site of the Undiagnosed Disease Network (UDN); connected Dr. Hugo Bellen, professor of molecular and human genetics and neuroscience at Baylor College of Medicine, an investigator at the Howard Hughes Medical Institute and the Jan and Dan Duncan Neurological Research Institute (NRI) at Texas Children’s Hospital to a patient who presented with a severe neurological condition without a diagnosis.

St. Baldrick's Foundation awards seven faculty with pediatric cancer research grants (July 20, 2018)
The St. Baldrick’s Foundation, a volunteer-powered charity dedicated to raising money for childhood cancer research, has announced its 2018 summer grant recipients, including seven from Baylor College of Medicine and Texas Children’s Cancer and Hematology Centers.

The Teratology Society honors Dr. Lupo (July 17, 2018)
Dr. Philip Lupo, PhD, MPH, received the 2018 F. Clarke Fraser New Investigator Award from the Teratology Society, recognizing an early career scientist who has established a successful independent research career.

Dr. Judith Margolin receives a special biennial Founder's award from the Lymphangiomatosis and Gorham's Disease Alliance (July 15, 2018)
Dr. Judith Margolin, Co-Director of the Pediatric Hematology-Oncology Vascular Anomalies Program at Texas Children's receives the 2017-2018 recipient of the special biennial Founder's award from the Lymphangiomatosis and Gorham's Disease Alliance for her extraordinary professionalism, critical care, treatment and support of patients and their families affected by rare lymphatic disorders.

Pediatric neurosurgeon, Dr. Sandi Lam, develops a novel endoscopic procedure to treat intractable seizures (July 16, 2018)
Dr. Sandi Lam, pediatric neurosurgeon at Texas Children's Neuroscience Center, performed a novel endoscopic hemispherotomy as a surgical treatment to treat intractable seizures that has significantly reduced seizure episodes.

Dr. Hsiao-Tuan Chao receives a Child Neurology Foundation grant to study pediatric epilepsy and intellectual disability (July 3, 2018)
Dr. Hsiao-Tuan Chao, MD/PhD, clinical child neurologist and associate director of the child neurology residency program at Texas Children’s Hospital was awarded the Child Neurology Foundation’s 2018 Pediatric Epilepsy Research Foundation Grant.

Mutations in gene TRAF7 associated with multisystem disorder (June 28, 2018)
A group of seven patients presenting with a similar disorder of unknown origin now know of a possible genetic root of their condition.

Rice University, Texas Children's Hospital and Baylor College of Medicine win major NIH grant to study discrete subaortic stenosis (June 27, 2018)
Scientists at Rice University, Texas Children's Hospital and Baylor College of Medicine have won a prestigious National Institutes of Health grant to study the dynamic processes and cellular players linked to discrete subaortic stenosis (DSS), a congenital heart disease.

A new integrative approach finds viable therapeutic targets for Huntington's disease (June 21, 2018)
When a complex system goes awry and multiple parts are affected, how do you figure out which changes are causative and which are merely correlative? A team of scientists led by Dr. Juan Botas, professor at Baylor College of Medicine and investigator at the Jan and Dan Duncan Neurological Research Institute have developed a way to sort through the thousands of genetic alterations that accumulate in human brains affected by neurological disease.

A simple sugar delays neurodegeneration in lysosomal storage disorders (June 20, 2018)
A new study from the laboratory of Dr. Marco Sardiello, assistant professor in Baylor College of Medicine and investigator at the Jan and Dan Duncan Neurological Research Institute at Texas Children’s Hospital, has found oral administration of trehalose, a simple sugar, can alleviate the neurological symptoms associated with the deficiency of a lysosomal enzyme.

Ceramides may play a role in early-onset Parkinson’s disease (June 18, 2018)
The researchers in the laboratory of Dr. Hugo Bellen, professor at Baylor College of Medicine and an investigator at the Howard Hughes Medical Institute and at the Jan and Dan Duncan Neurological Research Institute at Texas Children’s Hospital, have found ceramides, a family of lipid molecules found within cell membranes, play an important role in an early-onset form of Parkinson’s disease. The study was published in the journal Cell Metabolism.

Dr. Zhandong Liu receives distinguished service award (June 18, 2018)
Dr. Zhandong Liu, assistant professor at Baylor College of Medicine and researcher at the Jan and Dan Duncan Neurological Research Institute and member of the Dan L Duncan Comprehensive Cancer Center, has received a distinguished service award from the International Association for Intelligent Biology and Medicine (IAIBM).

Dr. Mingshan Xue awarded 2018 McKnight Scholar Award (June 14, 2018)
Dr. Mingshan Xue, assistant professor of neuroscience at Baylor College of Medicine and a researcher with the Jan and Dan Duncan Neurological Research Institute (NRI) at Texas Children’s Hospital, has been awarded the prestigious 2018 McKnight Scholar Award.

Dr. Hsiao-Tuan Chao receives the prestigious Burroughs Wellcome Fund Career Award (June 11, 2018)
Dr. Hsiao-Tuan Chao, a child neurologist and postdoctoral researcher at the Jan and Dan Duncan Neurological Research Institute (NRI) at Texas Children’s Hospital, was awarded the highly-competitive 2018 Burroughs Wellcome Fund Career Award for Medical Scientists.

Tau aggregates activate “jumping genes” in Alzheimer’s disease (June 8, 2018)
A recent study published in Cell Reports has found a novel mechanism by which Tau protein aggregates might contribute to neurodegeneration. In this collaborative study, researchers found numerous transposons (a.k.a jumping genes) were activated in the brains of deceased subjects affected by Alzheimer’s disease (AD). Further, using fruit fly models of AD they showed Tau protein is sufficient to activate transposons contributing to genomic instability that likely, triggers neurodegeneration.

Fruit fly study links TBX2 to a new Mendelian disorder (May 23, 2018)
An interdisciplinary team led by Dr. Shinya Yamamoto has found a link between TBX2, a member of the T-box family of transcription factors, to a novel disorder that mainly affects the cardiac, skeletal, immune and endocrine systems. The study appears in the journal Human Molecular Genetics.

Texas Children's research trainees receive national awards (May 17, 2018)
The Society for Pediatric Research (SPR) recently gave two Texas Children’s trainees national research awards for their outstanding research on kidney disease at this year’s annual Pediatric Academic Societies’ SPR meeting.

Texas Children's surgeons use state-of-the-art real-time 3-D visualization technology to streamline surgeries (May 9, 2018)
In March, pediatric surgical specialists at Texas Children’s Hospital have used the Orbeye for pediatric plastic, urology and congenital heart surgeries. Orbeye is a new freestanding videomicroscope that displays high-resolution 3-D imaging of surgical procedures on an expansive 55-inch 4K monitor with stunning clarity. It’s like bringing the IMAX movie theater experience into the operating theater.

Fruit fly study identifies a new link to aortic aneurysms. (April 25, 2018)
An interdisciplinary team of researchers has identified a new gene linked to human aortic aneurysms. By combining comprehensive genetic studies in the fruit fly, dataset searches and analysis of diseased human aortic tissue, the researchers shed light on mechanisms not previously known to be associated with this devastating disease. The study appears in the journal Developmental Cell.

FDA approves everolimus for tuberous sclerosis-associated seizures, a treatment first discovered at Texas Children’s Hospital (April 20, 2018)
On April 10, 2018, the Food and Drug Administration (FDA) approved everolimus tablets for oral suspension (brand name: Afinitor Dispersz, Novartis Pharmaceuticals Corporation) as an adjunctive therapy to treat partial-onset refractory seizures in adults and children over 2 years of age with Tuberous Sclerosis Complex (TSC)

Dr. Peter Hotez and Dr. Huda Zoghbi elected as members of the American Academy of Arts and Sciences, one of nation's most prestigious honorary titles (April 18, 2018)
Dr. Peter J. Hotez, professor and dean of the National School of Tropical Medicine at Baylor College of Medicine, and Dr. Huda Y. Zoghbi, professor and Howard Hughes Medical Institute Investigator at Baylor, both also with Texas Children’s Hospital, have been elected as the newest members of the American Academy of Arts and Sciences, one of the nation’s most prestigious honorary titles.

Dasatinib is a safe and effective treatment for pediatric Chronic Myeloid Leukemia in chronic phase (CML-CP) (April 6, 2018)
In the largest prospective phase II trial till date of its kind, researchers report dasatinib to be safe and effective for children with CML-CP.

Dr. Stephen Mack receives the First-Time, Tenure-Track Faculty Members Award from CPRIT (April 5, 2018)
The Cancer Prevention and Research Institute of Texas (CPRIT) has conferred the First-time, Tenure-Track Faculty Members Award on Dr. Stephen Mack to recruit him to Baylor College of Medicine from Cleveland Clinic.

The Bellen lab generates a large "CRIMIC" library, a versatile tool to accelerate biomedical discovery (March 30, 2018)
An exciting study published in eLife reports the generation of a large versatile library of fruit flies that can be used to perform efficient and elegant in vivo gene-specific manipulations.

Dr. David Nelson elected as the President of the American Society of Human Genetics (March 21, 2018)
David L. Nelson, Ph.D.,a Cullen Foundation Professor of Molecular and Human Genetics at the Baylor College of Medicine (NCM), Associate Director of the BCM Intellectual and Developmental Disabilities Research Center, and Director of the BCM Integrative Molecular and Biomedical Sciences Graduate Program and an investigator at the Jan and Dan Duncan Neurological Research Institute, has been elected as the 2018 President of the American Society of Human Genetics (ASHG).

The Sardiello lab develops a new web tool to speed up biomedical discovery (March 12, 2018)
Researchers in the laboratory of Dr. Marco Sardiello, assistant professor at Baylor College of Medicine and investigator in the Jan and Dan Duncan Neurological Research Institute at Texas Children’s Hospital, have developed a new web tool called Aminode that will help researchers analyze and predict proteins’ evolutionarily conserved regions (ECRs).

Dystonia Medical Research Foundation interviews Dr. Roy Sillitoe about his research in dystonia (March 9, 2018)
Dystonia Medical Research Foundation interviews Dr. Roy Sillitoe to learn how recent discoveries from his lab on a brain region called cerebellum can help dystonia patients. Watch the video

Research reveals mechanism that drives ataxia type 1 (March 9, 2018)
A group of researchers at Baylor College of Medicine, Texas Children’s Hospital and the University of Minnesota reports in the journal Neuron that polyQ-ATAXIN1 and a partner molecule called capicua form a complex that is essential for neurodegeneration.

Molecular Medicine awards Ross Prize to Dr. Huda Zoghbi (Mar 8, 2018)
Dr. Huda Zoghbi, director of the Jan and Dan Duncan Neurological Research Institute at Texas Children’s Hospital, Howard Hughes Medical Institute investigator and professor at Baylor College of Medicine, has been awarded the sixth annual Ross Prize in Molecular Medicine by The Feinstein Institute for Medical Research at Northwell Health.

National Cancer Institute awards Dr. Susan Blaney (Mar 6, 2018)
Dr. Susan Blaney, Professor and Executive Vice Chair for the Department of Pediatrics at Baylor College of Medicine and the Deputy Director of Texas Children’s Cancer Center, received a Director’s Service Award from the National Cancer Institute for her outstanding and dedicated service to the Institute and the Clinical Trials and Translational Research Advisory Committee from 2015 to 2017.

Research reveals path to ependymoma chemotherapy (Feb 28, 2018)
Ependymoma is a type of brain tumor that is resistant to chemotherapy. Dr. Stephen Mack, assistant professor of pediatrics – oncology and new faculty member at Baylor College of Medicine and Texas Children’s Hospital, and colleagues have developed a framework for discovering targets in ependymomas, and other cancer that lack known genetic drivers, thereby also providing insights into treatment strategies.

Texas Children's raises awareness for rare diseases (Feb 28, 2018)
This past Saturday, Feb. 24, hundreds of patients, family members, community advocates, health care providers and more joined Texas Children’s Hospital and Baylor College of Medicine to gather at The Health Museum in Houston, raising awareness for rare diseases in observance of the upcoming Rare Disease Day.

Automated detection of HFOs identify source of seizures in epilepsy patients (Feb 26, 2018)
Drs. Daniel Curry and Michael Quach from Texas Children’s Division of Neurology and Neurosurgery co-authored an insightful epilepsy study that was recently published in Brain, a Journal of Neurology.

Texas Children’s celebrates Blue Bird Circle’s 95th birthday and partnership (Feb 24, 2018)
Since 1998, the Blue Bird Circle has supported the Clinic for Pediatric Neurology at Texas Children’s with millions of dollars in grants, as well as thousands of volunteer hours. On February 6, Macey and 100 Blue Bird members including many of Texas Children’s employees, staff and leaders, attended a special luncheon at the Pavilion for Women to celebrate the Blue Bird Circle’s 95th birthday.

Changes in Pumilio1 levels linked to distinct neurological disorders (Feb 22, 2018)
In a collaborative study published in Cell, mutations that alter the protein levels of PUMILIO1 (PUM1) can cause a variety of neurological symptoms ranging from mild adult-onset ataxia to childhood-onset ataxia, intellectual disability, developmental delay and seizures. This is the first study to uncover a role for PUM1 in human brain function and to implicate it in two novel neurological syndromes.

Dr. Peter Hotez appointed to U.S. - Israel Binational Science Foundation Board of Governors (Feb 12, 2018)
Dr. Peter Hotez, dean of the National School of Tropical Medicine at Baylor College of Medicine and Texas Children’s Hospital Endowed Chair in Tropical Pediatrics, has been appointed as a representative to the U.S.-Israel Binational Science Foundation Board of Governors. The announcement was made today by the U.S. Department of State.

One little boy's DNA brings the promise of a cure for Rett syndrome girls (Jan 30, 2018)
Dr. Huda Zoghbi's lab has found some tantalizing clues by studying the DNA of a little boy, Henry Engel, who has a mutation in MeCP2 gene. These findings could one day help researchers to develop treatment for girls suffering from Rett syndome. Learn more

Dr. Huda Zoghbi receives Lebanon's highest honor by Lebanese President General Michel Aoun (Jan 23, 2018)
Dr. Huda Zoghbi, director of the Jan and Dan Duncan Neurological Research Institute at Texas Children’s Hospital,Howard Hughes Medical Institute investigator, the Ralph D. Feigin Endowed Chair and professor at Baylor College of Medicine, was awarded Lebanon’s highest honor, the National Order of the Cedar, Knight grade, by Lebanese President General Michel Aoun.

Texas Children’s Hospital receives grant to continue developing a vaccine for Chagas disease (Jan 22, 2018)
Texas Children’s Hospital is excited to announce it received a $1.9 million grant from The Robert J. Kleberg Jr. and Helen C. Kleberg Foundation to support Texas Children’s Center for Vaccine Development (CVD), advancing the efforts to develop a therapeutic vaccine against Chagas disease.

Dr. Orange receives prestigious award for advancements in immunology research (Dec 14, 2017)
Dr. Jordan Orange, professor of pediatrics and chief of the section of Immunology, Allergy and Rheumatology at Baylor College of Medicine, and the director of the Center for Human Immunobiology at Texas Children’s, is the recipient of the 2018 Edith and Peter O’Donnell Award in Medicine from The Academy of Medicine, Engineering and Science of Texas (TAMEST).

Alex’s Lemonade Stand funds Phase 1 neuroblastoma trial (Dec 4, 2017)
Neuroblastoma is the most common solid tumor cancer in children and is challenging to treat regardless of existing therapies that are available. Dr. Andras Heczey, assistant professor of pediatrics - hematology and oncology, and Dr. Leonid Metelitsa, professor of pediatrics - oncology at Baylor College of Medicine, have received a $1.5 million grant from Alex’s Lemonade Stand Foundation to conduct a Phase 1, first-in-human clinical trial of a new form of immunotherapy to treat neuroblastoma using native and engineered properties of natural killer T-cells (NKTs).

Genetic mutations involved in childhood lysosomal storage disorders linked to increased risk of Parkinson's (Nov 17, 2017)
A new study by an international team of scientists led by Dr. Joshua Shulman, sheds new light on the genetic factors associated with Parkinson’s disease, a common neurodegenerative disorder.

Charif Souki funds neurological research through $10 million gift to the Jan and Dan Duncan Neurological Research Institute at Texas Children’s Hospital (Nov 15, 2017)
Texas Children’s Hospital is excited to announce a $10 million commitment from Charif Souki to the Jan and Dan Duncan Neurological Research Institute (NRI) to fund the Huda Y. Zoghbi, M.D. Director’s Endowment. In honor of this gift, the NRI atrium will be named in Charif Souki’s honor and will now be called the Charif Souki Atrium.

Texas Children’s Cancer Center to offer state-of-the-art I-131 MIBG therapy (Nov 7, 2017)
As one of only a select number of pediatric hospitals in the U.S., Texas Children’s Cancer Center will now offer I-131 MIBG, a targeted nuclear medicine therapy that combines metaiodobenzylguanidine (MIBG) with radioactive iodine (I-131) to treat patients with high-risk neuroblastoma. This is the first pediatric treatment location in Houston designed specifically for this therapy and one of only a handful in the southern U.S. For more information about MIBG therapy at Texas Children’s Cancer Center, visit the Neuroblastoma Program website.

Seizures cause memory and cognitive loss by blocking expression of a neuronal calcium sensor (Oct 30, 2017)
Dr. Jeannie Chin, assistant professor at Baylor College of Medicine has identified a novel mechanism that shows how recurrent seizures impair memory and cognition.

Dr. Hsiao-Tuan Chao named the 2017 STAT Wunderkind (Oct 16, 2017)
Dr. Hsiao-Tuan Chao, an instructor of pediatrics - neurology at Baylor College of Medicine and a postdoctoral researcher at the Jan and Dan Duncan Neurological Research Institute at Texas Children's Hospital, has been named a 2017 STAT Wunderkind.

Dr. Zoghbi receives The George W. Jacoby Lectureship (Oct 9, 2017)
The American Neurological Association has awarded The George W. Jacoby Lectureship to Dr. Huda Zoghbi, professor in the departments of pediatrics, molecular and human genetics, neurology, and neuroscience at Baylor and director of the Jan and Dan DuncanNeurological Research Insitute at Texas Children'sHospital.

Breakdown of brain cells’ metabolic collaboration linked to Alzheimer’s disease (Oct 1, 2017)
Researchers have discovered that impairing a critical partnership between brain cells can lead to neurodegeneration.

Novel regulatory DNA loops found in gliomas: a promising target for future therapies (Sept, 19.2017)
Researchers in the laboratory of Dr. Benjamin Deneen, investigator at the Jan and Dan Duncan Neurological Research Institute at Texas Children's Hospital and associate professor at Baylor College of Medicine, have identified mechanisms in which NFIA, a glial gene, is regulated during normal brain development and in brain tumors.

New study reveals how repeated early-life seizures alter microanatomy of neurons (Aug 24, 2017)
A recent study published in eNeuro from the laboratory of Dr. John Swann, director of the Gordon and Mary Cain Pediatric Neurology Research Foundation Laboratories at Texas Children’s Hospital shows how frequent seizures alter the microanatomy of neurons in the brain and that the activity of a specific enzyme may contribute to the cognitive and behavioral deficits observed in children with epileptic encephalopathies.

Next-generation probiotics may reduce the risk of gut bacterial infections (Aug 9, 2017)
C. difficile infections are the most common cause of diarrhea associated with the use of antibiotics and very difficult to eliminate. Texas Children's Drs. Jennifer Spinler and Tor Savidge in collaboration with co-author Dr. Robert A. Britton, professor of molecular virology and microbiology at Baylor and member of the Dan L Duncan Comprehensive Cancer Center have now found a way to selectively kill this bacteria using probiotics.

Ready-to-use cocktail of immune cells targeting specific viruses are safe and effective in transplant patients (Aug 8, 2017)
Transplant patients are at risk for developing potentially lethal viral infections, many of which are not easy to treat and carry significant side effects. Researchers at the Center for Cell and Gene Therapy at Baylor College of Medicine, Texas Children’s Hospital and Houston Methodist have developed an alternative treatment in which virus-specific cells protect patients against severe, drug-resistant viral infections

Dr. Jimmy Holder awarded by the Doris Duke Charitable Foundation (July 26, 2017)
Dr. Jimmy Holder, a clinician and investigator at the Jan and Dan Duncan Neurological Research Institute at Texas Children's Hospital and assistant professor at the Baylor College of Medicine, has been awarded the 2017 Clinical Scientist Development Award by the Doris Duke Charitable Foundation.

Lunatic Fringe plays a key role in renewing neurons (July 26, 2017)
In a study published in the journal Elife, Dr. Mirjana Maletic-Savatic and colleagues describe how Lunatic Fringe, a Notch ligand helps to maintain the optimal number of neurons in the brain.

Patient-inspired research links sugar metabolism to a rare genetic disorder (June 29, 2017)
A team of researchers at the Jan and Dan Duncan Neurological Research Institute at Texas Children's Hospital have found Zellweger syndrome or peroxisomal biogenesis disorder is linked to altered sugar metabolism and not fat metabolism, as believed earlier.

Dr. Benjamin Arenkiel receives the 2017 Michael E. DeBakey, M.D 'Excellence in Research' Award (June 6, 2017)
Dr. Benjamin Arenkiel, a researcher at the Jan and Dan Duncan Neurological Research Institute (NRI) at Texas Children’s has received a 2017 Michael E. DeBakey, M.D., Excellence in Research Award, a prestigious honor given annually to Baylor College of Medicine faculty who have made the most significant published scientific contribution to clinical or basic biomedical research during the past three years.

Dr. Jordan Orange receives E. Mead Johnson Award for Research in Pediatrics (May 30, 2017)

Dr. Jordan Orange, MD, PhD, Professor in the Department of Pediatrics at Baylor College of Medicine and Texas Children's Hospital, where he serves as the Director of the Center for Human Immunobiology and the Vice Chair for Research, received the prestigious E. Mead Johnson Award for Research in Pediatrics from the American Pediatric Society and the Society for Pediatric Research.

Bellen Lab researcher receives the Harold M. Weintraub Graduate Student Award (May 24, 2017)

Dr. Lucy Liu, graduate student in Dr. Hugo Bellen's lab received the prestigious award for her work that showed how lipid metabolism causes neurodegeneration.

Dr. Sundeep Keswani receives the March of Dimes award (May 17, 2017)

Dr. Sundeep Keswani, surgical director of Basic Research and pediatric surgeon at Texas Children’s Hospital and associate professor of surgery in the Division of Pediatric Surgery at Baylor College of Medicine, was recently awarded a $300,000 grant over a period of three years for his project “Targeting the Extracellular Matrix: an Innovative Strategy to Improve Pulmonary Hypertension in Congenital Diaphragmatic Hernia

Researchers at the Jan and Dan Duncan Neurological Research Institute create a new web tool to expediate gene discovery (May 15, 2017)
A group of researchers led by Drs. Hugo Bellen and Zhandong Liu have created a newopen-access, user-friendly web resource, MARRVEL (Model Organism Aggregated Resource for Rare Variant ExpLoration) that will help biomedical researchers speed up their pace of scientific discovery.

TCH/NRI physician-scientists receive prestigious research scholarships from the American Academy of Neurology (May 03, 2017)

Dr. Hsiao-Tuan Chao, MD PhD, medical resident of pediatric neurology and Dr. Laurie Robak, clinical instructor at the Baylor College of Medicine and researchers at the Jan and Dan Duncan Neurological Research Institute (NRI) at Texas Children's Hospital were selected to receive prestigious scholarship grants from the American Academy of Neurology to support studies on neurodevelopmental and neurodegenerative diseases

Research paves way for improved colorectal cancer test (April 19, 2017)
The type of bacteria in your gut may help diagnose colorectal cancer. Researchers at Baylor College of Medicine and other institutions have identified specific types of bacteria that seem to be abundant in individuals with colorectal cancer.

Texas Children's to offer brand new FDA-approved treatment for Spinal Muscular Atrophy (April 11, 2017)
Texas Children’s Hospital is among the first hospitals in the nation to offer the new FDA-approved treatment for Spinal Muscular Atrophy (SMA) to pediatric patients. Texas Children’s inaugurated this program in February 2017 for established and newly diagnosed SMA patients.

Mutations in an early embryonic development gene may cause female infertility (Mar 27, 2017)
A team of researchers headed by Dr. Ignatia Van den Veyver, professor and at Baylor College of Medicine, director of clinical prenatal genetics and investigator at the Jan and Dan Duncan Neurological Research Institute at Texas Children’s Hospital, have shown that mutations in an early embryonic development gene may provide some clue to unexplained female infertility and recurrent pregnancy loss.

Mutations in ABL1, a known leukemia gene, could cause a novel developmental disorder (Mar 20, 2017)
Researchers at the Neurological Research Institute at Texas Children’s Hospital and faculty at Baylor College of Medicine have identified a novel role for ABL1, a known tumorigenic gene, in a new developmental disorder.

Rats could a valuable addition to an autism researcher's toolkit (Mar 15, 2017)
Mice are the mainstay of autism research. However, some researchers, including Drs. Rodney Samaco and Surabi Veeraraghavan of the Jan and Dan Duncan Neurological Research Institute and Baylor College of Medicine, argue in a lay article that rats are not merely "larger mice".

Careful study of mouse models by Dr. Zoghbi's team has uncovered a new neurodevelopmental disorder in five patients (Mar 13, 2017)
A collaborative study spearheaded by Dr. Huda Zoghbi, professor at Baylor College of Medicine and director of Jan and Dan Duncan Neurological Research Institute at Texas Children’s Hospital, shows that loss of Ataxin1-Capicua (ATXN1-CIC) complex causes a previously unidentified neurodevelopmental disorder in mice and humans.

Texas Children's Hospital researchers identify five new candidate genes for Parkinson disease (Feb 20, 2017)
A large collaborative study led by Dr. Joshua Shulman, Assistant Professor at Baylor College of Medicine and investigator at the Jan and Dan Duncan Neurological Research Institute (NRI) at Texas Children’s Hospital and Dr. Peter Heutink, professor at the German Center for Neurodegenerative Diseases, has revealed five strong candidate genes for Parkinson’s disease.

A potential therapeutic target for juvenile Batten disease (Feb 6, 2017)
Researchers in the laboratory of Dr. Marco Sardiello, assistant professor at Baylor College of Medicine and an investigator at the Jan and Dan Duncan Neurological Research Institute (NRI) at Texas Children’s Hospital have identified a pharmacologically actionable target for juvenile Batten disease, a rare but fatal pediatric neurodegenerative disorder.

Maternal diet alters energy and behavior of her adult male mice offspring (Jan 19, 2017)
A team of researchers in the laboratory of Dr. Ignatia Van den Veyver, professor at the Baylor College of Medicine and investigator at the Jan and Dan Duncan Neurological Research Institute at Texas Children's Hospital found that adult male offspring of female mice that were fed chronic low-protein diet are sluggish, with increased body fat and anxiety-like behaviors even though they were on a normal diet since weaning.

Fighting sickle cell disease with Type 2 diabetes medication (Jan 10, 2017)
Sickle cell disease and the blood disorder beta thalassemia affect more than 180,000 Americans and millions more worldwide. Both diseases can be made milder or even cured by increasing fetal hemoglobin (HbF) levels, but current treatment to ramp up HbF is limited in its effectiveness.

Method of birth does not affect an infant's microbiome composition (Jan 23, 2017)
A new study published today by researchers at Baylor College of Medicine in Nature Medicine found that the microbiome of infants greatly expands between birth and 4 to 6 weeks of age.

Researchers find that inhibitory neural networks broaden with age and experience (Jan 9, 2017)
For the first time, in a study published in Nature Neuroscience, researchers in the laboratory of Dr. Benjamin Arenkiel, associate professor at Baylor College of Medicine and principal investigator at the Jan and Dan Duncan Neurological Research Institute (NRI) at Texas Children’s Hospital, have demonstrated how inhibitory circuits form and develop in live brains. To learn more watch this video

Dr. Huda Zoghbi wins the prestigious Breakthrough Prize in Life Sciences (Dec 4, 2017)
Huda Zoghbi never dreamed her research into the rarest of rare brain disorders might one day lay the groundwork for promising therapeutic candidates for Alzheimer's and autism. She just wanted to help the patients she first encountered as a pediatrician-in-training in the 1980s, girls who mysteriously lost the ability to walk, speak and use their hands.

Study reveals potential new strategy to prevent Alzheimer's disease (Oct 6, 2016)
Taking a pill that prevents the accumulation of toxic molecules in the brain might someday help prevent or delay Alzheimer’s disease, according to scientists at Baylor College of Medicine, Texas Children’s Hospital and Johns Hopkins University School of Medicine.

A small region of the brain plays a major role in controlling appetite (See the Wall Street Journal article, Oct 3, 2016).
A seminal study published in Nature from the laboratory of Dr. Benjamin Arenkiel, associate professor at Baylor College of Medicine and researcher at the Neurological Research Institute at Texas Children's Hospital, demonstrates that a subset of cholinergic neurons in the basal forebrain act as critical regulators to modulate food intake and control body weight.