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Research News
Examining Outcomes in Children with Down Syndrome and Acute Lymphoblastic Leukemia
A new report published in the Journal of Clinical Oncology examined outcomes for children with Down syndrome and acute lymphoblastic leukemia (ALL) who were enrolled in Children's Oncology Group clinical trials between 2009 and 2013. The study's lead author was Dr. Karen Rabin, Professor of Pediatric Hematology-Oncology at Baylor College of Medicine and Director of the Leukemia Program at Texas Children's Cancer Center. The researchers concluded that children with Down syndrome and ALL exhibit an increased rate of relapse and treatment-related mortality, underscoring the need for novel, less-toxic therapies to improve outcomes for this patient population.
Rouce Team Awarded $1.2 Million National Cancer Institute Grant
A team of researchers at Baylor College of Medicine's Dan L. Duncan Comprehensive Cancer Center, led by Dr. Rayne Rouce, was awarded a $1.2 million P20 grant from the National Cancer Institute to partner with Texas Southern University, a Historically Black College and University, to create the BCM/TSU Collaborative Union for Cancer Research, Education and Disparities (CURED). The program, co-led by Dr. Rouce and Dr. Veronica Ajewole, will expand on existing collaborations between the two institutions to create an optimal environment for cancer research, education, community outreach, career development, and promoting diversity in the biomedical workforce.
Humanitarian Health Care Network Wins $5 Million NIH ComPASS Award
Dr. Eric Russell, Assistant Professor, Pediatrics - Emergency Medicine, and Associate Director of the Program for Immigrant and Refugee Child Health (PIRCH) at Texas Children's Hospital/Baylor College of Medicine, and Dr. Karla Fredricks, Assistant Professor, Pediatrics - Global and Immigrant Health, Director of PIRCH—along with colleagues at the University of Texas Rio Grande Valley, Stanford, and the Migrant Clinicians Network—were recently awarded a five-year, $5 million NIH ComPASS (Community Partnerships to Advance Science for Society) grant for their long-standing, community-engaged border health work. Entitled "Humanitarian Health Care Network: Bringing the Most Vulnerable to Care," the program identifies newly arrived immigrants with complex and high risk conditions and connects them to medical care in their final destination. The award will also support research into healthcare access, barriers, resiliencies, and other health needs among this uniquely vulnerable group, and it may be renewed for an additional five years.
Excess ceramide and disrupted iron metabolism in neuronal mitochondria found to be the cause for MEPAN syndrome
A recent study published in Nature Metabolism has revealed the pathogenic mechanism underlying a rare pediatric neurodegenerative disorder known as mitochondrial enoyl reductase protein-associated neurodegeneration (MEPAN) syndrome. The study was led by Dr. Hugo J. Bellen, distinguished service professor at Baylor College of Medicine, and Chair of Neurogenetics at the Jan and Dan Duncan Neurological Research Institute at Texas Children’s Hospital (Duncan NRI), and Dr. Debdeep Dutta, a postdoctoral fellow in the Bellen lab. The Duncan NRI team found that in patients and animal models of this disorder, a large number of neurons die due to excessive accumulation of ceramide and defective iron metabolism, which results from disruptions in mitochondrial fatty acid synthesis. It is the first to provide a mechanistic link between disruptions in mitochondrial fatty acid synthesis, iron and ceramide metabolism, and neurodegeneration.
Dr. Davut Pehlivan awarded a prestigious research grant by the Doris Duke Foundation
Davut Pehlivan, MD, assistant professor at Baylor College of Medicine and instructor of Neurology at Texas Children’s Hospital, was among 21 physician-scientists across the nation who recently received funding support from the Doris Duke Foundation (DDF) for advancing the prevention, diagnosis, and treatment of diseases by strengthening and supporting the clinical research.
Duncan NRI scientists develop a new fly model to probe an epileptic brain disorder
Developmental and epileptic encephalopathy (DEE) refers to a group of neurodevelopmental conditions characterized by developmental delay, cognitive impairment, and seizures in children. In 2016, the first case linking variants in both the copies of UBA5 gene to DEE44 was reported. Since then, twelve distinct missense variants in the UBA5 gene have been identified in 25 patients. A recent study by Dr. Hugo J. Bellen and his team at the Jan and Dan Duncan Neurological Research Institute (Duncan NRI) at Texas Children’s Hospital and Baylor College of Medicine generated a new fruit fly model to assess the severity of symptoms caused by each of these variants. This systematic analysis of UBA5 pathogenic variants lays the foundation for better evaluation of the variants, which is important for the DEE44 patients in the future and for the development of drugs and gene therapy to treat this rare disorder. The study was published recently in the journal eLife.
A new pathway to regenerate myelin discovered
A study led by Dr. Hyun Kyoung Lee, associate professor at Baylor College of Medicine and investigator at the Jan and Dan Duncan Neurological Research Institute at Texas Children’s Hospital, has discovered a new biological mechanism to regenerate and repair myelin, a protective sheath that insulates neuronal fibers and plays a vital role in ensuring rapid and accurate neurotransmission.
Texas Children’s Hospital and Baylor College of Medicine launch a new clinical genomic sequencing program to help patients with an undiagnosable condition
Known disease-causing gene variants have been found in about 35% of children with severe developmental and severe childhood diseases. Texas Children’s Hospital and Baylor College of Medicine researchers, Dr. Hugo J. Bellen, Dr. Richard Gibbs, and Dr. Katherine Y. King have launched a new genomic sequencing initiative to identify the genetic mutations and the underlying mechanisms responsible for the remaining two-thirds of severe pediatric conditions. This transformational project is made possible by a two-year grant (potentially extendable) from the Chan Zuckerberg Initiative and is an extension of national collaborative initiatives such as the National Institutes of Health’s Undiagnosed Diseases Network (UDN) and the Genomics Research to Elucidate the Genetic of Rare Diseases programs (GREGoR).
Studying rare diseases can help many
Drs. Shinya Yamamoto, Oguz Kanca, Michael F. Wangler, and Hugo J. Bellen who lead the Model Organism Screening Center (Drosophila Core) at the Jan and Dan Duncan Neurological Research Institute at Texas Children’s Hospital and Baylor College of Medicine, recently published a comprehensive review in Nature Reviews Genetics highlighting the growing importance of non-mammalian animal models in rare disease research and the wide range of opportunities they offer in speeding up gene discovery and therapy development.
Allen, Lupo earn pediatric cancer grants
Dr. Carl Allen and Dr. Philip Lupo, both professors of pediatrics – hematology/oncology, have each received grants from the St. Baldrick’s Foundation. Allen received a $200,000 research grant for his project, “Molecular Analysis of Pediatric Burkitt Lymphoma from COG Studies,” and Lupo received a $100,000 consortium grant for his project, “Reducing Ethnic Disparities in Acute Leukemia (REDIAL).”
Scientists generate a new Drosophila toolkit to understand how SARS-CoV-2 virus impacts human health
The COVID-19 pandemic has caused catastrophic damage to public health and economies all over the world for nearly four years. While the pandemic is under control now, the SARS-CoV-2 virus continues to mutate and inflict unprecedented suffering for millions in the form of myriad lingering symptoms that are collectively termed ‘long-COVID’. Thus, identifying the underlying causes and therapies for long-COVID symptoms and devising strategies to protect against known and emerging coronavirus family members remains a top priority in biomedical research. While the fundamental machinery and mechanisms of how the SARS-CoV-2 virus multiplies are now relatively well-documented, mechanistic aspects of how the virus disrupts the function of a multitude of cells and organs remain a mystery. Researchers at Baylor College of Medicine, Texas Children’s Hospital, and the University of California at San Diego have now developed a comprehensive toolkit of Drosophilamelanogaster (fruit fly) COVID-19 Resources (DCR) to study viral and human proteins interact, with the ultimate goal of developing therapies to counteract symptoms caused by existing and new strains.
Rouce honored for DEI efforts
Dr. Rayne Rouce is the recipient of the Award for Excellence in Advancing Diversity, Equity and Inclusion from the American Society of Gene and Cell Therapy. This is one of three new awards from ASGCT to acknowledge individuals who have done exceptional work in the areas of service, DEI and mentorship. Rouce was selected unanimously by the awards committee. She is being recognized for her outstanding contributions toward the society’s principles and programs advancing diversity, equity, and inclusion. Rouce is an assistant professor of pediatrics – hematology and oncology and an associate director in Baylor’s Office of Institutional Diversity, Equity and Inclusion.
A new study reveals the cerebellum as a source of generalized convulsive seizures
Recurrent seizures are debilitating and can sometimes, be fatal. The onset and presentation of seizures vary significantly among epilepsy patients. Of more than 25 categories of seizure presentations within epilepsy, those with motor convulsions are perhaps the most disruptive and generally most feared among patients and caregivers. Unfortunately, convulsive seizures are also the most commonly occurring type of generalized seizures.
Texas Children’s Cancer Center researchers receive grants –
Drs. Sarah Kappa and Gengwen Tian were recently awarded NCI-funded K12 Pediatric Oncology Research Training Program Scholar grants which provides hematology/oncology faculty up to 3 years of at least 75% protected time to pursue in-depth studies and experience in clinical/translational research.
Dr. Alexandra Stevens, recently received a K08 grant from the National Cancer Institute for her project titled, Exploiting the Metabolic Dependencies of Pediatric AML (ASSIST).
Dr. Bilal Omer, recently received a grant from the ChadTough Defeat DIPG Foundation for his project titled, C7G-CD2 CAR TCells for DMG: Clinical Trial of Dual Route Strategy.
Dr. Austin Brown, recently received an R01 grant from the National Institutes of Health for his project titled, A Systems Epidemiology Approach for Predicting Methotrexate Neurotoxicity in Pediatric Acute Leukemia ASSIST
Dr. Jeremy Schraw, recently received an R01 grant from the National Institute on Minority Health and Health Disparities for his project titled, Leveraging Ethnic Anotia-microtia Disparities for Discovery (LEADD) Study.
Gene mutation underlies neurodevelopmental disorders (Aug 4, 2023)
A group of 20 patients with undiagnosed neurodevelopmental disorders, ranging from mild to severe, now have a genetic diagnosis thanks to an international team of researchers, including investigators from Baylor College of Medicine's GREGoR Research Center. The team analyzed the patients' genes and conducted family studies, finding they all had mutations of the gene DHX9. While mutations of this gene had been associated with neurodevelopmental problems in prior studies using animal models, this is the first time it has been associated with human disease. The team's findings appear in the American Journal of Human Genetics.
Baylor and Texas Children’s team presents major advances in treating OCD at a Congressional briefing (Jul 25, 2023)
A team of neuroscientists and neurosurgeons from Baylor College of Medicine and Texas Children’s Hospital recently made groundbreaking advances in treating patients suffering from obsessive compulsive disorder (OCD). These findings were presented at the bipartisan Congressional Neuroscience Caucus briefing on June 14, 2023 by Dr. Nicole Provenza, a postdoctoral fellow at the Baylor College of Medicine training under Dr. Sameer Sheth, professor at Baylor and the director of the Cain Foundation Labs for Pediatric Neurology Research located in the Jan and Dan Duncan Neurological Research Institute at Texas Children’s Hospital.
Dr. King invited to serve as Chair of NIH study section (Jul 12, 2023)
Dr. Katherine Y. King, associate professor at Baylor College of Medicine and Texas Children's Hospital, was recently invited by the National Institutes of Health to serve as Chair of the Basic Biology of Blood, Heart and Vasculature Study Section, Vascular and Hematology Integrated Review Group, at the Center of Scientific Review. She will serve a one-year term beginning in July. She was nominated for this role based on her demonstrated competence and achievement in her scientific discipline, as evidenced by the quality of her research accomplishments, publications in scientific journals, and other significant activities, achievements, and honors.
A protein implicated in Batten disease is critical for generating new lysosomes (Jul 6, 2023)
Neuronal Ceroid Lipofuscinosis are a group of devastating neurodegenerative lysosomal storage disorders that begin in childhood. Mutations in CLN3 gene lead to a NCL called Batten disease, characterized by the progressive loss of vision, movement, and cognition. Targeted effective therapies are not available for these disorders since the biological roles of most genes responsible for these disorders are not well defined. Researchers at Baylor College of Medicine and Texas Children’s Hospital in the US, Telethon Institute of Genetics and Medicine, and Federico II University in Italy have now discovered that CLN3 is critical for lysosome biogenesis and autophagic lysosomal reformation (ALR), uncovering a novel disease mechanism in Batten disease.
Long-range neuronal connections drive glioblastoma invasion (Jul 3, 2023)
Glioblastoma (GBM) is the most aggressive and lethal form of brain tumor. Despite treatment, GBM recurrence is inevitable and tends to occur outside surgical margins or in locations remote to the primary tumor, highlighting the central role played by tumor infiltration in this malicious disease. Little is known about the underlying molecular mechanisms driving GBM infiltration, but in a new study published in the journal Nature, researchers at Baylor College of Medicine working with animal models reveal a novel process by which neurons in locations remote to the primary tumor provoke expression of genes from glioma tumors that subsequently drive tumor infiltration.
Atoh1 is Critical to Establish the Diversity of Pontine Nuclei Neurons (Jul 3, 2023)
A recent study published in Science Advances by researchers at Baylor College of Medicine and Texas Children’s Hospital has discovered six distinct neuronal lineages in the pons region of the brainstem and revealed new insights into their differential vulnerability to the partial loss of Atoh1, a gene crucial for the development of pontine neurons.
Astrocyte processing of serotonin regulates olfactory perception (June 16, 2023)
To enjoy the scent of morning coffee and freshly baked cookies or to perceive the warning smell of something burning, the brain needs two types of cells, neurons and astrocytes, to work closely with each other. Research has shown a great deal of the changes that occur in neurons during olfactory, or smell, perception, but what are the astrocyte responses and how they contribute to the sensory experience remains unclear. Researchers at Baylor College of Medicine and collaborating institutions report in the journal Science the responses of astrocytes to olfactory stimulation, revealing a new mechanism that is required to maintain astrocyte-neuron communication and process olfactory sensation.
A new way to visualize force-sensing neurons (Jun14, 2023)
A recent study by researchers at Texas Children’s Hospital, Baylor College of Medicine, and Scripps Research Institute has discovered fluorescent dye FM 1-43 as an effective and versatile tool to visualize PIEZO2 ion channel activity in mechanosensory neurons.
Researchers find a way to reduce the side effects of deep brain stimulation (May 22, 2023)
Deep brain stimulation (DBS) has emerged as an effective therapy for severe, treatment-resistant obsessive-compulsive disorder (trOCD) and has received approval under a humanitarian device exemption from the U.S. FDA. However, there are adverse side effects associated with this procedure. While working on a recent case, a team led by Dr. Wayne Goodman, D.C. and Irene Ellwood Chair of Psychiatry and professor at Baylor College of Medicine, and Dr. Sameer Sheth, director of the Cain Foundation Labs in the Jan and Dan Duncan Neurological Research Institute (Duncan NRI) at Texas Children’s Hospital and professor at Baylor, found a way to reduce potential side effects associated with this procedure. The study was published in Brain Stimulation
CAR-NKT cell therapy shows promising results against neuroblastoma in phase 1 clinical trial (Baylor College of Medicine - May 15, 2023)
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Toronto neurosurgeon tapped to lead Texas Children's, Baylor research into childhood brain cancer (Houston Chronicle - February 8, 2023)
Texas Children's Hospital, Baylor College of Medicine name Dr. Michael Taylor director of research program (Becker's Spine Review - February 9, 2023)
Dr. Michael Taylor joins Texas Children’s Hospital and Baylor College of Medicine as Director of the Pediatric Neuro-Oncology Research Program (Texas Children's Hospital - February 8, 2023)
Dr. Michael Taylor, a molecular biologist and an international leader in pediatric neuro-oncology, has joined Texas Children’s Hospital and Baylor College of Medicine to become the inaugural director of the institutions’ childhood brain cancer research program. He will focus on pursuing therapies for difficult-to-treat brain tumors.
CPRIT approves $90 million to boost cancer research, prevention efforts across Texas (Cancer Prevention & Research Institute of Texas (CPRIT) - February 15, 2023)
‘Getting Close’ to making stem, bone marrow transplants safer for patients with blood cancer (Cure Today - January 16, 2023)
Novel T-cell therapy effective against common viruses after stem cell transplantation (HemOnc Today - January 12, 2023)
An investigational T-cell therapy shows promise against six viral infections common after stem cell transplants (American Association for Cancer Research - January 11, 2023)
Data science can help healthcare leaders navigate the global health landscape (Artificial Intelligence in Medicine - January 4, 2023)
Study reveals approach to testing adolescent girls for VWD (ASH Clinical News - January 3, 2023)
Promising approach to mitigate complications of leukemia treatment (Oct 6, 2022)
Researchers at Baylor College of Medicine and collaborating institutions have engineered immune cells to control two major life-threatening complications, namely graft-vs-host disease (GvHD) and cancer relapse, which typically emerge after treating leukemia with allogeneic hematopoietic stem cell transplantation (allo-HSCT).
A novel instructive role for the entorhinal cortex discovered (Nov 2, 2022)
A longstanding question in neuroscience is how mammalian brains (including ours) adapt to external environments, information, and experiences. In a paradigm-shifting study published in Nature, researchers at the Jan and Dan Duncan Neurological Research Institute (Duncan NRI) at Texas Children’s Hospital and Baylor College of Medicine have discovered the mechanistic steps underlying a new type of synaptic plasticity called behavioral timescale synaptic plasticity (BTSP). The study, led by Dr. Jeffrey Magee, professor at Baylor, who is also a Howard Hughes Medical Institute, and Duncan NRI investigator, reveals how the entorhinal cortex (EC) sends instructive signals to the hippocampus — the brain region critical for spatial navigation, memory encoding, and consolidation — and directs it to specifically re-organize the location and activity of a specific subset of its neurons to achieve altered behavior in response to its changing environment and spatial cues.
An animal model of West Syndrome exhibits a progressive increase in epileptic spasms and learning and memory deficits (Nov 2, 2022)
West syndrome, the most prevalent type of syndromic epileptic encephalopathy affecting infants, is a devastating and often fatal condition. It is characterized by a triad of symptoms – seizures/spasms, a signature brain activity between seizure events, and intellectual disabilities. Researchers in the laboratory of Dr. John Swann, professor at Baylor College of Medicine and investigator at the Jan and Dan Duncan Neurological Research Institute at Texas Children’s Hospital, have provided the first demonstration of a progressive increase in epileptic spasms along with learning and memory deficits in an animal model of this disorder. In addition, the study published in Epilepsia, establishes this as an ‘ideal’ model to identify the underlying molecular mechanisms and to discover targeted therapies for this condition.
Duraine wins an electron microscopy image contest (Oct 21, 2022)
Lita Duraine, a certified electron microscopist at Baylor College of Medicine and Jan and Dan Duncan Neurological Institute at Texas Children’s Hospital, won an image contest held by JEOL USA, a leading global supplier of electron microscopes, ion beam instruments, mass and NMR spectrometers.
Spontaneously arising variants in FRMD5 gene are associated with a novel neurological disease (Oct 7, 2022)
A study led by Dr. Hugo Bellen, investigator at the Jan and Dan Duncan Neurological Research Institute (NRI) at Texas Children’s Hospital (TCH) and distinguished service professor at the Baylor College of Medicine (BCM), reports de novo variants in a gene involved in regulating cellular motility to be the underlying cause of a new neurological disorder. The study was published in the American Journal of Human Genetics.
Researchers discover 50 novel Parkinson's disease candidate genes using an innovative multilayered approach (Oct 3, 2022)
Many neurodegenerative disorders such as Parkinson’s disease (PD) result from the combined effects of mutations in several genes (i.e., polygenic). Although previous studies have identified a few genes that are responsible for familial or sporadic cases of PD, we are still far from knowing the entire spectrum of genes that contribute to this complex disorder. Researchers at the Jan and Dan Duncan Neurological Research Institute at Texas Children’s Hospital and Baylor College of Medicine have recently developed an integrated functional genomics approach that led to the discovery of 50 genes that have been shown for the first time to modify PD pathology in a disease animal model. The study was published in Human Molecular Genetics.
Dr. Huda Zoghbi awarded 2nd Elaine Redding Brinster Prize in Science or Medicine (Sept 28, 2022)
Dr. Huda Zoghbi, director of the Jan and Dan Duncan Neurological Research Institute at Texas Children’s Hospital, a distinguished service professor at Baylor College of Medicine and an investigator with Howard Hughes Medical Institute, has been awarded the second Elaine Redding Brinster Prize in Science or Medicine from the Penn Institute for Regenerative Medicine at the University of Pennsylvania.
Global Collaboration: mapping the whole human brain (Sept 27, 2022)
Researchers in the Tolias Lab and the Xiaolong Jiang Lab are part of a global collaborative led by the Allen Institute to map the whole human brain. In what is being called the brain equivalent of the Human Genome Project, this initiative will map 200 billion cells in the human brain by their type and function as well as create a primate brain atlas.
Disruption of MTSS2 function causes a new syndromic intellectual disability (Sept 27, 2022)
An Undiagnosed Diseases Network (UDN) study led by Dr. Hugo Bellen, investigator at the Jan and Dan Duncan Neurological Research Institute (NRI) at Texas Children’s Hospital and distinguished service professor at the Baylor College of Medicine, has found a spontaneous mutation in MTSS2 gene to be the underlying cause of a new syndromic intellectual disability.
Dr. Gerarda Cappuccio receives a postdoctoral fellowship from Autism Speaks (Sept 15, 2022)
Dr. Gerarda Cappuccio, a physician-scientist at Baylor College of Medicine and the Jan and Dan Duncan Neurological Research Institute (Duncan NRI) at Texas Children’s Hospital, has been selected to receive the 2022 postdoctoral fellowship award from Autism Speaks to test gene therapy for a rare disorder, MeCP2 Duplication Syndrome, a condition related to the over-expression of a gene MeCP2 linked to autism.
Dr. Mirjana Maletic-Savatic announced as SFARI 2022 Pilot awardee (Sept 15, 2022)
Dr. Mirjana Maletic-Savatic, Duncan NRI investigator and associate professor at Baylor College of Medicine, is among the 16 investigators from the US and all over the world to be selected by The Simons Foundation Autism Research Initiative (SFARI) for the 2022 Pilot Awards. She received this grant award to support her project titled, "16p11.2 copy number variant effects on the gene-metabolome coupling."
Let's Talk About Alzheimer's Research: A Community Event (Aug 31, 2022)
Baylor College of Medicine, Houston Methodist Hospital, and the Alzheimer’s Association have partnered to provide the community information about ongoing research on Alzheimer’s Disease and related causes of dementia (loss of memory and thinking).
Researchers identify an Achilles’ heel in neuroblastoma (Baylor College of Medicine - August 16, 2022)
Dr. Gerarda Cappuccio selected as the co-recipient of the 2022 John M. Opitz Young Investigator Award (Aug 16, 2022)
Dr. Gerarda Cappuccio, a physician-scientist at Baylor College of Medicine and the Jan and Dan Duncan Neurological Research Institute (Duncan NRI) at Texas Children’s Hospital, has been selected as the co-recipient of the prestigious 2022 John M. Opitz Young Investigator Award by the American Journal of Medical Genetics, a leading source of original research on genetic disorders that has a worldwide following among physicians, geneticists, and associated research and medical professionals.
The 2022 HADDS Conference featured in the Houston Chronicle (Aug 2, 2022)
The second 2022 HADDS Family Conference that took place at the Jan and Dan Duncan Neurological Research Institute between July 21- 23 was featured in an article by the Houston Chronicle titled,"Kids with a rare neurological disorder converged in Houston to meet others from around the world". Read the article here.
Dr. David Nelson announced as the recipient of the 2022 Victor A. McKusick Leadership Award (Aug 2, 2022)
Dr. David L. Nelson, professor at Baylor College of Medicine, co-director of BCM Michigan Emory Fragile X Center, associate director of Baylor’s Intellectual and Developmental Disabilities Research Center, and investigator at the Jan and Dan Duncan Neurological Research Institute (Duncan NRI) at Texas Children’s Hospital is the recipient of the 2022 Victor A. McKusick Leadership Award from the American Society of Human Genetics (ASHG). This award is bestowed upon an individual who has exhibited exemplary leadership and vision in advancing the Society’s mission through the promotion and successful assimilation of genetics and genomics knowledge into the broader scientific community in areas ranging from science, medicine, public policy, and/or health.
Researchers discover novel light-gated potassium channel (Jul 20, 2022)
Researchers at the University of Texas Health Science Center at Houston McGovern Medical School, Baylor College of Medicine, Texas Children's Hospital, Rice University and the University of Guelph, Ontario, Canada, have reported a new class of light-gated channels that promise to pave the way for rapid and efficient optical neuronal silencing.
Disruptions in brain sphingolipid metabolism reveal new insights into cause of Gaucher disease (Jul 14, 2022)
A study by researchers at Baylor College of Medicine, the Jan and Dan Duncan Neurological Research Institute at Texas Children’s Hospital and collaborating institutions is the first to associate neuronal activity to the levels of sphingolipids, a type of fat, in brain cells. Furthermore, disruption of sphingolipid metabolism led to significant neuronal damage and neurodegeneration in animal models, revealing a new molecular perspective to the cause of Gaucher’s disease. Published in Science Advances, the work offers novel opportunities to develop therapies for this and other neurodegenerative disorders.
The Jan and Dan Duncan Neurological Research Institute at Texas Children’s Hospital announces a new Co-Director (Jul 12, 2022)
Dr. Joshua Shulman, Professor at Baylor College of Medicine, has been named the new Co-Director of the Jan and Dan Duncan Neurological Research Institute (Duncan NRI) at Texas Children’s Hospital. The Duncan NRI is a premier neurological research institution and a destination for families seeking answers and treatments for rare and undiagnosed neurological conditions, as well as for more common neurodegenerative and neuropsychiatric disorders.
Joo Hyun Kim receives a prestigious fellowship from the American Epilepsy Society (Jun 27, 2022)
Dr. Joo Hyun Kim, a postdoctoral fellow in the laboratory of Dr. Mingshan Xue who is a researcher at the Jan and Dan Duncan Neurological Research Institute research at Texas Children’s Hospital, and an assistant professor at Baylor College of Medicine, has received an early career grant and a postdoctoral fellowship award from the American Epilepsy Society (AES) to support her research and professional development activities.
Zoghbi receives International Prize for Translational Neuroscience (June 20, 2022)
Drs. Huda Zoghbi and Adrian Bird were awarded the International Prize for Translational Neuroscience by the Gertrud Reemtsma Foundation for their pioneering discoveries on the causes of Rett syndrome.
Texas Children's Hospital elevates national ranking to second among best children's hospitals by U.S. News & World Report. (June 14, 2022)
Texas Children’s Hospital is proud to consistently be recognized as a leader in pediatric care by U.S. News & World Report, placing second nationally in its new 2022-2023 Best Children’s Hospitals rankings. The hospital rose from a No. 3 ranking on this annual report in 2021-2022.
The McNair Medical Institute names Dr. Kara Marshall as a new McNair Scholar (June 7, 2022)
Dr. Kara Marshall, assistant professor of neuroscience at Baylor College of Medicine and faculty member at the Jan and Dan Duncan Neurological Research Institute at Texas Children’s Hospital, has been named the newest McNair Scholar.
A faster, less expensive approach identifies highly specific anti-cancer compounds (June 7, 2022)
Dr. Huda Zoghbi named the 2022 Kavli Prize Laureate in neuroscience (June 1, 2022)
Dr. Huda Zoghbi, pioneering neurologist at Baylor College of Medicine and Texas Children’s Hospital, has been awarded the prestigious 2022 Kavli Prize in the field of neuroscience. She is being recognized for two discoveries – first, of the gene responsible for spinocerebellar ataxia 1 (SCA1), a progressive and often deadly disease in which neurons in the cerebellum and brain stem degenerate, causing loss of balance and coordination as well as swallowing difficulties. Second, for her discoveryof the MECP2 gene responsible for Rett syndrome, a developmental disorder that strikes children, mostly girls, causing regression and disability. She is the first Kavli prize winner for Baylor and Texas Children’s.
Cross-disciplinary Alzheimer's Disease Research Symposium (May 25, 2022)
The Center for Alzheimer's and Neurodegenerative Diseases (CAND) will host a cross-disciplinary Alzheimer's disease research symposium on June 13th, 2022 from 12:00 - 4:30 PM at the conference center and auditorium located in the Jan and Dan Duncan Neurological Research Institute (Duncan NRI) at Texas Children's Hospital. Dr. Huda Zoghbi, director of Duncan NRI, will present the keynote address at the symposium. Further details about this symposium can be found here.
Molecular profiling identifies new high-risk subtype of pediatric liver cancer (May 24, 2022)
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) 24th Annual Meeting.
2022 EBF3 HADDS International Conference (May 17, 2022)
The EBF3-HADDS Foundation was created to promote awareness, research, and support for a rare genetic syndrome discovered in 2016. The foundation will host the annual international conference at the Duncan NRI auditorium on July 21-23, 2022.
Finding the right diagnosis for one child can help millions more (May 16, 2022)
Read the story of how breakthrough research by Duncan NRI researchers solved a patient's medical mystery and brought hope to many other patients and families.
A pioneering study discovers an underlying cause for infantile spasms and points to a novel therapy (May 11, 2022)
A groundbreaking study, conducted in the laboratory of Dr. John Swann, director of the Gordon and Mary Cain Pediatric Neurology Research Foundation labs, investigator at the Jan and Dan Duncan Neurological Research Institute at Texas Children’s Hospital and professor at Baylor College of Medicine, has found that the levels of insulin growth factor -1 (IGF-1) and its downstream signaling are reduced in the brains of both IS patients and animal models. Furthermore, they found that the administration of an IGF-1 analog to an IS animal model successfully eliminated spasms and abnormal brain activity. This exciting study, published in the Annals of Neurology, has the potential to transform the treatment landscape for babies with infantile spasms.
Texas Children’s Hospital and Baylor College of Medicine study opens the door to improved diagnosis, prognosis and potential therapies for EBF3 and other neurodevelopmental disorders (Apr 20, 2022)
A team of researchers at the Jan and Dan Duncan Neurological Research Institute (Duncan NRI) at Texas Children’s Hospital recently completed a study that provides an answer to a puzzling question: why some patients with Early B-Cell Factor 3 (EBF3) gene variants develop symptoms that are significantly more severe than those experienced by other individuals with the EBF3-related autism spectrum and neurodevelopmental disorders.
NCI-COG Pediatric MATCH study shows benefit of genetic screening in refractory tumors (Baylor College of Medicine - April 4, 2022)
Oleic acid, a key to activating the brain’s ‘fountain of youth’ (March 25, 2022)
Many people dread experiencing the cognitive and mood declines that often accompany reaching an advanced age, including memory disorders such as Alzheimer’s disease and mood conditions like depression. While searching for new ways to prevent or treat these and other related conditions, a team at Baylor College of Medicine and the Jan and Dan Duncan Neurological Research Institute (Duncan NRI) at Texas Children’s Hospital identified a missing piece of the puzzle of how memory and mood are sustained and regulated in the brain.
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.
Fruit fly study uncovers functional significance of gene mutations associated with autism (March 15, 2022)
About 1 in 44 children in the U.S. are diagnosed with autism spectrum disorder (ASD) by the age of 8, according to the 2018 Centers for Disease Control and Prevention surveillance. How a child’s DNA contributes to the development of ASD has been more of a mystery. Recently, clinicians and scientists have looked more closely at new, or de novo, DNA changes, meaning they only are present in affected individuals but not in the parents. Researchers have seen that these changes could be responsible for about 30% of ASD. However, which de novo variants play a role in causing ASD remains unknown. Researchers at Baylor College of Medicine and Texas Children’s Hospital have taken a new approach to looking at de novo ASD genetic variants. In this multi-institutional study published in the journal Cell Reports, they applied sophisticated genetic strategies in laboratory fruit flies to determine the functional consequences of de novo variants identified in the Simons Simplex Collection (SSC), which includes approximately 2,600 families affected by autism spectrum disorder. Surprisingly, their work also allowed them to uncover a new form of rare disease due to a gene called GLRA2.
Reduced inhibition of hippocampal neurons impairs long-term memory recall in Rett syndrome (March 14, 2022)
An exciting study by researchers in the laboratory of Dr. Huda Zoghbi, distinguished service professor at Baylor College of Medicine and director of the Jan and Dan Duncan Neurological Research Institute (Duncan NRI) at Texas Children’s Hospital, have discovered that diminished memory recall in Rett syndrome mice can be restored by activating specific inhibitory cells in the hippocampus. The findings are published in the current edition of Neuron.