Research and Breakthroughs

Learn about our research efforts in neuroscience

The physician-scientists of the Neuroscience Center are noted leaders in clinical and basic science research and have helped pioneer much advancement in the neurological and neurosurgical fields. The research efforts neurology and neurosurgery of Texas Children’s are among the best in the world. 

Jan and Dan Duncan Neurological Research Institute (NRI)

The NRI allows basic science and clinical research scientists to leverage emerging technologies and invent new ones. Researchers, clinicians and educators work together in close proximity – literally and figuratively – speeding the adoption of new therapies to treat and better understand these devastating neurological conditions.

Breakthroughs

State-of-the-art DNA sequencing technology examines causes of genetic disorders

The Whole Genome Laboratory at Baylor College of Medicine, academic partner of Texas Children’s Hospital, is applying the power of next-generation DNA sequencing technology to unanswered genetic questions that directly affect patient health. The laboratory not only makes available the advances of high tech DNA sequencing, it also provides the interpretation by trained clinical genetics experts who will transmit the findings to a referring physician.

For decades, Texas Children’s Hospital has seen patients with various genetic conditions but often has been unable to identify the abnormal gene. Now, by sequencing the exome of these patients, the hospital will have for the first time the potential to identify the abnormal gene in the majority of cases. Identifying the gene involved is the necessary first step in the path to developing meaningful treatment and prevention. “We have estimated that 25% of the patients in the Blue Bird Clinic for Pediatric Neurology have a genetic disorder that is either not previously described or does not have a discovered genetic basis,” said Dr. Gary Clark, chief of Neurology at Texas Children’s Hospital. “Whole exome sequencing in our clinic population has led and will lead to new discoveries. Further, this technology will assist our patients and their families in understanding the cause of neurologic disease and the risk of recurrence. Whole exome sequencing represents the next revolution in genetic testing, and Texas Children’s Hospital and Baylor College of Medicine are eagerly embracing this technology.”

Anti-rejection drug reduces seizures in patients with tuberous sclerosis

A study conducted by Texas Children’s Hospital, Baylor College of Medicine and Cincinnati Children’s Hospital Medical Center has determined that Everolimus, a drug used to treat cancers and prevent rejection of transplanted organs, reduces the occurrence of seizures in patients with the genetic disorder tuberous sclerosis.

Tuberous sclerosis complex results from faulty signaling in a key molecular pathway (mTOR), causing abnormal cell growth, and it can ultimately lead to seizures. Reported online in the journal Annals of Neurology, this study investigates the manner in which Everolimus suppresses the activity of the mTOR pathway, which is overactive in this disorder.

Twenty patients with the disorder and difficult-to-treat epilepsy received the drug for a 12-week trial, and their symptoms were monitored. Seizures were reduced on average (median) by 73% in 17 of the 20 patients. Patients and their parents also reported reduced frequency by 50% in 12 of the 20 patients.

Deep brain stimulation for movement disorders aids children suffering from cerebral palsy

Texas Children’s Hospital is using deep brain stimulation to help treat pediatric patients suffering from different forms of dystonia. These disorders are characterized by abnormal twisting movement of the body due to improper signals from the basal ganglia – a deep region of the brain that coordinates movements.

In almost 80 percent of the patients suffering from genetic dystonia, deep brain stimulation has markedly improved the symptoms these children are experiencing. For those patients with secondary dystonias, such as dyskinetic cerebral palsy or stroke, this procedure has also yielded favorable results.

Deep brain stimulation is a technique by which an electrode is implanted in a patient’s brain to help modify erroneous signals. This procedure has been done more than 100,000 times, though mainly in adults with Parkinson’s disease or tremors. For this treatment, the electrode used is connected to a small programmable battery which is implanted in the patient’s upper chest region, near the shoulder. This device is programmed in a series of monthly office visits to help maximize its effects. It typically takes six to 12 months to see the full effects of this treatment.

To date, the team at Texas Children’s, including Dr. Amber J. Stocco, medical director of the Movement Disorders Clinic and Dr. Daniel Curry, a pediatric functional neurosurgeon, has used deep brain stimulation in patients with 10 different etiologies of dystonia, placing Texas Children’s Hospital among the top institutions in the country treating children with these disorders.