2015 Neurological Research Round-up


This past year was exciting and productive for neuroscience researchers at Texas Children’s Hospital. Texas Children’s Neurology and Neurosurgery departments were once again ranked #2 among the nation’s children’s hospitals by U.S. News & World Report.

Undoubtedly, that is largely due to the highest quality of specialized clinical care we provide to patients with neurological and neurodevelopmental disorders. Another very important reason for our consistently high national rankings, is our cutting-edge basic and clinical research programs. Our physicians and scientists are constantly on a quest to identify new therapies for incurable neurological conditions and to improve the standard of care for our patients.

Here is a snapshot of the most important research discoveries from Texas Children’s Neuroscience Center this year.

Clinical research highlights:

  • The first successful interventional drug trial for Rett Syndrome was conducted at Texas Children’s this year. This trial symbolizes our commitment to finding a cure for Rett syndrome. From Dr. Huda Zoghbi’s discovery of the gene that causes Rett syndrome in the late 1990s, to being the lead site for the first clinical trial, Texas Children’s has been a pioneer in Rett syndrome research. Dr. Daniel Glaze led this trial which was cosponsored by Neuren Pharmaceuticals and International Rett Syndrome Foundation. The trial was conducted to test the safety, tolerability and efficacy of NNZ-2566 (trofenitide) among adolescent girls and women. The Food and Drug Administration (FDA) is currently reviewing the data and additional trials to test its safety and efficacy are being planned for 2016.
  • We successfully completed a year-long interventional trial of Ataluren, a novel gene modifying drug for Duchenne muscular dystrophy (DMD), a progressive muscle wasting disease. This trial was conducted by Dr. Timothy Lotze in collaboration with PTC Therapeutics. Ataluren is an orally administered small-molecule compound that targets a specific nonsense genetic mutation in dystrophin gene in some DMD and cystic fibrosis patients. This mutation creates a premature stop signal and prevents the production of a functional dystrophin protein. Ataluren interacts with ribosomes and allows them to skip over the stop signal to produce a full-length, functional dystrophin protein. This specifically targeted drug is anticipated to increase the quality of life of patients by retaining some mobility and increasing their life span, all with fewer undesirable side effects. In fact, the results of this study have been so encouraging that Texas Children’s has been invited by PTC Therapeutics to participate in a follow-up study.
  • Texas Children’s is the first pediatric institution in the state of Texas to be approved to study the clinical benefits of pharmaceutical grade cannabidiol, Epidiolex, in several trials sponsored by GW Pharmaceuticals. This year a team led by Dr. Angus Wilfong evaluated the use of Epidiolex as a treatment for various catastrophic epilepsies, including Dravet’s and Lennox-Gastaut syndromes. Texas Children’s has been at the forefront of studying the potential clinical benefits and risks of using Epidiolex because we believe it is crucial to thoroughly understand its safety and efficacy before prescribing it to patients, especially infants and young children.

Basic research highlights:

The Jan and Dan Duncan Neurological Research Institute (NRI) opened its doors in 2010 and is among the world’s leading basic research institutes dedicated to solving childhood neurological and neuropsychiatric diseases. Learn more about NRI.

The NRI welcomed 2015 with the launch of a brand new website that showcases cutting-edge epilepsy research, conducted by our researchers at the Cain Foundation Laboratories, as well as other neuroscientists from around the globe.

In its fifth year, scientists at the NRI continued to make rapid strides in understanding the cause of many currently incurable neurological diseases with the ultimate aim of developing therapies.

Read all exciting research discoveries and news stories from the NRI and Cain Labs.

Our physicians and scientists believe that in order to efficiently identify and develop novel therapies for some of these difficult neurological conditions, it is crucial to closely collaborate with patient families, pharmaceutical or biotech industry experts, as well as various private federal granting agencies like the NIH (National Institutes of Health). Therefore, since its inception, the NRI has been hosting biennial symposia that bring together various key stakeholders to facilitate and promote dialogue.

Our next such event is April 21-22, 2016 when the NRI will host the third biennial conference on “Neurodegeneration: From Cellular Concepts to Clinical Applications.” If you are interested in participating in this free symposium, please register here.


About Dr. Rajalaxmi Natarajan, Scientific Research Writer

I am a scientific research writer for all Texas Children’s Hospital websites including the NRI and Cain Labs. Previously, I worked as a neuroscientist and cell biologist in Washington University School of Medicine and The University of Texas Medical Branch. My passion is to keep patient families informed about breakthrough scientific discoveries, active clinical trials and latest therapies that are being developed at Texas Children’s.
Posted in Neurology, Neuroscience, Neurosurgery, News, NRI, Research, Surgery

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