Baylor College of Medicine, Texas Children's Hospital researchers receive over $880,000 for Rett syndrome research

HOUSTON - (Aug. 8, 2012) - Researchers at Baylor College of Medicine and Texas Children's Hospital have been awarded over $880,000 for two grants from The International Rett Syndrome Foundation to study therapeutic options for Rett syndrome.

Currently, there is no treatment for Rett syndrome, a unique developmental disorder that is first recognized in early childhood and primarily affects girls.


Rett syndrome causes problems in brain functions that are responsible for cognitive, sensory, emotional, motor and autonomic function. People with Rett syndrome are impaired in hand use and spoken language and have problems with sensory sensations, mood, movement, breathing, cardiac function, as well as chewing, swallowing and growth.


The research awards include:

Dr. Daniel Glaze, professor of pediatrics - neurology at BCM, and Dr. Jeffrey Neul, associate professor of pediatrics - neurology at BCM working in the Jan and Dan Duncan Neurological Research Institute (NRI) at Texas Children's Hospital, received at $600,000, two-year Angel Award to conduct a clinical trial on an experimental drug to see if it is safe and effective in the treatment of adult Rett syndrome patients. Glaze and Neul are co-directors of the Texas Children's Blue Bird Circle Rett Center respectively. Glaze is also director of the Texas Children's Sleep Center.


Additionally, Neul, the Petrello Scholar at the NRI, and his colleague Dr. N. Carolyn Schanen, of the Nemours Foundation, received a $600,000, two-year grant to study targeting a certain mutation on the MECP2 gene as a therapeutic approach to Rett syndrome. Neul will receive $286,298 of this award to conduct research at the NRI. Rett syndrome is caused by mutations in the MECP2 gene located on the X chromosome. Approximately one-third of Rett syndrome patients have nonsense mutations in the MECP2 gene, which prevent the formation of a complete protein. In animal studies, Neul will look at drugs that target these types of mutations and allow the complete protein to be made as a treatment for the disorder.


In this wave of funding, The International Rett Syndrome Foundation awarded a total of $1.3 million to support new grant funding for translational research ranging from clinical studies and developing outcome measures to preclinical studies of new compounds in mouse models of Rett syndrome.

About Texas Children’s Hospital

Texas Children’s Hospital, a not-for-profit health care organization, is committed to creating a healthier future for children and women throughout the global community by leading in patient care, education and research. Consistently ranked as the best children’s hospital in Texas, and among the top in the nation, Texas Children’s has garnered widespread recognition for its expertise and breakthroughs in pediatric and women’s health. The hospital includes the Jan and Dan Duncan Neurological Research Institute; the Feigin Center for pediatric research; Texas Children’s Pavilion for Women, a comprehensive obstetrics/gynecology facility focusing on high-risk births; Texas Children’s Hospital West Campus, a community hospital in suburban West Houston; and Texas Children’s Hospital The Woodlands, a second community hospital planned to open in 2017. The organization also created the nation’s first HMO for children, has the largest pediatric primary care network in the country and a global health program that’s channeling care to children and women all over the world. Texas Children’s Hospital is affiliated with Baylor College of Medicine. For more information, go to www.texaschildrens.org. Get the latest news by visiting the online newsroom and Twitter at twitter.com/texaschildrens.