Dr. Huda Y. Zoghbi, director of the Jan and Dan Duncan Neurological Research Institute at Texas Children's Hospital and Baylor College of Medicine, has been awarded the 2011 Neuroscience Prize of The Peter and Patricia Gruber Foundation for her pioneering work in unlocking the genetic and molecular mysteries behind a number of devastating neurological disorders, including Rett syndrome, spinocerebellar ataxia type 1, and brain tumors called medulloblastomas. Learn more about Dr. Zoghbi and her work.

"I am deeply honored by this award. The Gruber Foundation seeks to draw attention to work they believe helps better the world. That was my goal in entering research, and although we have a way to go in the biomedical sciences to really improve the lives of patients with neurological diseases, I am grateful for the foundation's support and for their ongoing efforts to promote awareness of advances in neuroscience," said Zoghbi, who is professor of neuroscience, pediatrics, molecular and human genetics, and neurology at Baylor and a Howard Hughes Medical Institute investigator. "I am grateful to my mentors, particularly Dr. Art Beaudet, and my colleagues and trainees at Baylor College of Medicine as well as my long-time collaborator Dr. Harry Orr. It was really the nurturing Baylor environment that transformed me from a physician into a well-rounded scientist. I would not have had such a rewarding career anywhere else."

Orr, director of the Institute for Human Genetics at the University of Minnesota, is a long-time collaborator with Zoghbi. They co-discovered the gene for spinocerebellar ataxia 1 in 1993 and have gone on to understand how the mutation in that gene contributes to patient symptoms. Zoghbi has also discovered the gene for Rett syndrome, a neurological disorder that affects mainly girls, and Math1, a gene that plays a key role in functions from respiration to hearing to the development of brain tumors.

Collaboration at every level

As the founding director of the NRI, a collaborative endeavor funded by Texas Children's Hospital and powered by BCM faculty, Zoghbi leads a team of researchers from diverse biological and computational backgrounds in the world's first basic research institute dedicated to childhood neurological diseases. Her vision for the NRI involves collaboration at every level—interdisciplinary, interdepartmental and inter-institutional—to increase the pace of discoveries by pioneering a multidisciplinary research approach to the complex challenges of understanding brain development and function.

"The NRI will provide the impetus to find answers to the neurological diseases that devastate the lives of children worldwide," she said. "The NRI is the intellectual child of both institutions – Texas Children's and Baylor – and will drive neurological research in the future."

Understanding nervous system

The Gruber Neuroscience Prize is a highly-coveted international research award presented annually by The Peter and Patricia Gruber Foundation. The award honors scientists who have significantly advanced our understanding of the nervous system.

The prize consists of a gold medal inscribed with the honoree's name, a citation describing her achievements and an unrestricted cash prize of $500,000. Zoghbi has chosen to give most of the award to support research at the Neurological Research Institute and Baylor College of Medicine. She will also donate some of the prize to the American University of Beirut, which Zoghbi credits for providing the foundation for a lifelong career of intellectual curiosity, and to private groups that support children with neurological disorders.

Distinguished leader

"Dr. Zoghbi is an incredibly talented, transformative leader in this field, and her own personal investment of resources in the work of her team is a remarkable reflection of her conviction and commitment to this cause," said Mark A. Wallace, president and CEO of Texas Children's Hospital. "She and her team at the NRI are making discoveries that will literally change the future for children and adults suffering from neurological disorders—not just here locally but all over the world."

"Dr. Zoghbi is a distinguished leader in the laboratory and in the community of scientists as whole," said Dr. Paul Klotman, president and CEO of Baylor College of Medicine. "She is a tremendous mentor to the next generation of scientists."

Zoghbi was chosen by a distinguished advisory board of neuroscience experts from among nominations received from around the world. "What stands out about Dr. Zoghbi's discoveries is that the original inspiration for her science was her clinical observations – and her determination to ‘go to the bench' to solve the mystery of the disorder. Her work has revealed probable underlying mechanisms of a number of postnatal neurologic disorders, for which the path to clinical treatment can now be realistically followed. This trajectory to discovery and potential treatment is simply inspirational," said Carol Barnes, chair of the Selection Advisory Board to the Neuroscience Prize.

Zoghbi will accept the award Nov. 13, 2011, at the Annual Meeting of the Society for Neuroscience in Washington, D.C.

The Peter and Patricia Gruber Foundation honors and encourages educational excellence, social justice and scientific achievements that better the human condition. For more information about the foundation and its priorities, please go to the Gruber Foundation web site.

For more information on Zoghbi's research, visit the NRI web site.