NATIONAL TAY-SACHS & ALLIED DISEASES ASSOCIATION AWARDS $500,000 IN RESEARCH GRANTS
Boston, Mass. (May 18, 2010) - The National Tay-Sachs & Allied Diseases Association (NTSAD) has awarded five research grants totaling nearly $500,000, the largest dollar amount awarded by the organization’s Research Initiative in a single year. The grants will fund basic research to develop promising treatments for Tay-Sachs, Sandhoff and Canavan diseases.
There is currently no cure for these progressive neurodegenerative genetic diseases. They are fatal in children and extremely debilitating in adults.
The grantees include:
• Jean-Pyo Lee, PhD / Evan Y. Snyder, MD, PhD, Burnham Medical Research Institute, for use of induced pluripotent stem cells (IPSCs) to treat Sandhoff Disease in mice
• Maria Traka, PhD, University of Chicago, for a study to identify molecular pathways in Canavan Disease
• Fran Platt, PhD, University of Oxford, for studying the role inflammation plays in the brains of Sandhoff affected mice, potential leading to an anti-inflammatory therapy
• Gustavo Maegawa, MD, PhD, Johns Hopkins University for development of a method to more quickly screen potential drugs in the treatment of Metachromatic Leukodystrophy.
• Doug Martin, PhD, Auburn University, for gene therapy research on a natural sheep model of Tay-Sachs disease. The NTSAD grant supplements a $3.5 million NIH grant award to the Tay-Sachs Gene Therapy Consortium in August 2009 to begin human clinical trials of an experimental gene therapy for Tay-Sachs and Sandhoff diseases.
Tay-Sachs and other lysososomal storage diseases occur in all ethnic groups. The diseases can be prevented through education and carrier screening, which the NTSAD promotes.
“Today there is more hope than ever before for finding effective treatments for those affected by Tay-Sachs and related diseases,” said Sue Kahn, Executive Director of NTSAD. “
About NTSAD’s Research Initiative
NTSAD’s Research Initiative funds innovative basic research, translational studies or clinical trials in the areas of neurodegenerative disorders affecting the central nervous system, especially lysosomal storage disorders and pediatric leukodystrophies (such as Canavan and Krabbe). The majority of the funds have been raised through family foundations: Cameron and Hayden Lord Foundation, Cure Tay-Sachs Foundation, Hoffman Family Fund, Mathew Forbes Romer Foundation, Pesotchinsky Family Fund, Ungerleider Family Fund, and the NTSAD New York chapter.
About National-Tay Sachs & Allied Diseases Association (NTSAD)
The NTSAD leads the fight to treat and cure Tay-Sachs, Canavan and related genetic diseases while supporting affected families and individuals to foster quality of life. Founded in 1957, NTSAD was a pioneer in the development of community education about Tay-Sachs disease, carrier screening programs, and laboratory quality control programs. Today more than two million people have been screened for Tay-Sachs disease. Numerous Tay-Sachs carriers have been identified and thousands of healthy children have been born to high-risk couples. For more information, go to www.NTSAD.org .
Provided by National-Tay Sachs & Allied Diseases Association
