Our goal is to cure Tay-Sachs, Sandhoff, GM-1, Canavan and related genetic diseases. The Research Strategy was developed by our Scientific Advisory Committee, together with other experts, to achieve that goal as quickly as possible.
The NTSAD Research Strategy will:
• Develop a broad variety of Potential Therapies
• Advance the most promising efforts to find a cure
• Maximize impact of our Research Initiative grant funding
• Fund meaningful projects of specific interest to us
NTSAD supports research on a broad variety of potential therapies for the best chance at finding the cure. Another important reason to develop a diverse portfolio of projects is because the scientific community generally agrees that a single approach will not cure these diseases, but rather a combination of approaches.
The NTSAD Scientific Advisory Committee is comprised of world-class researchers and clinicians who are leading experts in lysosomal and
leukodystrophy diseases. These experts provide broad insights into the scientific field and advise NTSAD on the efforts most likely to lead to a cure. Their expertise includes not only understanding the underlying disease causing mechanisms and potential therapies but also other areas that could impact our diseases.
In addition to funding grant projects with the most potential to cure Tay-Sachs and related diseases, NTSAD hosts bi-annual scientific meetings. These meetings are opportunities for scientists to collaborate, share ideas and generate new ground-breaking ways to find a cure.
NTSAD often awards small seed grants that are likely to lead to sources of more significant funding. NTSAD has awarded $1.9 million in grants. Several of these have lead to large NIH funding totaling over $6 million, thereby tripling our investment in finding a cure!
NTSAD reaches out to partner organizations to co-fund projects when we receive a compelling grant application that we are unable to fund in full. The most recent example is a breakthrough Canavan
gene therapy grant in partnership with the Canavan Research Foundation.
In 2005 NTSAD and the National MPS Society co-founded the Lysosomal Storage Disease Research Consortium (LSDRC) which, along with other patient advocacy groups, partnered with the National Institute of Neurological Disorders and Stroke (NINDS) to support research towards potential therapies specifically addressing the neurological aspects of lysosomal storage disorders like Tay-Sachs. This partnership resulted in 13 grants which might otherwise not have been funded, totaling $372,000, and specifically targeting lysosomal diseases. Five of these projects have gone on to receive larger NIH funding and two additional NIH grants are pending.
While some projects may have potential application to many diseases, others are narrow in scope and apply only to a specific disease. If NTSAD does not fund such a specific project, then the research may not get done. Examples are the natural history study of Tay-Sachs and Sandhoff disease and acetate supplementation therapy for Canavan disease.
