NTSAD Research Initiative Request for Proposals

National Tay-Sachs & Allied Diseases Association Inc. (NTSAD) is considered to be the nation’s first genetic disease nonprofit organization. The “Allied Diseases” represent a family of more than 40 diseases including lysosomal storage diseases (LSD) and leukodystrophies affecting the central nervous system.

In 2002, NTSAD launched its Research Initiative with the goals of focusing and accelerating research through a network of scientists, clinicians, and patient organizations. Over that time, we have funded a number of projects that populate a research roadmap spanning research/proof of science and animal trials. Some of these seed projects have also led to follow-on government funding to help us move toward clinical trials and to commercially available therapies.

NTSAD is soliciting proposals for innovative research projects that would generate strong preliminary data for major funding in the area of neurodegenerative disorders affecting the CNS, especially lysosomal storage disorders and pediatric leukodystrophies (such as Canavan and Krabbe). Projects can be basic or translational and can build off of previous work with such areas as neural stem cells, the blood brain barrier, molecular chaperones, gene therapy, and substrate inhibitors as well as exploring other novel therapeutic strategies. The funders are especially interested in projects that bridge potential treatment of LSDs and leukodystrophies of the CNS. Funding would be $30,000 to $45,000 for one year, inclusive of indirect costs not to exceed 10%. The deadline for proposals is 5:00 p.m. (EST), Friday, February 29, 2008.

Application Guidelines

Executive Summary: Major objectives and impact of research. (1 page)

Detailed Research Plan: Design of research; process for collecting and analyzing data; potential risks and their management; top level one-year schedule of research including defining milestones, which will be reported in one interim report to demonstrate progress relative to budget. (up to 5 pages)

Detailed Budget: To be linked to the top-level one-year schedule as described above. The first half-payment (50%) will occur at inception of project. A second quarter-payment (25%) will occur upon completion of the interim six-month progress and budget report, and the final quarter-payment (25%) will occur upon completion of a final report. No more than 10% of the project's budget can go toward indirect expenses. (1 page)

Curriculum Vitae with emphasis on related experience. Also include a list of any other personnel expected to be involved with the research project and a brief description of their experience. (2-3 page NIH bio-sketch format)

Facility description: An outline of the facility or laboratory in which research will take place with a listing of existing equipment applicable to project. (I page)

Other sources of financial and collaborative support: This should include support already applied for and/or received. (1 page)

Proposals should be received no later than 5:00 p.m. (EDT) February 29, 2008 to:
Susan Kahn, Executive Director
National Tay-Sachs & Allied Diseases Association
Research Initiative
2001 Beacon Street, Suite 204
Boston, MA 02135
Phone: 617-277-4463;
Fax: 617-277-0134
E-mail: skahn@ntsad.org for PDF files only