COLLABORATIVE PROGRAM TO ACCELERATE THERAPEUTICS DEVELOPMENT FOR SPINAL MUSCULAR
ATROPHY (SMA)
RELEASE DATE: April 8, 2003
NOTICE: NOT-NS-03-012
RFP Available: NIH-NINDS-RFP-03-03
National Institute of Neurological Disorders and Stroke (NINDS)
The National Institute of Neurological Disorders and Stroke (NINDS) has the
stated mission to reduce the burden of neurological disease a burden borne by
every age group, by every segment of society, by people all over the world. To
support this mission, NINDS:
o Conducts, fosters, coordinates, and guides research on the causes,
prevention, diagnosis, and treatment of neurological disorders and stroke, and
supports basic research in related scientific areas.
o Provides grants-in-aid to public and private institutions and individuals in
fields related to its areas of interest, including research project, program
project, and research center grants. Provides individual and institutional
fellowships to increase scientific expertise in neurological fields.
o Operates a program of contracts for the funding of research and research
support efforts in selected areas of institute need.
o Conducts a diversified program of intramural and collaborative research in
its own laboratories, branches, and clinics.
o Collects and disseminates research information related to neurological
disorders.
NINDS has targeted Spinal Muscular Atrophy (SMA) for a pilot program in
accelerated therapeutics development. SMA is a devastating neurological disease
of childhood for which no effective treatment is currently available. SMA causes
degeneration of motor neurons in the spinal cord and brainstem, leading to
muscle weakness and, in severe forms of the disease, death. It is the most
severe of the common genetic neurological diseases and is the leading heritable
cause of infant death. SMA is an ideal candidate for targeted therapeutics
development. It is caused by the loss of function of a known gene, SMN1. Thus,
there are tractable strategies for developing treatments based on replacement
of the lost function. Multiple therapeutic avenues are available, including
drug development, gene therapy and stem cell therapy.
NINDS' research goal for Spinal Muscular Atrophy is to efficiently translate a
mechanistic understanding of SMA into a testable therapy suitable for clinical
trials. The NINDS strategy for achieving its research goal is to fund parallel
therapeutic pathways in a rapid milestone-driven process that will identify a
promising therapeutic candidate. This project will require a high level of
scientific expertise and experience on the part of the contractor. Furthermore,
efficient progress will involve an unusual degree of acceleration and
integration of multiple research projects, and thus success will also require
that the contractor use creativity and flexibility in applying that scientific
expertise.
It is anticipated that one award may be made for a period of four years in
September 2003.
Request for Proposals (RFP) NIH-NINDS-03-03 will ONLY be AVAILABLE
ELECTRONICALLY at either one of the following websites:
http://www.FedBizOpps.gov/ or
http://www.ninds.nih.gov/funding/funding_announcements/RFP_all.htm,
15 or more calendar days after the issuance of this synopsis. OFFERORS ARE
RESPONSIBLE FOR ROUTINELY CHECKING EITHER WEBSITE FOR ANY POSSIBLE SOLICITATION
AMENDMENTS THAT MAY BE ISSUED. NO INDIVIDUAL NOTIFICATION OF ANY AMENDMENTS
WILL BE PROVIDED. All responsible sources may submit a proposal, which shall
be considered by the agency. Refer to numbered note 26.****
INQUIRIES
Inquiries may be directed to:
Patricia S. Denney, Contracting Officer
Contracts Management Branch
National Institute of Neurological Disorders and Stroke
Neuroscience Center, Room 3287
6001 Executive Boulevard, MSC 9531
Bethesda, MD 20892-9531
Tel: (301) 496-1813
Fax: (301) 402-4225
Email: PD22N@NIH.GOV
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