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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