Notice Number: NOT-NS-07-005

Key Dates
Release Date: February 8, 2007

Issued by
National Institute of Neurological Disorders and Stroke (NINDS), ( http://www.ninds.nih.gov )
National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS), ( http://www.niams.nih.gov )
National Institute of Child Health and Human Development (NICHD), ( http://www.nichd.nih.gov )
National Heart, Lung, and Blood Institute (NHLBI), ( http://www.nhlbi.nih.gov )

The Purpose of this Notice is to indicate the intent to issue a Request for Applications ( RFA) to continue the Senator Paul D. Wellstone Muscular Dystrophy Cooperative Research Centers Program and to alert potential applicants as to changes in the objectives and structure of this cooperative agreement program.

Policy

The National Institute of Neurological Disorders and Stroke (NINDS), the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) , the National Institute of Child Health and Human Development (NICHD), and the National Heart, Lung, and Blood Institute (NHLBI) intend to issue an RFA for new (type 1) or competitive renewal (type 2) applications for the support of Senator Paul D. Wellstone Muscular Dystrophy Cooperative Research Centers (Wellstone MDCRCs). The Wellstone MDCRCs funded through this program have contributed toward the goal of improving the detection, diagnosis, treatment, and prevention of the muscular dystrophies. The initial three Wellstone MDCRCs were funded in 2003. The RFA to continue the Wellstone MDCRCs is expected to be published in the Spring of 2007 with applications using the U54 Specialized Center Cooperative Agreement mechanism due in the Fall of 2007.

NINDS, NIAMS, and NICHD intend to fund up to three Wellstone MDCRCs. NHLBI will participate in support of meritorious cardiopulmonary research in successful applications. Grantees will join a network of existing Wellstone MDCRCs to foster the translation of new scientific findings and technological developments into novel treatments for the muscular dystrophies. As nationally recognized centers of excellence in muscular dystrophy, the Wellstone MDCRCs are also expected to play leadership roles by promoting communication and collaboration, by developing and sharing research resources, and by contributing to the training of new researchers for the muscular dystrophy field.

Research Objectives and Center Structure

To foster the translation of new scientific findings and technological developments into the clinical research setting, the NIH has modified the Wellstone MDCRC program. The goal of a Wellstone MDCRC is to elucidate the relevance of basic science to human disease. Appropriate activities address the steps required for therapeutic development, including therapeutic target (disease mechanism) identification and validation, development of diagnostics and biomarkers to characterize or stratify patient populations, in vitro assay development, animal model development, candidate therapeutic efficacy screening, preclinical therapeutic optimization and FDA-required activities leading to an investigational new drug (IND) application, natural history studies, clinical outcome measure validation, cohort characterization and other studies in support of clinical trials, and early stage clinical trials for one or more types of muscular dystrophy. Each MDCRC should involve patient-oriented research, preferably with direct interaction between the investigators and muscular dystrophy patients as research subjects or epidemiological or behavioral outcomes or health services research. Applicants also are encouraged to involve patients and patient advocates as advisors, through educational programs, or through referrals to other sites of clinical care and research.

Applicants are strongly encouraged to refer to the Muscular Dystrophy Coordinating Committee's Action Plan for the Muscular Dystrophies ( http://www.ninds.nih.gov/find_people/groups/mdcc/MDCC_Action_Plan.doc ) for potential research directions.

The minimum configuration of a Wellstone MDCRC includes:

• A Center Director and Co-Director, responsible for scientific and administrative oversight of the center.
• A minimum of four components, which can be projects or cores, including at least one research project and at least the three cores listed below.
• One project must involve substantial collaborations among investigators in a focused therapeutic development effort for muscular dystrophy. Cores should support these efforts.
• One project must involve patient-oriented research.
• An Administrative Core that promotes a center environment through enhanced communication and collaboration of investigators.
• One or more Scientific Research Resource Cores, at least one of which constitutes a unique resource or service for the national muscular dystrophy research community. Letters of support from potential users of this core should be included in the application to document how the core will fill a high priority need in the muscular dystrophy research community.
• A Research Training and Education Core to provide specialized training in translational and clinical research and stipend support for predoctoral and postdoctoral investigators, and to engage the patient and lay communities in educational and research activities.

At least one research project proposed in a Wellstone MDCRC application must address problems that require a substantial collaborative research effort, preferably involving teams comprised of both basic scientists and clinical investigators and/or investigators experienced in translational research. Applicants are strongly encouraged to propose fewer, scientifically comprehensive and collaborative projects, rather than a larger number of small projects that might otherwise be easily accomplished outside of the center mechanism. Collaborations should be arranged to bring the best expertise to bear on a problem, whether the proposed collaborations are all on-site or utilize consortium agreements with off-site investigators at existing Wellstone MDCRCs or off-site investigators not affiliated with a Wellstone MDCRC.

Applicants for Wellstone MDCRCs can request up to $1M per year in total direct costs, exclusive of F&A costs on consortium agreements. Up to three Wellstone MDCRCs will be funded through this FOA.

Inquiries

For further information regarding this upcoming FOA, please contact:

John D. Porter, Ph.D.
Channels, Synapses, and Circuits Cluster
National Institute of Neurological Disorders and Stroke
6001 Executive Boulevard., Room 2142
Bethesda , MD 20892
Telephone: (301) 496-1917
FAX: (301) 402-1501
Email: porterjo@ninds.nih.gov

Glen H. Nuckolls, Ph.D.
Director, Muscle Disorders and Therapies Program
Musculoskeletal Diseases Branch
National Institute of Arthritis and Musculoskeletal and Skin Diseases
One Democracy Plaza
6701 Democracy Boulevard, Suite 800
Bethesda , MD 20892-4872
Telephone: (301) 594-4974
Email: nuckollg@mail.nih.gov

James W. Hanson, M.D.
Director, Center for Developmental Biology and Perinatal Medicine
National Institute of Child Health and Human Development
6100 Executive Boulevard , Room 4A05A
Bethesda , MD 20892-7510
Telephone: (301) 496-8535
Email: hansonj@mail.nih.gov

Judith Massicot-Fisher, Ph.D.
Program Director
Heart Development and Structural Diseases Branch
National Heart, Lung, and Blood Institute
6701 Rockledge Drive , MSC 7940
Bethesda , MD 20892-7940
Telephone: (301) 435-0510
Email: massicoj@nhlbi.nih.gov