NIH Report on Support for Research on
Neuromuscular Diseases
Muscle Study Group
Sept. 22, 2010
Glen Nuckolls, PhD
Program Director for Muscle Diseases
National Institute of Arthritis and Musculoskeletal and Skin Diseases
National Institutes of Health
US Dept. of Health and Human Services
nuckollg@mail.nih.gov
Topics for Presentation
• NIH Funding for Neuromuscular Diseases
• Examples of Recently Funded Grants
• Funding Opportunities
• Changes in Peer Review
NIH Support for Neuromuscular Diseases
(Dollars in millions and rounded)
FY09
FY09
FY07
FY08
(non-ARRA)
(ARRA)
ALS
$40
$43
$43
$13
Myasthenia Gravis
$10
$9
$9
$3
Charcot-Marie-Tooth
$9
$12
$14
$2
Dystonia
$18
$15
$16
$2
Spinal Muscular Atrophy
$11
$10
$11
$3
Muscular Dystrophy
$58
$56
$66
$17
Duchenne/ Becker MD
$23
$22
$27
$6
Facioscapulohumeral MD
$3
$3
$3
$2
Myotonic Dystrophy
$9
$9
$9
$4
http://report.nih.gov
FY09 NIH Grants for the Muscular Dystrophies
Training/career
dev $2.2M
Wellstone Centers $8.7M
ARRA short-term
grants $8.0M
Multi-project grants $6.3M
Translational grants $8.5M
Small business $1.0M
Investigator initiated
single project grants $42.2M
Other $5.9M
$83M total
Wellstone Centers Awards
Pittsburgh
Rochester
Seattle
Penn/Hopkins
Iowa
Children’s National (DC)
North Carolina
Rochester
Boston
Penn
Iowa
Nationwide Children’s (OH)
2003
2005
2007
NIAMS
2009
NINDS
2011
NICHD
2013
NHLBI
2015
Wellstone Center Competition 2010
The Research Institute at
Nationwide Children’s Hospital
Jerry Mendell, Chris Walker, Zarife Sahenk, Paul Martin
Focus: Immune Response to Gene Therapy
University of Pennsylvania
Lee Sweeney, Elizabeth McNally, Melissa Spencer, Glenn Walter,
Krista Vandenborne, Beth Barton, Michael Ostap, Richard Finkel
Focus: Skeletal muscle and heart fibrosis in muscular dystrophies
The University of Iowa
Kevin Campbell, Steven Moore, Kathy Mathews, Carsten Bonnemann
Focus: Dystroglycan-related muscular dystrophies
www.wellstonemdcenters.nih.gov
Magnetic Resonance Imaging and
Biomarkers for Muscular Dystrophy
www.imagingdmd.org
PI: Krista Vandenborne
UF, Oregon HSU, CHOP, Penn
Longitudinal study of 100 boys with DMD
and 50 healthy controls
Lower limb MRI and MRS to evaluate
muscle structure and composition
Measures of muscle strength and physical
function
Monitor participants as they begin
standard corticosteroid treatment
Banking of immortalized fibroblasts to
facilitate subsequent genetic studies
RC3: Pre-Clinical and Clinical Evaluation of
Skeletal Muscle Activator, CK-2017357
for Myasthenia Gravis
PI: Fady Malik
CK-2017357 binds to the troponin complex and
sensitizes muscle to nerve stimulation, increasing
contractile force.
Aims:
Testing of pre-clinical efficacy in an AChR
immune rat model
Phase 2A clinical trial of single and multiple
doses in MG patients
Pre-Clinical Translational Research
NINDS
Exploratory/Developmental Projects in
Translational Research for Neuromuscular
Disease (R21) PAR-08-228
NIAMS
Cooperative Program in Translational Research
for Neuromuscular Disease (U01) PAR-08-229
Standard receipt dates through July 2011
Innovative Therapies and Tools for Screenable
Disorders in Newborns (R03,R21,R01)
PAR-10-230, PAR-10-231, PAR-10-232
NINDS
NICHD
SMA and DMD included
Pre-clinical, clinical studies and clinical trials
Standard receipt dates through Sept. 2013
Clinical Studies and Clinical Trials
NIAMS
Pilot and Feasibility Clinical Research Grants in
Arthritis and Musculoskeletal and Skin Diseases
(R21) PAR-10-282
Multiple receipt dates, next receipt Nov. 18, 2010
Clinical Studies of Safety and Effectiveness of
Orphan Products Research Project Grant (R01)
RFA FD-11-001
Two receipt dates: Feb. 2, 2011 and Feb. 1, 2012
NINDS Exploratory Clinical Trials (R01)
NINDS Phase III Investigator-Initiated Multi-Site
Clinical Trials (U01) PAR-10-198 & PAR-10-199
NINDS
Standard receipt dates through March 2013
Ancillary Studies
Ancillary Studies in Clinical Trials of CNS/PNS
Disorders NINDS Accelerated Awards Program (R01)
PAR-09-263
NINDS
Multiple receipt dates, next date is Dec. 16, 2010
Ancillary Studies to Large Ongoing Clinical Projects
(R01 & R21) RFAs AR-11-010 & AR-11-013
NIAMS
Letters of Intent Receipt Date(s): November 3, 2010; March 1,
2011
Peer Review Changes
Phase out of A2 applications almost complete
Guidance on making an application “new”
Clarification of individual criterion scores
Summary statement may report pre-discussion criterion
scores
Paragraph justification of impact/priority score
Limitations on post-submission application materials
(NOT-OD-10-115)
Elimination of error correction window (NOT-OD-10-123)
For questions and guidance about grant
applications and other NIH issues:
Glen Nuckolls
NIAMS
(nuckollg@mail.nih.gov)
John Porter
NINDS
(porterjo@ninds.nih.gov)