Department of Defense Duchenne Muscular Dystrophy Research Program (DMDRP)

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Wilfrid Warren
6 years ago
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1 Department of Defense Duchenne Muscular Dystrophy Research Program (DMDRP) Marielena McGuire, Ph.D. Program Manager Congressionally Directed Medical Research Programs The views expressed in this presentation are those of the authors and do not reflect official policy or position of the Department of the Army, Department of Defense or the U.S. Government.

2 Who is the CDMRP? DEPARTMENT OF DEFENSE DEPARTMENT OF THE ARMY ARMY MEDICAL COMMAND MEDICAL RESEARCH AND MATERIEL COMMAND CONGRESSIONALLY DIRECTED MEDICAL RESEARCH PROGRAMS U.S. Army Medical Research and Materiel Command 2

3 A Major Funder The CDMRP is THE leading U.S. funder for Neurofibromatosis research The CDMRP is the 2nd largest U.S. funder for research in: Breast cancer Gulf War Illness Lung cancer Ovarian cancer Prostate cancer Tuberous sclerosis U.S. Army Medical Research and Materiel Command 3

Scope of CDMRP Funding 700 *Applications received since 1992 = 85,877 *Applications funded since 1992 = 11,419 600 500 * 400

Medical R & D Spinal Cord Injury Prostate Cancer Psychological Health/Traumatic Brain Injury Neurofibromatosis Ovarian Cancer

4 Scope of CDMRP Funding 700 *Applications received since 1992 = 85,877 *Applications funded since 1992 = 11, * 400 Millions ($) Breast Cancer Peer Reviewed Medical Institutionally Based Programs Peer Reviewed Orthopaedic Defense Medical R & D Spinal Cord Injury Prostate Cancer Psychological Health/Traumatic Brain Injury Neurofibromatosis Ovarian Cancer Deployment Related Medical Peer Reviewed Cancer *Amount of ARIF/ DMRDP/PH/TBI approximated U.S. Army Medical Research and Materiel Command 4

Hallmarks of the CDMRP t Research funds added to DoD budget by

yearly, award mechanisms changed as needed t Consumer

high-impact research t Two-tier formal review of applications

funding agencies Fills Unfunded/Unmet Gaps and Niches t

5 Hallmarks of the CDMRP t Research funds added to DoD budget by Congress (not part of DoD s request) t Vision is adapted yearly, award mechanisms changed as needed t Consumer advocates participate process t Fund highly innovative, high-impact research t Two-tier formal review of applications Institute of Medicine model t Avoid Duplication with other funding agencies Fills Unfunded/Unmet Gaps and Niches t Employs Collaboration and Synergy throughout U.S. Army Medical Research and Materiel Command 5

Advocates, Consumers, and the CDMRP t Almost 2000

have served on Integration and peer review panels

.. t Contribute unique personal and community

sense of urgency t Ensure that human dimensions

considerations Program policy Investment strategy

understanding of the scientific process back to

6 Advocates, Consumers, and the CDMRP t Almost 2000 consumers representing over 900 organizations have served on Integration and peer review panels Panel members... t Contribute unique personal and community experiences t Add perspective, passion, and a sense of urgency t Ensure that human dimensions of disease are incorporated into: Scientific considerations Program policy Investment strategy Research focus t Take information and a greater understanding of the scientific process back to community Provide hope to survivors and family members 6 U.S. Army Medical Research and Materiel Command 6

7 Program Execution U.S. Army Medical Research and Materiel Command 7

8 Goal of Two-Tier Review Process To find scientifically meritorious proposals and fund those that best fulfill program goals Peer Review Panels Partnership Integration Panel Criterion-based evaluation of full proposal Determination of absolute scientific merit Outcome: Written critique and scores for individual criteria and overall merit Comparison among proposals of high scientific merit Determination of adherence to intent and program relevance Outcome: Funding recommendation U.S. Army Medical Research and Materiel Command 8

Duchenne Muscular Dystrophy Research Program Vision: To extend and improve the function, quality of life, and life span for all individuals diagnosed with DMD Mission: To better inform the FY13: $3.

2M 8 awards development of drugs, devices, and other interventions and promote their effective clinical testing Focus Areas Preclinical research for therapeutic development Clinical biomarkers to

9 Duchenne Muscular Dystrophy Research Program Vision: To extend and improve the function, quality of life, and life span for all individuals diagnosed with DMD Mission: To better inform the FY13: $3.2M FY11-12:$7.2M 8 awards development of drugs, devices, and other interventions and promote their effective clinical testing Focus Areas Preclinical research for therapeutic development Clinical biomarkers to improve evaluation of diagnosis, disease severity, disease progression, and/or response to treatment Assessment of clinical trial outcomes Novel interventions to improve clinical care and quality of life in the near term Investigator-Initiated Research Award: Supports mid-stage or later translational research projects, including earlyphase, proof-of-principle clinical trials and correlative studies that will accelerate the movement of promising ideas in DMD into clinical applications. $525,000 Period of performance 3 years Optional Nested Resident or Medical Student Traineeship U.S. Army Medical Research and Materiel Command 9

10 DMDRP Portfolio FY11 A Cox-Inhibiting NO Donor to Counteract Functional Muscle Ischemia in Duchenne/Becker Muscular Dystrophy PI: Gail Thomas Preclinical research for therapeutic development (2) Biomarkers Research (1) Optical Imaging of Dystrophic and Damaged Muscle PI: Glenn Walter Translational Studies of GALGT2 Gene Therapy for DMD PI: Paul Martin Assessment of clinical trial outcomes (1) New Clinical Outcome Measurements PI: Avital Cnaan U.S. Army Medical Research and Materiel Command 10

11 DMDRP Portfolio FY12 t Elizabeth McNally, M.D., Ph.D. Preclinical Testing of a Novel Method to Block TGF beta Family Proteins in DMD t Barry Byrne, M.D., Ph.D. Advanced Gene Therapy for Treatment of Cardiomyopathy and Respiratory Insufficiency in DMD t Jill Rafael-Fortney, Ph.D. Optimization of Renin-Angiotensin-Aldosterone Inhibitors as a Treatment for DMD t Carmen Bertoni, Ph.D. A Read-Through Drug for Duchenne Muscular Dystrophy U.S. Army Medical Research and Materiel Command 11

12 Other DoD Supported Muscular Dystrophy Research t Clinical Trials Coenzyme Q10 and Lisinopril in Muscular Dystrophies (Paula Clemens, M.D.) t Resources Mouse Drug Screening Core at CNMC (Kanneboyina Nagaraju, Ph.D.) Jackson Laboratory Repository of muscular dystrophy mouse models CINRG infrastructure (Avital Cnaan, Ph.D.) t Therapeutics AON exon skipping (Qi Lu, Ph.D., Eric Hoffman, Ph.D.) Gene therapy (Jerry Mendell, M.D.) Muscle-derived stem cell therapy (Johnny Huard, Ph.D.) Non-hormonal steroid ligands (VBP15; Reveragen) Osteopontin (Melissa Spencer, Ph.D., Kanneboyina Nagaraju, DVM, Ph.D., Yi- Wen Chen, DVM, Ph.D.) U.S. Army Medical Research and Materiel Command 12

13 A Proud Advocacy Legacy Thank you for helping to provide Hope 13 U.S. Army Medical Research and Materiel Command 13

Peer Reviewed Alzheimer s Research Program (PRARP)

Peer Reviewed Alzheimer s Research Program (PRARP) Col Wanda L. Salzer, USAF Director, CDMRP Anthony Pacifico, Ph.D. Program Manager, PRARP Disclaimer The information in this presentation is the opinion