Tuesday, December 5, 2017

Teleconference Only


or 1-650-479-3208 Access code: 622 734 375

Meeting Agenda

1:00 pm

Welcome and Introductions
Stephen I. Katz, MDCC Chair

New ad hoc members:

  • Gustavo Dziewczpolski, Cure CMD
  • Grace Pavlath, MDA
  • Eugene Freund, CMS
  • Kimberly Richey, Dept of Education

Recognition of retiring public members:

  • Valerie Cwik, MDA
  • Peter Wald
1:15 pm Conflicts of interest policies and general meeting information
Glen Nuckolls, MDCC Executive Secretary
1:20 pm

Meeting Reports

2017 Parent Project Muscular Dystrophy Connect Conference
Brian Denger, PPMD

2017 Myotonic Dystrophy Annual Conference
John Porter, MDF

2017 Meeting of the Wellstone Muscular Dystrophy Centers
Tom Cheever, NIAMS

1:50 pm

MDCC Support for Action Plan Topics

Analysis of 2016 Funding Data
Glen Nuckolls, NINDS

Discussion of 2016 MDCC Funding Data
MDCC members and ad hoc members

2:20 pm

Selection of MDCC Chair for 2018 & 2019

Call for nominations and vote by MDCC members
Glen Nuckolls

2:30 pm

Meeting Adjourned







Meeting Summary


The Muscular Dystrophy Coordinating Committee (MDCC) met on December 5, 2017, via webinar/teleconference.  As Chair of the Committee, Stephen I. Katz, M.D., Ph.D., Director of the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS), led the meeting with Glen Nuckolls, Ph.D., MDCC Executive Secretary.  The entire meeting was held in open session in accordance with Public Law 92-463.

Welcome and introductions

Dr. Katz noted that Dr. Valerie Cwik’s (Muscular Dystrophy Association) and Dr. Peter Wald’s terms end this winter. He thanked them for their participation, particularly during the creation of the 2015 Action Plan, and he invited them to participate in future MDCC events. 

He noted that the committee has four new members: Dr. Gustavo Dziewczapolski (CureCMD), Dr. Eugene Freund (Centers for Medicare and Medicaid Services), Dr. Grace Pavlath (Muscular Dystrophy Association), and Ms. Kimberly Richey (Department of Education).

Meeting updates

Mr. Brian Denger, Parent Project for Muscular Dystrophy, summarized several conferences organized by PPMD during the past year. He highlighted the Inflammation in Duchenne workshop (June 2017), which had two main themes: the roles of inflammation and immunity in Duchenne muscular dystrophy, and immune responses to gene therapy, gene correction, and biologics being developed to treat the disease. He also described the Clinical Trial Design meeting (March 2017), during which researchers and advocates discussed the importance of standardizing outcome measures, implementing best practices in trial design, and exploring the potential for a master protocol for Duchenne and Becker clinical trials. Mr. Denger also noted the locations and times of annual Connect Conference and the EndDuchenne tour outreach meetings.  Most meeting summaries are available on PPMD’s website.

Dr. John Porter, Myotonic Dystrophy Foundation, summarized two connected meetings; the International Myotonic Dystrophy Consortium (IDMC) and the Myotonic Dystrophy Foundation Annual Meeting in San Francisco.  The IDMC was primarily attended by researchers with 350 attendees, 60 podium presentations, and 200 posters. Main areas of focus were targeting RNA with therapeutics, lessons learned from other RNA disorders, microsatellite expansion, and a call for increased focus on DM type2. The MDF annual meeting focused on the neurological aspects of DM. Its 650 attendees included researchers, patients, families, and clinicians.

Dr. Tom Cheever, NIAMS, gave an update from the Senator Paul D. Wellstone Muscular Dystrophy Specialized Research Centers meeting in October. This meeting occurs every other year.  The centers, which are funded by NIAMS, NINDS, NICHD, and NHLBI, conduct basic, translational, and clinical research on various forms of muscular dystrophies. On the first day of the meeting, each center Director provided an overview their center highlighting research projects, shared research resource cores, and training cores.  The second day of the meeting included many student and trainee presentations and a keynote presentation describing advances in gene therapy for spinal muscular atrophy by Dr. Jerry Mendel from the Research Institute of Nationwide Children’s Hospital.

MDCC Support for Action Plan Topics

Dr. Nuckolls presented an analysis of funding by MDCC member organizations in 2016 relative to topics of the 2015 Action Plan for the Muscular Dystrophies.  He thanked the agencies and patient organizations for providing the data that were used in the analysis and are available on the MDCC website.  Overall, 2016 funding across the Action Plan areas was approximately $109M, a slight increase from 2015.  Dr. Nuckolls gave an overview of how the data are coded, noting that disease and activity categories are coded “yes/no”, which allows for a count of the number of grants in that category.  Action Plan topics are coded by percentage, which allows for calculation of estimated level of funding for each topic.  Most grants are coded as “Understanding Causes” and “Therapy Development”, but Dr. Nuckolls anticipates that this may shift into categories such as “Clinical Trial Readiness” and “Screening/Diagnosis” as the MD research fields mature.  Private organizations and federal sources support a comparable number of awards, but the average dollar amount is higher for awards from federal funding organizations.  In 2016, 73% of funded independent muscular dystrophy researchers had only one grant; 12% had both private and federal funding.  The MDCC plans to continue to collect this funding information from member organizations and make it available to the public to monitor the status of the muscular dystrophy workforce and progress of the field.  Dr. Cwik asked about tracking these investigators over time because grants from patient groups are often awarded to investigators who have not yet received their first NIH grants. The committee will track investigator progress over time as it collects more data.

Selection of MDCC Chair for 2018 & 2019

This was Dr. Katz’s last meeting as Chair of the MDCC.  Dr. Katz mentioned that while it was his pleasure to chair this Committee for the past two years, it is important for leadership to rotate.  Dr. Nuckolls stated that the NIH had nominated Dr. Walter Koroshetz, Director of National Institute of Neurological Disorders and Stroke, as chair, and asked for any other nominations.  There were no nominations from any members.  A motion to nominate Dr. Koroshetz was made by Dr. Valerie Cwik and seconded by Mr. Dan Perez.  All members (8 standing members) voted yes.  Dr. Koroshetz stated that he looks forward to chairing the committee and commended Dr. Katz on his time as chair.

Public Comment

There were no public comments.


Committee members thanked Dr. Katz for his time as chair. Dr. Katz briefly summarized the meeting and thanked the presenters and the committee for their participation. 

Committee Roster
Muscular Dystrophy Coordinating Committee

In attendance

Chairperson –
Katz, Stephen I., PhD, MD
National Institute of Arthritis and Musculoskeletal and Skin Diseases
National Institutes of Health

Bianchi, Diana, MD (Ad Hoc)
Eunice Kennedy Shriver National Institute of Child Health and Human Development
National Institutes of Health

Cwik, Valerie, MD
Medical Director and Executive Vice President for Research
Muscular Dystrophy Association

Denger, Brian
Collaborative Programs Administrator
Parent Project Muscular Dystrophy

Hesterlee, Sharon, PhD
Evp Patient Advocacy and Public Affairs
Pfizer, Inc.

Kiley, James P., PhD
Airway Biology and Division of Lung Diseases
National Heart, Lung, and Blood Institute
National Institutes of Health

Koroshetz, Walter Joseph, MD
National Institute of Neurological Disorders and Stroke
National Institutes Of Health

Perez, Daniel Paul
President and Chief Executive Officer
Facioscapulohumeral Muscular Dystrophy Society, Inc.

Riley, Catharine, PhD, MPH (Ad Hoc)
Newborn Screening, Genetics Services Branch, Maternal and Child Health Bureau
Health Resources and Services Administration

San Agustin, Theresa B., MD
Program Manager
National Institute on Disability, Independent Living and Rehabilitation Research
Administration For Community Living

Street, Natalie, Ms (Ad Hoc)
Health Scientist
National Center for Birth Defects and Developmental Disability
Centers for Disease Control and Prevention

Freund, Eugene, Md (Ad Hoc)
Medical Officer

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