May 2, 2019

DEPARTMENT OF HEALTH AND HUMAN SERVICES
PUBLIC HEALTH SERVICE
NATIONAL ARTHRITIS AND MUSCULOSKELETAL AND SKIN DISEASES ADVISORY COUNCIL

MINUTES OF THE 97th MEETING
February 5, 2019
8:30 a.m. to 3:00 p.m.

February 5, 2019 Council Webcast

I.   CALL TO ORDER

The 97th meeting of the National Arthritis and Musculoskeletal and Skin Diseases Advisory Council (NAMSAC) was held on February 5, 2019, at the National Institutes of Health (NIH) Campus, Building 45, Conference Rooms E1/E2. The meeting was chaired by Dr. Robert H. Carter, Acting Director, National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS).

Attendance

Council members present:

Dr. Joan E. Bechtold, Professor, Orthopaedic Surgery, University of Minnesota
Dr. Jill P. Buyon, Director, Division of Rheumatology, Department of Medicine, New York School of Medicine, and Director, NYU Lupus Center
Mr. Vincent Del Gaizo, patient advocate and founding member and past chair, Friends of Childhood Arthritis and Rheumatology Research Alliance
Dr. Michael Econs, Glenn W. Irwin, Jr., Professor of Endocrinology and Metabolism; Director, Division of Endocrinology and Metabolism; and Professor of Medicine and Medical and Molecular Genetics, Indiana University School of Medicine
Dr. James Elbaor, Director, American Institute Orthopedic Surgery and Sports Medicine (via telephone)

Dr. Michael V. Holers, Professor, Department of Medicine, University of Colorado Denver School of Medicine
Dr. Judith A. James, Vice President of Clinical Affairs, Oklahoma Medical Research Foundation
Dr. Sundeep Khosla, Dr. Francis Chucker and Nathan Landow Research Professor; Director, Mayo Clinic CTSA/Center for Clinical and Translational Science; Dean for Clinical and Translational Science, Mayo Clinic College of Medicine
Dr. Ethan A. Lerner, Associate Professor of Dermatology, Massachusetts General Hospital
Ms. Rosemary J. Markoff, Co-Chair, Scleroderma Foundation National Advocacy Committee
Mr. William J. Mulvihill, The Mulvihill Advisory Group
Dr. Anthony Oro, Eugene and Gloria Bauer Professor of Dermatology; Associate Director and Professor, Center for Definitive and Curative Medicine, Department of Dermatology, Stanford University School of Medicine
Dr. Amy Paller, Professor of Dermatology, Northwestern University Feinberg School of Medicine
Mr. Richard F. Seiden, Foley & Lardner, LLP
Dr. Stephen J. Tapscott, Professor, Fred Hutchinson Cancer Research Center
Dr. Michael J. Yaszemski, Professor, Orthopaedic Surgery and Biomedical Engineering, Mayo Clinic

 

Staff and Guests

The following NIAMS staff and guests attended:

Staff
 

Dr. D. Lee Alekel
Ms. Pamela Beheler
Dr. Alexey Belkin
Cpt. Robyn Bent
Ms. Elizabeth Bouras
Dr. Amanda Boyce
Dr. Nakia Brown
Dr. Stephanie Burrows
Ms. Justine Buschman
Ms. Cindy Caughman
Dr. Thomas Cheever
Ms. Robin DiLiello
Ms. Teresa Do
Dr. Jonelle Drugan
Ms. Colleen Dundas
Mr. Erik Edgerton
Dr. Yasuko Furumoto
Dr. Nancy Garrick
Ms. Aleisha James
Ms. Katie Joffee
Ms. Shahnaz Khan
Dr. Anthony Kirilusha
Ms. Stephanie Kreider
Ms. Jane Lee

Dr. Gayle Lester
Dr. Helen Lin
Dr. Kan Ma
Dr. Marie Mancini
Dr. Katy Marron
Ms. Melinda Nelson
Dr. Kristy Nicks
Ms. Nicole Nyack
Dr. Heiyoung Park
Mr. Rick Phillips
Ms. Reaya Reuss
Mr. Neil Roberts
Dr. Kathy Salaita
Ms. Sheila Simmons
Dr. Asur Srinath
Ms. Robyn Strachan
Ms. Yen Thach
Ms. Jamie Thompson
Dr. Hung Tseng
Dr. Fei Wang
Dr. Xibin Wang
Dr. Charles Washabaugh
Dr. Ted Zhang

 

Guests 

Dr. Alan Beggs, Boston Children’s Hospital (via phone)
Dr. Walter Koroshetz, Director, National Institute of Neurological Disorders and Stroke, NIH
Dr. Michael Lauer, Deputy Director for Extramural Research, NIH
Mr. Glen Nuckolls, National Institute of Neurological Disorders and Stroke, NIH
Dr. James Ostell, Director, National Center for Biotechnology Information, National Library of
Medicine, NIH
Dr. Danuta Krotoski, Eunice Kennedy Shriver National Institute of Child Health and Human Development, NIH


II.   CONSIDERATION OF MINUTES

A motion was made, seconded, and passed to approve the minutes of the 96th NAMSAC meeting, held on September 5, 2018.


III.   FUTURE COUNCIL MEETING DATES

Future Council meetings are currently planned for the following dates:

June 5, 2019
September 10, 2019
February 4, 2020
June 9, 2020
September 1, 2020


IV.   IN REMEMBRANCE OF DR. STEPHEN I. KATZ, M.D., PH.D.

Dr. Carter led the Advisory Council in remembering the life and legacy of NIAMS Director Dr. Stephen Katz, who passed away recently after a sudden illness. Advisory Council members shared their memories of Dr. Katz, whose impact over decades of service to NIAMS and the NIH cannot be overstated.

V.   DIRECTORS REPORT

Budget and Congressional Activities

Dr. Carter began the Director’s Report by providing an update on NIAMS’ budget.  For the first time in 22 years, the Department of Health and Human Services (and, by extension, NIAMS) began the fiscal year with full-year budget in place. NIH as a whole saw a 5.4% increase in its budget, or approximately two billion dollars. A significant portion of this increase was allocated to specific programs and initiatives, such as the BRAIN Initiative, the Cancer Moonshot, and efforts to address the opioid crisis. NIAMS received a 3.1% budget increase, from $586.7 million to $605.1 million. NIH also was fortunate to avoid being directly affected by the recent government shutdown.

Dr. Carter presented NIAMS’ preliminary funding plan for FY 2019. Dr. Carter acknowledged the somewhat conservative stance of the plan, which is partially because Dr. Carter wanted to leave some flexibility in the budget for the new Director.

NIAMS is continuing to work on its next five-year strategic plan. NIAMS has received feedback on the plan’s content through a Request for Information and several stakeholder meetings.  NIAMS intends to have a draft strategic plan ready for Council to review prior to the next Advisory Council meeting. Dr. Carter has asked several Council members to serve on a working group that will review the draft strategic plan and provide feedback to the full Council. After the Institute receives the feedback, it will develop a second draft that will go out for public comment during the summer. Further changes will be incorporated into a final draft that NIAMS will submit to the Council at its September meeting.

Dr. Carter discussed the NIAMS-led NIH Back Pain Consortium (BACPAC) research program, which is part of NIH’s HEAL (Helping to End Addiction Long-term) initiative that has been developed to address the ongoing opioid epidemic in America. Four BACPAC Funding Opportunity Announcements (FOAs) have been issued related to new interventions, technologies, and clinical trials. The goal of the FOAs is to help better understand changes related to lower back pain and what the mechanisms and drivers of pain are in its early stages. Ultimately, the goal is to integrate findings into a more comprehensive model of lower back pain that inform longitudinal observational cohort studies and multimodal randomized clinical trials. This process has been fast-tracked; the funding decisions are to be made before the end of FY 2019.

Dr. Carter provided an update on the Pathways to Prevention workshop on “Appropriate Use of Drug Therapies for Osteoporotic Fracture Prevention”, which was held in October. The workshop panel’s draft report is currently available for public comment. NIAMS, the National Institute on Aging, and the NIH Office of Disease Prevention will hold a federal partner’s meeting this summer with other NIH and HHS components to discuss opportunities for collaboration and possible implementation of the panel’s recommendations.

Dr. Carter briefly described the NIH Advisory Committee to the Director’s (ACD) Working Group for Foreign Influences on Research Integrity and its subsequent report and recommendations. The working group was convened in light of recent issues related to unreported external funding sources and potential negative impacts on research integrity. The working group’s recommendations called for improved communication and increased awareness, development of risk mitigation practices, and increased monitoring.

The ACD also recently convened an Artificial Intelligence (AI) Working Group that has been tasked with exploring ways NIH can best harness AI and machine learning to advance biomedical research. Interim recommendations are expected from the working group this summer.
 

Personnel Changes

Dr. Carter briefed the Advisory Council on recent personnel changes at the NIH and NIAMS. At the NIH level:

  • Dr. Bruce Tromberg has been announced as the new Director of the National Institute of Biomedical Imaging and Bioengineering (NIBIB). Dr. Tromberg comes to NIH from the University of California-Irvine, where he held dual appointments in biomedical engineering and surgery and was Director of the Laser Microbeam and Medical Program at the Beckman Laser Institute and Medical Clinic. His research focused on the use of optics for imaging in biology and medicine and had previously been involved in NIAMS-funded research.

At the NIAMS level:

  • Mr. Chris Nee has been named NIAMS’ new Budget Officer. He comes to NIAMS from the National Human Genome Research Institute (NHGRI), where he served as Deputy Budget Officer.
  • Dr. Alexey Belkin is a new Program Officer on the Skin Biology and Diseases Team who previously worked in the NIH Center on Scientific Review (CSR) as the Scientific Review Officer for the Arthritis, Connective Tissue and Skin Sciences (ACTS) Study Section.  
  • Dr. Anthony Kirilusha joins NIAMS as the new Program Officer on the Joint Biology, Joint Diseases, and Orthopedics Team. Dr. Kirilusha previously worked at NHGRI in their intramural research program.
  • Dr. Xibin Wang has been named Program Officer for the Special Programs Team, which includes Training and SBIR/STTR Programs. 
  • Ms. Stephanie Hazlett from the American Academy of Orthopaedic Surgeons has completed her two-year term as co-chair of the NIAMS Coalition. Dr. Carter thanked her for her service and announced that Mr. Blake McDonald, from the American Academy of Dermatology, will be the new co-chair.

Highlights of Selected Recent Scientific Advances

Dr. Carter mentioned three recent scientific publications:

  • Dr. Betty Diamond and colleagues at the Feinstein Institute for Medical Research discovered that a subset of lupus anti-DNA antibodies, termed DNRAbs, cross react with the N-methyl-d-aspartate receptor (NMDAR) and enhance NMDAR signaling. Research shows that activated microglia and C1q are critical mediators of neuronal damage. Dr. Diamond’s research found that centrally acting inhibitors of angiotensin-converting enzyme (ACE) can prevent microglial activation and preserve neuronal function and cognitive performance. This indicates ACE inhibition represents a strong candidate for clinical trials aimed at mitigating cognitive dysfunction.
  • Dr. Bradley Olwin and his team at the University of Colorado Boulder recently published a paper in Nature called “TDP-43 and RNA form amyloid-like myo-granules in regenerating muscle.” Their research shows that TDP-43 is an essential protein for normal skeletal muscle formation that unexpectedly forms cytoplasmic, amyloid-like oligomeric assemblies, which the research team has dubbed myo-granules, during regeneration of skeletal muscle. Although myo-granules occur during normal skeletal-muscle regeneration, myo-granules can seed TDP-43 amyloid fibrils in vitro and are increased in a mouse model of inclusion body myopathy. Therefore, the research suggests increased assembly or decreased clearance of functionally normal myo-granules could be the source of cytoplasmic TDP-43 aggregates that commonly occur in neuromuscular disease.

Dr. Maria Morasso and her colleagues in the NIAMS IRP released a paper called “Transcriptional signature primes human oral mucosa for rapid wound healing,” which describes a comparative analysis between human oral and cutaneous wound healing using paired and sequential biopsies during the repair process. The study showed that wound-activated transcriptional networks are present at basal state in the oral mucosa, priming the epithelium for wound repair. Oral mucosal wound-related networks control epithelial cell differentiation and regulate inflammatory responses, highlighting fundamental global mechanisms of repair and inflammatory responses in humans. In addition, the research indicated that show that SOX2 and PITX1 transcriptional function has the potential to reprogram skin keratinocytes to increase cell migration and improve wound resolution in

VI.   INNOVATIONS IN PUBMED AND PUBMED CENTRAL

Dr. Carter introduced Dr. James Ostell, the Director of the National Center for Biotechnology Information (NCBI) at the National Library of Medicine (NLM). Throughout of his career, Dr. Ostell has been involved in the development of many of the online publication and research databases used by the medical research community.

Dr. Ostell began by emphasizing the increased importance of data and access to data in the current biomedical research environment. PubMed, which is hosted and maintained by the NLM, is visited by approximately 5.5 million users a day and at peak times receives 7,000 web hits a second. More often than not, these numbers make PubMed the most heavily trafficked website in the entire federal government. PubMed is known as repository for journal articles and
publications, but has been recently focusing on storing data as well, which has been one of the goals of the Scientific Data Council.

Dr. Ostell gave a brief overview of PubMed and PubMed Central. PubMed provides mainly titles and abstracts, while PubMed Central contains the full articles. PubMed Central can include supplementary data, data availability statements, and data citations. PubMed Central entries often end up populating PubMed, and efforts are underway to eventually merge the two sites from the user perspective. To support this effort, and to make the data more easily accessible in a user-friendly manner, NCBI is working on a new PubMed website based on modern standards for usability. The new site is currently called PubMed Labs and it can be accessed now in its beta form via Google search. Later in 2019, the new PubMed site will officially launch taking into account the usability and user preference data gleaned from the beta period.

As part of the general push towards improved access to data and increased usability, Dr. Ostell’s staff has been working to improve data citation through new citation guidance to researchers. The team is also working to link publications to datasets in the bibliography section. PubMed Central now has an associated data section which has already led to an increase of downloads of supplementary data. The new site will also have a focus on data citations in the references section. An ongoing focus will be to ensure consistent data between resources.

Discussion

Dr. James asked about the potential inclusion of clinical data. Dr. Ostell acknowledged that that is huge question in the field. One issue is that the quality of clinical data can be mixed and medical record information can be difficult to compare across sources. Data from longitudinal and interventional studies get stored in the Database of Genotypes and Phenotypes (dbGaP) but its use is limited to the quality of the data submitted. Dr. Ostell also discussed the issue of narrow consents limiting data usage. Dr. Econs noted that deidentification can be impossible for rare diseases. He also expressed concerns that future technological developments are not adequately addressed in current consent documents and raised issues related to the potential impacts of commercial genotyping. The community needs to be vigilant about these issues going forward, perhaps by limiting access to legitimate researchers. Dr. Ostell argued that the expectation of privacy may be a thing of the past given the current technological landscape of smartphones, the internet, and data and visual surveillance. He used credit cards as an analogy: we give out the number every day to vendors; what protects us isn’t keeping our number secret, it’s the laws in place against misuse and protections provided by the credit card companies. Dr. Ostell argued that this is the state of affairs in healthcare now too. One potential option to limit improper use would be to create a licensing system for access to the federally-hosted data.

 

VII.     2020 NIAMS POTENTIAL INITIATIVES

 Dr. Gayle Lester, Acting Director of the Division of Extramural Research, presented the FY 2020 NIAMS extramural funding initiatives to the Council for their information. Dr. Lester noted that these are all re-issues while FY 2021 initiatives will likely address new scientific topics.

  • Small Business Innovation Research on Rare Musculoskeletal, Rheumatic and Skin Diseases (Leads: Drs. Ricardo Cibotti and Xibin Wang). This initiative would encourage Small Business Innovation Research grant applications from small business concerns that propose translational studies to advance the development of products for diagnosis, treatment, and prevention of rare musculoskeletal, rheumatic or skin diseases.
  • Mechanistic Ancillary Studies to Ongoing Clinical Projects (Lead: Dr. Heiyoung Park). This initiative would encourage applications that propose time-sensitive mechanistic ancillary studies related to the NIAMS mission in conjunction with ongoing clinical projects. The ongoing “parent” project would have to be a clinical study that can provide a sufficient cohort of well-characterized patients, infrastructure, data, and biological samples. Applications submitted in response to this initiative would undergo an accelerated review and award process.
  • Limited Competition: Small Grant Program for NIAMS K08 and K23 Recipients (Lead: Dr. Su Yau-Mao). This initiative would continue the NIAMS small grant program for investigators to develop preliminary data for their first R01 application. The program would provide NIAMS-supported K08 and K23 recipients the opportunity to apply for support in the second to fourth year of their K awards. 

NIAMS Clinical Trial Planning Grant (Lead: Dr. Tom Cheever). This initiative would provide the necessary administrative and planning support for investigator-initiated clinical intervention clinical trials. Completion of the required milestones of a planning grant is a prerequisite for acceptance of a U01 clinical trial application, which supports the implementation and conduct of the study. Consultation with NIAMS staff would continue to be strongly encouraged prior to the submission of the planning grant

VIII.   UPDATE ON THE NIH NEXT GENERATION RESEARCHERS' INITIATIVE

Ms. Shahnaz Khan presented an overview of the NIH policy and guidelines on the inclusion of women and minorities as subjects in clinical research, and then discussed the triennial adherence report mandated under the policy.

Ms. Khan briefly discussed the origin of the inclusion policy, which can be traced back to the 1993 NIH Revitalization Act. The following year, the NIH implemented its inclusion guidelines, the underlying goal of which is to include individuals in clinical research in a manner that is appropriate to the scientific question under study. In 2001, the policy was amended with four major updates: the addition of a definition of clinical research, updating of the racial and ethnic categories, clarification for NIH-defined Phase III trials, and language regarding the role and responsibilities of NIH staff and the extramural community. The policy was again amended as part of the 21st Century Cures Act of 2016, under which Phase III trials are now mandated to submit results of valid analyses to clinicaltrials.gov and a triennial report is required (instead of biennial).

The 21st Century Cures Act also established the Inclusion Across the Lifespan policy, which is an update to the Inclusion of Children as Participants in Clinical Research policy. The new policy requires individuals of all ages to be included in clinical research studies unless there is a scientific or ethical justification, clarifies potential justifications for age-based exclusion criteria, requires participant age at enrollment to be provided in progress reports, mandates deidentified individual-level participant data, and allows for age at enrollment to be reported in units ranging from hours to years. This policy is effective for grants submitted for due dates on or after January 25, 2019.

Ms. Khan next discussed some of the ways NIAMS monitors adherence to the inclusion policy at the extramural level. One of the primary ways is through peer review, during which scientific review officers instruct peer reviewers to evaluate plans for inclusion by sex, gender, race, and ethnicity. Proposals that are lacking acceptable inclusion plans cannot be funded until appropriate plans are in place. Once studies are funded, NIAMS monitors compliance with the proposed inclusion plan via review of enrollment reports. This data is compiled as part of the triennial report to Congress certifying compliance to the policy, which is ultimately prepared by Office of Research on Women’s Health (ORWH) on behalf of the NIH Director.

Ms. Khan discussed the role of the Advisory Council under the policy, which is to review the aggregate IC data every three years and raise any questions or concerns. The Council then certifies or approves that NIAMS has complied with provisions of the statute, and then the NIAMS Director reviews the report and certifies Council approval.

Finally, Ms. Khan presented the total participant enrollment numbers for NIAMS over the last three years. The data shows a decrease in 2018, but year-to-year fluctuations of this type are to be expected due to the nature of clinical research.

Discussion

Dr. James asked how the 30% minority participation number compares to other ICs at the NIH. Ms. Khan said the full report for NIH will be published in April and she’ll be happy to forward that report to the Council when it becomes available.

The Advisory Council voted unanimously to approve the triennial inclusion report.

X.   WELLSTONE CENTERS EVALUATION

Dr. Carter invited Dr. Joan Bechtold to present the evaluation report to the Council. NIH has funded the Paul D. Wellstone Muscular Dystrophy Research Centers since it established the program in 2003. Six active Centers—with support from NIAMS, the National Institute of Neurological Disorders and Stroke (NINDS), the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD), and the National Heart, Lung, and Blood Institute (NHLBI)—comprise the program. Although NIH has refined the program over the past 15 years, NIH determined that a comprehensive review of the Wellstone Centers program was appropriate because of significant changes in the neuromuscular disease research landscape since 2003. The NIH convened a Working Group of the NIAMS Advisory Council that reviewed data collected from various sources including Wellstone Center progress reports, NIH administrative data, information extracted from publications and Wellstone Center websites, interviews with principal investigators and other stakeholders, and a Request for Information that was advertised to the broad research and patient advocacy communities.

The Muscular Dystrophy Community Assistance Research and Education Amendments of 2001, also known as the MD-CARE Act, directed NIH to expand, intensify, and coordinate research activities for the muscular dystrophies, established the interagency Muscular Dystrophy Coordinating Committee, and called for Centers of Excellence for research on muscular dystrophies. These latter entities were created and later renamed in honor of Paul D. Wellstone, the late Senator from Minnesota, who had championed the MD-CARE Act and other muscular dystrophy-related research. 

NIH has issued eight Funding Opportunity Announcements (FOAs) since 2003. The goal has been to have six Centers active at any given time with three Centers being the focus of each funding cycle. To date, NIH has funded 11 distinct Wellstone Center awards. NIAMS, NINDS, and NICHD each fund two Centers, while NHLBI provides additional support for mission-relevant projects. Funding obligations amount to approximately $1 million in direct costs per award year. Per the 2015 FOA, each Center is required to fund at least two projects, to maintain research resource cores, to provide a research training and education core, and to maintain an administrative core to coordinate efforts and engage patients and/or public.

Dr. Bechtold discussed the objectives of the Wellstone Evaluation Working Group. These were to identify best practices for achieving the Wellstone Centers goals of:

  • Supporting impactful basic, preclinical translational, and clinical research in the muscular dystrophies through synergistic projects;
  • Developing and broadly distributing resources that accelerate muscular dystrophy research;
  • Facilitating the training of the next generation of muscular dystrophy researchers and clinical scientists; and
  • Enabling connections with the patient community.

The Working Group held its first meeting in June 2018 and submitted its proposed recommendations in November 2018. The Working Group broke its recommendations down into several categories: research portfolio, structure and implementation, community engagement, research resources, and training.

For the research portfolio, the Working Group recommendation that NIH:

  • Continue to support a Wellstone Centers program that covers a range of diseases, including the rarer forms of muscular dystrophy.
  • Expand the scope of the Wellstone Centers program to encompass muscle diseases with related pathophysiological mechanisms and similar clinical manifestations.
  • Maintain a balance between clinical and preclinical research.
  • Strongly encourage Wellstone Centers to pursue research activities to advance clinical trial readiness, including the development or leveraging of natural history studies and linkages to patient registries.

Regarding structure and implementation, the Working Group recommended that NIH:

  • Maintain flexibility regarding the extent to which individual Center personnel should be involved in multiple Center projects and shared resource cores.
  • Continue to require that each Center contain at least one clinical research project while allowing the Centers to have flexibility regarding the type of project.
  • Encourage Centers to seek out opportunities for interdisciplinary collaborations and to partner with external scientists with complementary expertise.
  • Work closely with Wellstone Center principal investigators (PIs) to plan the Wellstone Center biennial meetings so that they include non-Wellstone PIs and other MD stakeholders who could add expertise or perspective.

For community engagement, the Working Group recommended that NIH:

  • Urge Centers to listen to the concerns of the patient community and to clearly communicate the role of and opportunities for the patient community to collaborate with the Centers.
  • Encourage Centers to bring groups together to advance shared scientific interests.
  • Encourage Wellstone PIs to strengthen interactions with the broader muscular dystrophy patient advocacy stakeholder community on both a national and a local level.
  • Encourage Centers to work with patient groups to develop appropriate patient-reported outcomes (PROs).

In terms of research resources, the Working Group recommended that NIH:

  • Require shared scientific research resource cores to maintain an active website that clearly lists resources, the process by which the resource can be obtained (e.g., a standard form), how requests are prioritized, resource cost, and potential wait times.
  • Ensure that Centers make preclinical research resources from research cores and from individual projects broadly available.
  • Highlight the importance of sharing clinical research resources either through research resource cores or as part of a scientific project.

In regards to training, the Working Group recommended that NIH:

  • Continue to require that Wellstone training cores provide stipend and salary support to trainees.
  • Encourage Centers to organize training activities that could be utilized by the entire muscular dystrophy scientific community.
  • Continue to encourage Centers to leverage existing training resources and financial support through their home institutions.
  • Encourage Centers to periodically convene meetings for trainees at the level of the Wellstone network.
  • Encourage Centers to expose non-clinical trainees at all levels (i.e., graduate students and postdoctoral fellows) to clinical aspects of muscular dystrophy research.
  • Encourage participation of clinical fellows, residents, and other clinicians on Wellstone projects and activities.

Discussion

Dr. Carter opened discussion by emphasizing the importance of the outstanding science that has been produced by the Wellstone Centers, in particular noting the work on dystrophin.

Dr. Tapscott talked about his involvement with the Wellstone Centers and similarly lauded their contribution to the advancement of science in the field of muscular dystrophy. He noted their work on developing pre-clinical models, establishing the scientific basis for oligonucleotide-based therapeutic trials for myotonic dystrophy, determining the molecular basis of facioscapulohumeral dystrophy, and creating a strong national network of institutions with muscular dystrophy expertise.

Dr. Walter Koroshetz, Director of NINDS, asked whether the Working Group considered definitional issues around what types of muscular disorders should fall under the purview of the Wellstone Centers. Dr. Bechtold invited Dr. Alan Beggs, chair of the Wellstone Evaluation Working Group, to respond to this question. Dr. Beggs said the existing definitions are based on clinicopathological definitions. As more is learned about the molecular basis of muscular disorders, some overlap is being discovered between conditions considered to be muscular dystrophy and some that are not. The Working Group’s view was that the core muscular dystrophies share a common pathophysiological mechanism that is often related to the underlying genetics.

Dr. Koroshetz noted that funding has not increased for the Wellstone Centers in 15 years and asked whether the Centers of Excellence might need further resources. Dr. Beggs acknowledged that has been an issue and was a subject of discussion in the Working Group. It is hard to dispute the fact that the purchasing power of $1 million has decreased in 15 years.

Dr. Khosla asked whether the Wellstone Centers were originally intended to spur new research or if it was intended to be an indefinite program. Dr. Koroshetz said the purpose of the evaluation was partly to see if the Centers were continuing to produce valuable science.

Dr. Holers asked, given the interest in orphan diseases and external funding and the need to develop patient resources, if there was discussion to use the intersection of industry, therapeutics and existing funding mechanisms to promote therapeutic development. Dr. Bechtold said that issue was discussed briefly and partly expressed in the Working Group encouraging collaboration with other muscular dystrophy research centers and the sharing of data.

Dr. Carter led the Advisory Council in thanking the Working Group for their great work on the evaluation report and expressed the sense of the Council to accept the report.

XI.   UPDATE ON THE RESTRUCTURING OF THE NIAMS EXTRAMURAL PROGRAM

This portion of the meeting occurred during closed session.

XII.   STAR AWARDS DISCUSSION

This portion of the meeting occurred during closed session.

XIII.   SPECIAL ACTIONS

This portion of the meeting occurring during closed session.

XIV. CONSIDERATION OF APPLICATIONS

In closed session, the Council reviewed and recommended a total of 927 primary and secondary applications.  The total cost requested in year -01 for all applications was $345,144,240.

XV.   ADJOURNMENT

The 97th National Arthritis and Musculoskeletal and Skin Diseases Advisory Council Meeting was adjourned at 3:00 p.m. Proceedings of the public portion of this meeting are recorded in this summary.

 

I hereby certify that, to the best of my knowledge, the foregoing summary and attachments are accurate and complete.

Melinda Nelson
Executive Secretary, National Arthritis and
Musculoskeletal and Skin Diseases Advisory Council
Acting Director, Division of Extramural Research
Activities, National Institute of Arthritis and
Musculoskeletal and Skin Diseases

Robert H. Carter, M.D
Acting Chairman, National Arthritis
and Musculoskeletal and Skin Diseases
Advisory Council
Acting Director,
National Institute of Arthritis and
Musculoskeletal and Skin Diseases