2016 SMA Researcher Meeting Included Cutting-edge Science, Navigating FDA Process


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2016 SMA Researcher Meeting

The 2016 SMA Researcher Meeting, the largest gathering specifically dedicated to spinal muscular atrophy (SMA), set a record with 350 attendees who discussed cutting-edge research and the future of SMA patients’ needs.

The goal of the annual event, held this year in Anaheim, California, is to create an open forum for the communication of unpublished results and sharing experiences across different disciplines.

The 2016 special session, titled “The Changing Landscape of SMA: Consideration of Future Needs,” was moderated by Dr. Stephen Kolb, MD, PhD, Cure SMA scientific advisory board member from Ohio State University, who debated the changing landscape of SMA in an imagined future where the first drug approvals for SMA had already occurred.

According to a press release, in the final panel discussion, Kolb summed up the topics debated and outlined important future directions, including:

  • Identifying novel drug targets for SMA;
  • Ensuring there are outcome measures or means that allow drug approvals for all SMA types;
  • Making patient-focused drug development efforts to debate risk/benefit from the patients’ perspective;
  • Setting up post-marketing registries to collect long-term information on drug efficacy and safety;
  • Determining SOCs for clinical trials and overall care;
  • Reducing the time to diagnosis with newborn screening;
  • Finding ways to make current drug candidates more openly available for pre-clinical research by promoting industry and research collaboration initiatives.

There were five key presentations at the SMA Researcher Meeting, including two sessions sharing experiences from different disease communities.

Healther Clark, MS, from Vertex Pharmaceuticals, gave a talk where researchers and clinicians learned from the experience of a different disease community, cystic fibrosis, about the efforts behind the development of a combination therapy and which regulatory strategies they used to obtain approval. Cure SMA believes their work offered valuable insights into strategies that can also be used by SMA drug developers to expedite combination drug development.

John Porter, PhD, from the Myotonic Dystrophy Association, hosted a session where he shared lessons from the first new drug application submissions to the FDA in Duchenne muscular dystrophy (DMD).

Porter’s presentation helped inform the SMA community about how to best navigate the U.S. FDA approval process by debating issues like calculating the strength of the drug’s impact in primary outcome measures, validating the outcome measures, understanding the natural history around these outcomes through validation of biomarkers, trial size, inclusion and exclusion criteria, and the use of placebo controls, as well as using partnerships to secure expertise and resources.

One session debated considerations for the development of combination therapies:

Karen Chen, PhD, from the SMA Foundation, talked about important points for developing combination therapies. As the SMA drug pipeline grows in diversity and complexity, Chen explained how the biological rationale and therapeutic need for multiple approaches has become increasingly apparent. In addition, she advocated for care and consideration when administering any drug to any patient and to understand how each drug works alone and together with others to know how they will affect their targets.

One presentation focused on the importance of patient registries. Katherine Klinger, Ph.D., from Genzyme, discussed the importance of registries in rare diseases, based on her experience analyzing data collected from a CF registry over the past 20 years, which helped to improve lung function and life span.

She mentioned how registries facilitate patient recruitment for clinical trials, help patients gain knowledge on long-term consequences of a drug therapy, teach them how clinical care impacts disease progression and provide a link to the community, and offer information directly relevant to their condition.

A final talk presented the latest updates to SMA standards of care. Dr. Richard Finkel, MD, from Nemours Children’s Hospital, spoke about the use of natural history data to inform the standardization of patient care. Updated standards of care can impact drug development as patients given the same treatment reduce clinical trial variability.

Still, standards of care are guidelines, and the presentation emphasized that each patient is free to decide on their preferred therapy. Finkel is currently working on updating SMA’s standards of care to reflect the knowledge gained since the last revision. He expects to publish them later this year.