Key Updates From Cure SMA’s 2021 Virtual SMA Research & Clinical Care Meeting

Cure SMA serves as the largest network of individuals, families, clinicians, and research scientists working together to support individuals with spinal muscular atrophy (SMA) and their families. The Annual SMA Conference is an invaluable resource for the entire SMA community—bringing together researchers, healthcare providers, people with SMA and their families to network, learn, and collaborate. In conjunction with the Conference, Cure SMA hosts the Annual International SMA Research & Clinical Care Meeting, which offers insightful discussion around new SMA research and clinical care practices.

Given restrictions related to ongoing pandemic and COVID-19 vaccination roll-out, Cure SMA held its 2021 Annual SMA Conference and SMA Research & Clinical Care Meeting virtually from June 7-11, 2021. The event was well received by all attendees, capturing 7,800 live household views, a number that has now grown to more than 9,500 total household views since the event ended two weeks prior.

New Clinical Trial Data Released at the 2021 Virtual Researcher Meeting

The SMA Researcher Meeting consisted of the Special Session and Basic Research Session on Thursday, June 10, and the Clinical Drug Development Virtual Session on Friday, June 11. The goal is to create open communication of scientific data, accelerating the pace of research. The meeting also builds productive collaborations—including cross-disciplinary dialogue, partnerships, integration of new researchers and drug companies, and educational opportunities for junior researchers.

This year’s Special Session, entitled "Rehabilitation and Regenerative Therapies/Technologies,” looked at why and how motor neurons degenerate within the context of low levels of SMN protein and how developmental factors influence this degeneration, both critical for better understanding the pathology of SMA. Furthermore, understanding degeneration may lead to potential therapies aimed at preventing or halting this degeneration, giving insight into therapies aimed at regeneration. The goal of this session was to explore what is known about degeneration and regeneration within the context of neuromuscular diseases and discuss how this knowledge may impact future therapeutic development. Speakers included: Joe Lewcock, PhD; Michael Coleman, PhD; Richard Robitaille, PhD; and Ahmet Hoke, MD, PhD.

At this year’s virtual meeting, several companies released new data from their programs. The data released covers the entire SMA population—from infants to adults—and includes different therapeutic approaches, including those that target the underlying genetic cause of SMA and those that work on the muscles affected by SMA. Clinical trial updates included:

• Scholar Rock Presents TOPAZ Phase 2 Data Showing the Transformative Potential of Apitegromab in Patients with Type 2 and 3 SMA https://www.curesma.org/scholar-rock-topaz-data-for-apitegromab-researcher-meeting-2021/
• New Genentech Data for Evrysdi Show Improved Motor Function in Pre-Symptomatic Babies After One Year and Confirm Safety Profile in Previously Treated People with SMA https://www.curesma.org/genentech-data-for-evrysdi-researcher-meeting-2021/
• Biogen Presents New Spinraza Data at the 2021 Virtual SMA Conference https://www.curesma.org/biogen-presents-new-spinraza-data-at-the-2021-virtual-sma-conference/
• Novartis Data Demonstrate Age-Appropriate Development When Zolgensma Is Used Presymptomatically and Rapid, Clinically Meaningful Efficacy in Symptomatic Children* https://www.curesma.org/novartis-data-zolgensma-used-presymptomatically-june21/

Cure SMA appreciates the sponsors that made the Virtual SMA Researcher Meeting possible. Thank you to our Presenting Sponsors, Biogen, Genentech, and Novartis Gene Therapies. We would also like to recognize Platinum Sponsors, Cytokinetics and Scholar Rock.

Clinical Care Continuing Medical Education Session

Cure SMA remains committed to its mission and serving the SMA community through research, care, advocacy, and support. On Wednesday, June 9, Cure SMA hosted a one-day Clinical Care CME Session as part of the 2021 Virtual SMA Research & Clinical Care Meeting.

The goals of the session were to provide a valuable opportunity for SMA healthcare providers across disciplines to share knowledge, facilitate collaborative discussions between healthcare providers to achieve improved care for SMA, foster clinical care research collaboration across all care centers, and promote interactions between new clinicians and leaders in clinical care for SMA.

The meeting featured special sessions, title “Ethics in SMA Clinical Care” and “Mental Health Challenges in SMA-Affected Patients and Families.” The ethics session was led by Julie Parsons, MD with panelists John Brandsema, MD, Tom Crawford, MD, and Bakri Elsheikh, MD, as well as ethical considerations guidance by Brian Jackson, MD. The panel explored five frequent clinical scenarios in SMA care.

Vamshi Rao led the mental health session, with panelists Pam Mosher, MD and Vanessa Battista, RN, MS, CPNP-PC, CHPPN providing an overview of mental health and SMA. They were joined by Diane Murrell, LCSW and Al Freedman, PhD, to discuss a case study of an adolescent with SMA and considerations. Additional focused speaker panels presented on the topics of Musculoskeletal Care, including hip pain, scoliosis management, and physical therapist education; Impact of New Therapies including nusinersen in adults, swallowing physiology and function, use of combination therapies, and nusinersen delivered by subcutaneous catheter; and SMA Newborn Screening with reports of the experiences in Australia, Wisconsin, Colorado, and Wyoming, with further exploration into the SMA patient community attitude toward fetal therapy.

The Clinical Care Session offered continuing education hours for 617 professionals dedicated to the SMA community. If you missed the live program, you may view and receive continuing education hours at University of Wisconsin-Madison Interprofessional Continuing Education Partnership between now and June 2022.

The Clinical Care CME Session was made possible through a partnership with the University of Wisconsin–Madison Interprofessional Continuing Education Partnership (ICEP) and Cure SMA. We also gratefully acknowledge the educational grants provided by Genentech and Novartis Gene Therapies.

Additionally, we are excited to share that the 2022 Annual SMA Conference dates have been officially announced. Next year, the SMA community will gather from Thursday, June 16 to Sunday, June 19, 2022, at the Disneyland Hotel in Anaheim, CA. The 2022 SMA Research & Clinical Care Meeting will be held June 15-17, 2022. We hope to see you there!

About Cure SMA

Since 1984, Cure SMA has grown to be the largest network of individuals, families, clinicians, and research scientists working together to advance SMA research, support individuals with SMA and their caregivers, as well as educate public and professional communities about SMA. The organization has directed and invested in comprehensive research that has shaped the scientific community’s understanding of SMA, led to breakthroughs in treatment and care, and provided individuals and families the support they need today. We are currently on the verge of breakthroughs that will strengthen bodies, extend life, and lead to a cure. To learn more, visit us at www.curesma.org.

*The data was presented at the European Academy for Neurology (EAN) Virtual Congress 2021. Novartis Gene Therapies presented similar data on the SPRINT trial to attendees of the 2021 Cure SMA Research & Clinical Care Meeting.