Highlights of HSG 2016: Discovering Our Future

The Huntington Study Group held its 23rd Annual Meeting and 10th Annual Clinical Research Symposium November 2-5, in Nashville, TN.

By: Molly J. Elson, BA

HSG 2016 brought together world-renowned clinicians, scientists, coordinators, and community members united by a common passion for improving therapies for HD. Together over the course of several days in November, members gathered to celebrate achievements, explore new therapies, and welcome a new generation of scientists and families into the community. The theme of the conference chaired by Drs. Ray Dorsey and Blair Leavitt was “Progress in HD research and care.” Keynote presentations, interactive working group sessions, and educational courses explored novel therapeutic interventions and emerging models of care. Other members of the HD community joined on the meeting’s final day.

Photo: Researchers presented their findings at the research symposium.

The open sessions began on Wednesday, November 2 with a discussion of the merits of the modified UHDRS as an efficacy endpoint. Results from two major prospective cohort studies (TRACK-HD and COHORT) and clinical trials (CARE-HD) were used as examples. Panels brought together nonprofits, academia, and the pharmaceutical industry to discuss progress in capturing clinically meaningful data and optimizing trial efficiency. “HD Insights of the Year,” an open session exploring breakthrough papers, brought researchers from Harvard, King’s College London, and CHA University in South Korea to the table. Drs. Jong-Min Lee and Flavia Niccolini presented their research into genetic modifiers of time to clinical onset, and pre-symptomatic biomarkers. Similarly, Dr. Jihwan Song posited that mHTT propagates in a prion-like manner. Veteran project manager, Ms. Elise Kayson, led a forum looking back on the results of PHAROS and PREDICT and what individuals at risk for HD can learn. Later, Dr. Martha Nance led a session in which the audience was invited to bring their most challenging cases for group discussion. Ethical dilemmas discussed included how to give genetic testing results to a parent who wished their underage child to be present, and the importance of maintaining contact with research participants post-study.

Photo: Dr. David Oakes received a well-deserved award from Drs. Ray Dorsey and Ira Shoulson in appreciation of his years of service.

A warm reception concluded the second day of activities. Drs. Ira Shoulson and Ray Dorsey presented well-deserved awards to Dr. David Oakes, Dr. Jody Corey-Bloom, Ms. Paula Wasserman, and Ms. Elise Kayson.

The “Digital Biomarkers for HD” panel, chaired by Dr. Ralf Reilmann, stood out for its presentation of some of the most promising technologies scientists are bringing to HD research. Drs. Max Little, Larsson Omberg, Spyros Papetropoulos, and Guarav Sharma described their development of novel objective motor assessments using tools such as Apple’s ResearchKit and MC10’s wearable sensors. Dr. Dorsey’s “Progress and directions” lunch laid out a five-year plan with concrete, achievable objectives to make HD a treatable condition. He recognized key accomplishments in recent HSG trials, and challenged researchers to embrace novel technologies and revolutionize clinical trial conduct.

Dr. Bob Beall, former president and CEO of the Cystic Fibrosis Foundation (CFF), gave the HSG 2016 keynote address, “Lessons learned from cystic fibrosis.” Dr. Beall transformed care and research for cystic fibrosis by raising money to identify genes responsible for the condition, partnering with the pharmaceutical industry, and establishing a network of care centers to increase trial efficiency. His leadership helped to increase the average life expectancy of cystic fibrosis patients from 18 years to 41 years. Dr. Beall challenged HSG to leverage CFF’s successful strategies towards HSG’s own goals.

The 10th annual HD Research Symposium opened the final day of the conference with an education panel, “Regulatory path for development of medical devices and drugs.” Members of the FDA discussed the regulatory pathway through which new therapeutic technologies are evaluated. Dr. Bonnie Hennig led a panel presenting the latest in stem cell research for HD. Attendees left with an appreciation of the challenges faced by stem-cell researchers. When the scientific portion of the conference concluded, Family Education Day began. Families and friends now sat at the tables as Peter Rosenberger shared his story of compassion, hope, and humor as a caregiver. Other talks focused on how to talk about HD as a family, provide palliative care, and test those at risk. The community gathering at the event’s conclusion reinforced the conference’s theme of working together and looking towards the future in order to halt the progression of this disease.

Photo: Everyone gathered for a group photo on Thursday afternoon.