By: Meredith A. Achey, BM
HSG 2014 highlighted “Ideas for the future” of the HSG, exploring education, innovation, and discovery in HD research. The meeting honored outgoing leaders Dr. Ira Shoulson and Dr. Steven Hersch, and welcomed new Chair Dr. Ray Dorsey and Co-chair Dr. Blair Leavitt.
Educational programming catered to both clinicians and researchers. Dr. Martha Nance, Clinical Professor of Neurology at the University of Minnesota, led a panel discussion on genetic testing and reproductive technology. The panel comprised genetic counselor Ms. Michelle Fox from the UCLA HD Center of Excellence; Ms. Debra Lovecky, Director of Program Services and Advocacy for HDSA; and Mr. Matt Bower, a University of Minnesota genetic counselor who also works in a diagnostic molecular genetics laboratory. Panelists brought attendees up to date on the latest genetic testing protocols and sparked a spirited debate.
The meeting included several training sessions for clinical trial teams. Dr. Ralf Reilmann from the George Huntington Institut, Münster, Germany, gave an overview of the quantitative motor (Q-motor) assessment system. Dr. David Craufurd from the University of Manchester, England, led an interactive session titled “Who’s afraid of the Problem Behaviors Assessment?”, describing the development and application of this rating scale. An informative and humorous session led by a panel of HSG coordinators and investigators on the finer points of creating and sustaining a top-notch HD research site followed. Through humorous skits featuring “Dr. P. Eye,” his team of harried coordinators, and auditor “Sawyer Data, FDA,” the panel illustrated the challenges and considerations involved in conducting a “top notch” HD trial.
Several sessions highlighted innovations in HD clinical care and clinical research. The HSG Clinical Trials Update gave members an overview of ongoing and recently concluded clinical trials. Mr. Joe Giuliano, Director of Clinical Operations at CHDI, presented on the opportunities for research available to the HD research community through ENROLL-HD. Ms. Bonnie Hennig, Associate Director of the University of Connecticut Huntington’s Disease Program, led a session titled “What’s hot at your center?”, where Dr. Jean-Marc Burgunder, Chair of the European Huntington’s Disease Network, described the integration of registry studies with clinical care in Europe. Dr. Craufurd described the development of a comprehensive HD care center from his genetic counseling unit in the UK, highlighting their traveling services for HD patients.
Recent scientific discoveries were highlighted throughout the meeting. In the first Insights of the Year panel, Drs. Francesca Cicchetti, X. William Yang, and Chris Tang described their groundbreaking HD research (see HD Insights, Vol. 9). The Innovators’ Forum, with presentations by representatives from Adamas, Intra-Cellular Therapies, Isis, Raptor, and Vaccinex, gave a glimpse into ongoing pharmaceutical research in HD. From new formulations of old compounds, to targeted gene therapeutic approaches and more, these innovators demonstrated the ongoing evolution of the approach to HD therapy.
Caring for young people in the HD community emerged as an important theme. Ms. Chandler Swope spoke about her work as the first HD Youth Organization Youth Worker. She uses counseling and group activities that help youth from HD families to network and share their stories, assisting them in coping with the challenges they face. She spoke of the need for increased involvement of youth in the HD research and clinical care community, in particular for young caregivers. Ms. Kristen Powers, producer of the documentary film Twitch, opened the 8th Annual HD Clinical Research Symposium on the final day of the meeting with a lively keynote presentation about taking a can-do approach to life and HD research, and the necessity of involving youth in the HD community.
Chaired by Drs. Andrew Feigin and Claudia Testa, the 8th Annual HD Clinical Research Symposium further updated members on ongoing research. Dr. Bindhu Paul presented her work on the neuroprotective role of cysteine. Dr. Vicki Wheelock described her team’s work to bring adult mesenchymal stem cells engineered to produce brain-derived neurotrophic factor, to human trials in HD patients. Dr. Diana Rosas described the findings of the PRE-CREST study, highlighting their development of new imaging markers. Dr. Kimberly Quaid described the ethical dilemmas that arise when considering the release of research results to trial participants. Finally, Dr. Dominique Bonneau presented interim results the ongoing CYST-HD trial of cysteamine bitartrate (see HD Insights, Vol. 9).
The meeting concluded with a half-day session for HD community members to learn about ongoing research, and to view Twitch. Led by Dr. Nance, the community events helped remind all HSG attendees of the importance of their ongoing work to find effective treatments for HD.