Welcome to the 11th edition of HD Insights. We are pleased to continue our mission to promote, disseminate, and facilitate research in Huntington disease. We are grateful to the Huntington Study Group (HSG), our sponsors, and our more than 2800 subscribers for their continuing support.
This issue investigates potential therapeutic strategies for HD treatment and provides a detailed update on disease-modifying strategies set to enter clinical trials in the upcoming year. We discuss Teva’s recent acquisition of Auspex following the promising results of FIRST-HD released earlier this year. We bring you an interview with former FDA attorney Mr. Frank Sasinowski, on his work with the 1983 Orphan Drug Act, his hopes for orphan drug development, and his advice for researchers and companies working to develop new therapies for rare diseases. Dr. Shaun Sanders describes the therapeutic potential of increasing palmitoylation of mHTT, thereby decreasing its toxicity and aggregation within HD patients. Dr. Dagmar Ehrnhoefer details several studies showing that wild-type HTT plays an important role in myristoylation and autophagy, and promotes the clearance of misfolded mHTT. Dr. Lise Munsie summarizes ongoing HD research and Dr. Daniel Zielonka discusses promising diseasemodifying strategies aimed at lowering HTT. Dr. Francesca Cicchetti shares the highlights of February’s CHDI meeting. Finally, we continue to provide an up-to-date status report on ongoing and recently concluded HD clinical trials.
The HD Insights team continues to rely on the support of firms dedicated to developing novel treatments to support our efforts to reach and educate the international HD research community. If you are interested in becoming a supporter, please contact me at firstname.lastname@example.org. We welcome new contributors, suggestions for topics, and ideas about how we can better serve you. If you or someone you know would like to share their ideas with over 2800 HD researchers and clinicians around the world, please contact me at email@example.com . Finally, subscription to HD Insights is always free: simply send us an email at firstname.lastname@example.org.
Ray Dorsey, MD
Editor, HD INSIGHTSTM
HD DISCLOSURES: DR. DORSEY RECEIVES GRANT SUPPORT FROM AUSPEX PHARMACEUTICALS, THE HUNTINGTON STUDY GROUP, AND PRANA BIOTECHNOLOGY.
Editorial Board Members
Ray Dorsey, MD — Editor, University of Rochester
Donald Kennedy, PhD — Stanford University
Nobuyuki Nukina, MD, PhD — Riken Brain Science Institute
Rodrigo Osorio — Agrupación Chilena de Huntington
Bernard Ravina, MD — Voyager Therapeutics
Ralf Reilmann, MD, PhD — George Huntington Institute
Sarah Tabrizi, MBChB, PhD — University College London
Leslie Thompson, PhD — University of California Irvine
Huntington Study Group
Shari Kinel, JD — HSG Executive Director
Liz McCarthy, BA — HSG Director of Programs & Communications
Kristin Strazdins — HSG Accounting and Office Manager
Francesca Cicchetti, PhD
Dagmar E. Ehrnhoefer, PhD
Ryan E. Korn, BA
Lise Munsie, PhD
Shaun S. Sanders, PhD
Daniel C. Zielonka, MD, PhD
Meredith A. Achey, BM — Deputy Editor
Ryan E. Korn, BA — Associate Editor
Robin Taylor — Production Editor
Martin Holmes — Technical Editor
David Kolko — Distribution Specialist