By Daniel Claassen, MD
Welcome to a new edition of HD Insights. These are interesting times. As I write this, I am conscious that many of us are personally affected by the coronavirus, and the impacts on travel, research, and clinical burden. Perhaps it is ironic that this edition, in part, focuses on a virus—one that is harnessed for a new treatment in HD.
One of the joys of being a clinician scientist is to be on the front lines of investigating new therapeutic options for patients. Already, many of us have administered monoclonal antibodies and intrathecal medications, but now we in the HD world enter a new therapeutic world: genetic treatments administered intracerebrally via viral vectors.
The potential implications of this treatment for our patients and our clinical practice are vast. In this edition we explore various perspectives on AAV treatments from: a neurologist who works in basic science, a neurosurgeon who administers these treatments, a clinical coordinator who must take the journey with patients through the clinical trials, and a clinician scientist who consider the AAV vector as a potential treatment, and leads the uniQure trial. I am indebted to Drs. McFarland, Larson and Reilmann, and to Danielle Buchanan for taking time out of their hectic schedules and writing such informative articles. I think you will find these perspectives really useful as you consider this new approach.
Also, we are excited to do a piece on Jack Griffin, who many of you know. Jack has individually encouraged many clinicians, social workers, and HD centers in getting much needed funding to folks that are trying to expand care. I think you will also enjoy the uplifting story of Lysle Turner.
As always, thank you for your encouragement and kind words regarding HD Insights. Our podcast continues to be a great success, as does our periodical.
In these trying times that may be isolating to some, I hope you will be encouraged as we press on in our care for HD.
Dr. Claassen is Associate Professor of Neurology at Vanderbilt University, and Chief Editor of HD Insights.