Recently, I was scheduled to dose a patient for an intrathecal antisense oligonucleotide (ASO) study. As usual, I was running from one end of campus to another (fashionably late as my team would put it), but when I came to the clinical research center, I noticed a crowd of about 20 people standing in the lobby. Aside from a momentary frustration that their presence slowed me down, I nonchalantly thought the Clinical Research Center was full of participants, thus explaining the crowd. To my surprise, after talking to my patient, I discovered that this crowd had accompanied her to this visit. They were aunts, uncles, cousins, and friends from her home town. They took off work, skipped school, and left their daily routines to support her participation in this experimental procedure.
To me, nothing captures the excitement and potential of these new ASO disease-modifying trials like this experience. These trials have been a long time coming, and as a community, can you sense the collective excitement?
In this edition of HD Insights, we highlight stories, perspectives, and considerations to clinical trial design of ASO studies that define this new era of HD care. I want to introduce a new clinical coordinator section, authored by Danielle Buchanan, a fantastic coordinator who works with me at Vanderbilt. Danielle talks about her experiences as a coordinator managing patient expectations in disease-modifying studies. Also, I hope you enjoy the wonderful story of a psychology student (Jessica Klein) meeting her first HD support group, and the insightful article about the “Right to Try” legislation by Alma Farooque. I find the article by Mark Guttman about his experiences with intrathecal drug delivery to be a testament to how, as clinicians, we need to adapt and learn new techniques, like ultrasound-guided lumbar punctures. Our feature story is on the subject of ASO trials, and I learned much from the insights provided by Karl Kieburtz and Amber Southwell.
Please also let me introduce you to Kevin Gregory, the new Director of Education, Communications, and Outreach at the Huntington Study Group. In this issue, Kevin reviews the recent HSG meeting. He helps in the outreach efforts of the HSG, including working with HD Insights. Finally, this will be the last periodical with our deputy editor Sara LaJeunesse. Sara is such a delight to work with, and we will miss her presence at the HSG and EHDN meetings, and I will miss her insightful guidance for HD Insights. We wish her well as she begins her new role at Penn State.
Keep your suggestions coming! I look forward to more of your comments and articles for HD Insights.
See you soon,
Daniel Claassen, MD
Associate Professor of Neurology