This article was first published at http://www.raredr.com/
George Yohrling, PhD, has served as the Senior Director, Mission and Scientific Affairs for the Huntington’s Disease Society of America (HDSA) since 2012.
With altering the immune system in Huntington’s disease (HD) patients emerging as such a hot topic in the space, Rare Disease Report wanted to sit down with Yohrling to get his thoughts on whether this method is worth pursuing.
RDR: Are there any studies currently ongoing that would be able to prove the effectiveness of immunotherapies in HD?
Yohrling: There’s this idea based on an observation that was made nearly 10 years ago that in HD patients, there’s an increase in neuroinflammatory markers; they’re called proinflammatory cytokines. They include TNF-ALFA and IL-6, and other things like that. Fast forward 10 years, and now there’s a Teva study called LEGATO-HD, which is studying a drug called laquinimod, which was in clinic for multiple sclerosis (MS) and failed to meet its endpoints. Now, it’s the subject of this LEGATO-HD study, and the idea and the hope is that the drug will get into the brains of HD patients, and correct some of the symptoms associated with HD.
RDR: Are there any other, more original, approaches that are being evaluated?
Yohrling: There is another approach, which is novel in the case of Huntington’s disease, and that’s the SIGNAL trial. SIGNAL evaluates the semaphorin 4D (SEMA4D) antibody, which seems to have a number of different biological functions, but specifically seems to be directly involved in a neuroinflammatory response. This study is currently ongoing and recruiting in the United States.
RDR: In your opinion, is targeting the immune system a worthwhile tactic to study?
Yohrling: I think it’s something that deserves being pursued. Certainly because of the data that we’ve alluded to, there’s ample information to suggest that early proinflammatory markers are elevated. It makes sense to me that our community of scientists and researchers should at least pursue that and determine whether early response to those markers could have a beneficial effect on patients with HD. At the end of the day, I think that if we continue research, we can prevent, or at least reduce, these issues in HD that have been observed in patients and are are related to the immune system, like proinflammatory cytokines.