Posted on January 24, 2019
This week, we learned that the SIGNAL trial has completed recruitment for its Phase 2 study of VX15/2503 (pepinemab), a drug made by Vaccinex. VX15 is an antibody designed to bind to the semaphorin 4D (SEMA4D) protein, a molecule that guides the activation and movement of cells within the body, and which may be responsible for inflammation in the brains of individuals who develop HD. The trial is designed to test whether reducing brain inflammation could slow the preogression of HD. A total of 240 people were enrolled in the trial. The study is ongoing for many participants, but we look forward to more news about whether it will be helpful for treating HD symptoms.
We also had news this week that a Boston-based company called UniQURE has received FDA clearance to move forward with a small clinical trial of its gene therapy drug for HD. The drug, called AMT-130, is a little piece of man-made genetic code called a microRNA. Once it enters a cell, it can find and chop up huntingtin RNA, so that the cell makes less toxic huntingtin protein. It would be packaged inside a type of harmless virus called an AAV, and the virus would be injected into the brain via a surgery. The idea is that with only one procedure (a brain surgery), the drug would keep attacking that huntingtin culprit for a very long time. This is extremely experimental but it has so far been successful at lowering huntingtin in animals like mice and minipigs. The next step is human testing, likely in just a few individuals.
Participate (from anywhere!) in an HDSA-Funded Research Study on Gut Bacteria
There is compelling evidence that having an abundance of harmful bacteria in the gut may worsen symptoms of certain brain diseases, such as Parkinson’s. But whether “disease-promoting bacteria” grow in the gut of HD patients remains unknown. Through the Human Biology Project, HDSA is funding Dr. Ali Khoshnan, a Senior Research Scientist at California Institute of Technology, to better understand what kinds of micro-organisms live in the gut and influence HD.
Dr. Khoshnan is examining the feces of people with and without HD for the presence of harmful bacteria. We are inviting individuals with an HD diagnosis along with an HD-negative household member to participate in this project and help us to identify potential modifiers of HD. Participation involves receiving a kit in the mail, filling out a consent form, donating a small swab of fecal matter, and mailing back in a pre-paid return envelope. Easy instructions and all collection materials needed will be provided. If the findings are interesting, participants may be asked to submit a second sample.
Any person with an HD diagnosis or positive genetic test can participate along with a household members or caregiver who is negative for HD. No restrictions with diet or medications. If you are willing to participate in this important research, please contact Dr. Ali Khoshnan, 626-395-1705, Khoshnan@caltech.edu.
New Survey on HDSA’s Website
Responding to a survey about your experience with HD is another great way to participate in research. We have posted a new university-approved survey study on our website this week:
Neli Dragneva, a student at Liverpool Hope University in the UK, is studying how optimism, beliefs about illness, and stage of disease affect quality of life for people with Huntington’s disease. In order to be eligible to participate, you must be over 18 years old and have a clinical diagnosis of HD. The survey consists of 3 short questionnaires and some general questions about yourself and should take approximately 20 minutes. Participation will be entirely anonymous and you are free to withdraw at any time even after completion of the survey.