Thank you to all who attended HDSA’s virtual 36th Annual Convention! If you missed us this time around, don’t stress—recorded sessions will be available on our YouTube channel in the coming weeks. While we await the video highlights, be sure to check out the local and online surveys page to see what HD research trials are happening in your area or that you can complete virtually from the comfort of your own home. Also be sure to check out the new virtual health community for those living with HD or caring for individuals with HD recently launched by HDSA in partnership with PatientsLikeMe. Register to join here.
uniQure begins high dose treatment in AMT-130 trial
uniQure issued a press release this week stating that two more patients have been added to their clinical trial of HD gene therapy AMT-130. So far there are twelve participants in the trial, all of whom have undergone a surgery to reach the deep areas of the brain most vulnerable to HD. Seven participants have received the drug and five have received an imitation surgery.
The two participants highlighted this week are the first to receive a higher dose of the drug – an exciting step, because it means that everything has gone safely and smoothly with lower doses so far. Each of 26 total participants in this Phase 1 / 2 trial will be monitored very closely for a year and then less frequently for up to five years. Through this unprecedented trial of the first HD gene therapy uniQure hopes to learn whether AMT-130 is safe and whether it can lower levels of huntingtin protein.
HDBuzz on Pridopidine: How it Works
Pridopidine is a drug that has been developed for treatment of HD and was initially studied as a medication for chorea. Initial motor studies found that pridopidine was safe and tolerable. Although the drug did not meet its key goal of improving motor symptoms, it had positive effects on Total Functional Capacity (TFC) in people with HD. A Phase III clinical trial, PROOF-HD, is currently recruiting participants to look for conclusive evidence that pridopidine does improve TFC in individuals with HD, and scientists have been evaluating the drug in the hopes of identifying exactly how pridopidine might affect these changes.
HDBuzz gave us the summary on three recently published peer-reviewed journal articles that highlight sigma-1 receptor (S1R) proteins as likely targets for pridopidine’s effects on neurodegeneration in HD. S1R proteins are found in brain regions in areas affected by HD and help nerve cells survive under stress. These studies have found that pridopidine helped to improve indicators of stress in cells and animal models of HD. For the full scoop on pridopidine and the latest research on how it works, check out HDBuzz’s newest article here.