Written by Bev Heim-Myers, HSC CEO
Yesterday we heard about the learnings from the Pfizer Amaryllis study and the extensive analysis that was carried out before the study was stopped. It is always disappointing for our community when a trial is stopped but it is important to remember that the decision is never made lightly. Even stopped trials add to the overall understanding of HD.
Today I met with Wendy Erler from WAVE Life Sciences. The first clinical study site for this exciting trial started in Canada with Dr. Mark Guttman leading the study. A study site in Poland has recently also been identified. The first doses have been given to some courageous leading edge participants and there seems to be good tolerance for the drug, to date – although it is early days.
Unlike the Ionis study that targets both the normal and mutant huntingtin, the WAVE study only targets the mutant HTT huntingtin protein with specific bio criteria identified for all study participants. About 70% of the HD community with the HD mutation are candidates for the study.
The good news is that they hope to have top line data by early 2019.
It was refreshing to meet with Wendy. WAVE Life Sciences is keenly aware of the challenges of living with HD. Wendy is the VP of Patient Advocacy and as such meets with many families and continues to ensure that the research meets the needs of the families. Wendy will also work on a communication update that I will be able to share with our community.
I am off to talk about HD-Cope for the production of “Postcards from Palm Springs”.
Read more about day two of the Conference from HDBuzz here.
Read more about day three of the Conference from HDBuzz here.