We were saddened to learn on March 22nd via a press release and community statement from Roche that their Phase 3 study of tominersen, known as the GENERATION-HD1 trial, has been halted early, meaning that participants will no longer receive the study drug or placebo. The decision to halt dosing of this genetic therapy came as a shock to the community, from participants and their loved ones, to doctors, researchers, and medical professionals. It was based on the advice of an independent data monitoring committee (iDMC), which exists for every large clinical trial. This is a group that analyzes the data regularly to monitor safety and efficacy, without having a personal or economic interest in the outcome of the trial. Not even Roche had access to the data that led to this decision – we know that no new safety concerns emerged, but we don’t yet understand why the recommendation was made to stop the trial.
If you are interested in a bit of a deeper dive into the language in the press release, HDBuzz released an article about the news, which is available here.
Within the coming months, as Roche gains the opportunity to analyze the data, we hope to be able to share more information with the community as to the facts behind this decision. It is difficult to overstate the hope that many people had placed in this trial, and many reactions and emotions have arisen. Although we have little information at the moment, a variety of community organizations, including HDSA, are offering Q&A sessions and webinars this week to discuss the news and to provide as much clarity as possible given the information available.
Today, Thursday, March 25th, at 11 AM Eastern. HDSA Webinar: Generation-HD1 Trial News – What does this mean and where do we go from here? Jennifer Simpson, LCSW (Assistant Director of Youth and Community Services at HDSA), George Yohrling, PhD (Chief Scientific and Mission Officer at HDSA) and Vicki Wheelock, MD (HDSA Center of Excellence Director at UC-Davis and Site Investigator for the Generation-HD1 trial) will participate in the discussion and take questions from audience. Register here to join.
Today, Thursday, March 25th, at 3 PM Eastern (7 PM UK time). Matt Ellison and Dr. Lauren Byrne from HDYO will be available for an hour session to chat and answer questions on the Roche research update with the information we currently have. Register here to join.
Today, Thursday, March 25 at 5:30 PM Eastern. Facebook live presentation organized by HDReach. Dr. Bonnie Hennig-Trestman will address mental health concerns by leading a conversation on handling disappointment. Dr. Bob Trestman (psychiatrist) will join her. Join via Facebook.
Friday, March 26th at 12 PM Eastern (4 PM UK time). The European Huntington Association has invited professor Sarah Tabrizi (lead neurologist in the GENERATION-HD1 study) and professor Anne Rosser to explain what has happened based on the information available, and also help us understand what this means for HD research in a more general perspective. Register here to join.
Saturday, March 27 at 4:10pm Eastern. As part of the Huntington Society of Canada (HSC) national conference, Dr. Ed Wild, Dr. Leora Fox, Dr. Rachel Harding, and Dr. Jeff Carroll of HDBuzz will be panelists in an “Ask the Scientist” Q&A session. Register here to take part in the conference (free to all).
Whatever you’re experiencing, you’re not alone, and we look forward to communicating more in the days and weeks ahead.