36th Annual HDSA Convention: Updates on Tominersen Trial

Patient Worthy signed up to cover several sessions at the Huntington’s Disease Society of America’s (HDSA) 36th Annual Convention, which was held virtually this year. This event features informative presentations and info sessions about the latest Huntington’s disease research geared towards the Huntington’s patient community.

The first session we listened to was on the Generation-HD1 clinical trial, which was evaluating the investigational therapy tominersen as a treatment for the disease.

About Huntington’s Disease

Huntington’s disease is a heritable disorder that causes brain cells to die. This is a long term, progressive, and ultimately lethal disease that causes severe debilitation over time. The disease is caused by a genetic mutation that affects the HTT gene. It normally appears between 30 and 50 years, but in rare cases it can occur before age 20. Symptoms of Huntington’s may first appear as subtle mood and behavioral changes and loss of coordination. Other symptoms include random movements called chorea, abnormal posture, sleep issues, trouble chewing, swallowing, and speaking, dementia, anxiety, depression, and impulsivity. Nine percent of deaths are the result of suicide. Treatment for Huntington’s disease is symptomatic, with no cure or disease altering therapies available. Most patients die around 15 to 20 years after their diagnosis. To learn more about Huntington’s disease, click here.

About The Trial

Dosing in this clinical trial was halted following a recommendation from an independent data monitoring committee. Dosing was also halted for the open-label extension portion of the study as well. While dosing for this trial has been halted, this does not mean that the entire study has been halted or cancelled, and the patients that are participating will continue to be monitored.

The 25 month study was evaluating two dose regimens: 120 mg every two months and 120 mg every four months. Both were being compared to a placebo group.

The recommendation from the committee to halt dosing was based on data presented to them in March. The trial had been running for 17 months at that juncture. In effect, they decided to halt dosing because the perceived balance between risk to the patients and the benefit of the treatment was not acceptable; in effect, the drug didn’t seem to be producing enough benefit. The data demonstrated, for example, that patient outcomes appeared worse in the two month dose group than in the placebo group.

In the four month group, the treatment didn’t seem to be markedly worse, but there was also no significant benefit in comparison to placebo. While these findings are far from encouraging, it’s still important for participants and their physicians to continue their participation and monitoring in this trial so that a complete data set can be captured.

Genentech, the sponsor of this trial, will release further data on the outcomes of this study in the future.

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