Finding the silver linings in the GENERATION HD1 cloud

The decision to permanently stop dosing with the drug tominersen and placebo in the GENERATION HD1 study is bitterly disappointing for HD families, who have waited so long and so patiently for a treatment.

The aim of this phase III trial was to establish if tominersen was effective in lowering huntingtin protein in a larger group of patients, and if it helped improve signs of HD in patients already showing symptoms. Roche and Ionis have stated that they have stopped dosing on the advice of the Independent Data Monitoring Committee, an impartial body of experts whose role is to monitor and decide whether drugs trials should continue. At this early stage we do not yet know why.

Alistair Haw, SHA Chief Executive Officer, said: “Despite the overwhelming sense of sadness we share over this outcome, there are still positives to focus upon. Firstly, there appears to be no sign of worrying side effects for those involved in this trial.

“Secondly, when researchers come to search for another treatment option, they will not be starting from square one. Lessons have been learned that will take us closer to the success the global HD community so longs for.

“In the meantime, Scottish Huntington’s Association is here for our families, supporting them through this difficult news – and accompanying them upwards and onwards to the day when Huntington’s disease is finally conquered.

“When that day dawns the family members who volunteered for this trial and the staff who helped them, including those at sites in Glasgow and Aberdeen, will be remembered amongst the heroes of the global HD community.”

HD Buzz has published an excellent article summarising the situation in layman’s terms, co-authored by editor-in-chief Dr Jeff Carroll, who says:

“This community – both HD families and HD scientists – have proven that they can do hard stuff together, so we’ll shake ourselves off and do it again. And we’ll keep doing it until HD is no longer a threat to ourselves and our loved ones.”


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