‘Game-changing failure’: Trials of two Huntington’s disease-fighting gene therapy drugs halted, in ‘crushing blow’ to sufferers

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Credit: NHGRI
Credit: NHGRI

Researchers had hoped that [a new treatment in trials] —known as antisense oligonucleotides (ASOs)—would be a game changer for [Huntington’s disease], an incurable genetic condition that affects cognition, behaviour and movement. But back-to-back announcements from Roche, headquartered in Basel, Switzerland, and Wave Life Sciences, in Cambridge, Massachusetts, have dealt a crushing blow to those affected by the disease.

“It’s the saddest possible result,” says Claudia Testa, a neurologist at Virginia Commonwealth University in Richmond, who has received consulting fees from Wave Life Sciences. “It’s clearly the right decision to halt dosing, even though I’m sure that was not the outcome anyone hoped for.”

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Several factors could have contributed to tominersen’s failure, according to Sarah Tabrizi, a neurologist at University College London and one of the investigators in the Roche trial. The drug suppresses production of the healthy, as well as the mutant, form of huntingtin, and a decrease in levels of the normal protein could have caused problems. Other possibilities are that the ASO did not reach the right parts of the brain, or that the disease had simply progressed too far in the trial participants for the drug to be beneficial.

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