Researchers are studying a new treatment for Huntington's disease that silences the affected gene, NPR reports.
The project is a joint effort between Roche and Ionis Pharmaceuticals. As NPR notes, Genentech, a part of Roche, reported last year that its antisense oligonucleotide drug could reduce the level of mutant huntingtin in patients' cerebrospinal fluid samples in a small study that has recently been published in the New England Journal of Medicine. It adds that Ionis developed the approach to deliver the antisense oligonucleotide drug to patients' brains via their CSF.
In their new trial, the companies are running a 25-month trial with more than 650 participants to determine whether the treatment stops or slows their symptoms from advancing, NPR adds, noting that they had to tweak the study earlier this year after they realized the drug didn't have to be given as often as they'd thought.
This isn't the only effort underway to treat Huntington's disease, it says. "A lot of different approaches are being worked on in different stages of drug discovery across the world," George Yohrling, a scientist at the Huntington's Disease Society of America, tells NPR. "It's really quite exciting."