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Cambridge: Biogen — Ruby Wallau for STAT

A plan to use new genetic techniques to treat non-genetic forms of ALS failed in an early study, Ionis and Biogen announced Thursday. The companies said they would discontinue the program.

In the randomized, 99-person trial, the companies tested a drug designed to silence a gene called ATXN2. Over the last decade, numerous drugs have been built to silence genes in ALS patients, but most of them have, fittingly, focused on rare genetic forms of ALS, where neurons die largely because of the work of a single mutated stretch of DNA.

This approach has led to an approved drug for one very small subset of ALS cases, driven by the gene SOD1, and drugs for other genes are in trials. But only perhaps 10% — or at most 20% — of ALS cases are genetic. The hope, based on some genetic data and promising mice experiments, was that lowering ATXN2 would help any patient who develops ALS.

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