I can't comment much on Brainstorm but I followed a different biotech Sarepta Therapeutics, for years, and their gene altering drug Eteplirsen quite closely as the trials were unfolding. If you read their statements, they said the drug increases dystropin and therefore improves Ducchene Muscular Dystrophy to the point where it is more like Becker's (huge improvement for these kids). All claims were "this drug improves dystropin production" and "walk test results improved on this drug." And it does work. The drug is a breakthrough. By the way, Eteplirsen works at the genetic level as well.
Perhaps with Brainstorm, the CSF markers they evaluated (what I saw them reporting in the search I did on them) were not a good marker for the effectiveness of treating ALS or it is too heterogeneous of a disease. It wasn't false to report the CSF changes but perhaps not all that relevant (unfortunately for your family member
).
We are way behind on hearing loss research but we *do* know functional hair cells are directly related to hearing ability. I see this as a huge difference between Frequency Therapeutics and Brainstorm. Frequency Therapeutics has shown in vitro it can regrow human cochlear hair cells. If they can show changes in audiogram after treatment, their claims are credible and they have accomplished hearing regeneration. There isn't as much wiggle room in their findings here. Either people could hear better or they couldn't.