Top-line results from BrainStorm’s NurOwn Phase III ALS study show the cell therapy missed the primary efficacy endpoint, but clinically meaningful responses were seen in a pre-specified subgroup which investigators considered potentially a significant step forward in therapy.

King & Spalding Partner David Farber called for amending Medicare and Medicaid statutes so that unapproved treatments can be reimbursed for terminally ill patients who have exhausted all other options.

CBER director said unlicensed stem cell clinics often report adverse events not made public, which is a concern under Right To Try.

Early engagement with US payers suggests openness to covering a liquid form of riluzole, because the tablet version is proven, but it's impossible to take for many ALS patients, says Italfarmaco subsidiary ITF Pharma.