Drugmaker Backs Off Plan to Charge for Unproven ALS Therapy

(Bloomberg) -- BrainStorm Cell Therapeutics Inc. has decided not to make its unproven therapy for Lou Gehrig’s disease available to patients under a new U.S. law after struggling to find funding for patients who couldn’t afford the medicine on their own.

The company had planned to charge patients for the therapy, the company’s chief executive told Bloomberg earlier this month, at a price that could have run into the hundreds of thousands of dollars. At the time, Brainstorm CEO Chaim Lebovits said the company wouldn’t proceed if only wealthy patients would have access to the therapy. He also said the company needed a financial incentive to provide the therapy.

“Companies cannot be NGOs,” Lebovits said in a phone interview with Bloomberg last week, referring to nonprofits. “We have to have an incentive.”

The therapy, called NurOwn, was to be available under the Right to Try law, which President Donald Trump signed earlier this year to give dying patients access to experimental therapies outside the oversight of the U.S. Food and Drug Administration.

The company said that while it received interest in helping provide the experimental therapy, “it has not yet materialized into a complete commitment, and as a consequence I sadly say we are unable to initiate provisions of NurOwn Right to Try pathway,” Lebovits said on a conference call Tuesday.

The company does plan to give Matt Bellina, a former U.S. naval aviator, access to the medicine. Bellina, whose ALS is too advanced for him to participate in clinical trials of NurOwn, campaigned for the Right to Try legislation. The company said Bellina was one of more than 1,000 patients who were hoping to get access to the drug.

“While it’s heartbreaking for me not to be able to currently provide access to more patients, we couldn’t see ourselves denying treatment to Matt,” Lebovits said. “We have a committed sponsor for this treatment, so Matt will make history and he will be the first person to receive treatment through this pathway.”

Ethics Controversy

The BrainStorm CEO’s comment that he was considering charging for the experimental drug created controversy, with medical ethicists saying it put desperate patients in a difficult position of paying potentially large sums for a treatment that may not work. Advocates for the law have said that dying patients should be allowed to make their own decisions.

BrainStorm’s treatment attempts to treat amyotrophic lateral sclerosis, or ALS. The progressive disease erodes nerve cells, weakening the muscles throughout the body and gradually leaving patients unable to walk, speak and breathe.

NurOwn’s mechanism doesn’t reverse the underlying cause of ALS, meaning it’s unlikely to cure the disease. A mid-stage study involving 48 patients found those given the drug appeared to respond, though the benefit didn’t last for most. The final results may not support approval.

Brainstorm, whose stock is listed in the U.S., is run from New York and Israel. The shares fell 5.3 percent to $3.95 at 10 a.m. in New York.

©2018 Bloomberg L.P.