BrainStorm Cell Therapeutics is considering providing an experimental therapy to amyotrophic lateral sclerosis (ALS) patients under the recently passed U.S. right-to-try law, Bloomberg reported. The Israeli biotech would expect to generate modest profits from the initiative, making it a test case for whether the law has turned unproven drugs into semicommercial products.
The right-to-try law signed by President Trump late last month states that anyone who provides drugs on a right-to-try basis can only recover direct costs, a term defined by the FDA as outlays “that can be specifically and exclusively attributed to providing the drug.” While that language appears to restrict the commercial opportunity created by the law, BrainStorm thinks it can—and, in fact, must—benefit financially from providing its cell therapy on a right-to-try basis.
“Companies cannot be NGOs,” BrainStorm CEO Chaim Lebovits told Bloomberg. “We have to have an incentive.”
The Bloomberg article went on to describe Lebovits’ vision for the planned right-to-try operation as a “semi-commercial enterprise with modest profits.” Lebovits pointed to the $300,000-plus prices of personalized cell therapies used in the treatment of cancer as a guide for what patients may have to pay to access BrainStorm’s ALS candidate, NurOwn.
BrainStorm is testing the modified mesenchymal stem cells in a phase 3 ALS trial. But following the passage of the right-to-try law it received multiple requests from people seeking access to the drug outside of this setting, leading it to set up calls with patients and investors to discuss how to proceed. BrainStorm is still ironing out the details but plans to work with nonprofits to help cover the costs of one out of every three patients.
The value of paying anything for the drug is uncertain. BrainStorm thinks NurOwn can improve the lives of patients with ALS by getting nerve growth factors to damaged neurons. But a phase 2 clinical trial found limited evidence of efficacy, with placebo matching NurOwn on measures of lung function and ALS progression.
BrainStorm saw some signs of efficacy, though. When paired to a clean safety profile, those signs were enough to encourage BrainStorm to move the candidate into phase 3. And, under the new law, the results are tantalizing enough to tempt some patients to pay serious sums of money to access the drug.