After "careful evaluation" of the right-to-try act, BrainStorm Cell Therapeutics has decided not to offer its experimental cell therapy for amyotrophic lateral sclerosis (ALS) under the newly passed law. The Israeli biotech considered ethical, legal and practical aspects of providing the treatment and said the clincher was its inability to fund the treatment for all patients who wanted to try it.
The news comes barely a week after Bloomberg reported that BrainStorm was thinking about offering the treatment, called NurOwn, under right-to-try. BrainStorm CEO Chaim Lebovits envisioned "a semicommercial enterprise with modest profits that wouldn’t exploit patients’ desperation," Bloomberg reported.
NurOwn, a personalized cell therapy based on mesenchymal stem cells, is currently in a phase 3 study. Based on discussions with clinicians, regulatory and legal experts and "a few hundred patients," the company came up with four "key conditions that must be satisfied" before the company can provide NurOwn under right-to-try, Lebovits said on a conference call Tuesday.
These are: providing NurOwn to patients at clinical trial-experienced sites, ensuring patients are educated on the risks and benefits of the treatment, only offering it to patients who do not meet the phase 3 clinical trial inclusion criteria and finding funding solutions for patients who can't afford the regenerative treatment. While there isn't yet a price tag on NurOwn, Lebovits said it could be similar to the prices of the CAR-T treatments approved for blood cancers last year. Kymriah and Yescarta have sticker prices of $475,000 and $373,000 respectively.
BrainStorm found a solution for every issue except for funding.
"Though many expressed a will to fund patients, many approached us and gave us detailed figures and plans on how to proceed, this has not yet materialized into concrete commitments," Lebovits said on the call. "As a consequence […] we are unable to provide it until all conditions are fully satisfied."
The company said it will focus on completing the phase 3 study of NurOwn so it can bring the treatment to patients with ALS. It expects topline data at the end of 2019 or the beginning of 2020.