For Peter Pitts, a former associate commissioner at the FDA, the appointment to the board of BrainStorm Cell Therapeutics is an opportunity to fulfill a promise he made long ago to a patient with ALS.
When BrainStorm Cell Therapeutics called with an offer to sit on its board of directors, Peter Pitts was caught off guard. He had previously been a vocal critic of how the company ran an expanded access program for NurOwn, its experimental stem cell therapy for ALS.
“I was pretty harsh relative to what I thought was a very bad idea, charging people new commercial prices for access to an experimental medicine,” Pitts said. “So I was surprised when they contacted me.”
But the company ultimately convinced Pitts that it was on track to help patients with ALS—something that struck a chord with him on a personal level. He steps into the role just as BrainStorm gears up for a Phase 3b trial—and another FDA run for ALS therapy NurOwn.
The New York and Jerusalem–based company made the announcement last week.
Pitts, who served as associate commissioner for external relations at the FDA from February 2003 to June 2004, went on to found the Center for Medicine in the Public Interest, where he has served as president for the past 22 years, according to his LinkedIn profile.
“The science [behind NurOwn] is very cool and looks very robust as well,” he told BioSpace last Thursday, adding that he finds the people around NurOwn to be “very impressive.”
“We had long conversations that showed me a lot of work that they were doing, and it was really impressive,” Pitts continued. “And their attitude seems to have changed from trying to mollify investors to really doing some very exciting science with patients really in the middle of the whole conversation.”
Pitts also has a long-standing passion for helping patients with ALS, a devastating, fatal neurodegenerative disease that is recognized in the U.S. on June 2 as Lou Gehrig’s Day, in honor of the former Yankees first baseman who died from the disease in 1941.
Pitts’ personal connection to ALS crystalized in 2001, when he spoke at an advocacy event in Washington, DC. “After I gave my usual kind of FDA stump speech, a guy comes up to me in a wheelchair with all the various paraphernalia, and he says to me, ‘I’m gonna be dead in a year. Please do everything you can to help move these programs forward,’” he recalled. “It was a very moving moment, and it really kind of stuck with me, and this [board appointment] kind of gives me the opportunity to help to fulfill that promise.”
NurOwn’s troubled past
NurOwn is a drug with a checkered past—and a dedicated fan club.
In November 2020, NurOwn’s first Phase 3 trial failed to meet statistical significance for the primary efficacy endpoint. The 34.7% response rate was only slightly better than the unusually high 27.7% placebo response. In a prespecified subgroup of patients with early-stage disease, however, the separation was much larger, with 34.6% of NurOwn recipients having a clinically significant response compared with 15.6% in the placebo group.
BrainStorm executives have consistently pointed to a floor effect—which occurs when the scale of measurement is unable to capture patients’ progression as their condition becomes severe—to explain the missed endpoint.
Still, in September 2023, an FDA advisory committee voted 17-1 against approving NurOwn, saying there was not substantial evidence to support its efficacy as a treatment for mild-to-moderate ALS. Instead of pushing the therapy forward to its December 2023 PDUFA date, BrainStorm elected to withdraw the biologics license application and conduct a Phase 3b trial—an endeavor it’s been raising money for ever since.
In June 2025, a new opportunity appeared to present itself when the biotech reported data from 10 Phase 3 participants who entered NurOwn’s expanded access program (EAP). Nine of these patients survived more than five years from ALS symptom onset, with median survival of 6.8 years. According to other published estimates, only about 10% of people with ALS survive past 5 years, BrainStorm said in its press release.
Following these EAP data, several people with ALS submitted a Citizens’ Petition to the FDA requesting that the agency approve NurOwn based on “new evidence and totality of evidence.” NurOwn trial participants and their families have been staunch advocates for BrainStorm, often speaking with BioSpace about the results they attribute to the cell therapy.
For his part, Pitts said the EAP data is “extremely promising. A lot of work yet to do, but very promising.”
However, with no further regulatory updates regarding NurOwn and the Phase 3b trial still stalled by funding issues, all has been quiet on the BrainStorm front of late.
“Financially we are far better off than a year ago, but awaiting final funding to start the trial,” BrainStorm president and CEO Chaim Lebovits told BioSpace last week. He expressed optimism that the study will be well underway this year. “If we find the funding very fast—which hopefully we will—we will be quite advanced in the Phase 3b trial” by the end of 2026, he added.
The trial will be run under a special protocol assessment (SPA), “significantly de-risking the regulatory aspects of the program,” BrainStorm said in a June 2024 press release.
This is the first-ever special protocol assessment (SPA) for an ALS trial, Lebovits said. An SPA is a legally binding agreement that the FDA is aligned on the trial’s design, he explained. The trial’s primary endpoint will be change in the Revised Amyotrophic Lateral Sclerosis Functional Rating (ALSFRS-R) total score from baseline to Week 24. The study will also look at survival.
Mizuho analyst Graig Suvannavejh told BioSpace in July 2024 that the SPA for NurOwn “is a good thing.” However, said, “I don’t know if it’s a great thing. The promise of a SPA is, if you show the data that the FDA has said that they want to see, the quid pro quo is that you should get your drug approved. What I’ve seen in the past is FDA changes their mind.”
Asked about the recent departure of FDA Commissioner Marty Makary and former CBER chief Vinay Prasad, and NurOwn’s prospects under the new leadership, Lebovits said, “we see these changes more in favor of rare disease and different products, including ALS.”
