Dr. Diana Zuckerman’s Testimony at the FDA Advisory Committee on NurOwn for ALS Treatment

September 27, 2023

I’m Dr. Diana Zuckerman, president of the National Center for Health Research. Prior to my current position, I was trained in epidemiology and on the faculty at Yale, a research director at Harvard, and a bioethics fellow at the University of Pennsylvania.  I am also a founding board member of the Alliance for a Stronger FDA, which is a coalition of industry and nonprofit organizations focused on ensuring funding for this very important agency.

Our public health think tank focuses on the safety and effectiveness of medical products and we do not accept funding from companies that make those products. So I have no conflicts of interest.

Thank you for serving on this important Advisory Committee.  I know how challenging it is to balance the desire to help patients who have a devastating disease while focusing on your role to provide your expertise, especially given the conflicting information presented today – and the implications for FDA’s reputation as a gold standard.

I’ll focus on a few key results from the Phase 3 study, which is the only controlled study to evaluate the treatment using the intended route and dose interval.

There was no benefit for the primary efficacy endpoint – in fact, the chance that treatment was better than placebo was about 50% – that’s like flipping a coin.  None of the key secondary efficacy endpoints showed any benefit. With all due respect, it is not appropriate to combine nonsignificant results into a significant trend because the more comparisons you do, the more likely one of them would have been statistically significant – but none of them were.

Perhaps most important:  More patients taking the treatment died: At 28 weeks 10 patients receiving treatment (of 95) had died, compared to 3 deaths of 94 patients the placebo group.   More than triple the number of deaths – and the chi square is statistically significant!  The patients who died on this treatment are not here today, and I want to represent those patients’ results, which haven’t gotten much attention so far today.

The improvement at week 28 was exactly the same for treatment group as the placebo group – 14% improved.  This should remind us that some ALS patients will improve even with no treatment.  That’s why a controlled trial is so important, and why individual success stories – however heartening — can be misleading.

In conclusion, patients deserve clear evidence so they can make informed decisions.   And we all deserve an FDA that approves treatments based on scientific evidence.  Patients should have the option of being in a free clinical trial or expanded access program, but it would be unconscionable for patients to pay for this unproven treatment – which they would do if FDA approved it.


The FDA Advisory Committee voted 17-1 that the data did not support the company’s claim that NurOwn was effective. The consumer representative was the only vote in favor, citing patients who lobbied in favor of NurOwn, whereas the physicians, researchers, and the patient representative who had ALS all voted against it because of the risks and lack of efficacy.  The company subsequently withdrew its application for approval.