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ALS community petitions BrainStorm and the FDA to urgently determine a path forward for NurOwn


Amyotrophic Lateral Sclerosis (ALS) is a rapidly progressive, 100% fatal disease that kills most people within two to five years of diagnosis. For the tens of thousands of Americans living with ALS today and the thousands more who will be diagnosed this year, little progress has been made in effective treatment options. In fact, the prognosis of an ALS diagnosis is essentially the same today as it was when the disease was first discovered more than 150 years ago. ALS is a serious disease with unmet medical need. People living with ALS deserve access to safe and effective treatments as quickly as possible.  

NurOwn is an investigational stem cell-based treatment developed by BrainStorm Cell Therapeutics that has shown encouraging results in Phase II and Phase III clinical trials. Although NurOwn did not achieve its primary endpoint in the Phase III trial, it proved safe and shows considerable promise in people with less advanced ALS. According to the peer-reviewed study

  • NurOwn treatment led to a trend toward slowed progression in a pre-specified subgroup of people with an ALSFRS score of 35 or higher.
  • Cerebrospinal fluid collected from all participants showed robust and sustained biomarker improvements with NurOwn, but not with placebo.
  • Post-hoc analysis suggests that NurOwn could have clinical benefits for participants with an ALSFRS score above 25. This is further supported by patient reported outcomes from a separate expanded access program.

Given these results and the critical unmet medical need of the ALS community, we strongly urge BrainStorm and the FDA to work together to determine the best path forward for NurOwn, ensuring urgent access to those who may benefit. 

In delivering this petition, I AM ALS, partnering ALS organizations and the ALS community insist on safe and effective treatments supported by rigorous science, while also recognizing (1) the critical and unmet need for effective treatments in this 100% fatal disease, and (2) the reality that ALS is a highly heterogeneous disease and treatments that work for a subpopulation of people should be pursued urgently. 

 Petition 

I AM ALS, partnering ALS organizations and the ALS community call on BrainStorm Cell Therapeutics and the Food and Drug Administration (FDA) to urgently move NurOwn through the regulatory process.  

We, the undersigned, demand the following actions: 

  • BrainStorm must work with the FDA to urgently establish a path forward for expeditious NurOwn review and, if needed, consideration of whether confirmatory evidence could appropriately be collected in post marketing requirements.

Clinical trial data show that NurOwn is safe, well-tolerated and highly promising for a community with few other treatment options. Although the Phase III trial did not meet its primary endpoint, the results, including a peer-reviewed study and patient reported outcomes, suggest that NurOwn has a clinical benefit for at least some people. As such, it is imperative that BrainStorm and the FDA work together urgently to determine the most expeditious path forward for the program, to include filing a Biologics License Application (BLA).  

  • The FDA must exercise appropriate flexibility to expedite the development and review of NurOwn, a safe and promising investigational ALS treatment. 

ALS is highly heterogeneous and 100% fatal. As FDA considers the path forward, and once a BLA for NurOwn is submitted, the FDA must act swiftly and in accordance with its own 2019 Guidance for drug development in ALS (including but not limited to careful consideration of patient-reported outcomes and biomarker data). In addition, we encourage FDA to leverage all appropriate authorities and flexibilities afforded under the Public Health Services Act and the Federal Food, Drug and Cosmetics Act to expedite the development of drugs for serious, life-threatening, rare conditions with minimal available treatment options and critical unmet medical need. 

Supporting ALS Organizations

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