September 20 – Update to I AM ALS statement regarding NurOwn Advisory Committee Meeting

UPDATE September 20, 2023 – I AM ALS is pleased to announce that over 10,000 ALS advocates and 20+ ALS non-profits have signed on to our NurOwn Advisory Committee Meeting statement below as of today! The following nonprofit organizations have signed on:

August 21, 2023 – Amyotrophic Lateral Sclerosis (ALS) is a 100% fatal disease that causes increased paralysis over time and usually progresses rapidly. Ultimately, people living with ALS lose the ability to eat, talk, breathe, or move on their own. Most people typically live only 2 to 5 years after diagnosis. Half of people living with ALS die within 18 months of diagnosis.(1)

The community of individuals living with and affected by ALS has been waiting years for the upcoming FDA Advisory Committee review of the experimental cell therapy, debamestrocel (NurOwn). All the while, many of these same individuals are progressing in their illness and – unfortunately – many of them have died of ALS during our wait. It is imperative that Advisory Committee members and FDA’s Center for Biologics Evaluation and Research (CBER) recognize the urgency of this disease and how very important this Advisory Committee meeting is for the ALS community. 

The FDA has long stressed the appropriateness of regulatory flexibility in applying the statutory standards to drugs for serious diseases with unmet medical needs, while preserving appropriate assurance of safety and effectiveness. I AM ALS and its community are grateful that regulatory flexibility has been exercised for previous ALS therapy evaluations and approvals. We thank the FDA for that. As we work together towards new therapies and cures, we urge Advisory Committee members and CBER to continue the pattern of regulatory flexibility for debamestrocel and other ALS treatments for several reasons: 

1. While no cure has been identified, each treatment helps move ALS further from a fatal disease to becoming a chronic disease. The importance of this cannot be understated. 
2. Each new ALS therapy approval gives those living with ALS more longevity and better quality of life. 
3. Any increases or preservation in functionality measured by an ALSFRS-R score – big or small – are monumental for individuals living with the disease, their loved ones, and the entire ALS community. 
4. Evidence considered must include all types of data – including trial data from the manufacturer, the landscape of ALS treatments, limitations of ALSFRS-R scoring system as a clinical trial endpoint, and the science – including the biomarkers AND the human experience from patient-reported improvements in functionality. Human experiences are critical data points that cannot be ignored in clinical research analysis.

I AM ALS and the ALS community are asking for a fair Advisory Committee meeting on September 27th that allows debamestrocel’s totality of evidence be considered. We ask the Advisory Committee to recommend that NurOwn receive approval consistent with recent ALS therapy approval processes.  We also ask for a confirmatory trial that includes the community in its creation and implementation.

(1)  Rodríguez de Rivera, F. J., C. Oreja Guevara, I. Sanz Gallego, B. San José Valiente, A. Santiago Recuerda, M. A. Gómez Mendieta, J. Arpa, and E. Díez Tejedor. “Outcome of Patients with Amyotrophic Lateral Sclerosis Attending in a Multidisciplinary Care Unit.” Neurología (English Edition) 26, no. 8 (October 1, 2011): 455–60. https://doi.org/10.1016/j.nrleng.2011.01.010.