We Cannot Wait: FDA Needs to Act on ALS

There are 30,000 Americans living with Amyotrophic Lateral Sclerosis (ALS), a 100 percent fatal diagnosis that usually kills people in two to five years. Despite this currently grim outlook for patients, the Food and Drug Administration (FDA) has refused to exercise regulatory flexibility that would lead to faster approval and availability for ALS treatments. Hopefully, a recent announcement from FDA signals the agency may be moving beyond a “one size fits all” approach in order to responsibly help more patients.

With the welcome news that the FDA has reversed its position on AMX0035 – saying it will now review the treatment – ALS patients have new hope that a treatment that has shown itself to have positive effects could be increasingly available. These patients have traveled a long road to get to this point.

The House Energy and Commerce Subcommittee on Health convened a hearing in July to question the FDA’s slow approval process for treatments for neurodegenerative diseases. The hearing was an important step in recognizing the urgent need for FDA to use regulatory flexibility for promising ALS treatments, with lawmakers on both sides of the aisle urging the FDA to help patients living with ALS today.

Although the FDA has incorrectly stated that the science is not there for ALS, leading ALS neurologist Merit Cudkowicz is hopeful, saying “there had previously been no hope for ALS patients, but now there is.” We agree; we are living proof that the science for ALS is there today.

As participants in a Phase 3 clinical trial and the Expanded Access program for an investigational therapy called NurOwn, we were fortunate to gain access to this individualized stem cell therapy. For us and others, NurOwn improved physical function and even halted the progression of ALS, particularly among patients with less advanced cases of our disease. But, our ability to continue receiving this life-changing treatment is gone. We need more doses in order to live, but FDA’s current stance on this therapy ensures that we won’t get any nor will anyone else.

The situation is dire. Only 1,300 clinical trial seats are available for people living with ALS in the United States, and many ALS patients will receive a placebo rather than an experimental treatment. There are even fewer opportunities for expanded access. We all know the outcome without treatments; we will lose our ability to walk and speak before succumbing to paralysis and an inability to breathe, followed by death. 

There is important legislation before Congress that would broaden expanded access programs, while investing in the research and development of neurodegenerative treatments. The ALS community needs Congress to pass this legislation and to pressure the FDA to expedite approval for NurOwn and AMX0035. Expanded Access offers a viable path to access for some patients, but it is no substitute for FDA approval.

Yet, FDA’s current posture on promising ALS treatments means they may not be approved for at least another four years. During his testimony, ALS patient and advocate Brian Wallach noted by the time these critically needed medicines are approved, every American living with ALS today will have died without access to treatments that could have improved their quality of life or even halted their disease progression.  

Denying patients a treatment that could extend their lives by even six or 12 months robs them of the opportunity to be with their families for another birthday, graduation, or wedding. As fathers and husbands ourselves, we know these milestones are the moments that make up a life. We know a cure for ALS has not yet been discovered, but until one is, we cannot accept that treatments that would help keep our families whole – even if just for a short amount of time – remain out of reach due to bureaucratic red tape.  

We are pleased to see the FDA’s Center for Drug Evaluation and Research, led by Patrizia Cavazzoni, M.D., reverse its position on AMX0035, and we are hopeful the agency’s biologics division, directed by Peter Marks, M.D., will do the same for NurOwn. Even if these treatments benefit only a small population of patients, it creates a chance to extend the lives of terminal patients who have no other options. For us, these treatments provide hope, but we need the FDA to approve them for patients living today. We do not have time to wait.

Phil Green is a former collegiate athlete and has been living with ALS since August 2018. Eric Stevens is a former NFL player and LAFD firefighter diagnosed with ALS in August 2019.