The US Food and Drug Administration (FDA) is responsible for developing guidance for amyotrophic lateral sclerosis (ALS).  The guidance provides a roadmap to those in the ALS industry to navigate through the development of treatments. To develop new treatments for ALS, the guidance would incentivize industry sponsors by directing trials to be more efficient, predictable, and effective at assessing drug safety and efficacy outcomes. The FDA has failed to deliver the guidance in a timely fashion and is now culpable to the inhumane treatment of ALS patients.

ALS is a progressive terminal neurological disease that can strike anyone at any time. The disease currently has no meaningful treatment. Patients are often told to get their affairs in order, and that they have 2-5 years left to live. You have a 1:400 chance of developing ALS in your lifetime and only 10% of patients live longer than 5 years. There is no typical progression of ALS; however, it eventually paralyzes every aspect of a patient’s ability to move. Eventually, patients are no longer able to walk, talk, swallow, and breathe on their own.

A federal law in 2012 allowed for patient-focused drug development (PFDD). After extreme pressure in May of 2015, ALS patients finally met with the FDA to discuss the drafting of a guidance document. Due to the lack of urgency from the FDA, the ALS Association (ALSA) took ownership of creation to expedite the development of the guidance. In August of 2017, the ALS community submitted the results to the FDA. In February 2018, the FDA published the Draft Guidance that completely ignored the work of the ALS community. The open comment section closed in May of 2018 after receiving hundreds of comments related to the published inhumane boilerplate Draft Guidance. In July, ALSA convened with the FDA and advocates to discuss the guidance. Due to the lack of results, ALS patients and advocates met with the FDA in February 2019. At that time Dr. Janet Woodcock and Dr. Billy Dunn stated that the ALS Guidance would be published within 2 months. After this deadline passed without publishing the ALS Guidance many patients and advocates reached out to the FDA. The lack of acknowledgement by the FDA for missing their deadline truly demonstrated that the ALS community is not a priority. In June of 2019, another ALS advocacy group, No More Excuses met with some of the same people at the FDA. ALS patients and advocates are doing everything possible. However, the FDA ALS Guidance remains extremely elusive. The FDA must be held accountable and publish the ALS Guidance.

We need you to sign our petition to hold the FDA accountable of their negligence to the ALS community.

Below are immediate requirements of the ALS Guidance along with an example from just one current clinical trial (NurOwn):

• Using historical controls for placebo groups – FDA inhumanely required 100 terminal patients to get a placebo (fake medicine) injected into their spinal cord (3x) and receive bone marrow aspiration. There are clinical trials for cancer and HIV/AIDS that eliminate the requirement of placebo due to the inhumane nature of testing fake medicine on a terminal patient. Historical controls allow the use of more actual placebo data than the use of placebo groups themselves. It also prevents the deterioration that ALS patients are forced to undergo as they receive a placebo. Placebo trials in ALS are akin to forcing people to die "in the name of science".
• Wider onset criteria – Trials usually have inclusion criteria that are set less than 18 months from first symptom. It typically takes 12-18 months to be diagnosed with ALS, patients often have symptoms longer than that. This excludes many from clinical trials. This is inhumane and forces trials to take longer to enroll participants.
• Requirement of crossover or open-label – Patients have no access once they have completed the trial. Patients on placebo also have no access to the treatment after completion. There are patients who show improvement or slowing of ALS in trials that are forced to go home and continue deteriorating after the trial.
• Remove penalties for unmasking data to apply for Accelerated Approval – Due to the additional 100 patients and strict onset criteria, BrainStorm is still trying to complete the NurOwn trial as ALS patients die. If BrainStorm decides to unmask the data early to apply for Accelerated Approval they are at risk of jeopardizing the trial.
• Incentivize Agencies to provide Right to Try and Expanded Access to patients that don’t qualify for trials – Outside of one patient, BrainStorm has chosen not to allow Right to Try or Expanded Access. There are no incentives to allow terminal patients that don’t qualify for the trial access to this treatment.
• Innovative Guidance – The FDA routinely calls for innovative trials but requires ALS to have archaic designs. The ALS Guidance needs to be a responsive document that is quickly amendable as innovation evolves.  A living breathing document will expedite updates and remove the slow federal bureaucracy that is occurring now.

We ask for Executive Order or Congressional action to order the FDA to expeditiously publish humane and ethical Guidance for ALS and to hold those responsible for these failures accountable. 

Since 2012, roughly 50,000 US ALS patients have died waiting for humane ALS Guidance from the FDA. Please sign this petition to show your support.

Authors

Twitter: Corey Polen  , End ALS 

Facebook:  #StandForCorey  , End ALS