Today, a group of ALS patients will come together to protest at the Food and Drug Administration building in Maryland to say they have not seen a response in research from the $115 million raised globally from the ice bucket challenge.
“We as ALS patients are just tired of no action being taken on our part,” said Mike Henson, one of the organizers. “I don’t know what’s worse, getting ALS 10 years ago and just knowing you’re going to die or getting it now and knowing that you could be saved if only people would take this seriously.”
The group is looking for answers to three potential drugs currently in the development process. They are: BrainStorm’s stem cell treatment NurOwn, Collaborative Medicinal Development’s synthetic molecule CuATSM, and Stanley Appel’s cell therapy known as Tregs. So far, BrainStorm is enrolling a Phase 3 clinical trial, Collaborative Medicinal Development has completed Phase 1 testing and Appel’s Tregs has only been studied in three patients.
The group wants to know what is taking so long for the FDA to approve these drugs. Henson points to the drug Zolgensma, a gene therapy for spinal muscular atrophy approved with only testing on 15 patients.
“That drug was approved very quickly with a very small trial,” Henson said. “So, we know this can be done, if we have the will to do this.”
