California-based biotechnology company Genervon has announced results of a Phase 2 clinical trial involving 12 amyotrophic lateral sclerosis (ALS) patients. The experimental drug, called GM604, has produced “very robust” and “dramatic” results, according to a research note.
Recently the company applied to the U.S. Food and Drug Administration (FDA) for accelerated approval. If granted this would mean that the drug could skip a Phase 3 clinical trial and head straight to market.
Patients who typically only live two to five years past diagnosis and who have no effective treatment options, quickly have begun calling on the FDA to grant the company’s request.
This view is not supported by all scientists, with some arguing that a larger trial is necessary before the drug becomes widely available, according to The Washington Post.
For example, David Gortler, a pharmacology expert, is quoted as saying: “All this petitioning and press releases over such little data is premature. I think Genervon is preying on the lack of information that the average person has about the drug-development process. . . . You can’t rush the scientific process. Good science takes time.”
On the other hand, if Phase 3 is adopted, most of the people currently diagnosed with ALS will not be around. As part of a campaign to have the drug released, over half a million signatures have been collected on an on-line petition hosted by Change.org.
The patients and their supporters have also made a video, calling for the drug to be released:
