ALS patients are now pushing the FDA to allow GM604 to be used. Patients are pushing for “accelerated approval” which will allow the drug to bypass large-scale trials.
A small Californian company named Genevron made this new drug. GM604 controls over 4,000 genes and modifies ALS progression. The drug was tested on mice with the SOD1 strain of ALS. Genevron said that GM604 was able to delay onset of symptoms by 27% and extend life for up to 30%.
The preclinical and Phase 1 trials also have good news. The results show statistically significant results with no side effects. The campaign for accelerated approval would make the drug available to all patients immediately.
One particular patient and his mother have made a very strong case for the approval. Anthony Carbajal and his mother made a video that went viral and caught thousands of attention. This particular video has over 530,000 supporters. In this video, Carbajal talks about how his grandmother and thousands of others died.
Although there is a solid case for accelerated approval, some are skeptical. Steve Perrin, a chief scientific officer of the ALS Therapy Development Institute, is such a skeptic. “The bottom line with the Genervon drug is there is absolutely no data.” He said. “There is no mathematical way or statistical way that they could measure a drug effect.”
Even with a few critics, this is surely a step forward to a cure for ALS.
To see Carbajal’s video click here