• ALS Crowd News
    • Apr 16, 2015

    FDA Pressed to Allow Access GM604

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

About Author

Seth Christensen

Seth is an ALS patient and founder of ALS Crowd, a division of the CrowdCare Foundation. As host of the ALS Crowd Radio show, he interviews top ALS researchers and focuses his efforts on the aggregation of big data to help researchers and patients find clues that will drive to a cure.


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