On March 25, 2015, ALS patients and their families held a rally on Capitol Hill demanding accelerated approval of GM604, a drug developed by California biotechnology company Genervon. The drug is a short peptide derived from motoneuronotrophic factor (MNTF), a protein involved in embryonic development, and it modulates over 80 ALS-related genes. Last fall, Genervon announced via press release results of a 12 patient Phase II study, as well as a single compassionate use patient (see Jan 2015 news story). In February this year, the company filed a formal request for an Accelerated Approval Program from the FDA, which would grant drug approval based on a surrogate endpoint, without the need to conduct lengthy, expensive clinical trials. As the ALS community awaits the decision by the FDA about GM604, the debate around access to this drug continues. Leaders in the clinical and research community have voiced concern that the due to the small scale of the Phase II trial, further confirmation of the results in a larger study of longer duration is necessary, while from the perspective of ALS patients, the promise of benefits from the experimental drug far outweigh the risks of side effects or lack of efficacy.
Click here read to read the recent Washington Post article on the debate.