Pasadena Company Says A Drug They are Developing Successfully Slows Down Progression of ALS

A pharmaceuticals company based in Pasadena announced today that a drug they are developing to combat neurodegenerative disorders has successfully slowed down the progression of ALS in a clinical test and can modify genes associated with the nervous system.

ALS, amyotrophic lateral sclerosis, is a progressive neurodegenerative disease that affects nerve cells in the brain and the spinal cord.

Genervon Biopharmaceuticals provided data to an external contract research organization that confirmed the drug — known as GM6 — can edit genes in the nervous system that have an impact on defense against stress and upkeep of cellular balance, according to a statement released to the media.

About 1,200 genes showed a strong response to GM6 treatment, based on the most stringent of statistical thresholds, according to the press release. Using a less conservative criteria, approximately 3,000 genes showed change.

These numbers affirm that GM6 has major regulatory effects in neuronal cells and can be classified as a “master regulator” of the human CNS, according to the media statement.

Despite the company’s claims, Steve Perrin, the CEO of the ALS Therapy Development Institute and John Carroll of Fiercebiotech have challenged the significance of the trial Genervon conducted, saying the number of ALS patients — 12 — was too small for the early-phase test run.

However, there is currently a petition on Change.org with 800,00 signatures asking the FDA to speed up approval of the drug.

GM6 is a peptide derived from HGMF, another peptide that is responsible for the development, protection and correction of the CNS during fetal development, according to the press release. It was discovered by Genervon using a technique called “protein band-fishing by cells” and is known to be highly specific to the human nervous system, rapidly releasing itself during the third trimester of pregnancy.

Genervon is developing the drug to combat prominent neurological disorders, such as Lou Gehrig’s Disease, Parkinson’s Disease, Ischemic Stroke, Muscular Dystrophy, Huntington’s Disease and Alzheimer’s Disease.