GM6 (previously known as GM604) is a potential treatment for amyotrophic lateral sclerosis (ALS) being developed by Genervon. The company is also developing GM6 as a potential therapy for Alzheimer’s disease. The U.S. Food and Drug Administration (FDA) has granted orphan drug status and fast-track status to GM6.
How GM6 works
ALS is a condition characterized by the loss of motor neurons (nerve cells that carry signals from the brain to the muscles, controlling their movement). The disease can sometimes be caused by mutations in a gene that encodes for an enzyme called SOD1. The mutations can lead to the enzyme being made incorrectly and clumping together, forming aggregates that are poisonous to nerve cells.
GM6 is an artificially created peptide. In contrast to other therapies, GM6 does not have a discrete target, but rather affects multiple pathways to treat ALS and promote motor neuron survival by slowing degeneration through an unknown mechanism.
GM6 in clinical trials
Research has shown that GM6 can be effective in disease modification in a mouse model of ALS. GM6 increased survival in this model and conferred dose-dependent improvements in physical performance measures of disease progression.
A Phase 2A randomized, double-blind, placebo-controlled clinical trial (NCT01854294) assessed the efficacy, safety, and tolerability of GM6 in 12 patients with ALS. The participants received six doses (three per week for two weeks) of either GM6 or placebo. Efficacy was measured by the percent change in protein markers for ALS measured from cerebrospinal fluid. Safety and tolerability were assessed by the number and severity of adverse events.
The results, published in F1000Research, confirmed the safety of GM6 in humans, with no serious treatment-emergent adverse effects. Additionally, favorable trends were observed in GM6-treated patients, including a slowed decline in ALS functional rating scale (a measure of disease progression). These findings suggest that GM6 may help motor neuron survival in ALS patients.
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