Genervon Biopharmaceuticals has published a previously confidential list of Parkinson’s disease-associated (PD) genes it says are controlled by GM6, its investigational treatment for PD and other central nervous system (CNS) diseases.
The list was released during the 35th annual JP Morgan Healthcare Conference, held recently in San Francisco. Genervon also recently published lists of genes it says are associated with amyotrophic lateral sclerosis (ALS) and Alzheimer’s disease.
“Genervon decided to share our confidential research findings, including the specific gene names, with the research communities with the goal of persuading neurological researchers to go beyond the single target drug development paradigm,” the company said in a press release.
GM6 is made up of six amino acids that act on multiple pathways that play important roles in the development of neurons and the human nervous system during the embryonic stage. The amino acids bind to a group of insulin receptors (IGF1 and IGF2 receptors) without altering insulin signaling. The mechanism of action for GM6 appears to involve many different pathways, and it promotes homeostasis and correction of multiple disease states, the company said in the release.
In the Phase 2a trial involving six PD patients, results showed the drug was well-tolerated and caused positive changes in patients’ blood biomarkers. Genervon summarized the findings in the recently published poster, “GM604 for Parkinson’s Disease treatment: pre-clinical findings and results from a pilot placebo-controlled trial.”
Researchers used multiple experimental mouse models to evaluate GM6 as a PD drug candidate. They found that it has protective effects that help improve patients’ motor performance and dopaminergic neuron survival.
They used DNA microarrays to identify GM6-regulated genes and found that a significant number of them are associated with PD. (DNA microarrays are a collection of microscopic DNA spots attached to a solid surface, such as a plastic or glass slide.)
Based on all their findings, the researchers said they believe GM6 can prevent dopaminergic neuron death through multiple means, including by activating developmental, pro-survival pathways, and correcting mitochondrial dysfunction.