Genervon Biopharmaceuticals LLC (“Genervon”) today announced that it has successfully completed its Phase 2a clinical trial for amyotrophic lateral sclerosis (“ALS”) disease modification. A full analysis of the trial’s results is expected to be completed in the third quarter of 2014, but preliminary data suggests that Genervon’s novel, proprietary, multi-target biological drug candidate, GM604, shows significant promise for treating ALS.

As many of you are aware, Genervon is researching GM604, 606, and 608 for ALS, Stroke and Parkinson's patients. I participated in the GM608 PD trial. Results should be released this summer. As they haven't yet broken the drug/placebo labels, I don't want to discuss my experience in detail. However, its suffice to say that today's news could be very important for Parkinson's patients.

About GM6:

Recent genomic, proteomic, and systems biology studies suggest that CNS disorders and diseases implicate a highly complex, multi-factorial process that involves the interplay of many non-dominant effectors in an interwoven dynamic network. That expounds the futile efforts of single action drugs.

MNTF is a specific, functional, regulatory, endogenous peptide for the protection of the nervous system from disorders and diseases including neurovascular and neurodegenerative diseases. Having discovered MNTF by its function, Genervon used DNA micro-array analysis to find the genes that are activated by its analogue GM6. That analysis indicated that GM6 up or down regulates, by at least two fold, more than 4,000 genes. Many of the modulated genes are related to nervous system disorders and diseases, and neurodegenerative diseases, including those in command of pathways for apoptosis, oxidative stress, inflammation, lipid and immune response.

These analyses showed that GM6 activates genes involved in neurogenesis, neural development, neuronal signaling plus many specific biological functions. GM6 modulated significantly genes through many important pathways. GM6’s coordinated modulation of such a large number of genes suggests a master-regulator that effects on this extremely intricate network of disease-related pathways.

http://www.news-medical.net/news/201...sclerosis.aspx

Does anyone know if the placebo response is as robust in ALS as it is in Parkinson's?

Yes, that certainly is the question that comes to mind when reading the press release and wondering why there was no mention about the control group. The little knowledge I have about ALS trials is that they do have placebo response issues, no different than most other disease studies. I have tried getting more information from both the company and the trial team, but to no avail. I was told that there will be a more detailed report coming out soon. Also, they are definitely moving forward with a larger, longer-term trial. As they are a small company, yet self-sponsoring the trial at their own expense, I'm hoping that they are satisfied that they have seen more than just a placebo response.

With regard to our main interest, PD, I was also told that they are planning a larger trial to begin later this year.