Clinical Trial Results Support Genervon’s GM6 as Promising ALS Treatment

Clinical Trial Results Support Genervon’s GM6 as Promising ALS Treatment

Genervon Biopharmaceuticals has published the results of a somewhat promising Phase 2a clinical trial investigating the effects of GM604 (or GM6) in patients with amyotrophic lateral sclerosis (ALS).

The study, “A Phase 2A randomized, double-blind, placebo-controlled pilot trial of GM604 in patients with Amyotrophic Lateral Sclerosis (ALS Protocol GALS-001) and a single compassionate patient treatment (Protocol GALS-C),” appeared in the journal F1000 Research.

The data showed that treatment with GM6 improved ALS biomarkers — as well as clinical and functional measures in the ALSFRS-R (a scale that measures disease status) and FVC (a measure of respiratory capacity) — in ALS patients, including those with advanced forms of the disease.

GM6 is a synthetic drug containing six amino acids that act on multiple pathways crucial to the development of neurons and the human nervous system during the embryonic stage. These amino acids specifically bind to a group of  insulin receptors (IGF1 and IGF2), activating pathways that regulate and repair the brain.

Previous work by Genervon showed that GM6 acts by regulating the activity of 89 genes involved in signaling pathways affected in ALS, such as neuronal generation, regulation of neuron death and regulation of oxidative stress-induced death in mitochondria, the cell’s powerhouse.

One of these genes is SOD1, a known ALS gene that accumulates in neurons, causing damage. According to the Pasadena, Calif.-based company, expression levels of SOD1 decreased with GM6 treatment in ALS patients.

“Our findings suggest a tentative tripartate mechanism of action by which GM6 could prolong motor neuron survival in ALS patients,” Genervon stated in a news release. “First, by reducing SOD1 expression, GM6 may block accumulation of pathologic SOD1 aggregates in motor neurons. Second, by reducing mitochondrial gene expression and potentially mitochondrial abundance (decreasing total tau), GM6 may disrupt the mitochondrial (intrinsic) apoptotic pathway. Third, GM6 appears to activate developmental/mitotic pathways (Cystatin C), which may promote cellular repair, axonogenesis, and neuron projection.”

Together, these results support GM6 as a promising new treatment for ALS, but more studies are necessary to confirm these effects. Genervon plans to open a Phase 3 ALS trial in the United States this year.

Joana brings more than 8 years of academic research and experience as well as Scientific writing and editing to her role as a Science and Research writer. She also served as a Postdoctoral Researcher at the Center for Neuroscience and Cell Biology in Coimbra, Portugal, where she also received her PhD in Health Science and Technologies, with a specialty in Molecular and Cellular Biology.
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Joana brings more than 8 years of academic research and experience as well as Scientific writing and editing to her role as a Science and Research writer. She also served as a Postdoctoral Researcher at the Center for Neuroscience and Cell Biology in Coimbra, Portugal, where she also received her PhD in Health Science and Technologies, with a specialty in Molecular and Cellular Biology.

25 comments

  1. Robin Davis says:

    Thank you ! A very interesting and hopeful article. Were an c9orf72 ALS persons included in this or any GM6 research ?

  2. Debbie McCravy says:

    More studies, more studies, more studies. It’s ironic to say that I am “sick” of it when my husband is the one who has ALS. If there is a chance of a cure or even stopping the progression….it’s the patient’s right to try a treatment.

  3. Robert Cebaus says:

    This all sounds great, will look forward to more info. I am new to all this. It seem that this may help, I did not see anything about side affects. Is there or do they know of a down side to this. I have been told about 8 weeks ago that I have ALS so am in the early stage and have a lot to learn.
    Bob

  4. DON NG says:

    why so many trials going on fda should make approval fast people are dying of AlS i m the sufferer too pls help us we do not want so many trials we want treatment

  5. Ismael Fernandez says:

    Please help.People with ALS dont have time for FDA approval.If it has already shown that it has been beneficial why wait.

    • Karen says:

      I have gone from a person who could walk 5 miles a day in May to less than a mile now and I can get no help. I try to be hopeful for some help but really feel hopeless.

  6. Jasmyn Romao says:

    I have someone very close t0 me suffering from this asshole ALS. I would love to know more . He’s in the aggressive stage. Please help now

  7. PAULA DIANN CLARK says:

    My husband is dying of ALS right before my eyes. He has rapid progressive ALS. Can anybody help him? He is a great man, i’ll be lost without him, Dennis God wants you in heaven.

  8. ZINAR says:

    please help us. my mom is diagnosed with ALS and i can’t loose her. She is all I have. any possible trial we are volunteer. HELP HELP HELP….

  9. AChang says:

    Hi,
    Unfortunately the ALS is more progressive than ” how long we should wait to be healed”. I am from Portugal and my syster was diagnosis with ALS, in case you know when will be approved the GM6, and if some can tell which write food she could eat, she started to lose height, and we need to find a correctly “fat diet” for her.
    Thank you
    AC

    • Oops…in my last reply to you, the text says sALSs. My voice-to-text is out of control and they don’t have an edit button on this site. That should have said “I come from a sALS and fALS family.

  10. A.Chang says:

    Hi,
    Unfortunately the ALS is more progressive than ” how long we should wait to be healed”. I am from Portugal and my syster was diagnosis with ALS, in case you know when will be approved the GM6, and if some can tell which write food she could eat, she started to lose height, and we need to find a correctly “fat diet” for her.
    Thank you
    AC

  11. Rodney Harry says:

    If the compound is any good (and it looks to be both good and safe), then they’ll be resistance (unless the company has investors who can pull strings with the regulatory agencies). I suspect that they have at least some friends in high places to make it this far (better connections than the poor scientists who don’t get their compounds launched or companies shut down…even by our “charities”)…but, who knows? I consider our regulatory body unpredictable.
    Along those lines, anyone know anything about the RCH4 shut-down? What they said about ALSTDI not picking up their product to study without huge extortion-prices (at 240K for one mouse study) was not good (then the scientists got the compound studied at a top US lab for only 68K?). I’m taking screenshots of the dialogue on the RCH4 website before the commentary on the events is forcefully removed.
    There are wonderful people at ALSTDI and they don’t have a clue that something is terribly wrong going on there. I’ve remained neutral over them the last few years, in silent observation. Time it’s self is revealing.
    This community, collectively, is disturbing. Anything valuable that advances is outside the USA (unless backed by big money). There are no non-profits in the real sense. Everyone has to make money to survive. But, we need to hold these charities to higher scrutiny and PALS alone aren’t equipped to do this. Who can help?

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