Genervon Biopharmaceuticals has published a list of 89 genes associated with amyotrophic lateral sclerosis (ALS) that are modulated by its drug candidate GM6. The identification of the genes regulated by GM6 helps to explain the mechanism of action of this treatment, which aims to improve neuronal repair and survival, and restore their proper function.
This information was presented at the 35th Annual JP Morgan Healthcare Conference, held in San Francisco on Jan 9–12.
Many clinical trials for ALS have failed because conventional drugs are designed to target a single gene or protein. This narrows down the efficiency of the drug because, in people whose brains are affected by ALS, several pathways are disrupted. By targeting several genes, GM6 may be a promising therapy approach that tackles different disease-associated pathways at the same time.
GM6 is a synthetic drug containing six amino acids that act on multiple pathways that play an important role in the development of neurons and the human nervous system during the embryonic stage. These amino acids specifically bind to a group of receptors, called the insulin receptors (IGF1 and IGF2 receptors), thereby activating pathways that regulate and repair the brain.
Several laboratory analyses showed that GM6 regulates the activity of 89 genes that have been previously associated with ALS. These genes are involved in signaling pathways that include neuronal generation, regulation of neuron death, and the regulation of oxidative stress-induced death.
They also found that GM6 helps to lower expression levels of SOD1, a known ALS gene. Previous studies have shown that mutations in the SOD1 gene account for 20 percent of inherited ALS cases, but misfolded versions of the SOD1 protein may also lead to the development of this disease.
Results obtained while studying the impact of GM6 treatment in the genetic profile of neurons support that this drug has a three-part mechanism of action. That mechanism includes: reducing SOD1 expression (avoiding its toxic accumulation in motor neurons), protecting the mitochondria (the cells’ powerhouse), and activating neuronal repair, growth and maturation. Details of the study can be found here.
GM6 was developed to target several neurological disorders, such as ALS, Parkinson’s, Alzheimer’s and Huntington’s diseases. It has been shown to have a safe profile in a Phase 1 and three Phase 2 trials with patients with ALS, Parkinson’s and stroke.
According to Genervon, a Phase 3 trial for ALS is expected to start this year.
So when do you think we will see this in real use ?
We do not know at this time Neil. We will certainly report on it when we have a definite time frame from the researchers.
Hurry, please.
The FDA has fallen asleep at the wheel in regards to ALS, as well as other diseases!^ Call your congressman and meet with them in person so they can see your face and the mental pain we are in. Help them put a face with the suffering…take family with you so they can see the number of people affected by one dreaded, hopeless diagnosis.
Thank you for sharing that encouragement.
I need itnow … in 7 years with ALS this is the best news that I have … thanks god… could I be a volunteer for phase 3?
Ask your doctor Reynaldo… we hope you are able to get some relief soon.
Where will the phase 3 trial be. Would like to participate in the GEnervon trial
Any idea when this year the trial will begin and where? Do trials only take place at one location or might multiple ALS clinics try the same drug?
Very encouraging .
Isnt GM6 being used in some countries already? Think it was Italy, Argentina, South Africa and Australia. Any reports from those patients?
We have not seen any news on this, but will keep searching. Thank you for the info.
Genervon Releases ALS, PD and AD Disease-Associated Gene Lists Modulated by GM6: http://www.businesswire.com/news/home/20170110005650/en
International PALS case reports: http://www.genervon.com/genervon/medicines_20160919A.php
During the JPM Healthcare Conference in San Francisco a couple weeks ago, Genervon decided to publish the very confidential ALS, PD and AD associated gene lists modulated by GM6 in order to encourage neurological researchers to explore and discover beyond the single target paradigm for multifactorial disorders of the brain and nervous system.
Genervon Releases ALS, PD and AD Disease-Associated Gene Lists Modulated by GM6: http://www.businesswire.com/news/home/20170110005650/en
International ALS patient case reports: http://www.genervon.com/genervon/medicines_20160919A.php
Why is a phase 3 trial necessary? Just distribute the treatment! What’s the worst thing that can happen to someone with ALS?!
My brother cannot wait that long!! He will not live long enough for you to do one more trial. At least, please, include VCU ALS Research Center in Richmond, VA to participate in the study. Please.
i’m wating for the phase 3 also but more so the treatment! been dealing with a c9orf72 for a year and a half now. will travel!
My partner is newly diagnosed with ALS (today). She has the bulbar form, with the first symptoms being slurred speech and arm/hand fasciculations. Is this drug administered IV or in pill form?
We are very sorry to hear about your partner and her diagnosis. To answer your question, I am not sure what form this medication is in.
Susan, I am sorry to hear about your partner having ALS, my girlfriend was diagnosed 2 months after yours. Having the same symptoms. Hand weakness and dexterity loss, now her speech is starting to slur. I’d like to know how your partner and you are dealing with things. Would like to talk and share stories of what works and what doesn’t. Contact info is [email protected]ast.net