Genervon’s GM6

Over the last couple of weeks, you may have seen information regarding Genervon’s GM6 therapy. GM6 is a naturally occurring peptide that regulates multiple genes and has shown no health risks.  Results of the latest trial suggest that it is effective at hitting its targets and has a statistically significant impact on patients’ progressions. Because of this, Genervon is seeking conditional approval from the FDA so they can make the treatment broadly available to patients while they continue to collect data to ensure that the drug is safe and effective (http://friends4eric.blogspot.com/2015/01/condition-green.html). Over 75,000 members of the ALS community have signed a petition encouraging the FDA to grant the approval (https://www.change.org/p/food-and-drug-administration-accelerated-fda-approval-of-genervon-s-gm6-for-use-in-als).

If I went into a complicated surgery, I could tell the doctors to go to extraordinary measures to save my legs or my voice. It would be a tragedy if they couldn’t accomplish that goal. If I woke up without the ability to stand or to walk or to speak, the doctors would feel that they had failed. Yet supporting the status quo and suggesting Genervon should do a Phase 2b trial, and then go from there, is essentially resigning me to the same fate, if not much worse. There is nothing in the immediate research pipeline with the capability of truly regenerating nerve cells. Even the stem cell trials work by creating a healthier environment for the remaining motor neurons. So slowing or stopping the disease is the immediate goal. And the immediacy and the urgency is real. Without an effective treatment I know where I, and many of my friends, are heading. Life expectancy is 2-5 years after diagnosis.

GM6 may not eventually prove to be effective for everyone.  However, two safety trials have found no significant safety risks. The pathways that it affects are not inherently dangerous. The company laid out pre-specified biomarkers and clinical primary and secondary outcome measures. This 12-week study actually had one of the most extensive biomarker plans of any trial I am aware of. So the researchers could not just monitor symptoms, but could measure what was occurring around the motor neurons.  In the trial GM6 had a statistically significant impact on a number of these outcomes. The results were consistent with what they said the treatment should do and were consistent with its performance in animal models.

I have a PhD in an empirical field. Over the past 10 years or so I have worked with data for a living. I understand the limitations of a 12 person trial, but also understand how unlikely it would be to get these results at random. Something positive is going on and we have lots of evidence that the treatment is safe.  This is a perfect candidate for accelerated conditional approval. This type of approval would require full data collection and surveillance going forward and final approval would be based on the data. If serious adverse events occurred or the data didn’t hold, approval could be quickly pulled. Inaction in all likelihood would be much more dangerous than action. “Do no harm” takes on a different meaning in this community. The pharmaceutical company wants to move forward and 75,000 members of the community are ready to act.

Suggesting we wait two years for the results of another trial is suggesting I tell the doctors I don’t want them to go to extraordinary measures. It is suggesting I should resign myself to what this disease will do to me over the near-term. And when you have ALS two years isn’t the near-term, it is a lifetime. How many of us won’t survive the next two years?

Yes, the standard protocol would be for another small-scale trial, but two years ago at a hearing with the ALS patients, the FDA said they recognized the urgent need for an effective treatment and would be flexible in working with the ALS community.  The FDA was flexible in addressing the Ebola outbreak last year, and because of the heroic actions of doctors and governments, less than 10,000 people died from the disease. Without any changes, roughly 75,000 to 100,000 people will die of ALS worldwide this year. Let’s let someone try to be a hero. Please urge the FDA to grant GM6 accelerated approval.

https://www.change.org/p/food-and-drug-administration-accelerated-fda-approval-of-genervon-s-gm6-for-use-in-als

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15 thoughts on “Genervon’s GM6

  1. Stephen,:,what you have articulated is difficult for the positioned decision makers to dispute ,. You have covered all bases even allowing for a compromise if there were negative side effects,. I kept up very closely with the “extraordinary” rapid,approvals and treatment of the fortunate responsive patients with Ebola ,, of course why not here and now,. Is it basically the same arm of the FDA which has to agree for this treatment, although experimental?? I will be contacting as close to the source about getting this approved ,. Appreciate your updates and your guidance understanding and navigating the systems that deal with ALS,. I may be in the Carolina’s ,. Hey to your gorgeous. Family,. Mitchell

    Sent from my iPhone

  2. As a friend and fellow empiricist I agree 100% with your outlined approach. I signed the change.org petition and will urge others to do so, too. Sending you my best, Lindsey Leininger

  3. SO incredibly well said Stephen! Much Love – Anthony

  4. Thanks to you, I don’t see how FDA can dispute your logic. I was diagnosed in august.very thankful for people like you who are making the effort.

  5. Joe. Sorry to hear about your diagnosis. Just know that you are not alone and there are a lot of incredible people involved in this fight.

  6. Thank you Lindsey! Hope y’all are doing well!

  7. Tricia Showerman

    Very well written! Thank you. My husband was diagnosed in 2012. My hope is that 2015 is the year for a cure. Tricia and David Showerman

  8. Thanks Stephen for your blogs and support for GM6. I found out about GM6 October 19, 2014 and I contacted them immediately. They told me to contact the FDA and the ALS Association which I did but didn’t hear back from either source. If I, as a lay person, knew about GM6 at that time, I find it inexcusable that the ALS Association was not aware of GM6 and did not contact them until recently. More than 3 months passed…3 months of further deterioration means untold hardships and pain for many ALS patients and their families, and possible death for many.

  9. Kirsten Ekdahl Hull

    Thank you for your incredible efforts to heighten awareness of ALS from not only a scientific and biological perspective but also from a deeply personal place. Sharing your story takes superhuman courage, diligence, and patience and I thank you for opening my eyes to the challenges but even more importantly to the possibilities to inspire hope. I was so moved by your award acceptance speech. You have found your calling, my friend: you are no longer just a father, husband, son, friend, and teacher, but you are now a true leader. Sending you and your family all of our best vibes from Chicago where you are happy not to be right now as I have ice forming on the inside of my windows…
    Keep smiling and keep giving us ways to make an impact. It felt great to sign that petition and fire off more letters and emails! – Kirsten

  10. Stephen, It was so nice to meet you today. You and your family have a positive energy and I hope our paths cross again. Who knows, maybe at the Italian Store? I know my dad needs more encouraging people in his life. I’m so proud of the work done through Change.org and hope that we can also get a response from the White House. Again, here is the link to the petition. We have 30 days to get 100,000 signatures and would love your support: http://wh.gov/iWMcT

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