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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Logistics

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It is now almost 2 years since I was diagnosed. All in all, my progression continues to be relatively slow. I am almost embarrassed to talk about many of my symptoms given what some of my friends are going through. I can still drive, get dressed, and feed myself. I can still do most things I need to do for myself in my day to day life. However, each of these things is getting more and more difficult. They have moved from being harder than they should be, to hard. Showering and drying off and getting dressed is a tiring proposition, so I don’t do it that often. Just ask my wife. At this point we can see that I won’t be able to do some of these for too much longer. Driving the kids to school, taking them to the zoo on my own, this may be the year I have to give these things up. Not being able to drive is obviously scary because of what it means to my freedom and the time I get alone with the kids, but it’s more than that. It also takes away one of the ways I can still contribute to keeping the trains moving on time for our family.

For example, we have our house on the market right now. It’s not the easiest thing to keep the house in “showing” condition with crazies running around with toys galore. So while we typically keep the house in pretty good shape, it’s a scramble when someone calls and wants to see it. The most efficient way to clean up is for me to take the kids and get out of Dodge. Then Cara’s left to actually get things done. This has been our strategy for lots of things around the house. I disappear with the kids, and she works. Obviously, this isn’t an equitable division of labor, but it has become the unfair default. When I can no longer drive, we will have to change the dynamics once again.

So in many ways, this year is going to be about logistics. What do we have to do to keep our heads above water? Steve Gleason is famous for saying that given where medicine is right now for ALS, “Technology is the cure”. I can see that day coming, but for us right now, logistics is the cure. Figuring out the hired help we need, how to get it consistently, and how to make sure everything doesn’t just fall on Cara. How do we make our daily lives manageable enough so that we can focus on other things?

If the kids are fed, laundry is done, and everyone gets a bath at least once a week, then we are winning. Then days are again about doing puzzles and reading stories and playing in the yard and going to ballet and getting the kids down and having a moment together of peace and quiet on the couch. Then physical limitations are just annoyances. These aren’t insurmountable tasks, but ones that need to get done.

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Once we accomplish them, then I know we will spend another year making incredible memories.  Like the afternoon in the fall when we had our family pictures taken and the kids somehow acted perfectly. Afterwards we noticed a brewpub at the edge of a pond in the same complex. So we sat on the dock in the sun, the kids feeding the blue gills and turtles and Cara and I enjoying a beer, laughing, waiting for one of the kids to fall in, and absolutely amazed when neither did. Memories like these:

Once we figure out the logistics, my third year with this disease will be remembered as the one in which we find a cure!