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.