Here we go again! Lots of conversation, lots of activism and unfortunately lots of disagreement about whether GM 604 should be made available to those living with ALS today. Keep in mind I don’t specialize in research, I am not a scientist nor a doctor. My knowledge and expertise is on tending to those living the ALS experience from diagnosis to end of life and all that happens in between.
I have profound thoughts and opinions on the institutionalization of our healthcare system as I write in my book, Sacred Bullet: Transforming Trauma to Grace While Tending the Terminally Ill.
In 1997-98, Myotrophin was a drug in the pipeline that showed great promise. Gordon Heald, the gentleman I cared for, wanted very much to be part of this trial. The trial was closed, yet Gordon was adamant about obtaining the drug at whatever the cost. For him it was about hope, a last chance at life. The drug was being tested in Japan at the time, very much out of his reach even though he would have written a check for any amount. Unfortunately, the check did not matter as he was unable to procure the drug. The fact it was found to be ineffective mattered not. It was his choice, his decision, his desire. He had absolutely nothing to lose, except hope.
GM 604 has been shown to have some positive results. Eric Valor, a man living with ALS, picked up the mantle in leading the charge to make it available to those living with ALS now without waiting for the three plus year process involved in clinical trials. A handful of individuals including my friend Matt Bellina have joined the cause. I have read that Genervon, the biotech company, has been working on this for 20 years. Accelerated Approval, in the world of big pharma, is a big ask, yet until we are willing to think outside the box, those living with ALS and their families continue to live in extraordinarily difficult circumstances beyond imagination, often suffering in ways most people have no reference for. To diminish hope can be a cruel punishment.
As the situation now stands, the eventuality of death is inevitable. So I ask the FDA and I ask those who oppose to making GM 604 available, what is the harm in researching as you normally would and approving accelerated approval?
Outside the box? …Yes!
Make it happen, Now!