As you all know, we had been hoping to get Grayson enrolled in a clinical trial for a drug that would hopefully slow or stop the progression of his disease. But unfortunately, at this time, they are only accepting kids with a confirmed genetic diagnosis of Leighs, which we don’t have.
However, the pharmaceutical company making the drug and doing the trial has run into a problem. They need 30 kids with confirmed genetic diagnoses for the next phase, and right now they have 12. Twelve- in the entire United States. Because they can’t find enough kids, the future of the trial (and the lives all the Leighs kids who are/could be benefiting from this drug) are in jeopardy.
My friend Lori wrote a great post about this on her blog. Read her post here.
I talked to the director of MitoAction today and she got Grayson’s information. She told me that 9 out of 10 families she knows of do NOT have the genetic diagnosis and this is why we need to get the FDA to allow the criteria for who can participate in the trial to be expanded.
What I am trying to wrap my brain around tonight is how rare this disease is and the fact that MY child has it.
It’s just crazy.