Clinical Trial Update

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.

And unfair.


  1. thenelipotmommy on February 27, 2013 at 5:39 am

    Oh sweetie. *hugs* I'm so sorry… about all of it. The stinking trial, the obtuse FDA, the future of these precious children being ignored, and above all… the heartbreaking unfairness that makes your child suffer. Just so, so sorry. You are amazing and strong beyond words… Blessings to you Mama.

  2. babyfoodsteps on February 27, 2013 at 2:16 pm

    praying today that the grass root efforts move mountains…for Grayson and for so many other kiddos… hugs

  3. Erin on February 27, 2013 at 3:22 pm

    Oh my goodness, that sure seems backwards, especially if they need more participants, OPEN IT UP! Hoping hoping it changes and G can get in. Goodness. So hard because they see it as diagnosis and numbers and not as people. :o(

  4. Laurie on February 27, 2013 at 4:45 pm

    I'm praying they open up the trial!! It's so important for Grayson and all those with Mito. It's just crazy it's that rare – I mean only 12 kids in the US??? HUGS to you girl!!!

  5. J o s e y on February 27, 2013 at 4:53 pm

    This just makes my heart HURT for you. So not fair. 🙁

  6. Cristy on February 27, 2013 at 9:32 pm

    The FDA is doing their job and Edison Pharma is incredibly generous and supportive and really pioneering the way for mitochondrial therapeutics. But our job right now as "Mito Moms" is to help bring forward EVERY SINGLE CHILD with Leighs into our database which MitoAction is compiling right now, and also to work together to voice our concerns. We have an active voice as a community. Please ask parents of children with Leighs – regardless of whether or not you would qualify now – to contact me so that we know who is out there. Cristy Balcells ( (mom to Eva, age 8 with Leighs)

  7. Kyla on March 1, 2013 at 10:04 pm

    I hope they open up the trial! Clinical DX/Genetic DX…whatever! If this could help those kiddos now or in the future, I hope they all get that chance.

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