All of us who have a child with mitochondrial disease, found out last week that the drug that is on trial in a study, announced there would be a change in the criteria to qualify for admission into the study.
This is great news...and a step in the right direction....but there is a lot of conflicting information as to what this means for us, and many other mito families.
I emailed locally a few times...
bless Pam's heart, she answered me with what she knows as of right now, in spite of the bazillion other families that are hounding her too. Seattle Children's is one of the hospitals that has this available in study, and she did say she will be sending out a letter or email to all of Saneto's families so everyone is clear.
So I thought I would share the info I know as of now....
Prior to the announcement last week, the 2 major qualifications were
(1) a child has to have a genetic dx for their mitochondrial disease
(2) a child has to be deemed 90 days from death
So...
they lifted the genetic component, which would qualify Hudson....
although we have no genetic link, we do know has the electron transport chain deficiency.
Thankfully Hudson is not near death, and so this will continue him to not qualify for the study today.
I was told that the 90 days had not changed.
This week there is a conference for the UMDF in Chicago, where Edison will present the latest information.
Because Hudson was in a study for a drug for I.S. way back when, I know the nurse who runs the trials at Children's, and so I put an email in to her today as well, asking if she knew anything different, and the specifics as to how it works.
I do know part of the study requires the child to get off the "mito cocktail"....I am curious to know how much in-patient is required, as well as how often they have to have clinic visits.
If I hear from the nurse who runs these clinical trials I will update with that information.
You can bet Hudson will be in this study if they lift the 90 days from death!
I think that is so ridiculous....you have to wait to the "very end" to be able to try something that could help your child???
Again....confirmation why we keep Hudson in Seattle...SO grateful for Seattle Children's Hospital, everything they have to offer and their awesome doctors!
I would love to see something help my Hudson, and all the other kids who suffer so at the hands of this disease...please keep this in your prayers, that it becomes available to many and helps our babies who live daily with a disease that has no treatments.
2 comments:
thats more info than i have recieved!
Abigail is in the second stage of the dna testing, which could lead to her diagnosis... but have no clue when we will hear any more about that.
as far as the trial in the study, we hadn't gotten much info, but saneto acted as though Abigail may qualify.. ?? but i don't think she is 90 days from death, so hmmm....?
honestly, i don't even know much at all about the trial study, what it requires, etc. i am interested in anything you find out! :)
Honestly, none of us know if our child with Mito is 90 days from death! Sadly Jack wont qualify as it stands now due to a lack of hospitalizations. He is stable despite some progression we're seeing.
Ive also heard additional info but its still stands as 90 days. I so wish I could be there in Chicago to hear Edison speak about it. Praying someone will let us know what was said exactly, thanks for sharing what you know.
The good news is its a step in the right direction. Praying all our littleones get accepted soon. HUGS to H. I would loved to of had your company today Debbie. We were there from 1:30 to 6:30pm, we both were beat!
Heidi & jack.
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