I'm not really sure what all of this means at this point, but one of my EB peeps (Thanks, Sara!) sent me this article this afternoon.
Stem Cell Therapy for Severe Forms of Epidermolysis Bullosa
This is a single institution trial to determine the effectiveness of stem cells from closely matched related and unrelated donors to replace missing or defective proteins responsible for severe forms of epidermolysis bullosa.
1. Patients aged < 25 years of age
2. Diagnosis of a severe form of EB characterized by collagen, laminin, integrin or plakin deficiency
3. Adequate organ function
4. HLA matched related or unrelated marrow donor or partially HLA matched unrelated umbilical cord blood donor
1. Active infection
2. History of HIV infection
3. Evidence of squamous cell carcinoma
If you or your child may meet the above criteria, you may be eligible to come to the University of Minnesota for the proposed treatment.
Prior to evaluation and screening to confirm eligibility, the following information is requested:
1. Clinical summary from a treating physician, including history of prior surgeries, transfusions, skin grafts, medications, allergies, infections
2. DNA mutation report if available
3. Skin biopsy report (including immunofluorescent staining)
4. HLA typing on child or adult with severe EB and unaffected siblings (instructions will be provided by our team)
The screening visit will involve skin biopsies, blood tests, photographs, assessment of skin fragility, and genetic testing. In addition, the visit will entail review of the clinical history and detailed to review of what is known and unknown regarding the proposed treatment procedure using stem cell populations from an allogeneic donor.
It sounds like, to me, they are opening up the transplants to Junctional EB patients as well. AND they are not requiring a sibling match for this trial. Now, I'm not saying this is something we would pursue for Jonah any time soon (considering how young he is), but this is HOPE. And maybe by the time he IS old enough that we felt it would be safe enough, they'll KNOW that it works with Junctional and without an exact sibling match.
We are so thankful, and consider this a direct answer to all the prayer that is going up for Jonah and others like him. With each passing day, I feel, more and more, that Jonah WILL be healed this side of Heaven... whether that be a "snap His fingers" sort of miracle, a gradual toughening of his skin, or a bone marrow transplant someday in the future.
Praise God for what He is doing through these amazing doctors and researchers!
Please be in prayer for us as we make contact with the University of Minnesota folks and find out more about our options... Pray that it will be clear what we should do and that we will be wise in the decisions we make for Jonah (now or in the future).
He's doing great, by the way! The nutritionist said he is in the 50th percentile for both height and weight, and has gone from the less than tenth percentile for head size to the 25th. This is no small miracle, to be sure.
No matter what we find out about this trial, we know that God will use Jonah's life to bring Him glory, and we feel honored to be his parents.
So many times, when people ask me how Jonah's doing, I say something along the lines of, "For now, it's just blisters, and that's all we can really ask." But it's not all I can ask. I ask for and fully expect a miracle... whatever form that takes.
And please keep all the other EB kids in your prayers too. There are so many out there struggling with even more than Jonah. I hope they are all going to bed with a little more hope tonight too.