Questions Answered!

Here are the questions you asked.  If you have a question, please email me at or post it in the comment section of the blog!

*is there as minimum or maximum patient age for the treatment?

The age range for the study is I believe under 18.  They still only have had 15 patients in the study, which by research numbers is nothing.  It is too soon to generalize any specifics about what the sweet spot might be for best age.  I would appear anecdotally that 4-6 would be the optimal age range for this population, but again, that is based on far too few to hold any real weight.

*since it's a study right now, are families responsible for cost? does insurance help?

Some insurance companies (like ours - Anthem BC/BS) will pay for the clinical trial, some won't.  It's up to the individual insurance provider.  The U has someone dedicated to working with insurance companies to help and assist with coverage.  
*what are some of the diseases other than EB that this treatment can help?

Auto-immune diseases, burns, and aging top the list.

*What is the success rate of BMT in children with EB?

So far, the results - meaning that stem cells are leaving the blood supply and turning themselves into skin cells for the first time in medical history - are worthy enough for publication in the New England Journal.  The spectrum of success is as broad as it could possibly be... from amazing success with several patients now, to tragic failure with several patients, to side effects as bad as EB, to graft failure.  This treatment in its current form is a part of a process. 

*If treatment is successful are the children cured or is it a means of improving quality of life?
It was never meant to be a cure or the end.  It has always been merely a stepping stone to better treatments and a better quality of life.

*Is the funding for MN used to research a cure for EB and /or to improve BMT procedures/outcomes?

 The money raised by PUCK goes directly to treating EB.
*What does the future look like for the children that have gone through the bmt?

Well, BMT unfortunately never really ends.  LOL.  What I mean by that is that there is follow up care that may be as infrequent as annually if all goes well and no side effects are present.  Really, in this case, there is a study to monitor the changes in skin 100 days after transplant, so that is why there is a lid on this study.  The patients still need to come back for biopsies every year.  The whole initial concept is that the Collagen VII will continue to grow into wounded areas over time, so the more wounds that continue, the more healing with working collagen. 

*I don't know much about BMT - does it have to be a sibling (or other relative) match? Or can anyone be a donor?

Transplant doesn't require a sibling donor.  An unrelated donor's cod blood can work as well.
*Are Drs. Wagner and Tolar focusing their research solely on BMT, or are they looking at other treatments?

*Is there a "point of no return" with EB patients? Meaning that even if BMT is successful, they might not recover from earlier EB damage?

Yes, unfortunately, there is a point of no return for EB kids.  Transplant can't undo what's already been done in regards to webbing of hands.