Epidermolysis bullosa (EB) is a rare, and exceedingly painful, skin condition that I’ve written about here and here. Children with EB are sometimes referred to as “butterfly children” because their skin is said to be as fragile as a butterfly’s wings. The severity of the condition ranges from mild to fatal.
Jonathan Pitre is a Canadian teen with an aggressive form of the disease. He travelled to Minnesota in August 2016 to have a potentially life-saving stem cell transplant at the University of Minnesota Masonic Children’s Hospital with Jakub Tolar, Jonathan’s doctor and a pediatric transplant specialist who has pioneered the use of stem cells to treat severe cases of EB.
Unfortunately, test results from his first transplant showed that the white blood cells in his body and bone marrow were his rather than his mother’s (the donor). The proposed solution was another transplant, when Jonathan was strong enough to undergo the procedure.
In January 2017, the Pitre family received the welcome news that the Ontario government will cover the $1 million cost of a second transplant back in Minnesota, the only facility in the world where Jonathan can have this treatment.
There’s no question that $1 million sounds like a big price tag for one treatment for one person. I spoke to my colleague at CCRM, Pat Bedford, Manager of Clinical Translation and Regulatory Affairs, and he had this to say:
“This story highlights the hopes and dreams that stem cell therapies hold for many people. It also brings into focus an important dilemma. People with rare, life threatening/seriously debilitating diseases cannot afford to wait for sufficient evidence of clinical safety and effectiveness, but public and private payers cannot afford to provide for them without taking funds from elsewhere.
We can strive to reduce the costs of these procedures as much as possible, but the issues remain. Compassion will continue to war with reason and there are very important and nuanced arguments to be made for both sides. Jonathan’s story provides a face for this discussion, which must never be forgotten in boardrooms where logical decisions must be made,” argues Bedford.
Debra Canada, a charity supporting people who have EB, has been providing financial support to the Pitre family for their trips to Minnesota. Click here if you’d like to donate to the Canadian chapter.
Hear from Jonathan himself, in the video below. His second transplant is scheduled to take place in March 2017.
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