Hematologists, sickle cell patients, and families with loved ones living with sickle cell disorders are hoping for a universal cure for sickle cell patients. In 1982, doctors at St Jude Children’s Research Hospital were the first to cure the genetic blood disorder using a bone marrow transplant. In fact, in 2014, I had the privilege of meeting Kimberlin, the first person cured of sickle cell. The experience made me hopeful that one day I’ll also see my daughter receive a cure as well.
Now years several years later, a cure for sickle cell patients via a bone marrow transplant hasn’t been a viable option. Mainly because for many sickle cell patients it is very hard to find an exact match among direct siblings to perform a bone marrow transplant. In addition, there are also a lot of problems associated with long-term care to make sure that the bone-marrow is not rejected by the new recipient. So, when I heard about the world-first procedure at Necker Children’s Hospital in Paris, which has shown promising results, I was ecstatic.
What’s amazing about the procedure performed on a teenager in Paris is it uses his own bone marrow. Thus, using the best match possible, the doctors changed the code of his bone marrow using a virus to correct the code that before made his red blood cells crescent-shaped. So far, for about 15 months, the correction has worked and he is living sickle cell pain free. This procedure is a part of a new trend seen in the medical world that uses gene therapy to help reverse genetic disorders.
Not Calling It a Complete Cure Yet
The hospital isn’t using the word “cure,” but is modestly keeping an eye on the patient in hopes that this procedure will be able to hope others, suffering from the debilitating effects of sickle cell, like the teen. While this gene therapy procedure is only a small step and many more patients will have to go through similar clinical trials, knowledge of the procedure provides hope for Hematologists, sickle cell patients, and families with loved ones living with sickle cell disorders.
Read the full story from BBC News here.
I was live on the BBC World Service Radio, Listen Below
Listen to Dr. Philippe Leboulch, the leading scientist who performed this gene therapy procedure in Paris. I had the honor of being able on the same radio show with him. I discussed parenting my daughter who is 8 years old and living with sickle cell anemia. My segment is towards the end (the last 25 minutes of the audio recording below).
- BBC News: Gallagher, James. (2017, March 2). Teenager’s sickle cell reversed with world-first therapy. Retrieved from http://www.bbc.com/news/health-39142971.
- My Friend Jen. YouTube. Online video. 2017, March 8. Retrieved from https://youtu.be/2BAZSdke7bs.
Are you hopeful for a medical cure, if so what disease are you hoping doctors will find a cure for and why?