Sunday, November 26, 2017

Gene Therapy for Bubble Babies

David Vetter, the baby in the photograph above, has Severe Combine Immunodeficiency disease. This disease became well-known in the 70s and 80s because of this young boy. He lived in this germ-free bubble for 12 years thus giving the disease the name "bubble baby" syndrome. Recently, within the past week, The National Institute of Health and Care Excellence published an article stating that they can possibly cure this rare disease using gene therapy. However, the cost of the treatment would be 500,000 euros which is approximately 600,000 US dollars. This gene therapy is very specialized and is only offered in one hospital in Italy. This is an alternative for those who do not have stem cell donors.

I believe that due to the cost of this therapy, the public would not make much use of this innovative technology. Hopefully in the future the cost of this therapy will decrease so that more people can take advantage of this option for treatment of rare diseases.


1 comment:

  1. WOW! A real life Bubble Boy. I find it interesting that there's a possible cure but I feel that if it was at all effective, there would be more than one place in the world to provide this treatment. The cost is unbelievably mind blowing for a treatment that MIGHT work, but some people are crazy enough to pay that much money because they want their child to be healthy and disease-free. If I had a child with this rare disease, I would want to more research to be done before I try it on my child. I think there should be more studies on this treatment and more research on this rare disease to get a better understanding on what treatments would work, if there is any.