The future of Hemophilia

The future of Hemophilia is Gene Therapy. Currently we treat those with Hemophilia by supplying them with the missing protein coagulation factor they lack, but this treatment does not actually cure the patient and has a very high treatment burden. The current goal of the future is to find a way to correct the harmful mutation that leads to the Hemophilia disorders manifestation. Positive results from gene therapy have been achieved in animals such as dogs and mice, but we have yet to break through with a humans.  

"Before the gene treatment, the animals experienced about five serious bleeding events a year. After receiving the novel gene therapy, though, they experienced substantially fewer bleeding events over three years, as reported in the journal Nature Communications."

"“We’ve cured mice 10 different ways,” says Trent Spencer, Ph.D., director of the Gene Therapy Laboratory at the Aflac Cancer Center and Assistant Professor of Pediatrics with Emory University School of Medicine.

“We can cure a mouse of hemophilia with our eyes closed. Getting from a mouse to a human, however, is a massive step and that is what we’re trying to do now.”

  

  

Currently the biggest block in the progression of gene therapy is the potential commercialism of a new treatment. Companies are less willing to invest in creating a product that would be very expensive initially to offset the development cost, as well as possibly unpopular due to moral concerns. Why risk bringing gene therapy treatments to the market when you already have a working, profitable traditional hemophilia treatments in place? Those with the most to gain from a gene therapy treatment for hemophilia would be individuals living in developmental countries. One hight-tech intervention is much more feasible than creating an infrastructure that would allow for an individual to receive daily treatments.   It will be interesting to see where Hemophilia is ten years from now, when the cost of gene therapy may be cheaper and the general public more accepting of gene manipulation. 

References:

Du, Lily M., et al. "Platelet-targeted gene therapy with human factor VIII establishes haemostasis in dogs with haemophilia A." Nature communications4 (2013).

"Gene Therapy and Hemophilia A - Children's Healthcare of Atlanta."Children's Healthcare of Atlanta. N.p., n.d. Web. 02 Aug. 2014.