The first effective means of treating hemophilia involved infusing cryoprecipitate, a cold-insoluble solid that forms when frozen blood plasma is thawed. Cryoprecipitate was first used to treat hemophilia in 1964 when it was recognized that it could be separated from the plasma by centrifugation and stored frozen, then dissolved by warming and infused. A major advance in the treatment of hemophilia occurred in 1966 with the development of lyophilized (freeze-dried) concentrates of factor VIII from large pools of cryoprecipitate. Compared to cryoprecipitate, the concentrates were much easier to use and store and had a much higher concentration of active factor VIII. Concentrates also enabled people with hemophilia to infuse themselves at home, resulting in faster, more convenient treatment and significantly increased autonomy.

The purity of factor VIII concentrates improved dramatically in 1988 with the introduction of monoclonal antibody affinity purification. Monoclonal antibodies are substances capable of binding with the factor VIII molecule and removing it from plasma or cryoprecipitate, after which the factor VIII is separated from the monoclonal antibodies. This purification procedure results in ultra pure factor VIII with almost no extraneous proteins, which may cause infusion reactions and adversely affect platelet and immune function.

The latest milestone in factor replacement therapy occurred late in 1992 when the FDA approved synthetically produced factor VIII, known as recombinant factor VIII. Synthetic factor VIII uses recombinant DNA technology to alter a group of culture cells genetically to allow them to produce factor VIII. The factor VIII is then purified in a manner similar to monoclonal factor VIII. Both of these types of products have been shown to be very pure.

What does the future hold? Current research is aimed at finding a temporary or permanent cure for hemophilia by utilizing gene therapy. Gene therapy involves inserting the gene for the missing factor into the cells of a person with hemophilia. These altered cells may produce normally active factor for several months or possibly years. There are still many obstacles to be overcome before the procedure can become a reality, however most researchers are confident that solutions to these problems will be found within the near future.