Treating Hemophilia with Biologics to Control Abnormal Blood Loss
Patients with hemophilia must contend with impaired clotting, the complications of which can lead to severe physical effects. In the most extreme instance, a situation that results in prolonged bleeding in the brain can be ultimately fatal. Or, as a more chronic example, sustained bleeding in joints can result in unwanted inflammatory responses and permanent damage. But despite these and other repercussions of abnormal blood loss, we have yet to develop a cure for hemophilia. Even worse, the effects of present methods of treating the disorder only last for limited amounts of time. Fortunately this gap leaves the door open for other, more potent therapeutics that can bring relief to patients—the most exciting of these therapeutics might just be biologics.
Rethinking Current Options of Treating Hemophilia
Hemophilia occurs approximately every 1 in 5,000 births. In the United States, Canada, and Europe alone, more than 30,000 people suffer from a moderate to severe form of the disorder. Even without a cure, treating hemophilia can cost a patient anywhere from $60,000 – $260,000 annually, leaving them with a significant economic burden as they manage the condition over the span of their lifetime.1
Currently, treating hemophilia involves infusions of the missing clotting factors. While administration on a regular basis has shown to be superior to on-demand treatment,2 blood concentrations of the clotting factors maintain normal levels for only a few hours before dropping off again. For this reason, it still leaves much to be desired in terms of improving the quality of life for afflicted patients, in spite of helping to control the abnormal and spontaneous bleeding that characterizes hemophilia.
Turning to Biologics as an Option for Treating Hemophilia
Last year, the FDA approved a biologic to control and prevent bleeding in patients with hemophilia B. In clinical studies, the drug demonstrated the ability to resolve over 70% of bleeding episodes after a single infusion. It also eliminated the need for blood transfusions in hemophilia patients requiring major surgery when administered as a continuous infusion.3
The drug’s success implies that biologics are a viable means of treating hemophilia. Research is currently underway to develop a therapeutic for the most common form of the disorder, hemophilia A. The method currently being studied involves taking the patient’s own cells, correcting the gene for the missing clotting factor, and then implanting a pouch containing those modified patient cells. The elegant administration method results in constant delivery of the necessary clotting factors into the bloodstream, allowing the protein levels to remain at normalized concentrations without the dips and spikes that characterize current infusion methods.
If this latest potential hemophilia therapy proves successful, it paves the way for the treatment of other disorders. Since the delivery vehicle relies on providing a constant supply of necessary proteins into the bloodstream, there is a high probability that it can be used in the management of other blood disorders or even metabolic conditions like diabetes. What makes the method particularly attractive is that it uses the patient’s own cells to administer the missing factors, thus lessening the chances of stimulating an immune response and increasing the therapy’s probability of success.
Thanks to the advent of biologics, we now have the opportunity to develop and provide better treatment options for many genetic conditions. Hemophilia, in particular, is one such disorder that presents a ripe opportunity for life sciences organizations because not only is it inherited, it can also manifest in patients as the result of a genetic mutation. While current methods of treating hemophilia can minimize the more adverse repercussions of abnormal bleeding, they also come with their own set of challenges in terms of costs and administration schedule for the patient. The unique innovation provided by biologics may be the key to treating hemophilia in better and more effective ways.
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- “New Funding Allows for the Clinical Development of Novel Cell Based Hemophilia Therapy,” December 25, 2015, http://www.newswise.com/articles/new-funding-allows-for-the-clinical-development-of-new-cell-based-hemophilia-therapy ↩
- “Prophylaxis vs. on-demand treatment with BAY 81-8973, a full-length plasma protein-free recombinant factor VIII product: results from a randomized trial (LEOPOLD II),” January 30, 2015, http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4671268/ ↩
- “New biologic to control bleeding for hemophilia B patients,” May 5, 2015, http://drugtopics.modernmedicine.com/drug-topics/news/new-biologic-control-bleeding-hemophilia-b-patients ↩