Hope for hemophiliacs on the horizon

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Hemophilia is a genetic bleeding disorder. It s X-linked, and therefore it affects males overwhelmingly. There are several different types depending on the factor that s lacking. They have one thing in common: the lack of one of the many proteins clotting factors that are involved with blood clotting makes the sufferer of hemophilia prone to bleeding sometimes fatal bleeding from minor injuries or even no injury at all. In addition, bleeding into the joints is not unusual, and can be excruciatingly painful. Approximately one in 5,000 to 10,000 male babies are born with hemophilia A (lack of clotting factor VIII), and one in 20,000 to 34,000 are born with hemophilia B (lack of clotting factor IX).

In the not-so-distant past, hemophilia patients could do nothing but be careful in order to cope with their condition. More recently, however, it has been possible to derive the appropriate clotting factors from human plasma, and since 2008, via genetic engineering, from hamster cells. These treatments work, but there are drawbacks. For example, they are not long-lasting and must be repeated regularly, perhaps every two weeks to prevent bleeding episodes and joint damage. Further, treatment with either type can be very expensive. Such a regimen can cost as much as $300,000 per year, and must be followed for the patient s lifetime.

Recently, as reported by Reuters, companies have been working on new types of longer-lasting treatments that could halve the number of treatments a patient would need, greatly reducing the price and hassle of treatment. Two companies are expected to file applications with the FDA next year for approval of such longer-acting treatments.

If these new treatments are approved by the FDA, commented ACSH s Dr. Gilbert Ross, they could be a substantial boon to hemophilia sufferers both in terms of frequency of required treatments, and perhaps from a financial viewpoint.