Dramatic AdvancesIn Hemophilia Treatment (NAPSA)—For millions of people with an inherited medical condition, the promise of gene therapy has never shone morebrightly. For those with hemophilia—a genetic blood clotting disorder that’s been aroundsince Biblical times—it represents the greatest hope for a normallife, free from bleeding episodes and the need for infusions. The recent discovery of genes that control the production of blood-clotting proteins naturally found in blood, such as factors VIII and IX, has brought hemophilia to the forefront of gene therapy trials. People with hemophilia don’t have enoughof, or are missing, the blood-clotting proteins factor VIII or factor IX. The result: they bleed for a longer period of time than normal, and the factor needs to be replaced through injections, called infusions. “Research is well underway to develop an effective gene therapy that will stimulate production of clotting factor,” says Dr. Bruce Ewenstein, global medical director for hemophilia for Baxter BioScience, a leading producer of hemophilia therapies. “If we succeed, we'll reduce, and ultimately may eliminate, the need to fill in for nature with infusions of replacement factor. Instead, people with hemophilia will produce the clotting factor in their bodies themselves.” Although that hope may be a decade or two from reality, even now people with hemophilia lead dramatically different lives than just a generation ago, thanksto the availability of home treatmentfor their infusions, and genetically engineered factor VIII replacement therapy. Because it is not made from human plasma, recombinant factor VIII, so named because of the recombinant DNA technology used to create it, has brought an unequaled level of purity and viral safety to factor concentrates. Since its introduction a decade ago, a new generation of people with hemo- Thanks to dramatic advances in treatment, people with hemophilia today lead activelives. philia is able to manage the condition without fear of potentially receiving the HIV virus along with their factor infusion. Nearing completion is the development of next generation therapy prepared without any human or animalproteins. So look around...that happy toddler riding his tricycle around the block, middle-schooler shooting hoops in his driveway, or teenager competing in a swim meet might just have hemophilia. Unlike the old days, when people with hemophilia sat on the sidelines for fear of starting a bleeding episode, exercise and sports, along with homeinfusions, are all part of normal life for today’s generation. And, with ever more sophisticated research and development of new therapies, tomorrow’s children mayscale even higher mountains with nary a look back. Interested in learning more about hemophilia? In honor of National Hemophilia Month this March, visit Baxter’s Web site at www.hemophiliagalaxy.com/news, or the National Hemophilia Foundation at www.hemophilia.org.
