Bright Promise for Advances in Hemophilia Therapy

New treatments promise better options for patients.

CSL Behring's Dr. Jerry Powell at the ASH Conference

Dr. Jerry Powell, left, discusses advances in hematology treatment at the American Society of Hematology Annual Meeting and Exposition in Atlanta. The meeting was attended by thousands of healthcare and industry professionals from around the world.

 

The future of hemophilia treatment is upon us.                                                                              

That was the consensus view of experts who gathered in Atlanta in December for the annual American Society of Hematology (ASH) meeting. We spoke with Jerry Powell, Medical Director of Coagulation, U.S. Commercial Operations at CSL Behring, about advances in hemophilia treatment and what the next several years might bring.

Q. During your time in the field, what have been some of the most important advances in treatment?

A. In recent years, innovation has fueled a number of welcome advances in hemophilia therapy. Thanks to newer treatments that provide greater molecular stability and consistently high and sustained factor activity levels, we are able to offer people with hemophilia long-lasting protection against bleeding, with fewer infusions and lower product consumption.

Q. How is a young man living with hemophilia living differently and better than he was, say, 25 years ago?

A. That young man is benefitting from the newer therapies' longer duration of action, long-lasting efficacy, lower bleeding rates and more convenient dosing. For patients, those features translate into improved compliance, treatment satisfaction and health-related quality of life, ultimately improving patient outcome allowing the patient to live an active life.

Q. Given the advances in therapy to date, what pressing medical needs remain for the bleeding disorders patient community, and how is the industry addressing these?

A. A committed hemophilia researcher's work is never done. Hemophilia is a complex and challenging disorder. We in the industry must continually innovate to develop new treatments and disease management tools to help patients lead full lives.

Q. Where do you see treatment headed 10 years from now?

A. One area of bright promise is gene therapy for hemophilia treatment. Researchers are looking at ways to insert better functioning factor VIII or factor IX genes into the cells of people living with hemophilia so their blood will clot more effectively. Trials of this approach have had mixed results to date, but studies are continuing. This possible breakthrough could yield several significant benefits to the hemophilia community:

• Fewer bleeding episodes

• Helping people with hemophilia to eventually begin producing their own clotting factor

• Reducing the number of or even eliminating the need for weekly infusions

• The potential for a person born with severe hemophilia to eventually experience much milder symptoms.

Q. Do you expect a cure for conditions like hemophilia and VWD in your lifetime?

A. Given the remarkable state of medical science and technology today, never say never. But at CSL Behring, our goal is making hemophilia and VWD as manageable as possible, so the people living with these bleeding disorders can enjoy rich, full and satisfying lives.