Patient Worthy attended the National Hemophilia Foundation’s 69th Annual meeting in Chicago and we are thrilled to report on some of the exciting new developments for patients with bleeding disorders.
Some of the highlights include:
- My Life, Our Future (referred to hence forth as MLOF)– A Genotyping and collaborative research initiative between the National Hemophilia Foundation, the American Thrombosis and Hemostasis Network, Bloodworks Northwest and Bioverativ. Over 7.500 people have already submitted data and samples, and this includes over 6,000 males with hemophilia A or B, as well as women who have hemophilia or who are confirmed carriers.
MLOF represents the largest hemophilia research depository resource. Patients who either have a bleeding disorder or who are first, second or third degree relatives may also participate. Understanding the relationship between the genotype and clinical expression of bleeding disorders can accelerate research for better treatments and potentially, a cure. Patients who want to learn more can watch this video or check out the MLOF website.
- Exciting developments toward a cure: Gene therapy may truly be available in our lifetime! Dr. Steven Pipe from the University of Michigan used the Amazon package delivery system as an analogy for explaining how gene therapy works. The audience, packed to a standing room only, could relate to this! The excitement in the room was palatable.
- Glen Pierce, MD, PhD also gave great encouragement in his presentation on treatment advances. He stated that the work by Spark and BioMarin is very promising. He believes capitalism has fueled the intense competition in treatment advances and the winners are the patients. He talked not only about gene therapy and gene editing, but also about non-factor replacement therapies, the inhibition of ant thrombin and small molecule approaches. He also cautioned pharma to avoid “unhelpful” marketing statements which are irrelevant to the clinical picture, confusing to patients, and unnecessary given the efficacy of what they have developed.
How would gene therapy be an advantage? It would be one and done; no need for on-going prophylactic treatment, literally freeing individuals and their families from time-consuming treatment regimes, and simply the stress of worrying about it. Many young men in the transition to adulthood have gaps in their compliance with treatment, which can lead to lifelong joint or head injuries. Additionally, gene therapy creates the potential to cure infants in developing countries who currently live short lives of pain and disability, as they are without a sophisticated system of hemophilia treatment centers (HTCs). This would obviously be another enormous humanitarian benefit of successful gene therapy. Amazon has it together… hopefully gene therapy will too!
Increased awareness, information, and support for women with bleeding disorders.
Women can have hemophilia, von Willebrand Disease (VWD) and a host of other rare and ultra-rare bleeding disorders, all of which can involve uncontrolled bleeding. Bleeding can be a special concern for females due to menstruation, pregnancy, childbirth and the interplay of female hormones creating variability over time. Women had many sessions to choose from, and many opportunities to ask questions and get feedback at this year’s meeting. If you are a woman with bleeding concerns do not hesitate to get in touch with your local HTC.