By Sandy Smith
Drugs and therapies at various stages of development offer hope for those with bleeding disorders.
Robert Sidonio Jr., MD, MSc, medical director of hemophilia at the Aflac Cancer and Blood Disorders Center, took attendees disorder by disorder, identifying drugs in the pipeline that may have an impact as part of the Friday session, Advancements in Treatment for Bleeding Disorders.
Sidonio began by focusing on treatments for rare disorders like Glanzmann thrombasthenia, “a space where just about everything is off-label.” He made his way through more common disorders like von Willebrand disease and hemophilia. While promising drugs are in development, gene therapy also is on the horizon.
In some ways, drug development remains hampered by funding—in which bleeding disorders compete for federal dollars with more prevalent diseases like cardiovascular—and a lack of participation in clinical trials by those with bleeding disorders.
A 45-minute panel discussion followed Sidonio’s presentation, with those topics recurring. The panel included Amy Dunn, MD, professor of pediatrics, director of hematology and director of the Hemophilia Treatment Center at Nationwide Children’s Hospital, Ohio State University; Kevin Mills, PhD, NHF chief scientific officer; Leonard Valentino, MD, NHF CEO and president; and Michael Recht, MD, PhD, chief science officer, American Thrombosis and Hemostasis Network.
The panel dove into the challenges of efficacy and safety for many of the drugs that Sidonio mentioned.
“It shows the importance of working with a treatment center that knows how to run clinical trials, so that patients can be watched very closely,” Dunn said.
Recht reiterated the need to follow patients over time, including those who participate in gene therapy. He would advocate, he said, that it would be considered a standard of care to enroll anyone receiving these medications in registries.
Valentino called for more diversity in clinical trials, well beyond the most typical white males. “As a community, we need to work harder. The physicians up here, the people who run hemophilia treatment centers, their hands are tied. You need to participate. This is your call to action. You want these drugs? You need to participate.”
Dunn followed up by pointing out the unique nature of bleeding disorders. “Your disease is different than someone else’s. You might have a different mutation. Your disease might respond differently. Unless you participate, we won’t know.”
Mills agreed. “We think about hemophilia and VWD and bleeding disorders as single-gene disorders. No two people bleed alike. There are 25,000 other genes that influence how you bleed, why you bleed. We’re in a really cool in time of trying to understand that biology. It really matters, that biology. The lab work taking place today is what is influencing the drugs that will be developed tomorrow.”
With gene therapy on the horizon, what is the long-term impact for drug development and HTCs? The panelists were asked if they’d be put out of business.
“Gene therapies are exciting,” Dunn said. “But these therapies are not for everyone now. There are going to be good patients for gene therapy. You need to have conversations with your treatment center staff who know you, who know about these drugs. They are not a ‘one-and-done.’ They may not work for you. You need to have all the information. It’s not a one-time conversation. It’s a series of conversations over time, where you have time to synthesize the information.” ■
It’s often said that if it’s not one thing, it’s another. But what if it’s actually a combination?
Symptoms of bleeding disorders can be impacted by a variety of other conditions that often affect women, such as endometriosis and polycystic ovary syndrome (PCOS). Learn more about what’s happening—and how to manage it—with the Saturday morning session, Coexisting Women’s Health Conditions. The consumer talk will feature Kalinda Woods, MD, FACOG, a practicing physician and assistant professor of gynecology and obstetrics at Emory University.
Saturday, 10:15 AM - 11:15 AM
Consumer – Women With Bleeding Disorders
GRBCC: 360 ABDE/BDC Virtual Platform (Hybrid)