Rare Disease Report

Guy Young, MD, on the Recent Revolutions in Hemophilia Treatment

DECEMBER 12, 2017
Guy Young, M.D.
Guy Young, M.D., Director at the Hemotosis and Thrombosis Center at the Children's Hospital of Los Angeles thinks that we're in the middle of a revolution.

Rare Disease Report caught up with the hematology and oncology expert at the 59th American Society of Hematology (ASH) Annual Meeting and Exposition in Atlanta, where he discussed the recent boom in product development for hemophilia. While the current therapies being evaluated are worth the excitement, the next revolution isn't far behind, he says, as gene therapy will be here sooner than later.


Rare Disease Report (RDR): Can you speak to the recent boom in regulatory action as it pertains to potential therapies for hemophilia?

Young: It’s interesting, because between 1999 and 2013, there really weren’t any new drugs other than “me too” drugs in hemophilia. Now, there’s been this huge boom in development and innovation. It’s hard to pinpoint exactly why that is, but I think there were unmet needs, and maybe it’s just that the technology wasn’t quite there to fulfill them. Now, one company, Genentech, has this product [Hemlibra (emicizumab-kxwh)], and then other companies have other products that look promising for managing hemophilia in patients with and without inhibitors. I think sometimes it just takes 1 domino to fall when it comes to innovation, and then, all of a sudden, the competitors become involved.

RDR: Does anything concern you about the potential of moving away from factor therapy?

Young: Of course, the companies that stand to lose a lot from this are those that make factor and bypassing agents. They’re going back to their scientists and back to their drawing boards, and they’re saying, “We need to come up with something; we need to improve.” I think that I’m going to call this the second revolution of hemophilia treatment. The first was when factor therapy came out, which was really before my time—in the ’70s. That dramatically changed treatment, because before factor therapy, hemophilia patients were basically spending much of their time in the hospital getting plasma or cryoprecipitate, managing their bleeds, and dealing with pain. Factor came and allowed not only for better treatment but also for home treatment. All of a sudden, people could be treated at home and not have to miss school or other activities. That was a revolution. It’s been “factor, factor, factor, let’s make the factor better” since the ’70s until now. That’s 40 years.

RDR: What can people expect from the world of hemophilia treatment in the near future?

Young: Now, we have another revolution: New products that are not factor are aiming to reduce the treatment burden and treat bleeding more effectively. This third revolution is coming, and it’s not too far away—it’s gene therapy. I think gene therapy offers the prospect of actually curing hemophilia. You get, in theory, 1 infusion and you don’t have hemophilia anymore. Ultimately, the goal of treating any disease is curing the disease. That’s the physician’s goal. With a lot of drugs, you make more money if it’s for a chronic treatment, but be that as it may, the ultimate goal of treatment is cure. This offers the prospect for cure, and emicizumab is not a cure. Fitusiran, another drug that’s coming along, is not a cure—you have to continue to take it. Granted, they’re working well and they’re fairly easy to take, but neither is a cure, and for patients with hemophilia, there is an emotional connection to being cured—to saying, ‘Hey, I don’t have hemophilia anymore; I can go live my life and not worry about it anymore.” I think it’s a huge prospect. There’s still a lot of work to be done, a lot of kinks to be worked out, and a lot of safety issues and concerns, but things definitely are moving in the right direction.


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