New Therapies Advance for Rare Blood Cancer Polycythemia Vera

Dr. Aaron Gerds, assistant professor of medicine at Cleveland Clinic Taussig Cancer Institute, emphasized the growing momentum in treatment development for polycythemia vera, a rare blood cancer. While the disease may not receive widespread attention, he noted that researchers are actively pursuing new therapies, including rusfertide and other agents targeting the hepcidin pathway.

Rusfertide, in particular, may reduce or eliminate the need for phlebotomies, potentially improving patients’ quality of life. Gerds highlighted that multiple new therapies are being studied — such as givinostat in a phase 3 trial — and stressed that even in rare diseases, progress is being made.

Transcript:

What would you like patients to take away from today's conversation?

There are a couple of important things to note. One is that we are developing — and continue to develop — new therapies for polycythemia vera. Even though it’s a rare disease and not often highlighted in the broader pantheon of diseases presented at ASCO, it’s still a very important condition, especially for the people who have it.

There is a group of us who are very invested in this disease and are working hard to develop new therapies for it. Rusfertide isn’t the only one in development — there are also other drugs targeting the hepcidin pathway, as well as agents outside that pathway. For example, givinostat is currently in a randomized phase 3 trial as well as a registration-type study, also seeking approval.

Even though it’s rare, we are working hard to develop better treatment options. That’s the most important takeaway.

Specifically, with rusfertide, it’s exciting because it’s a new therapy that reduces or even eliminates the need for phlebotomies. That can really improve patients’ quality of life, which is important. Having another tool in our arsenal to care for these patients is exciting and can offer a lot to our patients.

Transcript has been edited for clarity and conciseness.

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