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New Treatments Provide Hope for Patients with MPNs

New Treatments Provide Hope for Patients with MPNs
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Published on July 16, 2020

Key Takeaways

Recent advances in treatment for patients with myeloproliferative neoplasms (MPNs) have made it possible to treat the disease, as well as ease the symptoms, providing hope for patients diagnosed with one of the blood cancers.

New Treatments Provide Hope for Patients with MPNs

Treatment for patients with myeloproliferative neoplasms (MPNs) has generally focused on easing the symptoms. But recent advances have made it possible to also treat the disease, providing hope for patients diagnosed with one of the blood cancers.

“It's never good to have an MPN, but if you're going to have one, you're in a good time to have it,” said Dr. Jeanne Palmer, a hematologist-oncologist at the Mayo Clinic in Scottsdale, Arizona. Dr. Palmer participated in a webinar with Patient Power co-founder Andrew Schorr last month.

“I think right now we're not only coming up with ways to alleviate symptoms, which is what we see with the JAK inhibitors, but there's also a lot of studies looking at ways to try to modify the disease, to try to perhaps even benefit the biology of the disease, rather than just making people feel better.”

MPNs are blood cancers that occur when the body makes too many white or red blood cells, or platelets. They include myelofibrosis (MF), essential thrombocythemia (ET) and polycythemia vera (PV).

Clinical Trials for Myeloproliferative Neoplasms

Dr. Palmer said despite the COVID-19 pandemic, clinical trials are continuing for MPNs, including Sierra Oncology’s phase III MOMENTUM trial for patients with MF. The trial is designed to confirm the benefits of momelotinib — a JAK1, JAK2 and ACVR1 inhibitor — on the three hallmarks of myelofibrosis: symptoms, anemia and enlarged spleen. The enrollment goal is 180 patients who are symptomatic, anemic and have been treated previously with a JAK inhibitor.

Similarly, CTI BioPharma’s PACIFICA phase III trial is evaluating the safety and efficacy of 200 mg of pacritinib, a JAK2 inhibitor, administered twice daily in 180 patients with MF with severe thrombocytopenia (platelet counts of less than 50,000 per microliter). (Pacritinib is also being explored as a treatment for cytokine storm in hospitalized patients with severe COVID-19 in addition to JAK inhibitors.)

“Both of these drugs which provide an enormous benefit for patients will be able to continue to move forward, and hopefully be able to get approved,” Dr. Palmer said. “Because I think they both provide these wonderful opportunities for people to get relief from their symptoms without being tough on their counts.”

Dr. Naveen Pemmaraju, an associate professor in the Department of Leukemia at MD Anderson Cancer Center in Houston, Texas, who was also part of the panel, said ropeginterferon alfa-2b is also showing promise in patients with PV. The interferon was recently approved in Europe; the FDA is expected to approve the drug for use in the U.S. next year.

He said ropeginterferon alfa-2b is an improvement over peginterferon alfa-2a (Pegasys) because it is an injection once every two weeks, rather than once a week, and it has fewer side effects.

“…the results (from the trials) really look very encouraging now at four years plus follow up that people can stay on this drug for a long period of time,” he said. “Less side effects than we expected. And possibly a signal for less MPN events such as thrombosis, like blood clots. Of course, longer follow up is needed.”

For essential thrombocythemia, developing new treatments is more challenging, Dr. Pemmaraju said. He noted two studies on the role of JAK inhibitors in treating ETs produced mixed results.

“So, I think, by in large, the cornerstone for ET is still cardiovascular prevention, watching out for bleeds and clots, yes, (and) monitoring for development of pre-fibrotic MF or overt myelofibrosis,” he said.

Dr. Palmer said one of the positives from the outbreak of COVID-19 is the surge in telemedicine, which is allowing patients to continue participation in clinical trials.

“I even had a study that's basically said to me, ‘We'll do labs elsewhere. We'll take video visits,’” she said. “And they're really trying to be engaged to allow for more people to go on. And I'm hoping that since COVID is not going away anytime soon, I'm hoping to see more of this, which will enable more people to be on clinical studies.”

~Megan Trusdell


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