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Taking part in a clinical trial may be the best treatment choice for some polycythemia vera (PV) patients. Today's standard treatments for cancer are based on earlier clinical trials. LLS continues to invest funds in MPN research.

Clinical trials can involve new drugs, new combinations of drugs or approved drugs being studied to treat patients in new ways such as new drug doses or new schedules to administer the drugs. Clinical trials are conducted worldwide under rigorous guidelines to help doctors find out whether new cancer treatments are safe and effective or better than the standard treatment.

Current PV Research and Clinical Trials

Some examples of treatments being studied include:

  • Ruxolitinib (Jakafi®). This drug is a JAK2 inhibitor that is taken by mouth twice a day. Ruxolitinib is approved by the FDA to treat symptoms and signs of myelofibrosis, including an enlarged spleen, night sweats, itching and bone or muscle pain. It is indicated for treatment of patients with intermediate- or high-risk myelofibrosis, including primary myelofibrosis, post polycythemia vera myelofibrosis and post essential thrombocythemia myelofibrosis. It is being tested in a study in which patients are randomized between ruxolitinib and best available therapy (the traditional medication that a doctor determines is the best treatment choice for a given patient). Ruxolitinib is being studied for possible approval as new therapy for PV patients who have failed or are unable to tolerate treatment with hydroxyurea.
  • Possible genetic origin of MPNs. There is a theory that MPNs may occur in families; if so, they are a group of genetic diseases passed on from one generation to another. This idea is being studied to discover if abnormal genes cause MPNs.
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last updated on Thursday, June 28, 2012

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