Treating a Group of Rare Blood Cancers, Myeloproliferative Neoplasms, with N-Acetylcysteine

Principal Investigator: Dr. Angela Fleischman

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Disease: Myeloproliferative neoplasms: Polycythemia vera, essential thrombocythemia and myelofibrosis

Research Description: Myeloproliferative neoplasms (MPNs) are a group of chronic blood cancers that include polycythemia vera (PV), essential thrombocythemia (ET) and myelofibrosis (MF). MPN patients suffer from blood clots and chronic inflammation, and they are at risk of developing acute myeloid leukemia (AML), which has a dismal prognosis. There is a need for low-cost, low-risk treatments for early stage MPNs that address the major clinical issues and potentially prevent progression to AML. The nutraceutical N-Acetylcysteine (N-AC) is FDA approved to treat acetaminophen overdoses and has been shown to have anti-inflammatory and anti-thrombotic effects. This clinical trial will assess the ability of N-AC to reduce symptom burden and blood clotting in 50 MPN patients. N-AC is readily available over the counter in tablet and capsule formulations, which could help speed patient impact if positive results are seen.

Funding Partners: Goldman Philanthropic Partnerships

CWR funding role: Primary funder