Clinical Research Center for MPNs and Faculty

The Clinical Research Center for Myeloproliferative Neoplasms (MPNs) in the Leukemia department was founded in 2011 and is the largest single-institution research center for the care of MPNs worldwide. It is one of the world's leading MPN Clinical Research Centers that is at the forefront of developing novel treatments for MPNs. The Clinical Research Center for MPNs was one of the primary institutions that conducted pivotal clinical studies leading to regulatory approval of landmark therapeutics for MPNs, such as ruxolitinib, the first JAK1/2 inhibitor that was approved as a treatment for  intermediate or high-risk myelofibrosis, on November 16, 2011 (Verstovsek S., et al., New England Journal of Medicine 2012) and as a treatment for polycythemia vera, on December 4, 2014 (Vannucchi AM., et al., Verstovsek S. New England Journal of Medicine 2015).

Collectively, more than 200 early and advanced phase clinical trials on novel MPN medications have been conducted at the MPN Clinical Research Center. Notably, the MPN Team and the clinical research program in MPNs have grown exponentially since 2000. The MPN Team included one physician and one research nurse, and only one active MPN-specific clinical protocol was enrolling patients at that time. Since then, many important medications have been studied and several treatments received regulatory approval based on pivotal clinical studies that were conducted at the Clinical Research Center for MPNs, such as ruxolitinib for myelofibrosis and polycythemia vera, pacritinib for myelofibrosis and very low platelet counts (severe thrombocytopenia), momelotinib for myelofibrosis and anemia, and pemigatinib for myeloid/lymphoid neoplasms with FGFR1 rearrangement.

Please visit About MPN and MPN Awareness and Education and 7 Answers about Myelofibrosis to find educational materials about MPNs.

The results of the MPN clinical research studies conducted at the Clinical Research Center for MPNs have been published in prestigious medical journals and are frequently presented at the most important national and international conferences, such as those of the American Society of Hematology (ASH), the American Society of Clinical Oncology (ASCO), the Society of Hematologic Oncology (SOHO), and the European Hematology Association (EHA).

On February 28, 2022, pacritinib (JAK2/ACVR1/IRAK/FLT3 inhibitor) received accelerated regulatory approval as a medication to treat patients with intermediate or high-risk myelofibrosis and very low platelet counts (below 50x10⁹/L); the accelerated approval of pacritinib was based on the efficacy that pacritinib demonstrated as a treatment for patients with myelofibrosis and severe thrombocytopenia (platelet counts below 50x10⁹/L) in the Phase 3 PERSIST-2 clinical trial (NCT02055781). In a retrospective analysis of the PERSIST-2 study, pacritinib also demonstrated anemia benefits due to its potent inhibition of activin A receptor type 1 (ACVR1).

On September 15, 2023, momelotinib (JAK1/JAK2/ACVR1 inhibitor) received regulatory approval as a treatment for patients who have intermediate- or high-risk myelofibrosis and anemia based on the data acquired in the pivotal Phase 3 MOMENTUM clinical trial (NCT04173494) and the data from a cohort of patients who participated in the phase 3 SIMPLIFY-1 trial (NCT01969838). Momelotinib is a unique JAK1/2 inhibitor because it potently inhibits ACVR1 and thereby, expression of hepcidin (master regulator of iron in the body) in the liver, leading to marked and sustained anemia benefits, including red blood cell transfusion independence, besides reducing splenomegaly and improving symptoms. The regulatory approval of momelotinib was a major advancement in the treatment of anemic patients with myelofibrosis. Until momelotinib received regulatory approval, there was a critical unmet need for patients with myelofibrosis and anemia, which is one of the hallmarks of the disease.

Currently, many ongoing clinical trials are conducted at the Clinical Research Center for MPNs and are enrolling patients with MPNs or systemic mastocytosis. Our MPN experts are leading many important clinical trials on highly promising MPN treatments and aim to further improve the quality of life and the outcomes of patients worldwide. 

Please visit Approved MPN Medications and Clinical Trials for a detailed list of the active clinical trials in MPNs (myelofibrosis, polycythemia vera, and essential thrombocythemia) and systemic mastocytosis. Please visit Current MPN Research for more information on the MPN medications that have already received regulatory approval and the novel agents in clinical development. 

Faculty Treating MPNs and Systemic Mastocytosis (SM)

At MD Anderson, our leading MPN physicians have extensive experience and expertise in diagnosing and optimally treating all the MPN subtypes (myelofibrosis, polycythemia vera, essential thrombocythemia), systemic mastocytosis, and other myeloid neoplasms. Drawing upon their advanced expertise and the clinical trials that they lead at MD Anderson evaluating novel medications, our MPN physicians design optimal, personalized care plans that aim to improve the quality of life and the outcomes of our patients, and provide excellent patient care.

Prithviraj Bose, M.D., Professor, Leukemia; Chief, Section of MPNs

Naveen Pemmaraju, M.D., Professor, Leukemia, Co-Leader, Section of MPNs, Director of the BPDCN Program

Lucia Masarova, M.D., Associate Professor, Leukemia

Hussein A. Abbas, M.D., Ph.D., Assistant Professor, Leukemia (treating SM patients)