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 MPNs worldwide. 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 (first JAK1/2 inhibitor that was approved as a treatment for myelofibrosis on November 16, 2011). Collectively, more than 100 early/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 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 MPN medications were assessed and several received regulatory approval based on pivotal clinical studies that were conducted at the Clinical Research Center for MPNs at the MD Anderson Cancer Center.

The MPN research studies conducted at the Clinical Research Center for MPNs are published in prestigious medical journals and are frequently presented at the most important national and international conferences, such as 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). Furthermore, the Clinical Research Center for MPNs periodically publishes the newsletter MPN Focus with the goal to educate MPN patients, caregivers, and healthcare providers about novel MPN therapeutics and the latest research findings in MPNs. Please visit MPN Awareness and Education page to view the collection of MPN Focus newsletters and other MPN educational materials.

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 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 also 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. Regulatory approval of momelotinib was a major advancement in the treatment of anemic patients with myelofibrosis (MF). Until momelotinib received regulatory approval, there was a critical unmet need for MF patients with anemia, which is one of the hallmarks of the disease.

In February 2022, pacritinib (JAK2/ACVR1/IRAK/FLT3 inhibitor) received accelerated regulatory approval as a medication to treat patients with 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 thrombocytopenia (platelet counts below 100x10⁹/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 ACVR1.

Currently, other clinical trials are conducted at the Clinical Research Center for MPNs in which very promising MPN medications are evaluated. Please visit Approved MPN Medications and Clinical Trials, and Current MPN Research for more on MPN medications and recent advancements in MPN research.

Faculty Treating MPNs and Systemic Mastocytosis (SM)

Prithviraj Bose, M.D., Professor, Leukemia

Naveen Pemmaraju, M.D., Professor, Leukemia

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

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