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UT MD Anderson established the Hanns A. Pielenz Clinical Research Center for Myeloproliferative Neoplasms (MPNs) as a distinct operational unit within the Leukemia department and the Leukemia Outpatient Center in 2011. The goals of our MPN Faculty who treat patients in the Clinical Research Center for MPNs are to develop novel effective therapies for MPNs that will significantly improve patients' outcomes and quality of life, provide exceptional patient care and ultimately cure MPNs.

The Hanns A. Pielenz Clinical Research Center for MPNs has become the largest in the world for referrals of MPN patients and clinical research. Medications that have become the global standard of care for myelofibrosis (for example, ruxolitinib, the first and only JAK1/2 inhibitor until 2019) were developed for the first time at the Clinical Research Center for MPNs at UT MD Anderson. Our MPN faculty see more than 300 new patients with MPNs annually, far more than any other clinical center in the world, and are continuously engaged in many clinical research studies to develop novel therapies for our patients who have MPNs or systemic mastocytosis.

The main goals of the Hanns A. Pielenz Clinical Research Center for MPNs include the following:

• Conduct clinical trials to evaluate novel agents for patients with MPNs and provide exceptional patient care. For a comprehensive overview of novel MPN medications in clinical development at the Clinical Research Center of MPNs, please visit the site Current MPN Research.
• Develop novel effective medications that will receive regulatory approval for the treatment of MPNs, such as (JAK1/2 inhibitor) for the treatment of myelofibrosis (approved in November 2011) and polycythemia vera (approved in December 2014); and (JAK1/2 and ACVR1 inhibitor) for the treatment of myelofibrosis and anemia (approved in September 2023). To date, four JAK inhibitors (ruxolitinib, fedratinib, pacritinib, and momelotinib) have received regulatory approval as treatments for myelofibrosis in the US. Please read our recent article, from the "How I treat" Series, in  titled, "How I individualize selection of JAK inhibitors for patients with myelofibrosis". Several promising novel medications, which have different mechanisms of action from JAK inhibitors, are currently in early and advanced clinical development as treatments for myelofibrosis. Other novel agents are evaluated in clinical trials as treatments for polycythemia vera, essential thrombocythemia and systemic mastocytosis.
• Educate patients, caregivers, physicians and the broader community about MPNs and the latest findings regarding novel treatments in clinical development in MPNs and systemic mastocytosis at UT MD Anderson.
• Maintain a centralized MPN Tissue Biobank for the collection and storage of patient specimens, which are valuable resources to conduct basic and translational research in MPNs. Our MPN Tissue Biobank is a comprehensive repository of biospecimens, including peripheral blood samples (78%) and bone marrow aspirates (22%) from thousands of patients who have been treated at UT MD Anderson since 2006 when the MPN Tissue Biobank was established. As of September 1, 2024, our MPN Tissue Biobank has specimens and clinical data from more than 4,700 patients.

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. 

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 () and as a treatment for polycythemia vera, on December 4, 2014 ().

Collectively, more than 200 early and advanced phase clinical trials on novel MPN medications have been conducted at the Clinical Research Center for MPNs. Notably, the MPN Team and the clinical research program in MPNs advanced exponentially since 2000. At that time, only one physician and one research nurse treated MPNs, and only one active MPN-specific clinical protocol was enrolling patients. 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.

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  (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 (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 (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 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.

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