The latest developments in research and treatment of MPNs
Hanns A. Pielenz Clinical Research Center for Myeloproliferative Neoplasms
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The MD Anderson Cancer Center 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 the MD Anderson Cancer Center. 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. Please visit the highlighted link for a complete list of the active clinical trials in MPNs and systemic mastocytosis conducted at MD Anderson.
- Develop novel effective medications that will receive regulatory approval for the treatment of MPNs, such as ruxolitinib (JAK1/2 inhibitor) for the treatment of myelofibrosis (approved in November 2011) and polycythemia vera (approved in December 2014); and momelotinib (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 Blood (2025) 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 (please see Figure 1 below). 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 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 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.
This is a very exciting time because several novel promising medications are in clinical development to treat myeloproliferative neoplasms and systemic mastocytosis at the MD Anderson Cancer Center.
Clinical Research Center for MPNs Team
Figure 1
Targets of novel agents that currently are in clinical development for the treatment of myelofibrosis. Figure from our publication, Chifotides HT, Bose P, Masarova L, Pemmaraju N, Verstovsek S. SOHO State of the Art Updates and Next Questions: Novel Therapies in Development for Myelofibrosis. Clinical Lymphoma Myeloma & Leukemia 2022;22(4):210-223. Copyright: The University of Texas MD Anderson Cancer Center and Elsevier 2021.
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