title: Myeloproliferative neoplasms: new translational therapies.
abstract: The myeloproliferative neoplasms represent a diverse group of hematologic malignancies that have been the subject of intense investigation over the last decade. Although clinical trials of the much anticipated small molecule inhibitors of Janus kinase 2 have shown that these experimental agents are successful in palliating many of the symptoms associated with the myeloproliferative neoplasms, they have not been reported to affect the disease initiating hematopoietic stem cell population or to alter the natural history of these disorders. Investigators remain optimistic that new information about the genetic and cellular origins gained from the efforts of numerous laboratories will ultimately translate in to the identification of new drug targets and more effective therapies. We hypothesize that ultimately, the use of combinations of drugs including chromatin modifying agents, immunomodulatory agents, anti-apoptotic agents, cellular therapies and monoclonal antibodies will be required to effectively treat patients with myeloproliferative neoplasms.
authors.affiliation: Mount Sinai School of Medicine, New York, NY, USA. john.mascarenhas@mssm.edu
authors.forename: John
authors.initials: J
authors.lastname: Mascarenhas
authors.forename: Ronald
authors.initials: R
authors.lastname: Hoffman
mesh_terms: D018450:Disease Progression; D004359:Drug Therapy, Combination; D016313:Genes, abl / Q000235:genetics; D006801:Humans; D053614:Janus Kinase 2 / Q000235:genetics; D015464:Leukemia, Myelogenous, Chronic, BCR-ABL Positive / Q000235:genetics / Q000628:therapy; D009196:Myeloproliferative Disorders / Q000235:genetics / Q000628:therapy*; D011086:Polycythemia / Q000235:genetics / Q000628:therapy; D055728:Primary Myelofibrosis / Q000235:genetics / Q000628:therapy; D033581:Stem Cell Transplantation; D013920:Thrombocythemia, Essential / Q000235:genetics / Q000628:therapy; D057170:Translational Research, Biomedical
chemical_list: D053614:Janus Kinase 2