Preclinical and clinical activity of ATP mimetic JAK2 inhibitors

Martha Wadleigh, Ayalew Tefferi

Research output: Contribution to journalArticlepeer-review

10 Scopus citations


The discovery of a common Janus kinase 2 (JAK2) point mutation, JAK2V617F, in myeloproliferative neoplasms has generated enormous interest in the development and therapeutic use of small molecule JAK2 inhibitor-targeted therapy in these diseases. A handful of compounds are currently in clinical development in primary myelofibrosis or post-polycythemia vera (PV)/essential thrombocythemia (ET) myelofibrosis. To date, clinical benefit has been demonstrated in terms of reduction of splenomegaly, improvement in constitutional symptoms, and control of leukocytosis. Some of the drugs have also been evaluated in PV and ET, with demonstrated activity against erythrocytosis, thrombocytosis, pruritus, and splenomegaly. However, drug effect on bone marrow fibrosis or JAK2 allele burden has been modest so far. Regardless, it is important to keep in mind that current anti-JAK2 treatment trials constitute only the beginning of many upcoming similar clinical trials, and that it is premature to make generalizations or any form of comparative conclusions regarding drug activity or toxicity.

Original languageEnglish (US)
Pages (from-to)557-563
Number of pages7
JournalClinical Advances in Hematology and Oncology
Issue number8
StatePublished - Aug 2010


  • Jak2 inhibitors
  • Myelofibrosis
  • Myeloproliferative neoplasms

ASJC Scopus subject areas

  • Hematology
  • Oncology


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