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Engineering Oncogenic Heterozygous Gain-of-Function Mutations in Human Hematopoietic Stem and Progenitor Cells
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[Novel therapeutics in myeloproliferative neoplasms: beyond JAK inhibitors]

Yoko Edahiro1

  • 1Department of Hematology, Juntendo University School of Medicine.

[Rinsho Ketsueki] the Japanese Journal of Clinical Hematology
|October 4, 2023
PubMed

Insights

Myeloproliferative neoplasms (MPN) involve JAK/STAT signaling. While JAK inhibitors like ruxolitinib help, new therapies are needed for refractory myelofibrosis and to prevent leukemia progression.

Area of Science:

  • Hematology
  • Oncology
  • Molecular Biology

Context:

  • Myeloproliferative neoplasms (MPN) are driven by genetic mutations, notably JAK2.
  • Constitutive activation of the JAK/STAT pathway is a key mechanism in MPN pathogenesis.
  • Ruxolitinib, a JAK1/2 inhibitor, is approved for polycythemia vera and myelofibrosis, showing benefits in symptom control and disease markers.

Purpose:

  • To review the current understanding of MPN pathogenesis and the role of JAK inhibitors.
  • To highlight the limitations of existing JAK inhibitor therapies, including resistance and intolerance.
  • To introduce novel therapeutic strategies under investigation for myelofibrosis.

Summary:

  • JAK inhibitors have improved MPN management, but challenges remain, such as refractoriness and cytopenia in myelofibrosis patients.
  • JAK inhibitors do not prevent the progression to acute leukemia in myelofibrosis.
  • Emerging therapies targeting BCL, MDM2, LSD1, PI3K, BET, and telomerase show promise for myelofibrosis.

Impact:

  • Identifies unmet needs in myelofibrosis treatment.
  • Provides an overview of novel drug candidates for MPN.
  • Informs future research directions and clinical trial designs for myelofibrosis and related MPNs.
Keywords:
JAK inhibitorMyelofibrosisMyeloproliferative neoplasmsNovel agents

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