Onvansertib for the Treatment of Recurrent or Refractory Chronic Myelomonocytic Leukemia and Myelodysplastic Syndrome/MPN Overlap Neoplasms
Key claim: A phase I trial is evaluating onvansertib, a PLK1 inhibitor, for safety, optimal dosing, and efficacy in patients with recurrent or refractory CMML and MDS/MPN overlap neoplasms.
Abstract
This phase I trial evaluates the safety, effectiveness, and best dose of onvansertib for the treatment of patients with chronic myelomonocytic leukemia and Myelodysplastic syndrome/myeloproliferative neoplasm (MDS/MPN) overlap neoplasms that has come back (recurrent) or that does not respond to treatment (refractory). Onvansertib is a drug that binds to and inhibits an enzyme called PLK1, preventing cancer cell proliferation and causing cell death.
Why this matters
Recurrent/refractory CMML and MDS/MPN overlap neoplasms have few effective options once HMA-based therapy fails, and most emerging trials cluster around HMA/BCL-2, JAK, FLT3, or epigenetic combinations. Onvansertib introduces PLK1 (mitotic kinase) inhibition as a genuinely novel mechanistic axis for these specific subtypes, expanding the mechanistic diversity of the salvage pipeline. If tolerable, it could establish a new therapeutic class for a population where subtype-dedicated trials remain scarce.