The safety and efficacy of rigosertib in the treatment of myelodysplastic syndromes

Expert Rev Anticancer Ther. 2016 Aug;16(8):805-10. doi: 10.1080/14737140.2016.1209413. Epub 2016 Jul 15.


Introduction: Hypomethylating agents (HMAs) are the standard of care for patients with higher-risk myelodysplastic syndromes (MDS), but patients who relapse or are refractory have a poor prognosis with an estimated survival of 4-6 months. Rigosertib, a Ras mimetic that inhibits the phophoinositide 3-kinase and polo-like kinase pathways, has been tested in patients with higher-risk MDS following treatment with HMAs, where there are no approved second-line therapies.

Areas covered: This review will provide an overview of rigosertib, including safety and efficacy demonstrated in clinical trials. Expert commentary: There is an urgent need for new treatment options for patients who have failed or progressed on HMAs. Rigosertib is currently undergoing testing as a single agent in certain subsets of higher-risk MDS patients as well as in combination with azacitidine, where preliminary data show efficacy in patients with de novo MDS as well as HMA failures.

Keywords: DNA methyltransferase inhibitors; Myelodysplastic syndromes; ON 0910.Na; Ras-binding domain; hypomethylating agents; phosphatidylinositol 3-kinase; polo-like kinase; rigosertib.

Publication types

  • Review

MeSH terms

  • Azacitidine / administration & dosage*
  • Azacitidine / therapeutic use
  • Disease Progression
  • Drug Therapy, Combination
  • Glycine / administration & dosage
  • Glycine / adverse effects
  • Glycine / analogs & derivatives*
  • Glycine / pharmacology
  • Humans
  • Myelodysplastic Syndromes / drug therapy*
  • Myelodysplastic Syndromes / physiopathology
  • Prognosis
  • Sulfones / administration & dosage*
  • Sulfones / adverse effects
  • Sulfones / pharmacology
  • Survival Rate
  • Treatment Outcome


  • Sulfones
  • ON 01910
  • Azacitidine
  • Glycine