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Phase I/II and Pharmacological Study of Intravenous OTS167 in Patients with Refractory or Relapsed AML, Acute Lymphoblastic Leukemia, Advanced Myelodysplastic Syndromes, Advanced MPNs, or Advanced CML

Clinical Trial Details

The purpose of this study is to look at the safety and potential benefit of OTS167 in adults with relapsed or refractory Acute Myeloid Leukemia (AML), Acute Lymphoblastic Leukemia (ALL), Advanced Myelodysplastic Syndromes (MDS), Advanced Myeloproliferative Neoplasms (MPN) or Advanced Chronic Myelogenous Leukemia (CML). 

OTS167 is a novel kinase inhibitor which binds to the Maternal Embryonic Leucine Zipper Kinase (MELK). Studies have demonstrated expression of MELK in cancer stem cells-cancer initiating cells (CSC-CIC), as well as in other cancers, including central nervous system (CNS) tumors and breast cancer. In some studies, over-expression of MELK has been associated with increasing tumor severity, poor prognosis, or resistance to therapy. Studies have also suggested that MELK expression is associated with a variety of other genes that allow cancer cells to divide. It is thought that inhibition of MELK by OTS167 will impair cancer cells’ ability to proliferate, survive and evade other anticancer treatments. 

OTS167 has been given to animals with other cancers but this is the first clinical trial in which OTS167 is being given to humans with blood cancer. OTS167 is not approved for use by the U.S. Food and Drug Administration (FDA). Therefore, it can only be used in research studies such as this one. 

Treatment with OTS167 will be given by intravenous (IV) infusion over 30 minutes. Treatment will be administered in 21-day cycles in which subjects receive a daily dose of the study drug for 5 days, followed by 2 days of rest, daily dosing for 5 days, followed by 9 days of rest. 

Key Eligibility: 
  • Open to men and women aged 18 or older with one of the following diagnoses: Relapsed or refractory AML, ALL, acute biphenotypic leukemia, advanced MDS, advanced MPN, MDS/MPN overlap syndrome or advanced CML 
  • Potential patients must be refractory to all approved therapies or for which no approved or conventional therapies are available 
  • Detailed eligibility will be reviewed when you contact the study team

Study contact by location

Upper East Side - Manhattan


Yulia Dault, RN
(212) 746-4829

Primary Investigator(s)

Protocol ID(s)

Weill Cornell Medicine IRB #:






Open to Enrollment

Age Group