Mohamad Cherry, MD, medical director of hematology for Atlantic Health System Cancer Care, is leading two clinical trials that use novel approaches to target treatment resistance in acute myeloid leukemia (AML).

A phase 2 study is examining the response of an investigational drug that acts as a small molecule inhibitor of cyclin-dependent kinase 9 (CDK9).  CDK9 is a protein critical to the regulation of gene expression involved in cancer.  The study is also looking at MCL-1, a protein, which, in cancers, serves to block the induction of cell death and may drive resistance to chemotherapy.  Data from previous trials suggest that certain tumors are dependent upon the MCL-1 protein to survive, making it a compelling target.  Therefore, treatment which blocks production of the MCL-1 protein can be of benefit to AML patients. 

A phase 3 study is looking at combining novel cancer drug CPI-613 with high-dose chemotherapies for AML patients who have relapsed or not responded to standard treatments.  CPI-613 is a first-in-class drug that alters attacks hard-to-treat cancers by targeting the metabolic processes the disease needs to survive, grow and proliferate. CPI-613 affects the cancer cells’ ability to multiply while also making them more susceptible to lower doses of chemotherapy. CPI-613 has demonstrated promising results in other studies and has been granted orphan drug status by the FDA for the treatment of pancreatic cancer, acute myeloid leukemia (AML), myelodysplastic syndrome (MDS) and Burkitt’s and peripheral T-cell lymphomas.

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“These two trials are exciting because they explore small molecules in addition to standard chemotherapy for relapsed/refractory AML patients,” said Dr. Cherry. “These small molecules target novel growth pathways that are implicated in development and resistance of myeloid leukemia cells. Novel treatment approaches like these are urgently needed.”

Adult acute myeloid leukemia is the fastest growing and most common type of acute leukemia.  Mostly affecting cells that are not fully developed and can’t carry out their normal functions, AML progresses rapidly without treatment.  The adult form of AML has the lowest five-year survival rate of any type of leukemia: just over 28%, compared with survival rates for other leukemias that range from approximately 69% to 87%.  

Morristown Medical Center’s Carol G. Simon Cancer Center is the only site in New Jersey offering these two trials.  To learn more about these trials, please visit atlantichealth.org/cancer