New Drug Combination Shows Promise as Powerful Treatment for AML: Research

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  • The Leukemia & Lymphoma Society (LLS) is leading the groundbreaking precision medicine approach to improve cancer treatment
  • LLS’s goal is to test several investigational drugs in development to speed up approval

Scientists have identified two drugs that are potent against acute myeloid leukemia (AML) when combined, but only weakly effective when used alone. The researchers were able to significantly enhance cancer cell death by jointly administering the drugs that are only partially effective when used as single-agent therapies.

The study is a collaboration between Sanford Burnham Prebys Medical Discovery Institute and the University of Glasgow, which was recently published in the journal Nature Communication.

“Our study shows that two types of drugs, MDM2 inhibitors, and BET inhibitors, work synergistically to promote significant anti-leukemia activity,” says Peter Adams, Ph.D., a professor at Sanford Burnham Prebys, and senior author of the study. “The results were surprising because previous research had shown that each drug on its own had modest benefit against AML. The new research provides a scientific rationale to advance clinical studies of the drug combination in patients with AML.”

Notably, TP53, the most frequently mutated gene in all human cancers, is found unaltered in about 90% of AML patients. Since the product of the TP53 gene, p53, acts to suppress tumors, scientists have sought drugs that reactivate or boost its anti-cancer powers in AML, which should provide a clinical benefit. However, such drugs on their own have been disappointing in AML.

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