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Researchers at the Ludwig Center at Harvard demonstrate arsenic-based therapy can reverse drug tolerance in acute myeloid leukemia

Apr 30, 2019

APRIL 30, 2019, New York— Every year, nearly 11,000 Americans die of acute myeloid leukemia (AML), a blood cancer that affects mainly older adults. While most patients initially respond to chemotherapy, more than half of those who respond eventually relapse as AML cells develop resistance to treatment. In a new paper published on April 25 in Cell Research, a team of scientists led by Pier Paolo Pandolfi, a researcher with the Ludwig Center at Harvard and Director of the Cancer Center and Cancer Research Institute at Beth Israel Deaconess Medical Center, reveal vulnerabilities in a subset of AML cases that could help researchers overcome its drug resistance.

About 20% of AML cases involve cells with a mutation in a key metabolic enzyme called IDH. Pandolfi and colleagues identified key processes these cells use to develop drug resistance and identified vulnerabilities that they could exploit for therapeutic benefit in all stages of the disease’s evolution. Moreover, the team demonstrated that a combination of drugs already in use for treatment of another type of leukemia worked just as well against this form of AML in cell cultures and animal models.

Another form of leukemia known as acute promyelocytic leukemia (APL) was once a fatal diagnosis. It is now considered a curable disease, thanks to researchers including Pandolfi who demonstrated that arsenic trioxide (ATO) was effective against APL in combination with another drug called all-trans retinoic acid (ATRA). In the current study, Pandolfi and colleagues tested the combination in both the mouse model of AML with the IDH mutation and in human AML cells. They determined that, while the drugs target different vulnerabilities in the different forms of leukemia, the combination proved “powerfully and exquisitely effective” against this subset of AML. 

Pandolfi and his team are working to develop clinical trials to evaluate the combination for treatment of AML with mutation in IDH enzymes.

The BIDMC release from which this summary is derived can be found here.