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New drug may treat rare subtype of AML

AML is a cancer of the blood-forming bone marrow cells. Image credit – University of Helsinki

CANCER DIGEST – Feb. 25, 2023 – Researchers in Finland have identified a possible new targeted therapy that could improve treatment for rare subtypes of acute myeloid leukemia (AML), the most common type of leukemia in adults. The study was published in the journal Blood December 12, 2022.

While a newly approved drug in Finland, called Ventoclax, had been showing good outcomes for most patients with AML, a cancer of the blood forming cells in bone marrow, it was found that patients with rare subtypes of the disease fared poorly. These patients had erythroid and megakaryoblastic leukemias, subtypes that are particularly difficult to treat.

The challenge in using targeted drugs is in matching the therapy to the particular features of the disease. To identify a targeted therapy that would specifically target erythroid and megakaryoblastic AML the researchers screened more than 500 agents in laboratory tests to see which if any drugs were effective in killing erythroid and megakaryoblastic leukemia cells.

They found that BCL-XL protein inhibitors were particularly effective in eradicating these cell types. While BCL-XL protein inhibitors were not currrently approved for the treatment of any cancers, they were currently undergoing clinical trials for treatment of prostate, laryngeal, and neuroendocrine cancers.

“The laboratory findings provide evidence that patients with erythroid or megakaryoblastic acute leukemia would be a promising group for investigating the efficacy of BCL-XL inhibitors in clinical use,” postdoctoral researcher and lead author of the study Heikki Kuusanmäki, said in a press release.

The researchers believe that BCL-XL inhibitors will undergo clinical trials in the treatment of these leukemia types in the near future. The study was funded by the Academy of Finland, Cancer Foundation Finland, the Finnish Cancer Institute, Sigrid Jusélius Foundation and the Finnish Medical Foundation.

Sources: University of Helsinki press release and the journal Blood.


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