New Targeted Treatment for Acute Myeloid Leukemia

In addition to chemotherapy, targeted medications have been created to treat cancer. These medications only impact cancer cells, sparing normal cells. A novel targeted therapy for the treatment of acute myeloid leukemia is called venetoclax (AML). In Finland, Venetoclax just received marketing approval.

Venetoclax functions by making cancer cells more susceptible to programmed cell death. Venetoclax does not appear to be successful against erythroid and megakaryoblastic leukemias, two uncommon and challenging-to-treat subgroups of the illness, according to a recent study. Malignant cells mimic blood stem cells that make red blood cells or platelets in certain kinds of leukemia. These patients currently have few alternatives for treatment.

The study, conducted by the University of Helsinki, the HUS Comprehensive Cancer Center, and the University of Denmark, revealed a new targeted medication that may one day provide patients with these disease subtypes with a therapeutic choice. In December, the study was released in the journal Blood.

Further Investigation is Required

The scientists tested a wide range of pharmacological substances in the lab that might be particularly effective against erythroid or megakaryoblastic leukemia cells.

BCL-XL protein inhibitors were particularly successful in eliminating cancer cells isolated from various kinds of leukemia among the more than 500 medicines examined. Similar to BCL-2, the target of venetoclax, the BCL-XL protein blocks the induction of programmed cell death in cells.

At the moment, BCL-XL inhibitors are not used to treat patients, but their efficacy and safety are currently being investigated in clinical trials.

“The introduction of venetoclax has significantly improved the prognosis of AML patients. However, our research indicates that venetoclax is unlikely to function optimally against the subtypes of AML in our focus. Nevertheless, the finding should be verified in larger patient datasets,” says physician-scientist Olli Dufva.

The most prevalent form of acute leukemia in adults is Acute Myeloid Leukemia. Based on mutations and the level of leukemia cell development, it can be categorized into subtypes. Finding patients who will benefit from the new treatment alternatives is a challenge that comes with the usage of tailored therapies. This study makes it easier to choose precision-targeted medications.

“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,” says postdoctoral researcher Heikki Kuusanmäki.

The researchers believe that BCL-XL inhibitors will be trialed in the treatment of these leukemia types in the near future.

“This finding may in the future improve the prognosis of these very rare and difficult-to-treat leukemias,” says Professor of Translational Haematology Satu Mustjoki from the University of Helsinki and HUS Comprehensive Cancer Center.

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