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New promising targeted drug for a rare leukemia

February 23, 2023
University of Helsinki
Researchers have identified a possible new targeted drug for treating acute myeloid leukemia. In the future, the discovery may help patients with certain rare subtypes of the disease.

Targeted drugs have been developed to supplement chemotherapy in the treatment of cancer. These drugs only affect cancer cells, leaving healthy cells alone. Venetoclax is a new targeted therapy option for the treatment of acute myeloid leukemia (AML). Venetoclax was recently granted marketing authorisation in Finland.

Venetoclax works by sensitising cancer cells to programmed cell death. However, a new study now shows that venetoclax does not appear to be effective against erythroid and megakaryoblastic leukemias, two rare subtypes of the disease that are difficult to treat. In these leukemia types, malignant cells resemble blood stem cells that produce red blood cells or platelets. Currently, few treatment options are available to these patients.

The study carried out by the University of Helsinki, HUS Comprehensive Cancer Center and the University of Copenhagen identified a new targeted drug, which may in the future offer a therapeutic option to patients with these subtypes of the disease.The study was published in the Blood journal in December.

Further research needed

In the laboratory, the researchers screened a wide selection of pharmaceutical agents that could be effective specifically against erythroid or megakaryoblastic leukemia cells.

Among the more than 500 agents analysed, BCL-XL protein inhibitors in particular were effective in killing cancer cells isolated from these types of leukemia. The BCL-XL protein has a similar function of preventing cells from being driven to programmed cell death as BCL-2, the target of venetoclax. 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.

Potential to improve prognosis

AML is the most common type of acute leukemia in adults. It can be divided into subtypes based on mutations and the degree of differentiation of leukemia cells. One challenge associated with the use of targeted drugs is identifying patients who benefit from the new drug options. This study contributes to making the selection of targeted drugs more precise.

"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 trialled 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.

The study was funded by the Academy of Finland, Cancer Foundation Finland, the Finnish Cancer Institute, Sigrid Jusélius Foundation and the Finnish Medical Foundation. The study was carried out under the iCAN Digital Precision Cancer Medicine Flagship funded by the Academy of Finland.

Story Source:

Materials provided by University of Helsinki. Note: Content may be edited for style and length.

Journal Reference:

  1. Heikki Kuusanmäki, Olli Dufva, Markus Vähä-Koskela, Aino-Maija Leppä, Jani Huuhtanen, Ida Maria Vänttinen, Petra Johanna Nygren, Jay Klievink, Jonas Otto Vilhelm Bouhlal, Petri Pölönen, Qi Zhang, Shady Adnan Awad, Cristina Mancebo-Pérez, Joseph Saad, Juho J. Miettinen, Komal Kumar Javarappa, Sofia Aakko, Tanja Ruokoranta, Samuli Eldfors, Merja Heinäniemi, Kim Theilgaard-Mönch, Ulla Wartiovaara-Kautto, Mikko A I Keränen, Kimmo Porkka, Marina Konopleva, Krister Wennerberg, Mika Kontro, Caroline A. Heckman, Satu Mustjoki. Erythroid/megakaryocytic differentiation confers BCL-XL dependency and venetoclax resistance in acute myeloid leukemia. Blood, 2022; DOI: 10.1182/blood.2021011094

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University of Helsinki. "New promising targeted drug for a rare leukemia." ScienceDaily. ScienceDaily, 23 February 2023. <>.
University of Helsinki. (2023, February 23). New promising targeted drug for a rare leukemia. ScienceDaily. Retrieved May 28, 2024 from
University of Helsinki. "New promising targeted drug for a rare leukemia." ScienceDaily. (accessed May 28, 2024).

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