Study Finds Potential Therapeutic Target for Pediatric Acute Myeloid Leukemia

The researchers obtained genomic data from more than 2,000 pediatric patients with leukemia to identify associated gene variants.

A new study by the Nemours Center Children’s Health System found a gene expressed in children with acute myeloid leukemia (AML) that could serve as a new immunotherapy treatment target.

The study outlines the process and possible path for new immunotherapy drugs that improve survival and reduce treatment-related toxicity in children with AML. Leukemia is the most common cancer in children and teens, and AML accounts for nearly a quarter of those cases.

“Using genomic sequencing data, we have identified new targets for childhood cancer and collaborated with collaborators to develop new therapies for children with AML, instead of reusing adult cancer drugs that do not work well in children,” said senior study author E Anders Kolb, MD, director of Nemours’ Center for Childhood Cancer Research, in a press release.

The researchers obtained genomic data from more than 2,000 pediatric patients with leukemia to identify associated gene variants. Through genomic sequencing, they found that the gene mesothelin (MSLN) is abnormally expressed in more than a third of AML cases in children and young adults, but was absent from normal bone marrow cells, according to the study.

The researchers chose new immunotherapy drugs that would target MSLN to test in cell lines and animal models to further measure the preclinical effectiveness of leukemia therapies. Anetumab ravtansine (Bayer) and newer compound anti-MSLN-DGN462 (ImmunoGen) were tested, with each drug causing potent destruction of leukemia cells according to the study.

“We are working to show proof of the principle that we can create custom therapies for pediatric malignancies and turn the drugs we are testing in the lab into clinical trials,” said Sonali P. Barwe, PhD, the co lead author of the study and head of the preclinical leukemia testing laboratory at the Nemours’ Center for Childhood Cancer Research, in the press release.

REFERENCE

Study finds potential therapeutic target for pediatric acute myeloid leukemia. Nemours. Published May 3, 2020. Access May 6, 2021. http://nemours.mediaroom.com/media-releases?item=122879

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