Updates on Pediatric Leukemia Research

Researchers are making progress in identifying drugs that target certain mutations.

Leukemia is the most common childhood cancer and is responsible for nearly 1 in 3 childhood cancers. Most pediatric leukemias are acute lymphoblastic leukemia (ALL), which is sometimes referred to as acute lymphoblastic leukemia. Most of the remaining cases are acute myeloid leukemia (AML). Both ALL and AML are cancers that progress quickly and require immediate treatment. Standard treatments for childhood leukemia generally include chemotherapy, radiation therapy, and stem cell transplant. Research is now focusing on targeted drugs, immunotherapies, and newer chemotherapy drugs.

Targeted drugs

About 10% of children with AML have changes in the gene called FLT3, and a Phase 3 randomized trial is currently underway to evaluate Nexavar (sorafenib), which targets FLT3, in addition to standard chemotherapy in patients with newly diagnosed AML. Some pediatric leukemias also have gene fusions that can increase the growth of cancer cells. Vitrakvi (larotrectinib) is currently approved for adult and pediatric patients with solid tumors who have an NTRK gene fusion. Another drug, larotrectinib, is currently being studied in children with leukemia who have an NTRK gene fusion. The Pediatric Preclinical Testing Consortium is also addressing the challenges of developing novel targeted therapies for children by providing reliable preclinical data to lay the groundwork for robust clinical trials.


Researchers recently identified a new target for immunotherapy treatment known as mesothelin (MSLN) for AML in pediatric patients. A study on mesothelin recently published in Blood Advances included genomic data obtained from more than 2,000 children and young adults with AML. The researchers compared that data with comparable data from individuals with normal bone marrow. The study found that MSLN was manifested in 36% of study participants with AML. Next, the researchers selected new immunotherapy drugs that would target MSLN to test in cell lines and animal models to estimate the preclinical efficacy of leukemia treatments. Two experimental immunotherapies, anetumab ravtansine and a new compound, anti-MSLN-DGN462, produced potent destruction of leukemia cells in both laboratory tests and mouse models. “

“Using genomic sequencing data, we identified new targets for childhood cancer and worked with collaborators to develop new therapies for children with AML, rather than reusing drugs from the adult cancer realm that don’t work well in children,” said E Anders Kolb, MD, director of Nemours Center for Childhood Cancer Research and a study author, said in a press release. Further research is needed for this promising new target of immunotherapy treatment.


Researchers are working to develop new chemotherapy drugs for leukemia and are evaluating current drugs to find better ways to use existing drugs. Venclexta (venetoclax) is FDA-approved in adults with certain leukemias and is now being studied in children with AML who have failed to respond to previous treatments or who have relapsed.

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