Treatment Previously Approved for Leukemia Patients Shows Potential for Childhood Brain Cancer Patients
NFCR-supported researcher Dr. Rakesh K. Jain of Massachusetts General Hospital Cancer Center continues to make breakthroughs against some of the most difficult-to-treat cancers. In a recent study published in PNAS: Targeting EPHB2/ABL1 restores antitumor immunity in preclinical models of ependymoma, Dr. Jain and his team identified a potential new therapeutic approach for pediatric brain cancer.
Their research suggests that dasatinib (Sprycel), a drug already approved by the FDA for treating leukemia, could effectively inhibit the growth of a specific molecular subgroup of ependymoma (EPN), a rare and often treatment-resistant pediatric brain tumor. The study found that dasatinib potently inhibited the growth of EPN primarily by targeting EPHB2/ABL1 signaling, which is involved in tumor progression and immune suppression within the tumor microenvironment. By blocking these pathways, dasatinib not only slows tumor growth but also strengthens the body’s immune response by activating key immune cells (CD8+ T cells) and transforming certain tumor-associated immune cells from a “tumor-supporting” state to a “tumor-fighting” state. Their findings provide strong justification for advancing dasatinib into clinical trials for pediatric ependymoma, with the hope of expanding treatment options for children with this challenging disease.
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What is Ependymoma?
Ependymoma (EPN) is a rare tumor of the central nervous system that can develop in the supratentorial region, the posterior fossa of the brain, or the spinal cord. It is one of the most common types of pediatric brain tumors, yet many existing treatments fail due to its resistance to therapy. Given the lack of effective treatment options, repurposing dasatinib presents an exciting opportunity to improve patient outcomes.
About Dr. Rakesh K. Jain
Dr. Rakesh K. Jain, a world-renowned expert in tumor microenvironments and drug delivery, has received support from NFCR since 2014. His work has significantly advanced our understanding of how the tumor’s surrounding microenvironment impacts the immune system, drug efficacy, and treatment resistance. In recognition of his groundbreaking contributions, Dr. Jain was awarded the 2022 Szent-Györgyi Prize for Progress in Cancer Research.
Currently, with NFCR’s support, Dr. Jain is leading efforts to enhance CAR-T cell therapy for glioblastoma (GBM) patients.
