FGFR Inhibitors: Hope for Pediatric Brain Tumor Treatment
- Researchers have gained crucial insights into embryonal tumor with multilayered rosettes (ETMR), a rare and aggressive brain tumor affecting young children.Teh study, conducted by scientists at Dana-Farber/Boston Children's...
- The research, published in Nature Cancer, focuses on how a unique genetic alteration, the C19MC microRNA cluster, drives ETMRs by maintaining tumor cells in an immature state.
- Volker Hovestadt, co-lead author, said that FGFR and NOTCH receptors are expressed by proliferative NSC-like cells, while more differentiated malignant cells within the same tumor supply the corresponding...
new research spotlights the potential of FDA-approved FGFR inhibitors in treating embryonal tumor with multilayered rosettes (ETMR), a rare pediatric brain tumor. This groundbreaking study identifies vulnerabilities in ETMR cells, mirroring early brain development, which is crucial for tumor growth. Scientists believe targeting these cellular interactions,specifically with FGFR inhibitors,may become a key strategy for these arduous-to-treat cancers. According to News Directory 3, early results look promising. This research also advocates for upcoming clinical trials to evaluate FGFR adn NOTCH inhibitors. Discover what’s next in the fight against aggressive pediatric brain tumors.
FGFR Inhibitors Show Promise Against Pediatric Brain Tumor
Updated May 27, 2025
Researchers have gained crucial insights into embryonal tumor with multilayered rosettes (ETMR), a rare and aggressive brain tumor affecting young children.Teh study, conducted by scientists at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center and the Broad Institute, reveals potential new avenues for targeted therapies.
The research, published in Nature Cancer, focuses on how a unique genetic alteration, the C19MC microRNA cluster, drives ETMRs by maintaining tumor cells in an immature state. The team’s analysis showed that ETMR cells mirror early brain development, creating a hierarchy of stem-like and neuron-like cells. This cellular cooperation is vital for tumor growth, with mature cells supporting the stem-like cells.
Dr. Volker Hovestadt, co-lead author, said that FGFR and NOTCH receptors are expressed by proliferative NSC-like cells, while more differentiated malignant cells within the same tumor supply the corresponding ligands, indicating a coordinated role in sustaining tumor growth. He added that targeting these interactions may represent a critical vulnerability in ETMR.
The study suggests the potential for repurposing existing FDA-approved FGFR inhibitors. Early results have been promising in one patient treated under compassionate use.
Dr. mariella Filbin,co-director of the Brain Tumor Center at Dana-Farber/Boston Children’s cancer and Blood Disorders Center,emphasized the meaning of using single cell sequencing to examine this highly lethal tumor. She noted the fascinating way all tumor cells, even when different, help each other to survive and grow. Learning how to disrupt their cooperation, for example by blocking one of their communication lines will be imperative to treat this cancer in the future.
The study advocates for clinical trials to evaluate FGFR and NOTCH inhibitors in ETMR patients. Researchers are also exploring antisense therapies targeting C19MC miRNAs, which have demonstrated promise in laboratory settings. These findings provide a strong rationale for developing more effective targeted therapies, potentially revolutionizing the treatment of children with ETMR and improving overall survival.
What’s next
Further research will focus on clinical trials to test the effectiveness of FGFR and NOTCH inhibitors, as well as antisense therapies, offering hope for improved outcomes in children battling this aggressive cancer.