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Apr 23 2025
2A recent study has highlighted the potential of a repurposed gastrointestinal cancer drug to treat one of the most aggressive forms of brain tumors—high-grade gliomas (HGGs). These fast-growing tumors, particularly devastating in children, are notoriously difficult to treat and are associated with poor survival outcomes. A promising breakthrough now comes from a familiar source: a drug already approved by the FDA for a different cancer type.
Published in Cancer Cell, the study demonstrates how the existing cancer drug avapritinib, known for treating gastrointestinal stromal tumors, may be repurposed for glioma patients whose tumors carry mutations in the PDGFRA gene. Drug repurposing, the practice of finding new uses for existing medications, offers a valuable shortcut in the long, complex process of drug development—especially critical for patients battling aggressive diseases with limited treatment windows.
Dr. Mariella Filbin, a pediatric neuro-oncologist at Dana-Farber Cancer Institute and co-author of the study, spearheaded the effort to explore new treatment strategies for her young patients. Her team focused on PDGFRA, a gene commonly mutated in pediatric HGGs. Using genomic sequencing from pediatric glioma cases, the researchers found that several patients had elevated levels of PDGFRA in their cancerous cells compared to healthy cells, highlighting the gene as a potential therapeutic target.
The team identified four FDA-approved or investigational PDGFRA inhibitors and evaluated them in both laboratory models and patient-derived glioma cell lines. Among them, avapritinib stood out for its strong suppression of PDGFRA activity and its ability to trigger programmed cell death in tumor cells.
One of the key challenges in treating brain tumors is ensuring that drugs can cross the blood-brain barrier. In preclinical animal studies, avapritinib not only reached brain tissue but also significantly reduced tumor growth. This finding encouraged researchers to take the next step toward patient trials.
In collaboration with Blueprint Medicines—the Boston-based pharmaceutical company that developed avapritinib—Filbin’s team, along with researchers from the Medical University of Vienna and the University of Michigan, initiated a small-scale compassionate use study. Eight patients between the ages of four and 29, all with highly treatment-resistant HGGs, were given avapritinib. Seven of the patients had tumors with confirmed PDGFRA mutations.
The drug was well tolerated, and three of the patients showed changes in tumor size and shape, alongside extended survival compared to typical prognoses. These initial outcomes, while based on a small sample, are a meaningful step forward in a field where most patients succumb to the disease within nine to twelve months of diagnosis.
“This is a very hopeful development,” said Filbin. “But we now need rigorous clinical trials to confirm whether our early findings hold up.” Her team aims to launch a clinical trial for newly diagnosed glioma patients with PDGFRA mutations.
Experts emphasize that while this progress is exciting, single-agent therapies are rarely enough against aggressive cancers like HGGs. Ongoing preclinical research is focused on finding drug combinations that could work synergistically with avapritinib to improve treatment outcomes and extend survival.
“This sets the stage for combination strategies,” noted Dr. Oren Becher, a pediatric oncologist not involved in the study. “We need therapies that not only attack the tumor but also prevent its rapid evolution.”
Filbin’s lab continues to explore combination treatments, hoping to pave the way for longer-term survival and better quality of life for patients facing this devastating diagnosis.
Source:https://www.the-scientist.com/repurposed-cancer-drug-could-help-treat-brain-tumors-72931
This is non-financial/medical advice and made using AI so could be wrong.
