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Another encouraging update on GB13, a targeted immunotoxin that selectively kills brain tumor cells that express the IL13Ra2 receptor, which is found on many DIPG tumors but is largely absent from normal brain tissue.
In a newly published preclinical animal study, GB13 significantly reduced tumor size and improved survival across multiple DIPG models. The results were especially compelling when GB13 was given before and alongside radiation therapy. Notably, durable (and in some cases curative) responses were observed in human tumor–derived DIPG models. Researchers found that GB13 interferes with tumor cells’ ability to repair radiation-induced DNA damage, making radiation more effective at killing cancer cells. This radiosensitizing effect was consistent across multiple patient-derived DIPG cell lines and validated in both immunocompromised and immunocompetent mouse models. Together, these findings strongly support Targepeutics’ planned IND submission and upcoming Phase I clinical trial, with an expansion Phase II component, which will evaluate GB13 in children with DIPG.
As we’ve shared previously, The Musella Foundation helped support the early research behind GB13 more than a decade ago. It’s exciting to see this work continue to move steadily toward the clinic!