A new collaboration between Penn State College of Medicine and Targepeutics seeks to advance research into a promising combination therapy that offers hope to families with children diagnosed with DIPG (diffuse intrinsic pontine glioma).
This glioma is devastating and currently has limited therapy options. Patients diagnosed with this cancer survive for less than a year on average. A new combo therapy involving an immunotoxin dubbed GB13 and radiotherapy has shown promise in preclinical trials where approaches like chemotherapy or targeted therapy have had dismal results. The collaboration seeks to advance this treatment to clinical studies and eventual approval by the FDA.
The planned clinical trial seeks to recruit pediatric participants, a reflection of how urgent it is to get effective treatments for the disease. Under normal circumstances, trials often begin with adult subjects and then once successful, pediatric patients are enrolled. The effort being undertaken by Penn State and its collaborators highlights the role that this university is playing in ensuring that promising treatments move quickly from the research lab to the patients who need these treatments.
The FDA has designated GB13 as a treatment targeting a Rare Pediatric Disease and the innovation has also obtained Orphan Drug status. These designations have opened pathways for additional resources and prompt regulatory support in a bid to move the research process forward so that an effective treatment can become available to patients.
The FDA has already given the nod to the preclinical and clinical development proposals for the treatment. Targepeutics developed the drug candidate and Penn State is stepping in to participate as a fulfillment of their mission to undertake translational research that brings real solutions to families impacted by such devastating diagnoses. The target is to complete the planned preclinical work within the first half of 2026 and then expeditiously move the development process forward thereafter.
GB13 is delivered directly into the tumor, alongside radiotherapy. The drug kills cancer cells while leaving the surrounding healthy tissues unaffected. Lab tests have shown that this combination is effective and the planned clinical trials are intended to further test the efficacy and safety of the protocol.
Doctor Giselle Sholler, a pediatric oncologist and hematologist at Penn State University, says their study reflects the institution’s commitment to making innovative therapies accessible to patients in need.
Many entities like CNS Pharmaceuticals Inc. (NASDAQ: CNSP) are also advancing R&D programs aimed at addressing hard to treat pediatric brain cancers. These efforts promise to yield results that will alter the prospects of patients that have been diagnosed with these conditions.
NOTE TO INVESTORS: The latest news and updates relating to CNS Pharmaceuticals Inc. (NASDAQ: CNSP) are available in the company’s newsroom at https://ibn.fm/CNSP
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