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Posted On: 08/11/2025 5:14:54 PM
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Study Reveals Potential Way to Treat Pediatric Gliomas
A joint study between the Broad Institute and the Dana-Farber Cancer Institute has discovered a new and potentially effective way to treat pediatric gliomas. The research revealed that a percentage of children diagnosed with gliomas, an incredibly common type of pediatric brain cancer, may respond to certain inhibitors that have already been approved by the U.S. Food and Drug Administration.
According to the researchers, 8.9% of pediatric glioma patients exhibited fibroblast growth factor receptor alterations, a group of proteins that play a key role in cell differentiation, growth, and development. These alterations increased the gliomas’ sensitivity to inhibitors that can block the action of FGFR proteins.
Most importantly, since these inhibitors already have FDA approval, researchers can begin studying how they interact with pediatric gliomas at a relatively accelerated pace.
These findings could pave the way for the development of targeted therapies for gliomas in pediatric cancer patients, especially patients afflicted with pediatric low-grade gliomas (pLGG). Although FGFR-altered pediatric gliomas affect a significant portion of pediatric glioma patients, there are no clinical trials or FDA-approved treatments for them. Consequently, the recent findings could be critical to developing treatments for patients suffering from this particular kind of pediatric glioma.
Even so, further research will be necessary to improve the efficacy of the FDA-approved inhibitors against FGFR-altered pediatric gliomas before the therapy is deployed. Pratiti (Mimi) Bandopadhayay, MBBS, PhD, a Dana-Farber/Boston Children’s Cancer and Blood Disorders Center physician-scientist, Broad Institute member, and co-senior author, says the study was motivated by diagnoses of FGFR-altered pediatric gliomas around the world.
The research team wanted to see if there were already existing medications that could address this condition, Bandopadhayay says, and its results gave the team new insight into how to treat pediatric cancer patients with this specific type of glioma. According to Bandopadhayay, there is a major need for alternative treatments for FGFR-altered gliomas in pediatric patients, and the joint research effort led to the approval of tovorafenib as a therapy for BRAF-altered gliomas in children.
Co-headed by Keith Ligon, MD, PhD, a Broad Institute associate member and pathologist at Dana-Farber, the research involved carrying out genomic analyzes on over 10,000 gliomas, with alterations in the FGFR1 and FGFR2 genes being the most common in structural variants and point mutations. Co-first author April Apfelbaum says their results represented the first time preclinical data showed that FGFR-altered pediatric gliomas could respond to FGFR inhibitors.
The research adds to growing momentum in pediatric cancer studies that seek to repurpose existing drugs for faster clinical application. By narrowing in on genetic drivers like FGFR alterations, scientists can tailor treatments more precisely and reduce the need for side effect-prone conventional therapies.
With additional studies and eventual clinical trials, FGFR inhibitors may soon become a cornerstone in personalized care for children with gliomas. This could help ease long-term side effects and provide better quality of life for survivors navigating the challenges of pediatric cancer recovery.
As the search for new therapeutics targeting pediatric cancers continues through the work of companies like CNS Pharmaceuticals Inc. (NASDAQ: CNSP), the efforts being undertaken to find ways to repurpose existing drugs can help in quickly increasing the treatment options available to patients.
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
Please see full terms of use and disclaimers on the TinyGems website applicable to all content provided by TinyGems, wherever published or re-published: https://www.TinyGems.net/Disclaimer
A joint study between the Broad Institute and the Dana-Farber Cancer Institute has discovered a new and potentially effective way to treat pediatric gliomas. The research revealed that a percentage of children diagnosed with gliomas, an incredibly common type of pediatric brain cancer, may respond to certain inhibitors that have already been approved by the U.S. Food and Drug Administration.
According to the researchers, 8.9% of pediatric glioma patients exhibited fibroblast growth factor receptor alterations, a group of proteins that play a key role in cell differentiation, growth, and development. These alterations increased the gliomas’ sensitivity to inhibitors that can block the action of FGFR proteins.
Most importantly, since these inhibitors already have FDA approval, researchers can begin studying how they interact with pediatric gliomas at a relatively accelerated pace.
These findings could pave the way for the development of targeted therapies for gliomas in pediatric cancer patients, especially patients afflicted with pediatric low-grade gliomas (pLGG). Although FGFR-altered pediatric gliomas affect a significant portion of pediatric glioma patients, there are no clinical trials or FDA-approved treatments for them. Consequently, the recent findings could be critical to developing treatments for patients suffering from this particular kind of pediatric glioma.
Even so, further research will be necessary to improve the efficacy of the FDA-approved inhibitors against FGFR-altered pediatric gliomas before the therapy is deployed. Pratiti (Mimi) Bandopadhayay, MBBS, PhD, a Dana-Farber/Boston Children’s Cancer and Blood Disorders Center physician-scientist, Broad Institute member, and co-senior author, says the study was motivated by diagnoses of FGFR-altered pediatric gliomas around the world.
The research team wanted to see if there were already existing medications that could address this condition, Bandopadhayay says, and its results gave the team new insight into how to treat pediatric cancer patients with this specific type of glioma. According to Bandopadhayay, there is a major need for alternative treatments for FGFR-altered gliomas in pediatric patients, and the joint research effort led to the approval of tovorafenib as a therapy for BRAF-altered gliomas in children.
Co-headed by Keith Ligon, MD, PhD, a Broad Institute associate member and pathologist at Dana-Farber, the research involved carrying out genomic analyzes on over 10,000 gliomas, with alterations in the FGFR1 and FGFR2 genes being the most common in structural variants and point mutations. Co-first author April Apfelbaum says their results represented the first time preclinical data showed that FGFR-altered pediatric gliomas could respond to FGFR inhibitors.
The research adds to growing momentum in pediatric cancer studies that seek to repurpose existing drugs for faster clinical application. By narrowing in on genetic drivers like FGFR alterations, scientists can tailor treatments more precisely and reduce the need for side effect-prone conventional therapies.
With additional studies and eventual clinical trials, FGFR inhibitors may soon become a cornerstone in personalized care for children with gliomas. This could help ease long-term side effects and provide better quality of life for survivors navigating the challenges of pediatric cancer recovery.
As the search for new therapeutics targeting pediatric cancers continues through the work of companies like CNS Pharmaceuticals Inc. (NASDAQ: CNSP), the efforts being undertaken to find ways to repurpose existing drugs can help in quickly increasing the treatment options available to patients.
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
Please see full terms of use and disclaimers on the TinyGems website applicable to all content provided by TinyGems, wherever published or re-published: https://www.TinyGems.net/Disclaimer
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