Posted on 03/21/2025 10:14:47 PM PDT by ConservativeMind
High-grade glioma, an aggressive form of pediatric and adult brain cancer, is challenging to treat given the tumor location, incidence of recurrence and difficulty for drugs to cross the blood-brain barrier.
Researchers established a collaborative team to uncover a potential new avenue to address this disease.
The team's study shows that high-grade glioma tumor cells harboring DNA alterations in the gene PDGFRA responded to the drug avapritinib, which is already approved by the US FDA to treat gastrointestinal stromal tumors with a PDGFRA exon 18 mutation as well advanced systemic mastocytosis and indolent systemic mastocytosis.
"We were excited to see that avapritinib essentially shut off PDGFRA signaling in mouse brain tumors," said Carl Koschmann, M.D.
Aside from surgery and radiation, there aren't effective drugs to treat high-grade gliomas, especially upon recurrence. Koschmann and his collaborators targeted PDGFRA, which is one of the most commonly mutated genes, as a potential inroad to discover new drug therapies.
"We'd been doing screens with a lot of commercially available drugs that inhibit PDGFRA. We found avapritinib to be the strongest and most focused inhibitor that targets PDGFRA alterations," Koschmann said.
"When we gave mice the drug and showed that it reached the brain, we knew we were onto something," explained Kallen Schwark.
The team was able to treat some patients with high-grade glioma through an expanded access program established by Blueprint, while a clinical trial was not yet available.
"Across multiple international institutions, we treated the first eight patients with high-grade glioma with avapritinib," Koschmann explained. "The patients tolerated the drug well and in three of the eight patients, we were able to see their tumors shrink."
High-grade gliomas are very aggressive, with a prognosis of less than two years and limited treatment options.
(Excerpt) Read more at medicalxpress.com ...
It is available today.
Was just coming to post that. That stuff is getting hard to find here in North Texas lately. After 4 years in Scandinavia at various times, courtesy of Uncle Sam, I like to keep a bottle in the freezer. Thought maybe this article was going to lead to increased supplies, but....
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