New drug may boost survival for children with rare bone cancer
Children with a rare bone cancer may have better survival chances when treated with a drug that cuts off the blood supply to tumors, researchers report.
The medication, pazopanib (Votrient), appeared to extend the lives of a small group of children with Ewing sarcoma, a cancer that forms tumors in the bones, according to findings published in Frontiers in Oncology.
“While we wait for new treatment options, it is possible to implement this existing drug to improve outcomes in very high-risk patients,” said Anna Raciborska, head of oncology at Poland’s Warsaw Mother and Child Institute. “It opens the door to targeted therapies earlier in the disease course, potentially improving survival and quality of life.”
Typically, fewer than 25% of children with advanced Ewing sarcoma survive five years after diagnosis. Researchers explored pazopanib, originally developed for kidney cancer, as a potential way to slow the disease.
“Pazopanib is a pill that blocks the tumor’s ability to grow new blood vessels, which tumors need to survive and spread,” Raciborska said. “By cutting off this ‘blood supply,’ the drug presumably makes tumors weaker and more sensitive to chemotherapy and radiation, which may slow the disease and enhance the effectiveness of existing treatments.”
From 2016 to 2024, 11 children aged 5 to 18 received pazopanib alongside standard therapies, including chemotherapy, surgery, radiation, or stem cell transplants. Results showed that roughly 86% of the children survived two years after diagnosis, while about 68% experienced no progression in their cancer.
Currently, 10 of the children are still alive, with six continuing to take pazopanib. One patient’s cancer progressed, and two experienced a relapse. The drug also caused minimal side effects.
Researchers stressed that larger studies are needed to confirm the findings, though the rarity of advanced Ewing sarcoma makes enrolling patients in clinical trials challenging.
“While the results are encouraging, larger controlled trials are needed before changing standard practice,” Raciborska said. “Our study could serve as a basis for creating prospective, multicenter trials to confirm these promising results, but it will require significant work and resources.”
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