Ewing sarcoma is one of the most common types of bone cancer found in children and teenagers.
When the cancer spreads to different parts of the body—a condition known as multi-metastatic Ewing sarcoma—it becomes much harder to treat.
In the past, fewer than one in four children with this advanced form of the disease survived five years after diagnosis. But a new study has brought hope.
Researchers have discovered that a drug called pazopanib might help children with this aggressive cancer live longer.
In a small group of 11 young patients, 85% were still alive two years after diagnosis, and nearly 70% saw no further growth or return of their cancer during that time.
The study was led by Professor Anna Raciborska of the Warsaw Mother and Child Institute and published in the journal Frontiers in Oncology.
Pazopanib is not a new drug. It was first made to treat kidney cancer in adults. Later, doctors found it also had some success in treating adults with Ewing sarcoma.
This led Professor Raciborska and her team to wonder if it could help children too—especially when used early and in combination with standard treatments like chemotherapy, surgery, and radiation.
Pazopanib works by stopping tumors from growing new blood vessels. Like any living thing, tumors need a blood supply to grow and spread. By cutting off this supply, the drug can make tumors weaker and more sensitive to other treatments. This might slow the disease and give standard cancer treatments a better chance to work.
Between 2016 and 2024, 11 children between the ages of 5 and 17 were treated with pazopanib as part of their regular cancer care at the Warsaw Mother and Child Institute. They received the drug during and after chemotherapy.
Treatment was paused for surgeries or stopped if serious side effects occurred. Most of the children also received radiation therapy, and some had stem cell transplants. On average, they took pazopanib for about 1.7 years.
The results were encouraging. Ten of the 11 children responded well to treatment. Only one child’s cancer progressed during the study, and one sadly passed away. Two patients experienced a return of their cancer, but overall, most patients remained stable. Importantly, pazopanib was well tolerated.
The side effects were mild and manageable, which is especially important for young patients who are already dealing with strong treatments.
The two-year survival rate for the children in this study was 85.7%, and 68.2% remained free of new cancer events two years after starting treatment. These numbers are better than those seen in past studies on adult patients, suggesting that pazopanib may work better in children or when used earlier in the course of the disease.
However, the researchers caution that their study was small. More research is needed, especially in larger groups of patients, to confirm these promising results.
Because multi-metastatic Ewing sarcoma is rare, it can be difficult to run large clinical trials. But the team hopes that future studies—especially those supported by European research programs—can explore pazopanib’s potential even further.
Professor Raciborska believes this drug could be added to current treatment plans to help children with the most serious cases of Ewing sarcoma. It may not be a cure on its own, but it could give children more time and a better chance at recovery—without adding harsh side effects.
“While the results are encouraging, larger controlled trials are needed before changing standard practice,” she said. “We hope future studies will confirm what we’ve seen here and give more children a fighting chance.”
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The study is published in Frontiers in Oncology.
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