St. Baldrick’s Foundation Research Outcomes blogs highlight examples of the progress your donations are supporting. This quarterly edition focuses on research impacting kids with brain tumors, Ewing sarcoma, and Hodgkin lymphoma.
Thank you for making these advances – and more – possible.
Targeting Every Part of the Tumor in DMG
One of the biggest challenges in treating cancers like diffuse midline glioma (DMG) is that not all tumor cells are the same. Different groups of cells within the same tumor can respond differently to treatment, allowing some to survive and the cancer to come back. In this study, supported in part by St. Baldrick’s, researchers established and validated a new way to map these different cell types and identify the key proteins driving each one. By doing this, they were able to match existing drugs to target each group of cells more effectively.
When tested, combining drugs that target these different tumor cell types worked much better than using a single drug alone and significantly extended survival in preclinical models. Some combinations, including drugs already approved for other uses, showed especially promising results. This research offers a new strategy for designing smarter combination treatments for DMG and potentially other hard-to-treat cancers, bringing hope for more effective therapies in the future.
A Smarter Way to Target Ewing Sarcoma
Dr. Poul Sorensen and Dr. Haifeng Zhang have uncovered a promising new way to target cancers driven by genetic changes that create abnormal proteins and are often difficult to treat directly. Specifically, they looked at treatment for Ewing sarcoma. Instead of going after those hard-to-target proteins, the team found that many of these cancers share a common marker on the cell surface called IL1RAP. By using a type of targeted therapy known as an antibody–drug conjugate (ADC), they were able to deliver cancer-fighting drugs straight to the tumor cells while largely sparing healthy ones.
This approach showed encouraging results across several cancer types, including Ewing sarcoma and certain lymphomas, and worked in models of both early and advanced disease. Because IL1RAP is not commonly found on normal cells, this may offer a safer, more precise way to treat a range of tough cancers. These findings support further clinical testing and point to a potential new strategy that could benefit patients with fusion-driven cancers in the future.
This work was supported by the Better Ewing Sarcoma Treatment (BEST) Grant, funded by the family of Martha Riedel, D-Feet Cancer The Dalton Fox Foundation, The Faris Foundation, and The Shohet Family Fund for Ewing Sarcoma Research, a St. Baldrick’s Hero Fund.
Reducing the Risk of Second Cancers in Hodgkin Lymphoma
Researchers looked at the long-term risk of developing second cancers in children treated for Hodgkin lymphoma using today’s more modern radiation approaches. In the past, higher radiation doses and less precise techniques led to increased risks of cancers later in life. This study found that newer methods, especially more targeted forms like proton therapy, are linked to lower risks of developing secondary cancers such as breast, lung, and thyroid cancer.
While the risk isn’t completely gone and can vary from patient to patient, these findings are encouraging. They suggest that advances in radiation therapy are helping make treatment safer for survivors, while still effectively treating the original cancer. The results may also help doctors make more informed decisions about treatment planning and long-term follow-up care, including how best to monitor survivors for any future health risks.
This work was supported through a grant to the Children’s Oncology Group.
Improving Outcomes in a Rare and Aggressive Brain Tumor
Researchers, supported in part by St. Baldrick’s, studied children and young adults with relapsed or treatment-resistant central nervous system nongerminomatous germ cell tumors (NGGCTs), a rare brain tumor with very poor outcomes. Because there is no standard treatment for these patients, the team analyzed data from multiple international centers to better understand which approaches might improve survival after relapse.
They found that more intensive treatments were associated with better outcomes. Patients who received high-dose chemotherapy followed by stem cell rescue had a 3-year overall survival rate of 76%, compared with only 5.3% in those who did not receive this approach. Similarly, those who received repeat radiation had a 3-year survival of 33%, compared with 17% in patients who did not. While the study is limited by its very small size and other factors, these results suggest that select intensive therapies may offer meaningful benefit for some patients. The authors highlight the need for larger, collaborative studies to better identify which children are most likely to benefit and to guide future treatment strategies.
Not every publication of research supported by St. Baldrick’s makes the news, but each one adds to the body of scientific knowledge that takes us one step closer to better outcomes for kids with cancer. Your continued support will make more research possible to Conquer Kids’ Cancer.
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