St. Baldrick’s Childhood Cancer Research Foundation Research Outcomes blogs highlight examples of the progress your donations are supporting. In this edition, we’re sharing research aimed at uncovering new vulnerabilities in hard-to-treat cancers, protecting children from serious transplant complications, and refining treatment approaches to improve both survival and quality of life.
Thank you for helping move this progress forward.
Prebiotic Supplement May Help Protect Children During Stem Cell Transplants
Some children with cancer need a bone marrow (stem cell) transplant when their disease affects the blood and bone marrow, or when doctors need to use very intensive treatment to eliminate the cancer. However, the transplant process can disrupt the gut microbiome, increasing the risk of a serious complication called graft-versus-host disease (GVHD). In a study supported in part by St. Baldrick’s Foundation, researchers tested a naturally occurring sugar found in breast milk, called 2′-fucosyllactose (2′-FL), to see if it could help protect the gut during transplant. The supplement was safe, helped maintain healthy gut bacteria, and was associated with signs of improved intestinal healing. Notably, none of the children who received 2′-FL developed moderate-to-severe gastrointestinal GVHD, compared with 17% of similar patients who did not receive the supplement. These findings suggest that supporting the gut microbiome with 2′-FL may be a promising way to improve outcomes after pediatric stem cell transplantation.
Researchers Uncover a New Weak Spot in Aggressive Childhood Cancers
Researchers have identified a potential new weakness in some of the most aggressive childhood cancers, including certain forms of neuroblastoma, medulloblastoma, and Burkitt lymphoma. These cancers are often driven by a powerful cancer-promoting gene called MYC, which has historically been very difficult to target with drugs. In a study supported in part by St. Baldrick’s Foundation, scientists discovered a previously unknown protein, called Firefox (FFX), that helps keep MYC active and fuels tumor growth. When FFX was blocked in laboratory models, tumor growth slowed significantly. The findings suggest that targeting FFX could offer a new way to treat MYC-driven cancers and may eventually lead to more effective therapies for children with these hard-to-treat tumors.
Refining Risk Assessment in Wilms Tumor
Wilms tumor is the most common kidney cancer in children, and most children with the disease do very well with treatment. Researchers continue to look for better ways to identify which children have a higher risk of relapse so treatments can be tailored to each patient’s needs. In Europe, doctors have long used a tumor’s response to pre-surgery chemotherapy to help guide treatment decisions, but this approach has not traditionally been incorporated into Children’s Oncology Group (COG) treatment strategies in North America. In a study supported in part by St. Baldrick’s, researchers found that examining how much of the tumor remains alive after several weeks of chemotherapy provides important information about a patient’s outlook. Children whose tumors responded well to chemotherapy had excellent survival rates, while those with a higher amount of remaining active tumor faced a greater risk of relapse. These results support bringing this well-established international approach into future COG clinical trials, with the goal of improving risk assessment and helping tailor treatment more precisely for children with Wilms tumor.
Balancing Cure and Quality of Life in Rhabdomyosarcoma
Rhabdomyosarcoma is a rare childhood cancer that develops in muscle or soft tissues and is often treated with a combination of chemotherapy, surgery, and radiation therapy. While radiation can be an important part of treatment, it can also cause long-term side effects, especially in growing children. Researchers supported in part by St. Baldrick’s, presented findings showing that kids with intermediate-risk rhabdomyosarcoma whose tumors completely disappeared after chemotherapy appeared to do just as well with a lower dose of radiation as those who received the standard higher dose. The lower-dose group maintained excellent local tumor control and had favorable survival outcomes. Although these results will require further study, they support ongoing efforts to safely reduce treatment-related side effects while maintaining high cure rates and quality of life for kids with cancer.
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.
