St. Baldrick’s Foundation Research Outcomes blogs highlight examples of the progress your donations are supporting. In this edition, we’re sharing research that is helping uncover new treatment opportunities, improve care during cancer treatment, and address the long-term health needs of childhood cancer survivors.
Thank you for making these advances – and more – possible.
Finding a Common Weakness in Rhabdomyosarcoma
St. Baldrick’s Researcher Dr. Corinne Linardic is studying a rare and aggressive type of childhood cancer called fusion-positive rhabdomyosarcoma (FP-RMS). Researchers have known this cancer can be caused by several different gene fusions (genetic changes where two genes become joined together), but it wasn’t clear why these different changes all lead to the same disease.
The researchers found that, despite their differences, all of these gene fusions behave in much the same way. They switch on the same cancer-driving genes, interact with the same group of proteins, and use the same biological pathways to help the cancer grow.
Most importantly, the team identified a shared weakness across all forms of FP-RMS. The cancer cells rely heavily on an enzyme called thymidylate synthase to survive. When the researchers treated laboratory models of FP-RMS with pralatrexate (a drug that inhibits thymidylate synthase), tumor growth slowed significantly, regardless of which gene fusion was causing the cancer.
These findings suggest that children with fusion-positive rhabdomyosarcoma may not need different treatments based on the specific gene fusion driving their cancer. Instead, researchers have identified a common vulnerability shared across the major forms of the disease that could potentially be targeted with a single treatment approach. While the results are encouraging, the research is still in its early stages, and clinical trials are needed to determine whether pralatrexate is safe and effective for children with FP-RMS.
Helping Kids Report Treatment Side Effects
Children receiving cancer treatment often experience side effects that can be difficult to fully capture during clinic visits. Symptom reporting usually relies on doctors interpreting what children and caregivers describe, which can sometimes lead to incomplete or inaccurate records.
St. Baldrick’s Scholar, Dr. Stacey Crane and colleagues developed the Smart Pediatric Oncology Tracker of Symptoms (SPOTS), a child-friendly online tool that allows children with cancer and their caregivers to report symptoms directly. Children and families helped design the tool, ensuring it was easy to use and engaging. Participants described SPOTS as clear, easy to navigate, and appreciated features such as a customizable character and the opportunity for children to report their symptoms independently.
While the researchers identified areas for improvement, such as simplifying some terminology and navigation, the study showed that SPOTS has the potential to help children communicate their symptoms more accurately and completely. The next step is to test the tool in real-world clinical settings to determine whether it improves symptom reporting and helps healthcare teams better manage treatment side effects.
Understanding Ewing Sarcoma’s Surroundings
Researchers are working to better understand the environment surrounding Ewing sarcoma tumors, known as the tumor microenvironment. This network of cells, proteins, and other tissues can influence how cancers grow, spread, and respond to treatment.
By studying tumor samples from children with Ewing sarcoma, St. Baldrick’s Fellow Dr. Christopher Kuo and colleagues, found important differences between tumors that had already spread and those that had not. They discovered that tumors that remained localized contained more supportive tissue that appeared to help recruit immune cells to the tumor, suggesting it may play a protective role. The team also identified a signaling pathway, called MIF-CD74, that was active in all of the tumors they studied and may help the cancer hide from the immune system.
These findings provide new insight into how Ewing sarcoma interacts with its surrounding environment and identify MIF-CD74 as a promising target for future immunotherapy research. By better understanding how these tumors evade the immune system, researchers hope to develop more effective treatments for children with Ewing sarcoma.
Dr. Kuo’s Fellowship was supported by the Shohet Family Fund for Ewing Sarcoma Research, and the D-Feet Cancer Dalton Fox Foundation.
Helping Survivors Stay Heart Healthy
St. Baldrick’s Scholar, Dr. Hari Narayan and colleagues studied 112 childhood cancer survivors who had received anthracycline chemotherapy, a treatment that can increase the risk of heart problems later in life. Using activity trackers to measure physical activity and advanced heart imaging to evaluate heart structure and function, the team examined whether exercise was associated with better heart health after cancer treatment.
The researchers found that nearly two-thirds of survivors were not getting the recommended amount of moderate-to-vigorous physical activity each week. They also found that survivors who were more physically active had healthier heart structure, suggesting that regular exercise may help reduce some of the long-term effects of anthracycline treatment on the heart.
These findings highlight the importance of promoting physical activity as part of long-term survivorship care. While additional research is needed, the results suggest that regular exercise could play an important role in helping childhood cancer survivors maintain heart health after treatment.
Dr. Narayan’s Scholar award was supported by Team Abby Gives, a St. Baldrick’s Foundation Hero Fund.
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.
Donate now and help support research into better treatments for kids with cancer.
