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 neuroblastoma, AML, rhabdomyosarcoma, as well as research into survival disparities for kids with ALL.
Thank you for making these advances – and more – possible.
Engineering T Cells to Fight Neuroblastoma
Researchers have previously found that a protein called GPC2 is highly expressed on the surface of neuroblastoma cells, making it a potential therapeutic target. In a recent study, supported in part by the St. Baldrick’s Foundation, researchers developed an antibody that binds only to GPC2 on tumor cells, sparing healthy cells. They tested combining this antibody with CAR T-cell immunotherapy to assess safety and effectiveness. In preclinical models, this treatment caused significant tumor regression with no obvious toxic side effects. Based on these results, a first-in-human Phase 1 clinical trial is now underway for children with relapsed or refractory neuroblastoma (NCT05650749).
Dexrazoxane’s Impact In Pediatric AML
Dexrazoxane is a cardioprotective medication given to patients receiving chemotherapy to reduce the risk of heart damage. While its heart protective effects have been known for years, a recent study, conducted in part by two St. Baldrick’s Scholars, Dr. Daniel Zheng and Dr. Hari Narayan, is the first to show its impact on a national scale in children with acute myeloid leukemia (AML). The researchers analyzed data from children across the U.S. and found that dexrazoxane use rose from less than 1% in 2010 to nearly universal (98%) in 2021, and serious cardiac complications dropped significantly, with no evidence that the drug compromises treatment. This study confirms that, in a large population of children, dexrazoxane clearly provides more benefit than risk in treating pediatric AML.
Dr. Naryan’s Scholar award was supported by Team Abby Gives, a St. Baldrick’s Foundation Hero Fund.
Using Global Data To Improve Rhabdomyosarcoma Outcomes
A new study from the INternational Soft Tissue saRcoma ConsorTium (INSTRuCT) examined outcomes for children with metastatic rhabdomyosarcoma (RMS) who had cancer spread to only one site at diagnosis. Researchers found that these children had significantly better survival than those with multiple metastatic sites, with nearly half living five years after diagnosis. Outcomes were best for patients whose cancer had spread only to the lungs or lymph nodes, while those with bone or bone marrow involvement had the poorest survival. The study also revealed that certain genetic changes were linked to worse outcomes. These findings suggest that not all metastatic RMS is the same and that children with limited, single-site disease may benefit from more personalized treatment approaches.
This research was conducted through the INSTRuCT Consortium and the Pediatric Cancer Data Commons (PCDC), both supported in part by St. Baldrick’s Foundation.
Exploring Racial, Ethnic, and Socioeconomic Factors in B-ALL Outcomes
A new study, supported in part by the St. Baldrick’s Foundation, explored why children from Hispanic and Black backgrounds with B-cell acute lymphoblastic leukemia (B-ALL) have long been known to experience lower overall survival than white children. The researchers hypothesized that differences in survival after relapse might help explain these disparities and analyzed more than 2,000 patients treated on Children’s Oncology Group trials. They found that much of the survival gap was related to disease characteristics at the time of relapse, such as how quickly the cancer returned, but Hispanic children continued to experience poorer outcomes even after accounting for these factors. Children from lower-income areas also had lower survival overall, and Hispanic and Black patients were more likely to live in these neighborhoods. The researchers conclude that reducing disparities will require both improving frontline treatment to prevent relapse and ensuring equitable access to promising new relapse therapies, such as immunotherapy.
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.
