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 high-risk neuroblastoma, AML, and advanced sarcoma, as well as research into the quality of life for kids going through treatment.
Thank you for making these advances possible.
Targeting the Tumor Environment in High-Risk Neuroblastoma
Neuroblastoma is known for being complex and difficult to treat, especially in high-risk cases. About half of high-risk neuroblastoma patients have an extra copy of a gene called MYCN, which helps the cancer grow and avoid being destroyed by the body’s immune system. To better understand these cases, St. Baldrick’s supported researcher Dr. William Weiss and colleagues created a new lab model of high-risk MYCN-amplified neuroblastoma that provides insight into the tumor environment, specifically by including immune suppressing cells called macrophages.
Next, the researchers tested immunotherapy drugs in this new model. They found that one drug, anti–PD-L1, slowed tumor growth and changed the tumor environment in a good way: it removed the “bad” macrophages and let T-cells (the immune system’s attack cells) get in and do their job.
This research opens up new possibilities for treating high-risk neuroblastoma by focusing not just on the tumor itself, but on the immune cells that help the tumor grow and hide. Targeting these immune cells with the right combination of immunotherapies could make these hard-to-treat cancers more vulnerable to the immune system—and ultimately improve outcomes for children facing this aggressive disease.
Mapping AML to Save More Lives
Acute Myeloid Leukemia (AML) is a fast-moving type of cancer with poor outcomes. One reason it’s so hard to treat is because the cells that cause it change over time. Scientists know that these changes to the way DNA is “marked” (something called DNA methylation) play a big role in how AML develops. However, doctors haven’t had good tools to use this information when diagnosing or treating patients.
To help fix that, researchers, supported in part by an American Cancer Society St. Baldrick’s Foundation award, studied over 3,000 samples from patients with leukemia. They created something called the Acute Leukemia Methylome Atlas — a detailed map of these DNA changes. This map can help identify different types of leukemia more accurately and predict how likely a patient is to survive five years after diagnosis. The team also built a new test that can quickly read a patient’s DNA and these special chemical marks. This test is also faster and more affordable than older methods.
This research brings us closer to a future where leukemia can be diagnosed more quickly, treated more precisely, and better understood.
CAR‑T Breakthroughs Targeting Advanced Sarcomas
Advanced sarcomas have limited therapeutic options and poor outcomes in children. Thanks in part to an exciting study exploring a new CAR-T therapy that targets a protein called endoglin (ENG/CD105), researchers are now one step closer to improving treatment options.
This protein is found not only on sarcoma cells, but also on the supportive environment that helps tumors grow and spread. By designing CAR-T cells to recognize ENG, scientists supported in part by the St. Baldrick’s Foundation EPICC Team, saw strong cancer-fighting activity in the lab. The new ENG CAR-T cells were also able to break apart tightly packed cancer cell clusters and get past barriers that usually protect tumors from immune attack. This has been a big hurdle in treating sarcoma.
In lab models, this approach helped control tumor growth, reduce spread, and extend survival—offering a promising path forward for patients with few options today.
Supporting Kids through Leukemia Treatment
Children being treated for a common type of leukemia (standard-risk B-ALL) often face physical and emotional challenges during and after treatment. A recent COG study, supported in part by St. Baldrick’s, followed 420 young patients to see if giving one part of their treatment—vincristine and dexamethasone (VCR/DEX)—less often (every 12 weeks instead of every 4) would improve their quality of life. Notably, reducing how often VCR/DEX is given does not impact survival—children who got it every 12 weeks had equally strong outcomes as those who received it every 4 weeks.
In this study, parents completed surveys over several years to track their children’s well-being and school support needs. The study found that many children experienced physical or emotional difficulties regardless of how often they received VCR/DEX, and those who struggled early were more likely to continue struggling.
These findings show that while lighter treatment can ease the burden without compromising survival, kids with cancer still need early and ongoing support for their physical, emotional, and educational needs.
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.
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