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 medulloblastoma, AML, and LCH as well as research into survivorship and late effects.
Thank you for making these advances possible.
Stopping Medulloblastoma Spread
Medulloblastoma, a type of brain cancer, often spreads to the lining of the brain and spinal cord. Scientists don’t fully understand how this spread happens. Researchers on the St. Baldrick’s Foundation EPICC Team recently discovered that special cells in the brain’s lining—called fibroblasts—are drawn to the cancer by signals from the tumor. Once there, the tumor changes these fibroblasts into a new form that helps the cancer grow and spread. When researchers blocked one of these tumor signals (called PDGF) in models, the results were promising. These findings suggest that future strategies aimed at disrupting this pathway could lead to new targeted therapies.
A New Tool for AML Researchers
Researchers have created a detailed map of how normal blood cells develop, and used it to better understand how this process goes wrong in acute myeloid leukemia (AML), a type of blood cancer. By comparing healthy blood cells to leukemia cells, they found that AML can take on many different forms—even within the same person—and that these differences aren’t always caused by genetic mutations alone. This discovery helps explain why AML can be so unpredictable and hard to treat. The study provides a toolkit for other researchers to rapidly map and classify their own blood samples, which could lead to more personalized and effective treatments in the future. Dr. Charles Mullighan contributed to this study and his work was supported by a St. Baldrick’s Foundation Robert J. Arceci Innovation Award.
Helping Families Navigate the Process: Early Information about Late Effects
Many parents and childhood cancer survivors don’t feel that they got enough information about late effects to be ready for life after cancer treatment. To better understand their needs, St. Baldrick’s Scholar Dr. Katie Greenzang and colleagues interviewed childhood cancer patients, survivors, and parents. They found that while the topic can be emotionally tough, most families want to hear about these risks early so they can prepare. Those still in treatment often felt overwhelmed and preferred to get information gradually. In contrast, survivors and their parents often wished they had learned more from the start. Overall, families support having clear, personalized information early on, but their preferences for how and when to get it vary. This suggests more tailored communication is needed to help families feel informed and supported during this challenging time.
Genetic Testing to Identify Highest Risk LCH Patients
Langerhans cell histiocytosis (LCH) is a rare cancer in children that affects certain immune cells. Historically, doctors have staged patients according to whether certain “risk organs” like the liver or bone marrow were involved. Today, thanks to better treatment and supportive care, most kids with LCH survive. However, children who have widespread disease don’t respond well to current standard treatment, and treatment failure can lead to long-term problems, including a type of brain damage called LCH-associated neurodegeneration.
To find better ways to predict who’s at highest risk for treatment failure, St. Baldrick’s funded researchers looked at over 500 LCH patients. They found that children who have a specific genetic change, called BRAFV600E, present at the time of diagnosis are more likely to not respond to treatment and are at a high risk of developing LCH-associated neurodegeneration. Because of this finding, researchers suggest that doctors should use genetic testing in patients with LCH to predict which kids need more aggressive or targeted treatment early on.
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.
