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 diffuse midline glioma, rhabdoid tumor of the kidney, and retinoblastoma.
Thank you for making these advances – and more – possible.
Turning a Natural Compound into Hope for Kids with Brain Tumors
Preliminary studies suggest that derivatives of a natural fungal compound called verticillin A may hold promise against diffuse midline glioma (DMG), a rare and aggressive childhood brain tumor. Although verticillin A has long been recognized for its potential anticancer properties, researchers at MIT have only recently succeeded in synthesizing it in the lab for the first time, opening the door to more extensive study and modification.
Using these newly synthesized compounds, St. Baldrick’s funded researcher Dr. Jun Qi at Dana-Farber Cancer Institute tested verticillin derivatives against DMG cell lines, with encouraging early results. Dr. Qi and colleagues are now working to further validate how these compounds work at the molecular level and hope to begin testing them in models of pediatric brain cancers.
Focused Ultrasound Passes First Test in Treatment of Brain Cancer in Children
A key challenge in treating brain tumors is the blood-brain barrier, which protects the brain by blocking most chemotherapies from reaching tumor tissue at effective concentrations. Columbia University researchers have, for the first time, shown that focused ultrasound – a non-invasive technique using sound waves to temporarily open the blood-brain barrier and enhance drug delivery to the brain – can be safely applied in children undergoing treatment for brain cancer. Excitingly for pediatric patients, focused ultrasound does not require sedation and is entirely painless.
In a recent study involving St. Baldrick’s funded Dr. Cheng-Chia Wu, the technique was tested in combination with chemotherapy in three children with diffuse midline glioma (DMG), a rare and uniformly fatal pediatric brain cancer. In all three participants, focused ultrasound successfully opened the blood-brain barrier, allowing chemotherapy to penetrate the tumor, and some short-term improvements in mobility were observed – although all three children ultimately succumbed to their disease or related complications.
Establishing the safety and feasibility of focused ultrasound in kids paves the way for additional clinical trials, with the goal of improving survival outcomes for children with brain cancer.
A Promising New Lead for Treating Rhabdoid Tumor of the Kidney
New research is offering fresh hope for rhabdoid tumor of the kidney (RTK), a rare and very aggressive childhood cancer that desperately needs better treatments. St. Baldrick’s funded researcher Dr. Noriko Satake and colleagues zeroed in on a protein called CDK11, which plays a key role in how cells divide and make proteins. They found that CDK11 levels were especially high in RTK tumors, and even higher in patients with worse outcomes. When the team tested a drug that blocks CDK11 (called OTS964), they saw encouraging results: it slowed tumor growth in lab-grown cancer cells and improved survival, all with minimal side effects. These findings point to CDK11 as a promising new target that could one day lead to better treatments for children with RTK.
A New Liquid Biopsy Tool for Childhood Eye Cancer
A promising new liquid biopsy tool called LBSeq4Kids could change how doctors diagnose and monitor eye cancers in children. Instead of relying only on traditional tissue biopsies, this tool looks for tiny fragments of tumor DNA in fluids like the liquid inside the eye, spinal fluid, or blood.
In a study supported in part by St. Baldrick’s that included 60 patients with eye tumors (most with retinoblastoma), LBSeq4Kids was remarkably accurate, detecting cancer DNA in nearly all children with active disease and in every child who experienced a recurrence. Just as importantly, it did not flag false positives in children whose eyes were in remission or in those with non-cancerous conditions that can mimic retinoblastoma.
These findings suggest that LBSeq4Kids could become a powerful, less invasive way to help guide diagnosis, treatment decisions, and long-term monitoring for children with eye 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.
Donate now and help support research into better treatments for kids with cancer
