In 2023 Dr. Eric Thompson of Chicago Medical Center and Washington University proposed an innovative study focused on two compounds and how they might better cross the blood-brain-barrier for Medulloblastoma. This project was approved for funding by The Cure Starts Now and here are the results:
Treating brain cancer in children remains one of the biggest challenges in modern medicine. One major roadblock? The brain itself. It has a powerful defense system known as the blood-brain barrier, which acts like a security gate—keeping harmful substances out, but also stopping many helpful medications from getting in.
But a new study is giving researchers hope that we might finally have a way to get past that gate.
The Problem: Promising Drugs That Can’t Reach the Brain
Two cancer-fighting drugs, asciminib and nilotinib, have shown promise in killing medulloblastoma cells in lab settings. Medulloblastoma is the most common malignant brain tumor in children, and current treatments haven’t changed much in decades.
The catch? These drugs are blocked by a protein called P-glycoprotein, which is part of the blood-brain barrier. It works like a bouncer, pumping out medications before they can reach their target.
The Breakthrough: Adding the drug, Tariquidar
Researchers wondered: what if we could turn off that blocker?
Enter tariquidar—a compound that blocks the action of P-glycoprotein. In this study, scientists tested what would happen if they combined tariquidar with asciminib or nilotinib.
The results were exciting.
- More drug in the brain: Tariquidar helped both drugs reach the brain in significantly higher amounts without increasing dosing.
- Longer-lasting effects: The drugs stayed in the body and the brain much longer when combined with tariquidar.
- Stronger cancer-killing power: Lab-grown medulloblastoma cells were significantly more likely to die when treated with the combination than with the drugs alone.
Why This Matters
This could represent a major step forward in pediatric brain cancer treatment. Using tariquidar to enhance the delivery of existing cancer drugs could enhance their effectiveness, without the need to invent entirely new therapies.
What’s Next?
The study’s authors call for further research, including clinical trials, to explore how these combinations perform in human patients. If successful, it could open up new treatment avenues not just for medulloblastoma, but potentially for other brain cancers as well.
You can help make that future possible. Donate today to support groundbreaking research like this and bring us one step closer to a cure for childhood brain cancer.
This study was supported by The Cure Starts Now, a nonprofit dedicated to finding a cure for pediatric brain cancer by funding innovative, high-impact research.
