Michelle Longworth, MD

Pursuing Safer, More Effective Combination Therapies for Childhood Neuroblastoma

Neuroblastoma is a childhood cancer that usually appears before age 5. It is the most common solid tumor found outside the brain in children and is responsible for about 1 in 10 pediatric cancer deaths. Even with very intensive treatment, the five‑year survival rate for children with high‑risk neuroblastoma is still under 40 percent, and the harsh treatments often cause serious long‑term health problems. This makes it critical to find therapies that are both more effective and less toxic.

Dr. Longworth and her collaborator, Yogen Saunthararajah, MD, are focusing on testing new combinations of drugs and protein‑targeting treatments to see whether they can force neuroblastoma cells to mature into more normal cells or die, without harming healthy cells. Her team is working to identify the best combination of three therapies that can reliably kill or change neuroblastoma cells in the lab.

Once the most promising combination is identified, Dr. Longworth will move on to preclinical testing to see whether it works just as well in more advanced research systems that better reflect how neuroblastoma behaves in the body. This work aims to build the foundation for future treatments that are both safer and more effective for children with this challenging cancer.