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Ethan Lee M.D., Ph.D.

Researcher Photo

Funded: 07-01-2026 through 06-30-2028
Funding Type: Research Grant
Institution Location: Nashville, TN
Institution: Vanderbilt University Medical Center affiliated with Monroe Carell Jr. Children's Hospital at Vanderbilt

Pediatric craniopharyngioma (CP) is a rare brain tumor (5-10% of cases). CP is located near vital areas such as the eyes and the hormone control center, and recurs after surgery in 50% of cases. Standard treatments (surgery and radiation) lead to serious issues and reduced quality of life for most patients. No drugs are available for treating CP. Dr. Lee and colleagues will target the Wnt signaling pathway, a system that cells use to communicate and control when they grow or divide in CP tumors when Wnt signaling is overactive. Dr Lee and team will test promising drugs for CP: FDA-approved drug pyrvinium and a newer, unapproved drug SSTC3. These drugs block Wnt signaling and are effective in other cancers. Dr. Lee and team will use CP tumor cells from patients and CP models, and measure how the drugs block Wnt signaling in cells determining if the drugs shrink tumors. Dr. Lee and colleagues will pioneer targeted therapies and reduce the need for harsh CP treatments.