Research And Grants

Thyroid Hormone Therapy in Combination with Chemotherapy for Pediatric Medulloblastoma

Medulloblastoma is the most common malignant brain tumor in children. While initial treatments are often effective, options for cure after relapse remain limited. Our work has shown that medulloblastoma cells are highly sensitive to T3, the active form of thyroid hormone. in both cell lines and mouse models across medulloblastoma subtypes, T3 alone and in combination with chemotherapy significantly reduced tumor size and prolonged survival with minimal side effects. Building on this promising preclinical work, we propose a clinical trial using T3 therapy in patients with relapsed or treatment-refractory medulloblastoma. In addition, we will use cell-free DNA (cf-DNA) techniques to identify patients with microscopic disease not detectable by MRI, using cerebrospinal fluid (CSF) liquid biopsy. These patients will also receive T3 therapy. Our primary aims are to evaluate the safety of T3 in combination with chemotherapy and to assess its therapeutic effect. We will also examine T3’s potential to clear minimal residual disease in cf-DNA–positive patients. T3 is FDA-approved with pediatric formulations available, enabling immediate clinical use. This trial will be conducted through PNOC to facilitate access to many patients. If successful, this study could establish T3 as the first hormonal therapy for pediatric brain tumors.