Beat Childhood Cancer Research Consortium at Penn State College of Medicine and Aminex Therapeutics Announce National Clinical Trial for Pediatric Patients with High‑Risk Cancers
Combination therapy of DFMO and AMXT 1501 is being evaluated in a national pediatric clinical trial for high‑risk childhood cancers, including neuroblastoma, central nervous system tumors and sarcomas.
A newly launched national pediatric clinical trial will assess the safety, tolerability and preliminary efficacy of a combination of two oral agents, DFMO (a polyamine synthesis inhibitor) and AMXT 1501 (a polyamine uptake inhibitor), in children and young adults with high‑risk cancers such as neuroblastoma, diffuse intrinsic pontine glioma, atypical teratoid rhabdoid tumor, embryonal tumor with multilayer rosettes, Ewing sarcoma and osteosarcoma. The study, enrolling over 220 patients across up to 50 sites, follows encouraging signals from a phase I trial in adult patients. By targeting polyamine metabolism, the combination aims to reduce immunosuppressive cells in the tumor microenvironment and stimulate anti‑tumor immune responses. "We are excited to expand our clinical evaluation to children and are greatly encouraged by early signals of clinical efficacy demonstrated in our Phase I trial in adult cancer patients," said Dr. Giselle Sholler. The trial’s protocol is registered with ClinicalTrials.gov (NCT 06465199).
Pediatric patients with high‑risk neuro‑oncology and sarcoma subtypes have limited curative options. A dual‑drug regimen that harnesses polyamine metabolism and immune activation offers a novel therapeutic strategy that may improve survival and reduce relapse. By conducting a large, multicenter study, this regimen could rapidly transition from experimental to standard care if proven effective.
Bizonyítékszint: Klinikai vizsgálat. Formális klinikai vizsgálati eredmény.
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- Emerging clinical and research approaches in targeted therapies for high-risk neuroblastoma
- Frequency and Clinical Significance of Clonal and Subclonal Driver Mutations in High‑Risk Neuroblastoma at Diagnosis: A Children's Oncology Group Study