DALLAS — Lantern Pharma and its subsidiary Starlight Therapeutics said the U.S. Food and Drug Administration has cleared an investigational new drug application for STAR-001, allowing the companies to move forward with a planned Phase 1 clinical trial in children with relapsed or refractory brain cancers.
The early-stage trial will evaluate STAR-001 both as a standalone treatment and in combination with spironolactone in pediatric patients with central nervous system malignancies, including atypical teratoid/rhabdoid tumor, diffuse intrinsic pontine glioma, glioblastoma, medulloblastoma, and ependymoma.
The study will be conducted across multiple academic centers in collaboration with the Pediatric Oncology Experimental Therapeutics Investigators’ Consortium, a network of leading children’s cancer institutions across the United States, Canada, and Israel.
Pediatric brain tumors remain one of the most difficult cancers to treat, with limited options for patients whose disease has returned or failed to respond to standard therapies. In the United States alone, nearly 5,000 new pediatric brain tumor cases are expected in 2026, and the disease remains the leading cause of cancer-related death among children and adolescents.
“This IND clearance is a defining milestone for Starlight Therapeutics and a meaningful step forward for pediatric neuro-oncology. For children with relapsed or refractory CNS tumors, the options are desperately limited — and the science behind this planned trial was built to change that,” said Panna Sharma, CEO & President, Lantern Pharma Inc. and Founder & Chairman, Starlight Therapeutics.
STAR-001 is a precision oncology drug designed to selectively activate within tumor cells and induce DNA damage. Researchers believe combining it with spironolactone — a drug that disrupts a key DNA repair mechanism — could enhance its effectiveness by preventing cancer cells from repairing that damage.
Preclinical studies showed promising results, including a 181% increase in median survival in ATRT models when the combination therapy was used compared with control.
“High-grade pediatric brain tumors represent a significant unmet medical need, with few effective options for children whose disease has relapsed or become refractory to standard therapy,” said Marc Chamberlain, M.D., Chief Medical Officer of Starlight Therapeutics. “We look forward to initiating this planned trial and to the possibility of delivering a new therapeutic option to children who need it most.”
The trial is expected to enroll between 18 and 42 patients between the ages of 1 and 17, pending additional funding and site activation.
