FDA clears Lantern Pharma’s STAR-001 Phase 1 trial in pediatric CNS tumors, evaluating a novel precision therapy alone and with spironolactone.
Written By: Samiksha Jadhav, Bpharm
Reviewed By: Pharmacally Editorial Team
Lantern Pharma and its CNS-focused subsidiary Starlight Therapeutics have received clearance from the U.S. Food and Drug Administration (FDA) for an Investigational New Drug (IND) application to initiate a Phase 1 clinical trial of STAR-001 (LP-184) in pediatric patients with relapsed or refractory central nervous system (CNS) malignancies.
The open-label, dose-escalation study will assess STAR-001 as both a monotherapy and in combination with spironolactone. The trial will enroll patients aged 1 to 17 years with aggressive CNS tumors, including atypical teratoid/rhabdoid tumor (ATRT), diffuse intrinsic pontine glioma (DIPG), glioblastoma, medulloblastoma, and ependymoma.
The study will be conducted in collaboration with the Pediatric Oncology Experimental Therapeutics Investigators’ Consortium (POETIC), a multinational network of leading pediatric cancer centers across the United States, Canada, and Israel. Approximately 18 to 42 patients are expected to be enrolled across multiple sites.
Panna Sharma, CEO & President of Lantern Pharma and Founder & Chairman of Starlight Therapeutics, stated that the IND clearance represents a key milestone for both organizations and for pediatric neuro-oncology, highlighting the urgent need for new treatment options in relapsed CNS tumors. He noted that STAR-001 and its combination with spironolactone were developed using the RADR® AI platform to target ERCC3-mediated DNA repair, a key survival pathway in high-grade tumors. Preclinical findings demonstrated a 181% improvement in median survival in ATRT models, supporting a novel, mechanism-driven therapeutic strategy.
This approach addresses a significant unmet need. Pediatric CNS tumors remain one of the most challenging areas in oncology, with limited treatment options and poor survival outcomes, particularly in relapsed or refractory settings. Despite advances in molecular profiling and surgical techniques, outcomes for high-grade tumors have improved only marginally over the past decades.
STAR-001 is a precision oncology agent identified using Lantern’s proprietary RADR® AI platform. It is designed to selectively target tumor cells with high expression of PTGR1, where it is bioactivated into a DNA-crosslinking agent that induces lethal DNA damage.
A key feature of the study is the evaluation of STAR-001 in combination with spironolactone, which degrades ERCC3, a critical enzyme involved in DNA repair. By inhibiting this pathway, spironolactone enhances the accumulation of DNA damage induced by STAR-001, creating a synthetic lethality approach. Preclinical studies have shown significant improvements in survival and tumor cell death with the combination compared to monotherapy.
STAR-001 has received multiple orphan and rare pediatric disease designations from the FDA, underscoring its potential to address significant unmet needs in pediatric neuro-oncology. The Phase 1 study will primarily evaluate safety, tolerability, and preliminary antitumor activity, with the aim of advancing a novel therapeutic option for children with limited treatment alternatives.
References
Lantern Pharma and Starlight Therapeutics Announce FDA Clearance of IND for a Planned Phase 1 Pediatric CNS Cancer Trial of STAR-001, 27 March 2026, Lantern Pharma Inc – Lantern Pharma and Starlight Therapeutics Announce FDA Clearance of IND for a Planned Phase 1 Pediatric CNS Cancer Trial of STAR-001
About the Writer
Samiksha Vikram Jadhav is a B.Pharm graduate with a strong academic foundation in pharmaceutical sciences, pharmacology, and drug development. She has a keen interest in healthcare advancements, clinical research, medical writing, and emerging therapies. Her work focuses on presenting developments in the pharmaceutical and healthcare sectors through clear and accurate scientific communication.