NanOlogy Advances Novel Intratumoral Cisplatin Formulation for Fatal Pediatric Brain Cancer DIPG
Key Insights
NanOlogy (search) announced a drug development program for diffuse intrinsic pontine glioma (search) (DIPG (search)), a lethal pediatric brainstem tumor (search) affecting 150-300 children annually in the US with median survival under one year.
The company is developing Large Surface Area Microparticle (LSAM) Cisplatin for direct intratumoral delivery, designed to overcome cisplatin's poor blood-brain barrier penetration and systemic toxicity issues.
NanOlogy (search) is completing FDA-required IND enabling studies and plans to initiate clinical trials in late 2026 to evaluate safety and efficacy of stereotactic intratumoral LSAM-Cisplatin (search) administration.
NanOlogy (search), LLC, a private clinical-stage oncology company based in Fort Worth, Texas, has unveiled a drug development program targeting diffuse intrinsic pontine glioma (search) (DIPG (search)), one of the most aggressive and lethal pediatric brain cancers. The company is developing Large Surface Area Microparticle (LSAM) Cisplatin for stereotactic intratumoral administration, representing a novel approach to treating this devastating brainstem tumor (search).
Addressing an Urgent Medical Need
DIPG (search) presents one of pediatric oncology's most challenging scenarios. According to DIPG.org (search), the disease afflicts 150-300 children annually in the United States, with a median survival of less than one year. Current treatments, primarily radiation therapy, may delay disease progression but almost always fail to deliver long-term survival.
"We believe NanOlogy (search)'s innovative LSAM-Cisplatin (search) investigational drug designed for intratumoral administration can overcome the limitations of current treatment options with highly targeted delivery of drug into the tumor, continuous drug release, and minimal systemic toxicity," said H. Paul Dorman, founder and chairman of NanOlogy.
Overcoming Cisplatin's Limitations
Laboratory research studies have demonstrated cisplatin's ability to kill DIPG (search) tumors by binding to DNA (search) and disrupting cell replication, leading to cell death. However, systemically administered cisplatin formulations face significant challenges in treating brain tumors. The drug does not cross the blood-brain barrier effectively, limiting its efficacy in brain tumors, while systemic administration is associated with numerous severe side effects throughout the body.
NanOlogy (search)'s LSAM-Cisplatin (search) formulation is specifically designed to address these limitations through direct intratumoral delivery. The approach leverages advancements in minimally invasive surgical procedures and imaging that now allow local delivery of LSAMs to solid tumors (search) virtually anywhere in the body, including the brain.
Clinical Development Timeline
NanOlogy (search) is currently completing Investigational New Drug (IND) enabling studies required by the U.S. Food and Drug Administration (search) (FDA). Upon completion of these studies, the company plans to submit an IND application for LSAM-Cisplatin (search) to treat malignant neoplasms of the brain (search), including DIPG (search).
Subject to FDA allowance to proceed, NanOlogy (search) aims to initiate a clinical trial in late 2026 to evaluate the safety and response of stereotactic intratumoral administration of LSAM-Cisplatin (search) in children diagnosed with DIPG (search). The company is also assessing future opportunities to extend the use of LSAM-Cisplatin to treat other brain and solid tumors (search).
Technology Platform and Broader Applications
NanOlogy (search)'s approach is enabled by CritiTech Particle Engineering Solutions (search)' Purcision™ technology platform, which produces Large Surface Area Microparticles (LSAMs) of pure drug for direct local delivery. This technology aims to overcome issues of systemic toxicity and tumor bioavailability that plague conventional cancer treatments.
The company's clinical programs have advanced LSAM investigational drugs in multiple solid tumors (search) including pancreas, lung, bladder, peritoneal, ovarian, prostate, and dermal cancers. The feasibility of the Purcision™ platform has been established across various drug classes, including taxanes (search), platins (search), tyrosine kinase inhibitors, and poly (ADP-ribose) polymerase inhibitors (search).
NanOlogy (search)'s investigational drugs are protected by composition of matter patents issued in the United States and other major jurisdictions worldwide, including Canada, Europe, Japan, China, South Korea, Australia, and India. These patents form the foundation of an extensive intellectual property portfolio comprising over 100 issued patents protecting the company's investigational drugs, formulations, methods, and technology.