Preclinical study shows RNA therapy halts glioblastoma growth
Transcode's treatment induces tumor cell death, prolongs survival in mice
Written by |
TTX-MC138, an RNA-based therapy from Transcode Therapeutics that’s in early clinical development, induced tumor cell death and prolonged survival in a mouse model of glioblastoma.
That’s according to the study, “Nanotherapy Targeting miR-10b Improves Survival in Orthotopic Glioblastoma Models,” which was published in the Journal of Functional Biomaterials. The work was funded in part by a grant from Henry Ford Health and Michigan State University Health Sciences.
“This research represents an important step forward in targeting one of the most treatment-resistant forms of cancer,” Zdravka Medarova, PhD, chief science officer of Transcode and co-author of the study, said in a company press release. “By pairing our differentiated delivery approach with robust biological support, we are broadening the potential reach of our RNA-based therapeutics beyond metastatic solid tumors.”
Glioblastoma is an aggressive form of glioma caused by the uncontrolled growth of cells in the brain. Previous studies have shown that an RNA molecule called miR-10b is a key driver of cancerous cell growth in various types of cancer, including glioblastoma.
To target RNA molecules, scientists often use antisense oligonucleotides (ASOs), short pieces of genetic material that bind to a specific RNA molecule and inhibit its function. But getting ASOs into cancer cells within a living human body is no easy feat.
Phase 2a trial planned
To create TTX-MC138, scientists targeted miR-10b with ASOs and packaged them into magnetic nanoparticles engineered to home in on tumor cells. A perk of this approach is that the particles, being magnetic, will appear on MRI scans, making it easy to monitor their movement in the body.
In this study, the researchers tested TTX-MC138 in mouse models of glioblastoma. Results showed the therapy homed to the brain tumors and decreased miR-10b levels as intended, leading to tumor cell death and prolonging survival.
“Our therapeutic studies demonstrated that… TTX-MC138 … improved survival in [glioblastoma] tumors, with appreciable inhibition of the target and induction of tumor cell [death],” the researchers concluded.
Transcode is sponsoring a Phase 1 clinical trial (NCT06260774) evaluating TTX-MC138 in individuals with solid tumors that have metastasized (spread to other parts of the body). The company said it plans to launch a Phase 2a trial of the therapy in the first half of 2026.
