Breakthrough in Cancer Treatment: Combined Gene Silencing Shows Promise in Early Studies
CHAPEL HILL, NC – Researchers at the University of North Carolina (UNC) Lineberger Complete Cancer Center have announced a significant advancement in cancer therapy, demonstrating the triumphant simultaneous silencing of the KRAS and MYC genes in preclinical studies.This dual-gene targeting approach, utilizing novel RNA technology, offers a potential new strategy for treating cancers resistant to conventional therapies.
KRAS mutations are implicated in approximately 25% of all cancer cases, with a notably high prevalence in lung, colorectal, and pancreatic cancers. The MYC gene, a key regulator of cell growth and proliferation, is involved in the progress of 50-70% of all cancers. Despite its critical role, MYC has historically been considered “undruggable” due to the challenges in developing therapies that specifically target it without causing significant side effects.
“Together disabling KRAS and MYC is akin to severing both Achilles’ heels of the cancer cell,” explained Dr. Medhat V. Pecot, co-leader of the research team and a member of the UNC RNA Discovery Center.”We’ve not only shown that combined gene silencing is achievable, but also paved the way for a broader approach where multiple genetic targets can be addressed concurrently.”
The new RNA technology builds upon previous work by Dr. Pecot’s team, published in cancer Cell in 2025, which focused on a targeted RNA therapy for the specific KRAS G12V mutation. This latest innovation expands the therapy’s applicability by effectively silencing a wider range of KRAS variants. The initial research, funded in part by a $2.5 million grant from the National Cancer institute (NCI), focused on patient-derived xenografts of pancreatic ductal adenocarcinoma.
Precision and efficiency in Targeting Multiple genetic Drivers
The technology’s strength lies in its precision and efficiency. By simultaneously targeting multiple genetic drivers, it creates a powerful therapeutic tool for tumors that have developed resistance to standard treatments like chemotherapy and radiation. Researchers are now investigating the feasibility of extending the platform to target three or more genetic drivers within a single molecule. Early studies, detailed on the UNC lineberger website, showed a 70% reduction in tumor volume in mice treated with the combined therapy compared to control groups.
The development aligns with the UNC RNA Discovery Center’s mission to create innovative RNA therapies for challenging diseases. “Our findings demonstrate that combining targeted gene silencing with targeted delivery to tumors represents a promising path toward precision medicine,” stated Dr. Pecot. “This technology could offer genuine hope to patients with cancer types previously considered untreatable.” The team is currently working towards initiating Phase 1 clinical trials at UNC Hospitals in late 2026, focusing initially on patients with KRAS-mutated pancreatic cancer.
