Rochester, Minn, Mar 23: Researchers at Mayo Clinic have unveiled a promising new approach to treating Cholangiocarcinoma, a rare and aggressive cancer with limited treatment options. The study, published in JHEP Reports, highlights an innovative targeted therapy using milk-derived nanoparticles to deliver gene-silencing treatment directly to cancer cells.
The research introduces a novel method that combines precision targeting with genetic therapy. By leveraging small interfering RNA (siRNA), scientists aim to selectively switch off cancer-driving genes while minimizing damage to healthy tissue.
“One significant challenge in treating these cancers is the lack of therapies targeting specific genetic alterations,” said Dr. Rory Smoot, surgical oncologist at Mayo Clinic and senior author of the study. “Our approach is designed to silence those genes in cancer cells while sparing normal cells.”
To achieve this, the multidisciplinary team screened an extensive library of DNA molecules using a technique known as Cell-SELEX. This led to the discovery of a specialized DNA strand, or aptamer, capable of selectively binding to cholangiocarcinoma cells, acting as a molecular “homing device.
The aptamer was then attached to biocompatible, milk-derived nanoparticles developed by Dr. Tushar Patel. These tiny fat-based particles were loaded with siRNA and engineered to deliver the gene therapy precisely to tumor cells.
“We demonstrated that this system can deliver gene-silencing therapy directly into cancer cells,” said Dr. Brandon Wilbanks, first author of the study. “This resulted in reduced tumor growth and increased cancer cell death, without harming surrounding healthy tissue.”
While the findings are currently preclinical, the technology has been patented by Mayo Clinic. Researchers are now focused on refining gene targets and evaluating the approach across multiple forms of cholangiocarcinoma, with the long-term goal of developing personalized gene therapies for patients.
“These advances offer real hope,” added Dr. Smoot. “They suggest a future where safer, more targeted treatments can be developed for patients with very limited options.”
The research was supported by multiple Mayo Clinic programs and collaborating institutions, reinforcing ongoing efforts to advance precision medicine and innovative cancer therapies.
