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Mayo Clinic Develops Milk-Based Nanoparticles to Target Aggressive Bile Duct Cancer
New gene-silencing therapy shows promise in preclinical studies for hard-to-treat cholangiocarcinoma.
Mar. 17, 2026 at 3:15am
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Researchers at Mayo Clinic have developed a novel approach using milk-derived nanoparticles loaded with gene-silencing therapy to directly target and treat cholangiocarcinoma, a rare and aggressive form of bile duct cancer. The targeted delivery system was able to decrease cancer growth and increase cancer cell death in preclinical studies, without harming nearby healthy tissues.
Why it matters
Cholangiocarcinoma is a difficult-to-treat cancer with limited treatment options, so new targeted therapies like this could significantly improve outcomes for patients. The milk-based nanoparticle delivery system also represents a potentially safer and more biocompatible way to administer genetic therapies compared to other methods.
The details
The research team used a gene-therapy strategy involving small interfering RNA (siRNA) to silence specific cancer-driving genes. They screened a library of DNA molecules to find an aptamer that could selectively bind to cholangiocarcinoma cells, then attached that homing device to milk-derived nanoparticles loaded with the siRNA. This enabled direct delivery of the genetic therapy into the cancer cells.
- The findings were published in March 2026 in the journal JHEP Reports.
The players
Rory Smoot, M.D.
Surgical oncologist at Mayo Clinic in Rochester and senior author of the study.
Tushar Patel, M.B., Ch.B.
Transplant hepatologist and researcher at Mayo Clinic in Florida, who previously developed the milk-derived nanoparticle platform.
Brandon Wilbanks, Ph.D.
Postdoctoral research fellow at Mayo Clinic and first author of the study.
What they’re saying
“One significant issue is the lack of medications that treat the specific alterations in these cancers. Our approach is designed to turn off specific cancer-driving genes while leaving healthy tissue alone.”
— Rory Smoot, M.D., Surgical oncologist
“We showed that this system could deliver gene-silencing therapy straight to the cancer. This led to decreases in cancer growth and an increase in cancer cell death, without harming nearby healthy tissues.”
— Brandon Wilbanks, Ph.D., Postdoctoral research fellow
“These advances bring real hope. They show that it may be possible to develop safer, more personalized treatments for patients who currently have very limited options.”
— Rory Smoot, M.D., Surgical oncologist
What’s next
Researchers are now working to optimize gene targets and test the approach across multiple forms of cholangiocarcinoma, with the long-term goal of developing patient-specific gene therapies delivered via this milk-derived platform to improve outcomes for patients.
The takeaway
This innovative use of milk-based nanoparticles to precisely deliver gene-silencing therapy directly to cholangiocarcinoma cells represents a promising new avenue for treating this rare and aggressive form of bile duct cancer, which currently has limited treatment options.
