Chinese clinical trial shows breakthrough in liver cancer survival

A pioneering Chinese study has found that a new drug cocktail administered before surgery can nearly double the time patients with a deadly form of liver cancer live without the disease returning.

The study, published Thursday in the New England Journal of Medicine, focused on intrahepatic cholangiocarcinoma, or ICC. This cancer forms in the bile ducts inside the liver and is notorious for being "silent," often discovered only after it has reached an advanced stage.

Led by Zhongshan Hospital, affiliated with Fudan University, the trial involved 11 hospitals across China. Experts say the findings offer a new "standard of care" for a disease where the five-year survival rate after surgery is currently only 25 percent to 40 percent.

The researchers tested a neoadjuvant protocol, which is a treatment given as a first step to shrink a tumor before the main surgery. In this trial, 178 patients with a median age of 59 were recruited by February 2024. One group received three cycles of Gemox chemotherapy along with a targeted therapy drug and an immunotherapy drug, which helps the body's own immune system recognize and attack cancer cells. The other group underwent immediate surgery, which is the traditional standard practice.

The results showed that the drug combination significantly delayed the return of the cancer. The median event-free survival — the length of time after treatment that a patient remains free of specific complications or cancer recurrence — was 18 months for those who received the drug cocktail. In contrast, the group that went straight to surgery saw a median of only 8.7 months.

Shi Guoming, one of the lead researchers, noted that the tumors in the drug-treatment group significantly shrank, with an objective response rate of 55 percent. This term refers to the percentage of patients whose tumors partially or completely disappeared during the treatment phase.

China is disproportionately affected by this disease, seeing over 50,000 new cases annually, which accounts for more than half of the global total. The study, which began in 2021, also showed a promising trend in overall survival. The 24-month survival rate for the drug-protocol group was 79 percent, compared to 61 percent for the surgery-only group.

Zhou Jian, president of Zhongshan Hospital, said that as the study progresses, it may further demonstrate long-term benefits for patients. The team presented preliminary results at the annual meeting of the European Society for Medical Oncology in Germany in October 2024, where it garnered international attention. Shi expressed hope that the findings will be further validated through international studies so the Chinese protocol can eventually be used as a global cure.