Existing drug could speed new treatment for rare liver cancer

The study, published in the journal Gastroenterology, focused on fibrolamellar carcinoma, a rare liver cancer that accounts for about 2% of all liver cancer cases and primarily affects children and young adults. There is currently no cure, and the disease is often diagnosed only after it has spread, limiting treatment options.

Scientists from Cornell University and the University of Washington found that the cancer protects itself by preventing immune T cells from reaching the tumor. Instead, the cancer redirects and traps these cells within nearby fibrous tissue, stopping them from attacking cancer cells. This process, known as T cell exclusion, may explain why immune checkpoint inhibitors, a common form of cancer immunotherapy, have shown limited success in treating the disease.

To better understand how this happens, researchers used single-nucleus transcriptomics, a technology that analyzes gene activity in individual cells. The technique revealed that altered liver stellate cells produce the fibrous bands that give fibrolamellar carcinoma its name and release signals that draw T cells away from the tumor.

The team then tested AMD3100, an FDA-approved drug, on slices of tumor tissue taken from patients. The drug blocked the signals that trap T cells, allowing them to move back into the tumors.

When AMD3100 was combined with immune checkpoint inhibitors, researchers observed stronger T cell activity and a significant increase in cancer cell death compared with immunotherapy alone.

The findings offer one of the clearest explanations so far for why immunotherapy has struggled against fibrolamellar carcinoma and suggest that targeting T cell exclusion could improve treatment outcomes.

Researchers caution that further studies are needed before the approach can become a standard treatment. However, they say the fact that AMD3100 is already FDA-approved could help accelerate clinical trials because its safety profile is already well understood.

The research team is now seeking liver cancer specialists to launch clinical trials to evaluate the combination therapy in patients with fibrolamellar carcinoma.

Earlier, Qazinform News Agency reported that Türkiye achieved world’s first 8-way cross liver transplant.