FDA-approved drug shows promise against rare liver cancer

A study has identified how an existing medication could improve immunotherapy outcomes for fibrolamellar carcinoma. The rare liver cancer currently has no cure and often spreads before detection. Researchers demonstrated the approach using patient tumor samples.

Fibrolamellar carcinoma accounts for about 2 percent of liver cancer cases and mainly strikes children and young adults. Tumors create fibrous bands that trap immune T cells away from cancer cells, blocking the effects of checkpoint inhibitors.

Scientists at Cornell University and the University of Washington used single-nucleus transcriptomics to map these interactions. They found that stellate cells altered by the cancer send signals directing T cells into the fibrous tissue.

Treatment with AMD3100, an FDA-approved drug, redirected T cells into the tumors. When combined with immune checkpoint inhibition, the approach increased tumor cell death in the samples.

Praveen Sethupathy, co-senior author, said the results highlight T-cell exclusion as a key barrier in this cancer. The team is now recruiting specialists to begin clinical trials. The work was funded by the Fibrolamellar Cancer Foundation.

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