Existing Drug May Unlock Immunotherapy Potential Against Rare Liver Cancer

Cornell University study finds FDA-approved drug could help activate immune system to fight fibrolamellar carcinoma.

Published on Feb. 20, 2026

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Researchers at Cornell University have discovered that an existing FDA-approved drug called AMD3100 may be able to unlock the potential of immunotherapy to treat fibrolamellar carcinoma, a rare and fatal form of liver cancer. The study found that fibrolamellar tumors can sequester T cells away from the cancer, preventing the immune system from fighting the disease. However, AMD3100 was able to mobilize T cells into the core of the tumor, making immunotherapy more effective.

Why it matters

Fibrolamellar carcinoma is a particularly challenging cancer, as it often goes undetected until it has already metastasized, leaving patients with a poor prognosis. Immunotherapy has not been effective against this type of cancer, but this new research suggests that using an existing drug to overcome the tumor's ability to exclude T cells could make immunotherapy a viable treatment option.

The details

The study, published in the journal Gastroenterology, found that fibrolamellar tumors are able to rewire their local microenvironments in a way that sequesters T cells away from the cancer cells, preventing the immune system from attacking the tumor. The researchers tested the drug AMD3100, which is currently used to treat a different disorder, and found that it was able to prevent the tumors from excluding T cells, allowing the immune cells to infiltrate the core of the cancer.

  • The study was published on February 17, 2026.

The players

Praveen Sethupathy

Professor of physiological genomics and the paper's co-senior author.

AMD3100

An FDA-approved drug that was found to be able to mobilize T cells into the core of fibrolamellar carcinoma tumors.

Fibrolamellar carcinoma

A rare and fatal form of liver cancer that primarily affects children and young adults, accounting for up to 2% of all liver cancers.

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What they’re saying

“Our results provide among the first indications of why a type of immunotherapy called immune checkpoint inhibition hasn't worked well in these patients, and even if this particular drug isn't the end-all-be-all, it teaches us that this T-cell exclusion phenomenon is an important one to tackle in fibrolamellar carcinoma.”

— Praveen Sethupathy, Professor of physiological genomics and the paper's co-senior author (Gastroenterology)

What’s next

Sethupathy and colleagues are currently searching for liver cancer clinicians who might be interested in starting clinical trials for the new treatment using AMD3100 in combination with immune checkpoint inhibitors.

The takeaway

This research represents an important step forward in understanding why immunotherapy has not been effective against fibrolamellar carcinoma, and suggests that using an existing drug to overcome the tumor's ability to exclude T cells could make immunotherapy a viable treatment option for this rare and deadly form of liver cancer.