Sometimes, the biggest medical breakthroughs come from looking at familiar medicines in a completely new way. Instead of developing a drug from scratch, researchers are increasingly discovering that existing treatments may have untapped potential against other diseases.
That may now be the case for a rare and aggressive form of liver cancer, reports Science Daily.
According to researchers from Cornell University and the University of Washington, an FDA-approved drug could help immunotherapy work more effectively against fibrolamellar carcinoma. The findings were published in the journal Gastroenterology.
Why the treatment fails
Fibrolamellar carcinoma accounts for only a small percentage of liver cancer cases, but it primarily affects children and young adults. Because the disease is often diagnosed after it has spread, treatment options are limited and patient outcomes remain poor.
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The researchers found that the cancer creates a protective environment that prevents immune T cells from reaching the tumour. Instead of attacking cancer cells, the immune cells become trapped in the tumour's surrounding fibrous tissue, limiting the effectiveness of immunotherapy.
Using advanced single-nucleus transcriptomics, the team mapped the tumour environment in unprecedented detail, allowing them to identify how this process occurs at the cellular level.
An old drug, a new purpose
The scientists then tested AMD3100, a drug that has already been approved by the U.S. Food and Drug Administration for another medical condition.
In laboratory experiments using patient tumour samples, the drug enabled immune T cells to move back into the tumour rather than remaining trapped in surrounding tissue. When AMD3100 was combined with immune checkpoint inhibitors, tumour cell death increased significantly compared with immunotherapy alone.
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Next step: clinical trials
Although the research has so far been limited to laboratory studies, the findings suggest a promising new strategy for treating fibrolamellar carcinoma.
One advantage is that AMD3100 is already FDA-approved, meaning researchers may be able to move more quickly into clinical testing than would be possible with an entirely new drug.
The research team is now seeking collaborators to launch clinical trials and determine whether the combination treatment can improve outcomes for patients living with this rare form of liver cancer.
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