Researchers at Mount Sinai have achieved a significant breakthrough in treating hepatocellular carcinoma (HCC), the most common type of liver cancer.
Led by Dr. Josep M. Llovet, Professor of Medicine at the Icahn School of Medicine at Mount Sinai, the team tested a combination of treatments that could help patients live longer without their cancer worsening.
The findings, published in The Lancet, could change how this challenging cancer is treated.
Liver cancer is one of the deadliest cancers worldwide. In 2023, it was the fourth leading cause of cancer-related deaths globally, with over 800,000 deaths each year.
HCC accounts for about 90% of primary liver cancers. It often develops in people with chronic liver conditions such as viral hepatitis, heavy alcohol use, or a fatty liver disease linked to metabolic problems.
For more than 20 years, the standard treatment for intermediate-stage HCC has been transarterial chemoembolization (TACE), a localized therapy that blocks the blood supply to liver tumors while delivering chemotherapy directly to them.
However, while TACE slows tumor growth, its benefits are limited. Many patients eventually experience disease progression, highlighting the need for more effective treatments.
The new study, called “LEAP-012,” tested a combination of TACE with two systemic therapies: lenvatinib, a targeted therapy that blocks tumor growth signals, and pembrolizumab, an immunotherapy drug that helps the immune system fight cancer.
The trial focused on patients with unresectable, non-metastatic HCC, meaning their cancer could not be removed by surgery but had not yet spread to other parts of the body.
The results showed that combining TACE with lenvatinib and pembrolizumab significantly improved progression-free survival—the time patients lived without their cancer worsening—compared to TACE alone.
This represents a critical advance in treating intermediate-stage HCC. Although a final analysis of overall survival (how long patients live in total) is still underway, early results suggest a promising trend toward longer survival.
Dr. Llovet described the study as a “landmark trial” and emphasized its importance for both patients and clinicians. “For over 20 years, TACE has been the standard treatment for intermediate-stage HCC, but its efficacy has been limited,” he explained.
“Our study is the first to show a statistically significant improvement in progression-free survival when combining TACE with systemic therapies, paving the way for a potential new standard of care.”
This combination therapy is particularly significant for the 25% of liver cancer patients classified as intermediate stage.
For clinicians, it offers a new strategy that integrates immunotherapy and targeted therapies with existing local treatments. For patients, it brings hope for better disease control and potentially longer lives compared to the current standard treatments.
The researchers are planning further studies to confirm the overall survival benefits of this combination and to explore its effectiveness in treating other stages of liver cancer.
These future trials could expand the use of this approach to more patients and provide additional insights into how best to manage this challenging disease.
In summary, the Mount Sinai team’s findings represent a major step forward in the fight against liver cancer.
By combining established local therapies with modern systemic treatments, this new approach offers hope for patients with intermediate-stage HCC, providing better disease control and potentially longer survival.
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The research findings can be found in The Lancet.
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