Researchers at Mount Sinai have made a breakthrough in treating a type of liver cancer called hepatocellular carcinoma (HCC). This research, led by Josep M. Llovet, MD, PhD, Professor of Medicine (Liver Diseases) at the Icahn School of Medicine at Mount Sinai, tested a combination of treatments that could help patients live longer without their cancer getting worse. Their phase 3 study, titled "LEAP-012: Transarterial chemoembolisation combined with lenvatinib plus pembrolizumab versus dual placebo for unresectable, non-metastatic hepatocellular carcinoma," was published today in The Lancet.
Liver cancer is a serious and deadly cancer. In 2023, liver cancer was the fourth most common cause of death globally , accounting for more than 800,000 deaths annually. Hepatocellular carcinoma represents approximately 90 percent of primary liver cancers. This cancer is most commonly found in people living with chronic viral hepatitis, heavy alcohol use, or metabolic dysfunction-associated steatotic liver disease.
This landmark trial demonstrates that the novel combination of lenvatinib, pembrolizumab, and transarterial chemoembolization (TACE) significantly improves progression-free survival compared to TACE alone, addressing a critical unmet need in the treatment of intermediate-stage HCC.
"For over 20 years, TACE has been the standard treatment for intermediate-stage HCC, but its efficacy has been limited," said Dr. Llovet, who is Director of Mount Sinai's Liver Cancer Program and a member of The Tisch Cancer Institute, Mount Sinai's National Cancer Institute-designated clinical cancer center. "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 extended the time patients lived without disease progression compared to TACE alone. Additionally, a trend towards improved overall survival was observed, with longer follow-up planned to confirm these results.
"This approach represents a significant step forward in treating the 25 percent of liver cancer patients classified in the intermediate stage," said Dr. Llovet. "For clinicians, this study introduces a groundbreaking approach to managing intermediate-stage HCC by integrating immunotherapy and targeted therapies with established local treatments. For patients, it offers hope for improved disease control and potentially extended survival compared to current standards."
Future research will focus on evaluating overall survival and exploring the combination's efficacy in other liver cancer stages and patient populations.
The LEAP-012 study was conducted across leading institutions in North America, Europe, and Asia, including Kindai University (Japan), Zhongshan Hospital (China), the University of Barcelona (Spain), and Yale School of Medicine (USA). The study was funded by Merck & Co. and Eisai, emphasizing the global effort to advance liver cancer treatments.
About the Icahn School of Medicine at Mount Sinai
The Icahn School of Medicine at Mount Sinai is internationally renowned for its outstanding research, educational, and clinical care programs. It is the sole academic partner for the eight- member hospitals* of the Mount Sinai Health System, one of the largest academic health systems in the United States, providing care to New York City's large and diverse patient population.
The Icahn School of Medicine at Mount Sinai offers highly competitive MD, PhD, MD-PhD, and master's degree programs, with enrollment of more than 1,200 students. It has the largest graduate medical education program in the country, with more than 2,600 clinical residents and fellows training throughout the Health System. Its Graduate School of Biomedical Sciences offers 13 degree-granting programs, conducts innovative basic and translational research, and trains more than 500 postdoctoral research fellows.
Ranked 11th nationwide in National Institutes of Health (NIH) funding, the Icahn School of Medicine at Mount Sinai is among the 99th percentile in research dollars per investigator according to the Association of American Medical Colleges. More than 4,500 scientists, educators, and clinicians work within and across dozens of academic departments and multidisciplinary institutes with an emphasis on translational research and therapeutics. Through Mount Sinai Innovation Partners (MSIP), the Health System facilitates the real-world application and commercialization of medical breakthroughs made at Mount Sinai.
