(New York, NY – November 30, 2021) – The National Cancer Institute has awarded Mount Sinai researchers $3.15 million in grant funding to assess the potential of a multidisciplinary drug development platform to identify new biological targets for precision-based therapeutics for hepatocellular carcinoma (HCC). The platform includes precision mouse models, tumor 3D organoids, and a proprietary library of small molecule inhibitors.
HCC is the most common type of liver cancer among adults, accounting for approximately 80 percent of all cases and 800,000 deaths globally each year. It is a leading cause of cancer-related death worldwide, in particular among patients with cirrhosis.
Current HCC drugs approved by the U.S. Food and Drug Administration have limited efficacy, and patients are frequently diagnosed during advanced stages of cancer when they have developed cirrhosis, which further narrows therapeutic options. By combining an innovative drug design with a patient-derived platform developed by academic laboratories at Mount Sinai, the researchers hope to identify new leads for HCC drug development.
The co-Principal Investigators are Arvin Dar, PhD, Professor of Oncological Sciences, and Pharmacological Sciences; Ernesto Guccione, PhD, Professor of Oncological Sciences, and Pharmacological Sciences; and Amaia Lujambio, PhD, Associate Professor of Oncological Sciences, and Medicine (Liver Diseases), at The Tisch Cancer Institute at the Icahn School of Medicine at Mount Sinai. Through preliminary studies, they have identified a strong lead, WNTinib, a novel small molecule inhibitor that has the ability to reduce the growth of tumors.
“It is structurally similar to other clinical compounds, but what makes WNTinib unique is that we have optimized it against multiple targets that we want to inhibit in liver cancer while removing activity against anti-targets that we believe can make tumors more aggressive. Our goal now is to move forward with a clinical trial of WNTinib to better understand how it works, and to develop secondary compounds that would target other specific subtypes of liver cancer. This grant is critical in that it will support us in these efforts and potentially enable us to achieve a breakthrough in therapeutics for patients with liver cancer and a breakthrough in developing a new platform for exploring promising leads,” said Dr. Dar.
In addition to the study, Mount Sinai has launched the new Center of Excellence for Liver and Bile Duct Cancer to further deepen its commitment to quality care for liver and bile duct cancer patients. A leader in the study and treatment of liver and bile duct cancer, the Center sees approximately 400 new patients each year, making it the biggest center of its kind nationwide. It is part of The Tisch Cancer Institute at Mount Sinai, a National Cancer Institute-designated Cancer Center.
The Director of the Center is Myron Schwartz, MD, the Henry Kaufmann Professor of Surgery and Director of Liver Surgery in the Recanati-Miller Transplantation Institute. The Center is focused on bringing multi-disciplinary, cutting-edge, highly personalized care to patients with all forms of liver cancer. The Center brings together world-leading physicians to provide exceptional patient care that draws from their experience, innovation, clinical trials, research, and education.
“The Center of Excellence for Liver and Bile Duct Cancer offers Mount Sinai’s patients access to the most advanced diagnostic treatment approaches at a state-of-the-art facility,” Dr. Schwartz said. “Our multidisciplinary care teams include specialists such as hepatobiliary surgeons, medical oncologists, gastroenterologists, pathologists, and transplant surgeons, and our patients also have access to comprehensive support services, such as social workers, financial counselors, and clergy. Through this center, and our ongoing research, we will continue to make progress in the diagnosis and treatment of liver cancer among our patients.”
About the Mount Sinai Health System
The Mount Sinai Health System is New York City's largest academic medical system, encompassing eight hospitals, a leading medical school, and a vast network of ambulatory practices throughout the greater New York region. Mount Sinai advances medicine and health through unrivaled education and translational research and discovery to deliver care that is the safest, highest-quality, most accessible and equitable, and the best value of any health system in the nation. The Health System includes approximately 7,300 primary and specialty care physicians; 13 joint-venture ambulatory surgery centers; more than 415 ambulatory practices throughout the five boroughs of New York City, Westchester, Long Island, and Florida; and more than 30 affiliated community health centers. The Mount Sinai Hospital is ranked on U.S. News & World Report's "Honor Roll" of the top 20 U.S. hospitals and is top in the nation by specialty: No. 1 in Geriatrics and top 20 in Cardiology/Heart Surgery, Diabetes/Endocrinology, Gastroenterology/GI Surgery, Neurology/Neurosurgery, Orthopedics, Pulmonology/Lung Surgery, Rehabilitation, and Urology. New York Eye and Ear Infirmary of Mount Sinai is ranked No. 12 in Ophthalmology. Mount Sinai Kravis Children's Hospital is ranked in U.S. News & World Report’s “Best Children’s Hospitals” among the country’s best in four out of 10 pediatric specialties. The Icahn School of Medicine is one of three medical schools that have earned distinction by multiple indicators: ranked in the top 20 by U.S. News & World Report's "Best Medical Schools," aligned with a U.S. News & World Report "Honor Roll" Hospital, and No. 14 in the nation for National Institutes of Health funding. Newsweek’s “The World’s Best Smart Hospitals” ranks The Mount Sinai Hospital as No. 1 in New York and in the top five globally, and Mount Sinai Morningside in the top 20 globally.