FDA approves new drug to treat hepatocellular carcinoma, the most common form of liver cancer
UCLA research played a key role in the development of regorafenib
The U.S. Food & Drug Administration today approved a new drug to treat people with hepatocellular carcinoma, the most common form of liver cancer. The drug, regorafenib, is the first and only therapy approved by the FDA in over a decade to show significant improvement in survival for patients with the disease after standard treatments have failed.
The research that led to the approval was conducted at UCLA and 21 other sites in the U.S., Europe and Asia. UCLA was the leader of the U.S. arm of the international collaboration, and the clinical studies were led by Dr. Richard Finn, an associate professor of medicine at the David Geffen School of Medicine and Director of the Signal Transduction and Therapeutics Program at UCLA’s Jonsson Comprehensive Cancer Center.
"When liver cancer progresses to advanced stages, a cure is no longer possible," Finn said. "Traditional treatments can help slow and control the disease for a short period of time, but eventually tumor growth will continue. Today’s approval of regorafenib is a major clinical advancement for patients when initial therapies are no longer working."
Hepatocellular carcinoma (HCC) accounts for three quarters of liver cancers in the U.S. and is the second leading cause of cancer-related deaths worldwide. An estimated 40,000 Americans will be diagnosed with liver cancer in 2017, and more than 28,000 will die from the disease.
In the early and intermediate stages of the disease, doctors can treat patients only in the affected area of the liver with surgery, transplantation or traditional drug treatments such as chemotherapy. However, as the cancer progresses and localized treatments do not work, some people may be candidates for systemic therapies, Finn said
Ten years ago, the drug sorafenib was approved by the FDA as the first systemic therapy for people with advanced HCC. Today’s approval of regorafenib provides a second option—and encouraging new hope—for people whose liver cancer has progressed on prior traditional and systemic treatments.
In the two-year study, the drug was tested in 567 people with HCC in a phase III clinical trial (named RESORCE). Finn and colleagues divided the patients randomly into two groups, one receiving regorafenib (374 recipients) and the other receiving placebo (193 recipients).
Developed by Bayer, Regorafenib (brand name Stivarga) was shown to extend overall survival by 10.6 months compared to 7.8 months for those receiving placebo. This represents an average improvement in survival of 3.1 months in those taking regorafenib, and a 37 percent reduction in risk of death. Patients receiving the drug did experience side effects such as hypertension and fatigue, but they were generally manageable
While fewer Americans are developing and dying from many types of cancer, liver cancer rates have more than tripled since 1980 according to the American Cancer Society. The risk factors for the disease include heavy alcohol use and obesity, as well as common liver diseases like hepatitis B or C, and cirrhosis. The prevalence of these conditions and a general lack of awareness of risk factors may be contributing to the rise in liver cancer rates, Finn said.
Because of our diverse population, Los Angeles County has one of the largest liver cancer populations in the United States. The Pfleger Liver Cancer Center at UCLA has a multi-disciplinary team of specialists committed to offering patients the best available treatments and access to practice-changing clinical trials such as RESORCE.
Further researching is ongoing to explore combinations of regorafenib with other therapies, and help develop new treatment approaches for people with liver cancer and who may not benefit from the sequence of regorafenib and sorafenib.