Trial tests therapy that may protect transplanted livers from hepatitis C
Researchers at MassBiologics of the UMass Medical School have begun a Phase II clinical trial testing the ability of a human monoclonal antibody to prevent hepatitis C virus (HCV) infection of a donor liver in transplant patients. The first patients were enrolled in the study in December. The primary goal of this randomized, double-blind, placebo-controlled study is to test if the monoclonal antibody MassBiologics developed, designated MBL-HCV1, prevents re-infection of patients chronically infected with HCV who are undergoing liver transplantation. MassBiologics plans to enroll 16 patients in the first part of the study. “We are hopeful that positive results from this study will meet an important public health need, and we could not take this important step without the willing and thoughtful participation of these volunteers,” said Donna Ambrosino, MD, executive director of MassBiologics and professor of pediatrics at UMass Medical School. There are currently five hospitals participating in the trial—Massachusetts General Hospital and Beth Israel Deaconess Medical Center in Boston; Lahey Clinic in Burlington, Mass.; Yale-New Haven Hospital in Connecticut; and Mount Sinai Hospital in New York City—and others may join in the coming months. The first six patients enrolled have come from three of these sites. HCV damages the liver and is the leading indication for liver transplantation; it is diagnosed in about half of the 6,000 patients who receive liver transplants each year in the United States. According to the U.S. Centers for Disease Control and Prevention, 3.2 million Americans are chronically infected with HCV and approximately 10,000 die annually of the disease. Globally, as many as 170 million people are estimated to suffer from HCV infection.
What is a monoclonal antibody?
Antibodies are molecules made by the immune system to thwart potential disease-causing agents, such as viruses and bacteria. Antibodies are made in different configurations, designed by the immune system to attached to very specific targets and prevent those targets from causing illnesses. It’s similar to a lock and key process—an antibody made to fight a flu virus won’t attack the bacteria that cause strep throat, just like the key to a person’s car won’t open the door to his home. A “monoclonal antibody,” which is the class of medicine MassBiologics is developing to block hepatitis C, is a drug that has been created in large quantities from a single cell (mono-clone) that was engineered to produce a specific antibody aimed at a specific pathogen or cellular process. When the single cell that makes an effective antibody is developed, it is then grown under controlled conditions so that it divides, over and over again, yielding millions of identical cells, much like an unlimited number of duplicate keys can be made from an original master. All of those cells then produce the effective antibody, which is collected, purified and packaged as medicine to treat specific conditions.