Dr. William Lee uses a model of a liver to explain to patient Patrizia Cazzaniga the damage that hepatitis C can do without treatment. Mrs. Cazzaniga is now virus-free after taking part in a clinical trial at UT Southwestern testing new hepatitis C drugs.
Patrizia Cazzaniga had heard the horror stories about early treatments for hepatitis C – multiple daily pills and weekly shots for up to a year, side effects that could be debilitating, and a cure rate of only about 40 percent. But after a shorter and less intensive treatment with experimental drugs at UT Southwestern Medical Center that ended in October, Cazzaniga is now virus-free three months past treatment. She’s thrilled.
“If you don’t get treatment, you can get cancer or cirrhosis. That scared me. Now I feel great, my energy has come back, and I don’t have trouble with my stomach anymore,” says Cazzaniga, 57, who took part in one of 10 current clinical trials testing new hepatitis drugs at UT Southwestern.
“These new drugs are much more potent and effective,” says Dr. William M. Lee, professor of internal medicine at UT Southwestern and local site investigator for these ongoing national and international drug trials.
In the U.S., the Centers for Disease Control and Prevention (CDC) estimates that 4.1 million people carry the hepatitis C virus, with 3.2 million of them chronically infected. Further, CDC data show that about 15,000 Americans infected with the virus die annually from liver disease. About 75 percent of those infected do not know they have hepatitis C, since symptoms may not occur until later stages of the disease.
For years, the standard treatment was six ribavirin capsules daily and weekly peginterferon shots. The treatment period was long – nearly a year – and side effects could include body aches, fatigue, headaches, anxiety, or depression.
In 2011, the first new drugs were approved, boceprevir and telaprevir, which are protease inhibitors that interfere with the virus’ ability to replicate, but treatment with these drugs involved taking even more daily pills – 8 to 12 – and more side effects. In December 2013, two next-generation medications were approved: another protease inhibitor called simeprevir and the polymerase inhibitor sofosbuvir, which keeps the virus from multiplying within the liver. At least 10 other hepatitis drugs are in various phases of trial, including the drug combination that Cazzaniga took.
“It looks like by the end of this year we will be down to one pill a day and no shots, with a total treatment period of just 12 weeks. It’s going to change treatment dramatically,” says Lee. “It’s stunning how much more effective these newer drugs are. In some cases, success rates are close to 100 percent.”
In early results, all UT Southwestern participants in the trial that included Cazzaniga have tested virus-free at four weeks. Lee says while 24 weeks virus-free after treatment has been the gold standard to be considered “cured,” tests show that finding no detectable virus at 12 weeks after treatment is just as reliable a standard with the new drugs.
“These newer drugs are more potent; they are biochemically more powerful, and therefore require much lower daily dosing, which leads to fewer side effects,” he says. “We’re not going to completely eradicate hepatitis C, but we will have medicines available that have the potential to cure many who have the disease.”
Participation in the clinical trial at UT Southwestern was a lifesaver for Cazzaniga, who lacked medical insurance and otherwise would have returned to her native Italy for hepatitis treatment.
“I’m glad they are finding new, better ways to take care of people with hepatitis,” she says. “People need to get treated. A lot of times people think that hepatitis will just go away, but it doesn’t.”
Risk factors for the virus, which is transmitted mainly by blood, include intravenous drug use, blood transfusions, or getting a body piercing or tattoo. In Mrs. Cazzaniga’s case, she suspects a blood transfusion she received during childbirth 31 years ago infected her. Screening for the virus in blood did not start until 1992, which is, in part, why the CDC recommends Baby Boomers born between 1945 and 1965 get tested, regardless of any knowledge of risk factors.
Slight fatigue, loss of appetite, and a minor skin rash were the only side effects Cazzaniga reported during her treatment, which involved taking 12 pills daily. Trial participants took three drugs for four or six months: FDA-approved ribavirin, a new protease inhibitor called faldaprevir, and a new polymerase inhibitor called deleobuvir. The two new drugs are made by Boehringer Ingelheim, the sponsor of this nearly three-year drug trial that is taking place in 15 countries with 490 hepatitis C patients.
“Before treatment, I was getting close to developing cirrhosis. With the virus now gone, cirrhosis stops. I am very happy and felt very comfortable coming to UT Southwestern,” she says.
Source: UT Southwestern Medical Center