A new combination pill
could provide hope for hepatitis C patients who can't take or don't respond to
currently available treatments, researchers say.
The pill combines two
investigational drugs, sofosbuvir and ledipasvir, and in clinical trials it
eliminated the hepatitis C virus (HCV) in nearly all patients who took it,
according to findings published online in The Lancet.
of patients new to hepatitis C virus therapy who took eight weeks of the
sofosbuvir/ledipasvir combination tablet were HCV undetectable 24 weeks after
therapy ended, which means they are cured of HCV," said lead author Dr
Eric Lawitz, clinical professor of medicine at the University of Texas Health
Science Centre at San Antonio. "Similarly, among patients who had received
prior therapy and took 12 weeks of sofosbuvir/ledipasvir, 95% were
Hepatitis C, if left
untreated, can cause severe and potentially fatal liver damage. However, most
people recently infected with hepatitis C do not have symptoms. Instead, the
virus causes long-term damage, mainly scarring of the liver, a condition called
Complicated drug regimen
Combination drug treatments
currently are available for the most prevalent form of the virus, which affects
about 75% of Americans with the disease, Lawitz said. But these
combination treatments involve interferon and protease inhibitors, which can
have terrible side effects. The therapy also requires a complicated drug
regimen of pills and injections.
Fewer than half of
hepatitis C patients can undergo the existing combination therapy, given the
drugs' interactions and side effects, the authors said in background
information. For those patients, there are no treatment options at present.
"We've had nothing to
offer them," said Dr David Bernstein, chief of the division of hepatology
at North Shore University Hospital in Manhasset, NY "It's painted a
rather bleak picture for these patients."
Sofosbuvir and ledipasvir
are targeted, direct-acting agents that interfere directly with the life cycle
of the hepatitis C virus, Lawitz said.
Researchers combined the
two drugs into a single tablet taken once a day. They recruited 100 hepatitis C
patients to try the medication, including 60 who had never received treatment
and 40 who had been unsuccessfully treated using the current therapy. Some
patients also received ribavirin, currently a standard treatment, in addition
to the combination pill.
Just over half of the
previously treated patients had cirrhosis.
Sustained virological response
By 12 weeks, nearly all of
the patients had achieved what doctors call a sustained virological response,
in which the virus is eliminated and prevented from replicating – in essence,
a functional cure.
"These types of
advances are game-changers," Bernstein said. "We're going to be
curing [very high percentages of people] with simple oral agents, one pill once
a day with mild to no side effects."
About half of the patients
had at least one health problem during the study, with the highest rates seen
among patients who took ribavirin. The most common side effects were nausea, anaemia,
upper respiratory tract infection and headache. The treating physician rated
most of them as mild, and no one had to discontinue treatment because of side
The treatment of hepatitis
C is rapidly changing, Lawitz said. "We are moving from an era of
injectable medications with significant toxicity to an era of all-oral
combination pill therapy that provides the promise of being well-tolerated with
very high rates of cure," he said.
The combination drug is
still being evaluated in phase 3 clinical studies, so it is too soon to tell if
it will receive US Food and Drug Administration approval or how it would be
priced, Lawitz said.
Because hepatitis C progresses
without symptoms, the US Centres for Disease Control and Prevention recommends
that adults born from 1945 through 1965 get tested for the virus.
For more information on
hepatitis C, visit the U.S.
National Library of Medicine.
Picture: Hepatitis C from Shutterstock