New medicines are being developed that are expected to transform
the care of patients with hepatitis
C, making treatment far more effective and far less grueling.
The new drugs, which could start reaching the market as early
as next year, could help subdue a virus that infects roughly
four million Americans, most of them baby boomers, and 170 million
"I almost think this will be revolutionary, to be honest,"
said Dr. Fred Poordad, chief of hepatology at Cedars-Sinai Medical
Center in Los Angeles. "We are chomping at the bit to try
to treat as many patients as we can."
About two dozen pharmaceutical companies are now pursuing drugs
for hepatitis C, which an executive at Vertex Pharmaceuticals
recently called "one of the largest pharmaceutical opportunities
That is because the toll of the disease, which now kills about
12,000 Americans a year, is expected to rise in the coming decade.
Although new cases have dropped sharply, hundreds of thousands
of people who were infected decades ago are expected to start
experiencing the effects of liver damage.
New cases of liver cancer are already rising year by year. And
hepatitis C is the leading cause of liver transplants, like
the one recently received by the rock musician Gregg Allman.
Hopes for new treatments were buoyed in May by the first
results from a late-stage clinical trial of one of the new
from Vertex. When added to the existing treatment - a combination
of alpha interferon and ribavirin - telaprevir effectively cured
75 percent of patients, compared with 44 percent of those treated
with the existing drugs alone. And for many patients, the course
of treatment could be halved to 24 weeks.
Dr. Poordad, who is a consultant to some of the pharmaceutical
companies, said that one-fifth of his patients were being "warehoused,"
meaning they were forgoing treatment now to wait for the new
But even if the drugs do work, some experts and doctors warn
that this virus may be particularly tough to vanquish. Three-quarters
of the people who are infected do not know it because they are
not tested for the virus and because the infection can be asymptomatic
for years while it stealthily attacks the liver.
And because this disease is transmitted by blood, those infected
largely are former or current IV-drug users - a population that
characteristically has little or no health insurance - who may
not be the most able to stick to a lengthy treatment regimen
that can cause brutal side effects.
Pharmaceutical companies "completely ignore the real face
of hepatitis C," said Dr. Diana L. Sylvestre, who runs
a clinic in Oakland, Calif., that treats drug addicts and former
addicts with hepatitis C. "A minority of patients who have
hepatitis C will benefit from these drugs."
When she gave a recent talk at Vertex, Dr. Sylvestre's first
slide showed a man in a suit, meant to be a Vertex executive,
with his head in the sand.
Dr. Camilla Graham, a senior director of medical affairs at
Vertex, said that addicts accounted for less than 10 percent
of people with hepatitis C. While many people got infected by
trying drugs in the 1960s and 1970s, they have long since kicked
the habit, she said.
Hepatitis C can also be transmitted sexually, particularly when
men have sex with other men. And many people got the virus from
blood transfusions before 1992, when donated blood began being
tested for the virus.
Nevertheless, pharmaceutical companies realize that difficulties
getting patients screened and treated could limit the use of
their drugs. So they are contributing to a groundswell of activism
to raise awareness of what has long been known as a silent epidemic.
Also contributing to the new advocacy is the highly organized
HIV community, since 15 to 30 percent of those with HIV also
have hepatitis C.
A report issued by the Institute of Medicine in January urged
a new national strategy to improve prevention, detection and
treatment of hepatitis C and hepatitis
B which also causes liver disease. A hepatitis task force
created by the Department of Health and Human Services is preparing
an action plan by October. The House Oversight and Government
Reform Committee held a hearing on hepatitis last month.
Drug makers contribute to the National Viral Hepatitis Roundtable,
which helped pay for the Institute of Medicine report, and several
companies have banded together into the Corporate Hepatitis
Alliance to lobby for more government funding. In January, several
companies started the Viral Hepatitis Action Coalition, to help
finance research at the Centers for Disease Control and Prevention.
Vertex has commissioned studies projecting a rising toll from
hepatitis C. One such study, done by Milliman, a health insurance
consulting firm, projected that the number of people with advanced
liver disease from hepatitis C would quadruple in 20 years if
treatment did not improve.
Screening people for hepatitis C should become easier. In June,
the Food and Drug Administration approved a rapid blood test
developed by OraSure Technologies that gives an answer in 20
minutes rather than several hours needed if the sample is sent
to a lab. Future versions might use a mouth swab, allowing screening
to be done at churches, street fairs and other gatherings.
There is a risk that increased screening could result in treatment
for people who will never need it. Only 5 to 20 percent of people
with chronic infection develop cirrhosis
in about 20 to 30 years, and doctors cannot predict which patients
those will be.
"I think the companies have done a superb job of marketing
this disease," said Dr. Ronald L. Koretz, emeritus professor
of clinical medicine at the University of California, Los Angeles.
Dr. Koretz said there was no good evidence that treatment made
a difference since many patients cured by the drugs might never
have developed serious problems anyway.
The current treatment for hepatitis C consists of weekly injections
of alpha interferon - the leading brands are Roche's
Pegasys and Merck's PegIntron
- combined with ribavirin, a generic oral drug. It is not quite
clear how these drugs work.
The regimen usually lasts either 24 or 48 weeks and costs more
than $30,000. It can be rough, causing flulike symptoms, depression,
anemia and other problems. And the treatment fails to cure the
patient about half the time, either because it cannot clear
the virus from the body or because the patient cannot tolerate
The new drugs generally inhibit enzymes needed by the virus,
a strategy that has worked well against H.I.V. The two drugs
that could conceivably make it to the market by next year, Vertex's
telaprevir and Merck's boceprevir, are both pills that inhibit
the protease enzyme.
For a few years at least, the new drugs would have to be used
along with interferon. But doctors are hopeful that starting
perhaps in five years, combinations of the new pills will do
away with the need for interferon.
The drugs could offer new hope to an estimated 300,000 people
for whom the existing treatment has not worked. Some early data
suggests that telaprevir, when combined with the existing drugs,
could cure half of them.
"I was willing to try yesterday," said Kenny C. Charles,
58, of Woodbourne, N.Y., who said he got hepatitis C from blood
transfusions and had undergone four unsuccessful treatment attempts
with the existing drugs. Now, he said, his liver was starting
to show signs of cirrhosis, or scarring.
Some people with hemophilia, who were infected more than 25
years ago by blood-clotting drugs derived from human plasma,
are pressing the Food and Drug Administration to allow them
to be treated with combinations of the new drugs, without interferon,
even before the new drugs are approved. The F.D.A. held a public
hearing on the request in April and is now formulating a policy.
Mark Antell of Rosslyn, Va., one of the organizers of the petition,
said he had to stop taking interferon because of flulike symptoms,
loss of hair and creaking joints. "It was as though I was
aging very rapidly," he said.
Mr. Antell, 63, a retired Environmental Protection Agency employee,
said hemophiliacs were typically not allowed into clinical trials
to test the new drugs, so they needed another way to obtain
"I think there's a lot of guys in my situation, and we
don't have a lot of time," he said.
A Pollack. Hope
Against Hepatitis C. New York Times. July 21, 2010.