The benefits aren’t limited to cancer patients.
In a 2008 clinical trial of Enbrel (with the chem-ical-based drug methotrexate) conducted by
the developers of the drug—Amgen and Wyeth
Pharmaceuticals—half of patients with moderate to severe rheumatoid arthritis were in remission after one year, compared with 28 percent
of patients treated with methotrexate (brand
names Rheumatrex and Trexall) alone.
When Pat Novak Nelson of Basking Ridge, New
Jersey, was diagnosed with rheumatoid arthritis
in 1976, available treatments provided little relief
for the inflammatory disease. Starting with a
single swollen finger, Nelson developed such overwhelming pain that she had to give up her passions:
tennis, volleyball, and softball. She couldn’t even
play Frisbee with her young son. Back then,
treatment for the roughly 1. 3 million sufferers
of rheumatoid arthritis was aspirin and anti-inflammatory drugs such as Naprosyn (naproxen)—
which worked “for a while,” Nelson recalls.
Then, in 1998, the FDA approved Enbrel, a
biologic drug that blocks the protein receptors
that trigger inflammation, swelling, and joint
damage. “Without question, biologic agents
have had a profound impact on improving the
treatment of rheumatoid arthritis,” says John
Howard Klippel, M. D., president and CEO of the
Arthritis Foundation. “It can’t cure the disease,
but it stops it from progressing.”
As promising as Enbrel sounded, Nelson had
reservations. “I thought about it for a couple of
months because the possible side effects [infec-
tions, tumors, headaches] can be nasty,” she says.
But with no alternatives in the offing, she decided
to take the plunge, and now, twice a week, she self-
injects Enbrel. “It made a huge improvement in
the quality of my life,” she says. “I can’t remember
a day when I woke up with pain.”
HOW THEY’RE MADE
GENE IS SPLICED
The genes for a
disease-fighting
protein are insert-
ed into the cells of
a living organism.
CELLS EXTRACTED
The cells with that
new protein are
then removed
from the
organism.
COPIES MADE
The cells multiply,
slowly at first,
then in increas-
ingly larger
quantities.
For all the stories of miraculous recoveries
and pain-free lives, biologics represent a quandary
in modern medicine. The drugs are typically prohibitively expensive—Rituxan can cost $60,000 a
year; Enbrel, $32,000—partially because the FDA
has no process for approving generic versions of
biologics (see “What Price a Miracle?” page 57).
And, as patients such as Pat Nelson know, the
drugs can also trigger serious side effects, ranging
from heart attacks to infections.
Chicago oncologist Mark Kozloff, M.D., who
has been treating cancer patients for 30 years,
HARVESTED
The desired pro-
teins are extract-
ed from the rest
of the cells, puri-
fied, and made
into biologic
medicines.
does not dispute the effectiveness of the drugs
for his patients, but he often wonders “who
bears the cost and how does society pay for
it.” Even when patients are covered by health
insurance or Medicare, many of them face high
costs. Richard Oropeza Jr. is okay for now, but
his future is uncertain. With his brain cancer
stable, Oropeza continues to take Avastin, but he
doesn’t pay for the drug—yet. Oropeza receives
his biweekly IV infusions courtesy of Genentech,
the biotech company that developed Avastin. All
other costs—doctor fees, MRI scans—are covered under Oropeza’s private insurance plan, but
only after he pays a deductible. And Genentech’s
coverage of his treatment may end next year.
“I’m not going to worry about the cost now,”
Oropeza says. “I live day by day, and right now
I’m doing well and feeling good. I’m hoping that
less expensive generic versions of these drugs
will come along soon.”
The safety of biologics is another area of con-
cern. Roughly 13 percent of the drugs, including
Herceptin, Enbrel, and Rituxan, have received
FDA black-box-label warnings—the most
severe kind—for their potential serious side
effects, including not just infections and heart
attacks but also lymphoma and disorders of the
central nervous system and immune system.
Adverse effects sometimes do not surface for
five or more years after drugs have been on the
market. “The human body is in a constant state
of change,” wrote the editors of The Journal of
the American Medical Association in an edito-
rial accompanying a 2008 report on biologics,
“and the effects of some drugs will manifest
only after exposure over time.”
In clinical studies 6 out of 39 patients receiv-
ing Avastin for colorectal and other types of
cancer had bleeding complications and delayed
wound healing.
And while some patients such as Nelson have
had no side effects from Enbrel, others haven’t
been as lucky. “We know that the drug mediates
the immune system’s response, which in some
cases can have grave consequences,” says Debra
Lappin, president of the Council for American
Medical Innovation and an Enbrel user herself
(she has a rheumatic disease that causes arthritis of the spine and sacroiliac joints). “The drug
reduces the inflammatory response, thereby
preventing serious joint damage, but it can also
decrease the immune system’s ability to fight
infection.” In a 2007 report in the Archives of
Internal Medicine that (CONTINUED ON PAGE 78)
