8This year, gene therapy finally became a clinical reality. The U.S. Food and Drug Administration approved t wo personalized treatments that engineer a patient’s own immune system to hunt down and kill cancer cells. The treatments, the first gene therapies ever approved by the FDA,
work in people with certain blood cancers, even patients who
haven’t responded to other treatments.
Called CAR-T cell immunotherapy (for chimeric antigen
receptor T cell), one is for kids and young adults with B cell
acute lymphoblastic leukemia, or ALL, approved in August
(SN Online: 8/30/17). The other is for adults with non-Hodgkin
lymphoma, approved in October. Other CAR-T cell therapies
are in testing, including a treatment for multiple myeloma.
“It’s a completely different way of treating cancer,” says
pediatric oncologist Stephan Grupp, who directs the Cancer
Immunotherapy Program at the Children’s Hospital of
Philadelphia. Grupp spearheaded the clinical trials of the
newly approved ALL therapy, called Kymriah.
Researchers are developing many different versions of
CAR-T cell therapies, but the basic premise is the same: Doctors remove a patient’s T cells (immune system cells that attack
invaders) from a blood sample and genetically modify them to
produce artificial proteins on their surfaces. Those proteins,
called chimeric antigen receptors, recognize the cancer cells in
the patient’s body. After the modified T cells make many copies of themselves in the lab, they’re unleashed in the patient’s
bloodstream to find and kill cancer cells.
CAR-T cell therapy is particularly exciting because it works
well in people who haven’t responded to other available treatments, says Renier Brentjens, an oncologist at Memorial Sloan
Kettering Cancer Center in New York City. Of the 63 kids and
young adults treated in a clinical trial of Kymriah, 83 percent
had their cancers go into remission within three months.
Now that these therapies have been clinically approved,
there’s been an “explosion of interest” in the approach, says
Dario Campano, an immunopathologist at the National
University Cancer Institute in Singapore. Going forward, he
By Laurel Hamers
In CAR-T cell therapy, a
patient’s T cells (teal)
modified to hunt
down and kill
From the opioid epidemic to air pollution, public health
woes captured a lot of attention in 2017. Below are a
few key stats behind this year’s headlines.
Estimated number of U. S.
adults who misused or
abused opioids in 2015,
according to an August
report (SN: 9/2/17, p. 5)
Number of U.S. children
each day who die or are
medically treated in an
for gun-inflicted wounds
(SN Online: 6/26/17)
Portion of U. S. adults with high
blood pressure, according to a
new definition from the
American College of Cardiology
and the American Heart
Association (SN: 12/9/17, p. 13)
People who died from
pollution globally in 2015,
the Lancet Commission on
pollution and health
announced in October
(SN: 11/25/17, p. 5)
expects to see even more rapid progress in the technology.
Fifteen years ago, Campana helped develop the chimeric
antigen receptor that’s used in Kymriah today. For now, the
treatments are approved for use only when other treatments
have failed, but someday CAR-T cell therapy could be the first
treatment doctors try, he says.
One drawback is the price. Kymriah costs $475,000 for
a onetime treatment, according to Novartis, which makes
Kymriah. The non-Hodgkin lymphoma treatment made by
Gilead Sciences, called Yescarta, is listed at $373,000. The
total price tag for treatment could be higher when the costs
of dealing with side effects and complications are factored in.
The approach is approved only for blood cancers. Using
CAR-T cell therapy on solid tumors will require finding ways to
get the T cells past additional cellular roadblocks, Grupp says. s