Yahoo – AFP,
Kerry SHERIDAN, August 30, 2017
 |
| Six years ago Emily Whitehead -- pictured here in 2013 -- became the first child ever to receive the anti-cancer gene therapy treatment. Today at age 12 she is cancer free (AFP Photo/KARI WHITEHEAD) |
Miami (AFP)
- US regulators on Wednesday approved the first gene therapy against cancer --
a treatment that uses a patient's own immune cells to fight leukemia -- opening
a new era in the fight against one of the world's top killers.
The
treatment is made by Novartis and is called Kymriah (tisagenlecleucel).
This type
of anti-cancer immunotherapy, known as a CAR-T cell therapy, was known by
CTL019 until now.
"This marks
the first-ever CAR-T cell therapy to be approved anywhere in the world,"
Novartis CEO Joseph Jimenez told reporters on a conference call.
"It
uses a new approach that is wholly personalized by using a patient's own
T-cells."
Kymriah was
approved by the US Food and Drug Administration for children and young adult
patients up to age 25 with a form of acute lymphoblastic leukemia (ALL).
To qualify
for treatment, patients must have B-cell precursor ALL that is refractory, or
the patient has relapsed at least twice.
The FDA
described the approval as "a historic action" and a "new
approach to the treatment of cancer and other serious and life-threatening
diseases," said a statement.
How it
works
The
treatment is not a pill or a form of chemotherapy, which can weaken the body's
natural defenses.
Instead, it
harnesses a patient's own immune cells, called T-cells and white blood cells,
and trains them to recognize and fight cancer.
The
patient's immune cells are removed with a special blood filtration process,
sent to a lab, and genetically encoded to be able to hunt down cancer cells.
These
re-engineered T-cells are then transfused back into the patient, where they can
begin attacking leukemia.
Studies
have shown that 83 percent of patients responded to the treatment, achieving
remission within three months, Novartis said.
An
application with the European Medicines Agency is expected to be filed by the
end of the year.
$475,000
a dose
The price
of Kymriah -- which is delivered to a patient just once -- is $475,000, said
Bruno Strigini, CEO of Oncology at Novartis.
Patients
who do not respond to the treatment within the first month would not be
expected to pay, he told reporters.
The more common
treatment for leukemia -- bone marrow transplants -- can cost between $540,000
and $800,000 the first year in the United States, Strigini said.
Meanwhile,
outside analyses have set a cost-effective price for Kymriah between $600,000
and $750,000, he added.
"Recognizing
our responsibility we set the price below that level," said Strigini.
Most
patients who fit the criteria for treatment would likely be covered by
insurance, since they are under 25 and would either be on their parents'
insurance or covered by government-sponsored Medicaid, a Novartis spokesman
said.
The price
tag is high -- as are costs throughout the cancer industry -- a trend that is
"unsustainable," said Craig Devoe, acting chief of medical oncology
and hematology at Northwell Health Cancer Institute in New York.
Asked if he
thought Novartis's pricing was reasonable, he told AFP: "It's not."
Devoe also
said that while the positive results are exciting, many questions remain about
how long these novel cancer treatments -- developed mainly in the past decade
or so -- will last.
"Not
just responding but staying in response long term is what patients want,"
he said.
"We
really don't know how long these cells are going to stay active."
Breakthrough therapy
The
treatment was pioneered by Carl June at the University of Pennsylvania.
Its most
high-profile patient is Emily Whitehead, now 12.
Six years
ago, she was the first child to receive what was widely considered a risky
treatment.
She has
been cancer-free ever since.
In 2014, US
regulators designated CTL019 as a "breakthrough therapy" and put the
experimental immunotherapy agent on the fast track to market approval.
It was the
first cancer immunotherapy to receive the breakthrough designation. More are expected
to follow in the coming years as the field of immunotherapy grows.
Last month,
an advisory panel to the FDA unanimously urged approval of the cutting-edge
cancer therapy.
About two
dozen centers in the United States are expected to be able to treat patients
seeking Kymriah, with as many as 35 up and running by the end of the year.
ALL is the
most common form of childhood leukemia, with about 3,000 cases diagnosed in the
US each year.
Of those,
600 US patients would likely meet the criteria of being under 25 and having
relapsed or refractory ALL, according to Novartis.
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