years before it's been developed further and it
becomes afordable to all, but it's a massive step in the
drug,” and trains them to recognize and attack the
disease. It is part of the rapidly growing field of immunotherapy that bolsters the immune system
through drugs and other therapies and has, in some
cases, led to long remissions and possibly even cures.
the first-ever treatment that genetically alters a patient’s own
cells to fight cancer, a milestone that is expected to transform
treatment in the coming years.
approved for children and young adults for an aggressive type
of leukemia — B-cell acute lymphoblastic leukemia — that has
resisted standard treatment or relapsed. The F.D.A. called the
disease “devastating and deadly” and said the new treatment
fills an “unmet need.”
gene therapies for other types of cancers, and experts expect
more approvals in the near future. Dr. Scott Gottlieb, the F.D.A.
commissioner, said that more than 550 types of experimental
gene therapy were being studied.
have life-threatening side effects, including dangerous drops
in blood pressure, the F.D.A. is requiring that hospitals and
doctors be specially trained and certified to administer it,
and that they stock a certain drug needed to quell severe
made individually for each, will cost $475,000. Novartis said
that if a patient does not respond within the first month after
treatment, there will be no charge. The company also said it
would provide financial help to families who were uninsured
Novartis official noted that bone-marrow transplants, which
can cure some cases of leukemia, cost even more, from
$540,000 to $800,000.
would be candidates for the new treatment.
children and young adults who had a remission rate of 83
percent within three months — a high rate, given that relapsed
or treatment-resistant disease is often quickly fatal.
University of Pennsylvania and licensed to Novartis. It was
identified in previous reports as CAR-T cell therapy, CTL019
was 6 and near death from leukemia in 2012 when she was
treated, at the Children’s Hospital of Philadelphia. Now 12,
she has been free of leukemia for more than five years.
called T cells will be removed from a patient’s bloodstream at
an approved medical center, frozen, shipped to Novartis in
Morris Plains, N.J., for genetic engineering and multiplying,
frozen again and shipped back to the medical center to be
dripped into the patient. That processing is expected to take
network of 20 approved medical centers to be certified within
a month, a number that would be expanded to 32 by the end of
the year. Five centers will be ready to start extracting T cells
from patients within three to five days, the company said.
touch off an intense reaction, sometimes called a cytokine
storm, that can cause high fever, low blood pressure,
lung congestion, neurological problems and other life-
threatening complications. Medical staff members need
training to manage these reactions, and hospitals are being
told that before giving Kymriah to patients, they must be sure
that they have the drug needed to treat the problems,
tocilizumab, also called Actemra.
Kettering Cancer Center in Manhattan, said his hospital was
“99 percent” of the way through the certification process, and
would soon be offering Kymriah.
said. “It will provide a lot of hope. This is the beginning.”
would work for other, more common types of cancer, not just for
the treatment of cancer and other serious and life-threatening
diseases,” the agency said in a statement, noting that the new
therapy is “the first gene therapy available in the United States.”
University of Pennsylvania, recalled that in 2010, when tests
showed that the first patient was leukemia-free a month after
being treated, he and his colleagues did not believe it. They
ordered another biopsy to be sure.
Dr. June said, his voice breaking with emotion. “It was so
improbable that this would ever be a commercially approved
therapy, and now it’s the first gene therapy approved in the
United States. It’s so different from all the pharmaceutical
models. I think the cancer world is forever changed.”