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Sunday, December 8, 2013

Killing cancer like the common cold


 Nick Wilkins was diagnosed with leukemia when he was 4 years old, and when the cancer
kept bouncing back, impervious to all the different
treatments the doctors tried, his father sat him
down for a talk. John Wilkins explained to Nick, who was by then
14, that doctors had tried chemotherapy, radiation,
even a bone marrow transplant from his sister. "I explained to him that we're running out of
options," Wilkins remembers telling his son. There was one possible treatment they could try:
an experimental therapy at the University of Pennsylvania. He asked his son if he understood what it would
mean if this treatment didn't work. "He understood he could die," Wilkins says. "He
was very stoic." A few months later, Nick traveled from his home
in Virginia to Philadelphia to become a part of the
experiment. This new therapy was decidedly different from the
treatments he'd received before: Instead of
attacking his cancer with poisons like
chemotherapy and radiation, the Philadelphia
doctors taught Nick's own immune cells to
become more adept at killing the cancer. Two months later, he emerged cancer-free. It's
been six months since Nick, now 15, received the
personalized cell therapy, and doctors still can
find no trace of leukemia in his system. Trusting her intuition led to two cancer diagnoses Twenty-one other young people received the same
treatment at The Children's Hospital of
Philadelphia, and 18 of them, like Nick, went into
complete remission -- one of them has been
disease-free for 20 months. The Penn doctors
released their findings this weekend at the annual meeting of the American Society of Hematology. "It gives us hope that this is a cure," Nick's father
says. "They're really close. I think they're really
onto something." 'A whole new realm of medicine ' At the conference, two other cancer centers --
Memorial Sloan-Kettering in New York and the
National Cancer Institute -- will be announcing
results with immunotherapies like the one Nick
received. The results are promising, especially
considering that the patients had no success with practically every other therapy. "This is absolutely one of the more exciting
advances I've seen in cancer therapy in the last
20 years," said Dr. David Porter, a hematologist
and oncologist at Penn. "We've entered into a
whole new realm of medicine." In the therapy, doctors first remove the patient's
T-cells, which play a crucial role in the immune
system. They then reprogram the cells by
transferring in new genes. Once infused back into
the body, each modified cell multiplies to 10,000
cells. These "hunter" cells then track down and kill the cancer in a patient's body. Essentially, researchers are trying to train Nick's
body to fight off cancer in much the same way our
bodies fight off the common cold. Tumor Paint: Changing the way surgeons fight
cancer In addition to the pediatric patients, Penn
scientists tried the therapy out in 37 adults with
leukemia, and 12 went into complete remission.
Eight more patients went into partial remission and
saw some improvements in their disease. The treatment does make patients have flulike
symptoms for a short period of time -- Nick got so
sick he ended up in the intensive care unit for a
day -- but patients are spared some of the more
severe and long-lasting side effects of extensive
chemotherapy. Penn will now work with other medical centers to
test the therapy in more patients, and they plan to
try the therapy out in other types of blood cancers
and later in solid tumors. A university press release says it has a licensing
relationship with the pharmaceutical company
Novartis and "received significant financial
benefit" from the trial, and Porter and other
inventors of the technology "have benefited
financially and/or may benefit financially in the future." Searching for one-in-a-million cancer cells The big question is whether Nick's leukemia will
come back. Doctors are cautiously optimistic. The studies
have only been going on since 2010, but so far
relapse rates have been relatively low: of the 18
other pediatric patients who went into complete
remission, only five have relapsed and of the 12
adults who went into complete remission, only one relapsed. Some of the adult patients have been
cancer-free and without a relapse for more than
three years and counting. Relapses after this personalized cell therapy may
be more promising than relapses after
chemotherapy or a bone marrow transplant, Porter
explained. First, doctors have been delighted to find the
reengineered T-cells -- the ones that know how to
hunt down and attack cancer -- are still alive in the
patients' bodies after more than three years. Stigma lingers for deadliest cancer "The genetically modified T-cells have survived,"
Porter said. "They're still present and functional
and have the ability to protect against recurrence." Second, before declaring patients in remission,
Penn doctors scoured especially hard for errant
leukemia cells. Traditionally, for the kind of leukemia Nick has,
doctors can find one in 1,000 to one in 10,000
cancer cells. But Penn's technology could find one
in 100,000 to one in a million cancer cells, and
didn't find any in Nick or any of the patients who
went into complete remission. 'It's not a fluke' One of the best aspects of this new treatment is
that it won't be terribly difficult to reproduce at
other medical centers, Porter said, and one day,
instead of being used only experimentally, it could
be available to anyone who needed it. "Our hope is that this can progress really quite
quickly," he said. "It won't be available to
everyone next year, but I don't think it would take
a decade, either." Right now patients can only get this therapy if
they're in a study, but Dr. Renier Brentjens,
director for cellular therapeutics at Memorial
Sloan-Kettering, says he thinks it could become
available to all patients in just three to five years. "When you have three centers all with a
substantial number of patients seeing the same
thing -- that these cells work in this disease - you
know it's not a fluke," he said. Two days ago, Brentjens became the co-founder
of Juno Therapeutics, a for-profit biotech start-up
company that's working on immunotherapies. "Fifteen years ago I was in the lab looking at
these cells kill tumor cells in a petri dish and then
I saw them kill tumor cells in mice, and then
finally in humans," Brentjens said. He says he'll never forget the first patient he
treated, who initially had an enormous amount of
cancer cells in his bone marrow. Then after the
therapy, Brentjens looked under the microscope
and, in awe, realized he couldn't find a single
cancer cell. "I can't describe what that's like," he said. "It's
fantastic."

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