Page 28 - ARUBA TODAY

A28 SCIENCE
Friday 1 September 2017

U.S. clears breakthrough gene therapy for childhood leukemia

By LAURAN NEERGAARD take about three weeks.
AP Medical Writer The $475,000 price tag
WASHINGTON (AP) — doesn’t include the cost
Opening a new era in of needed hospitalizations,
cancer care, U.S. health travel to a certified hospital
officials have approved a and other expenses.
breakthrough treatment On a conference call
that genetically engineers Wednesday, Novartis ex-
patients’ own blood cells ecutives said the company
into an army of assassins is working with the Medic-
to seek and destroy child- aid program and private
hood leukemia. insurers and expects broad
The Food and Drug Admin- coverage, and will offer
istration said the approval some financial assistance
on Wednesday was histor- with such things as copay
ic, the first gene therapy to and travel costs. But they
hit the U.S. market. Made didn’t promise all patients
from scratch for every pa- would be able to get the
tient, it’s one of a wave of therapy.
“living drugs” under devel- This undated image made available by Novartis on Wednesday, Aug. 31, 2017 shows an IV bag For some patients, the new
opment to fight additional of their drug Kymriah. This photo provided by the Children’s Hospital of Philadelphia, taken in May CAR-T therapy might re-
blood cancers and other 2017, shows Dr. Stephan Grupp, right, who led a key study of a newly approved treatment for place bone marrow trans-
childhood leukemia.
tumors, too. Associated Press plants that cost more than
Novartis Pharmaceuticals half a million dollars, noted
set the price for its one- way to harness the immune benefit lasts: Some patients Sekeres of the American Grupp, who led the Novar-
time infusion of so-called system than popular im- did relapse months later. Society of Hematology. tis study.
“CAR-T cells” at $475,000, munotherapy drugs called The others still are being But, he cautioned that “I don’t want to be an apol-
but said there would be no “checkpoint inhibitors” tracked to see how they CAR-T “isn’t a panacea.” ogist for high drug prices in
charge for patients who that treat a variety of can- fare long-term. Among concerns, some- the U.S.,” Grupp stressed.
didn’t show a response cers by helping the body’s Still, “a far higher percent- times leukemia can devel- But if it’s the last treatment
within a month. natural T cells better spot age of patients go into re- op resistance, and some- they need, “that’s a really
“This is a brand new way tumors. CAR-T cell therapy mission with this therapy times patients worsen while significant one-time invest-
of treating cancer,” said gives patients stronger T than anything else we’ve waiting for their new cells, ment in their wellness, es-
Dr. Stephan Grupp of Chil- cells to do that job. seen to date with relapsed said Sekeres, who directs pecially in kids who have
dren’s Hospital of Philadel- “We’re entering a new leukemia,” said Dr. Ted La- the Cleveland Clinic’s leu- a whole lifetime ahead of
phia, who treated the first frontier in medical innova- etsch of the University of kemia program and wasn’t them.”
child with CAR-T cell ther- tion with the ability to re- Texas Southwestern Medi- involved with CAR-T testing. “This is a turning point in
apy — a girl who’d been program a patient’s own cal Center, one of the “Unfortunately leukemia the fight” against ALL, said
near death but now is can- cells to attack a deadly study sites. “I wouldn’t say grows so rapidly that it can Penn’s Dr. Carl June, who
cer-free for five years and cancer,” said FDA Commis- we know for sure how many evade even the smartest pioneered the therapy.
counting. “That’s enor- sioner Scott Gottlieb. will be cured yet by this of our technologies,” he But he and other research-
mously exciting.” The first CAR-T version, de- therapy. There certainly is added. ers say thousands more pa-
CAR-T treatment uses veloped by Novartis and a hope” that some will be. To better ensure patient tients eventually may ben-
gene therapy techniques the University of Pennsylva- Most patients suffered side safety, the FDA is requiring efit. Kite Pharma’s similar
not to fix disease-causing nia, is approved for use by effects that can be gruel- Novartis to offer CAR-T ther- CAR-T brand, developed
genes but to turbocharge several hundred patients a ing, even life-threatening. apy only through medical by the National Cancer In-
T cells, immune system sol- year who are desperately An immune overreaction centers specially trained stitute, is expected to win
diers that cancer too often ill with acute lymphoblastic called “cytokine release and certified to handle the approval later this year to
can evade. Researchers leukemia, or ALL. It strikes syndrome” can trigger high complicated treatment. treat aggressive lympho-
filter those cells from a pa- more than 3,000 children fevers, plummeting blood Novartis expects to have 32 ma, and Juno Therapeutics
tient’s blood, reprogram and young adults in the U.S. pressure and in severe centers around the coun- and other companies are
them to harbor a “chime- each year and while most cases organ damage, side try, mostly in large cities, studying their own versions
ric antigen receptor” or survive, about 15 percent effects that require sophis- running by year’s end, with against blood cancers in-
CAR that zeroes in on can- relapse despite today’s ticated care to help pa- the first 20 offering care cluding multiple myeloma.
cer, and grow hundreds of best treatments. tients without blocking the within the next month. Scientists around the coun-
millions of copies. Returned In a key study of 63 ad- cancer attack. The FDA Patients’ collected immune try also are trying to make
to the patient, the revved- vanced patients, 83 per- designated a treatment for cells will be frozen and CAR-T therapies that could
up cells can continue mul- cent went into remission those side effects Wednes- shipped to a Novartis fac- fight more common solid
tiplying to fight disease for soon after receiving the day. tory in New Jersey that cre- tumors such as brain, breast
months or years. CAR-T cells. Importantly, “This is remarkable tech- ates each dose, a process or pancreatic cancers — a
It’s a completely different it’s not clear how long that nology,” said Dr. Mikkael the company says should harder next step.q