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Health Rounds: CAR-T therapy shows early promise against brain cancer

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    By Nancy Lapid
       March 14 (Reuters) - Hello Health Rounds Readers! Today
we have potentially exciting early data for the treatment of one
of the most deadly forms of brain cancer from three separate
trials. We also report promising data on a new blood test for
detecting colorectal cancer that was as accurate as current
commercial home tests. 
    
    Early trials hint at benefit of CAR-T cells in glioblastoma
    First-in-human studies of CAR-T cell therapies for recurrent
glioblastoma suggest the approach may eventually become useful
for patients with this deadly type of brain cancer, researchers
say.
    The treatment involves removing a patients’ own T cells - a
key component of the immune system - genetically altering them,
and re-infusing them. The therapy, which has so far only been
approved for a variety of blood cancers, shrank tumors in three
separate early-stage glioblastoma trials designed primarily to
test the safety of the approach. 
    In one trial, reported on Wednesday in The New England
Journal of Medicine, three patients with recurrent glioblastoma
were treated with CAR-T cells engineered to target various forms
of epidermal growth factor receptor (EGFR) proteins on the tumor
cell surface. 
    “Tumor regression was dramatic and rapid, occurring within
days after receipt of a single... infusion,” researchers
reported. 
    The tumors began to regrow within one to two months in two
of the three participants, but the third patient’s response to
treatment persisted through 150 days of follow-up. 
    “These results are exciting, but they are also just the
beginning, they tell us that we are on the right track,” study
leader Dr. Marcela Maus of Mass General Cancer Center in Boston
said in a statement.
    Since CAR-T therapy first came on the scene, researchers
have been looking for ways it might treat solid tumor cancers. 
    In a separate early trial published on Wednesday in Nature
Medicine, CAR-T cells targeting EGFR and a second cell-surface
protein called Interleukin 13 receptor alpha 2 shrank tumors in
all six patients with recurrent glioblastoma, although none
shrank by at least 50%.
    “Stable disease was maintained on scans performed at least
2 months after CAR T cell therapy in three of the four patients
who had at least 2 months of follow-up time,” the researchers
said.
    The trial, funded in part by Gilead Sciences'  GILD.O  Kite
Pharma unit, is ongoing, with future participants receiving
incrementally higher doses of the treatment. 
    “We are energized by these results, and are eager to
continue our trial, which will give us a better understanding of
how this dual-target CAR T cell therapy affects a wider range of
individuals with recurrent (glioblastoma),” study leader Dr.
Donald O’Rourke of the University of Pennsylvania Perelman
School of Medicine in Philadelphia said in a statement.
    A third early stage trial published last week in Nature
Medicine tested various doses of CAR-T cells targeting
Interleukin 13 receptor alpha 2 in 65 patients with recurrent
glioblastoma. 
    The treatment, licensed by Fortress Biotech's  FBIO.O 
Mustang Bio  MBIO.O  division, achieved stable disease or better
in 50% of patients, the authors reported. Overall, half of the
participants survived more than 8 months. 
    “These were heavily pretreated patients, so we were not sure
how they would do with CAR T cell therapy,” senior author Dr.
Behnam Badie of City of Hope in Duarte, California said in a
statement. “But some of them even did better than how they
initially responded to standard of care treatments.”
    
    Blood test shows promise for colorectal cancer screening 
    An experimental blood test for detecting early colorectal
cancer was as accurate as currently available home stool tests
for people at average risk for the disease in a large trial,
researchers reported. 
    “The results of the study are a promising step toward
developing more convenient tools to detect colorectal cancer
early while it is more easily treated,” senior author Dr.
William Grady of Fred Hutchinson Cancer Center in Seattle said
in a statement. 
    In 7,861 volunteers, researchers compared the blood test 
from Guardant Health  GH.O  to gold-standard colonoscopy for
colorectal cancer screening.
    The blood test, from an emerging field known as liquid
biopsies, detects colorectal cancer signals in the blood from
DNA shed by tumors. 
    Overall, 83.1% of the participants with colorectal cancer
confirmed by colonoscopy had positive blood tests, while 16.9%
had false negative tests in which the colonoscopy showed cancer 
the blood test failed to catch.
    “These results are on par with the performance of other
guideline-recommended, non-invasive screening (tests), where
overall sensitivity in detecting colorectal cancer ranges from
74% to 92%,” Guardant said in a statement.
    The results were published in The New England Journal of
Medicine on Wednesday. 
    The test was less sensitive for advanced precancerous
lesions that are at high risk for becoming malignant.
    “Colorectal cancer is common and very preventable with
screening, but only about 50% to 60% of people who are eligible
for screening actually take those tests,” Grady said. 
    “Having a blood-based test for people to take during routine
doctor’s visits could be an opportunity to help more people be
screened,” he added. 
    A U.S. Food and Drug Administration advisory committee is
expected to review the test later this month. 

    





 (Reporting by Nancy Lapid; Editing by Bill Berkrot)
 ((nancy.lapid@thomsonreuters.com))

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