image provided by AstraZeneca shows the company’s drug Imfinzi, known
chemically as durvalumab. (AstraZeneca via AP)
Linda A. Johnson
Trenton, N.J. (AP) - U.S.
regulators have approved a new drug that harnesses the immune system to
treat bladder cancer that has spread after chemotherapy or surgery.
The Food and Drug Administration on
Monday approved Imfinzi for advanced bladder cancer, along with a
companion diagnostic test for identifying which patients are most likely
to benefit from it.
Imfinzi, also known as durvalumab,
is part of a new generation of drugs that stimulate the immune system to
help fight cancer. British drugmaker AstraZeneca PLC, which developed
the drug, is testing it for several other cancers.
The average monthly list price for
Imfinzi is roughly $15,000, but varies with the patient’s weight,
according to AstraZeneca. It’s given by IV, usually every two weeks.
Imfinizi works by binding to a
protein, found in varying levels on tumor cells, that blocks immune
system cells from attacking the tumor.
In a company-funded study, 26
percent of patients with high levels of the protein responded to
Imfinzi; their tumors shrank or stopped growing, some for 20 months or
more, according to the FDA. Only 4 percent of patients with low levels
of the protein responded to the drug. The diagnostic test, made by
Ventana Medical Systems, distinguishes between those groups.
The FDA gave the drug accelerated
approval and is requiring AstraZeneca to complete ongoing testing in
more patients to confirm the drug’s benefits and safety.
Common side effects include
fatigue, bone and muscle pain, nausea, swelling in hands and feet, and
urinary tract infections. Infections elsewhere in the body were
infrequent. However, two patients died, one from a lung infection and
the other from a liver infection.
Bladder cancer is the sixth most
common type of cancer in the U.S., with about 79,000 new cases and
nearly 17,000 deaths expected this year, according to the National
Cancer Institute. When bladder cancer has spread, only about 5 percent
of patients survive beyond five years, government figures show.
Until recently, treatment options
were limited to chemotherapy, radiation or surgery.
Fetal physiologist Marcus G. Davey of the Children’s Hospital of
Philadelphia, who helped design the artificial womb system, is shown
near giant tanks holding a liquid designed to simulate amniotic fluid.
(Ed Cunicelli/Children’s Hospital of Philadelphia via AP)
Washington (AP) -
Researchers are creating an artificial womb to improve care for
extremely premature babies - and remarkable animal testing suggests the
first-of-its-kind watery incubation so closely mimics mom that it just
Today, premature infants weighing
as little as a pound are hooked to ventilators and other machines inside
incubators. Children’s Hospital of Philadelphia is aiming for a gentler
solution, to give the tiniest preemies a few more weeks cocooned in a
womb-like environment - treating them more like fetuses than newborns in
hopes of giving them a better chance of healthy survival.
The researchers created a
fluid-filled transparent container to simulate how fetuses float in
amniotic fluid inside mom’s uterus, and attached it to a mechanical
placenta that keeps blood oxygenated.
In early-stage animal testing,
extremely premature lambs grew, apparently normally, inside the system
for three to four weeks, the team reported Tuesday.
“We start with a tiny fetus that is
pretty inert and spends most of its time sleeping. Over four weeks we
see that fetus open its eyes, grow wool, breathe, swim,” said Dr. Emily
Partridge, a CHOP research fellow and first author of the study
published in Nature Communications.
“It’s hard to describe actually how
uniquely awe-inspiring it is to see,” she added in an interview.
Human testing still is three to
five years away, although the team already is in discussions with the
Food and Drug Administration.
“We’re trying to extend normal
gestation,” said Dr. Alan Flake, a fetal surgeon at CHOP who is leading
the project and considers it a temporary bridge between the mother’s
womb and the outside world.
Increasingly hospitals attempt to
save the most critically premature infants, those born before 26 weeks
gestation and even those right at the limits of viability - 22 to 23
weeks. Extreme prematurity is a leading cause of infant mortality, and
those who do survive frequently have serious disabilities such as
The idea of treating preemies in
fluid-filled incubators may sound strange, but physiologically it makes
sense, said Dr. Catherine Spong, a fetal medicine specialist at the
National Institutes of Health.
“This is really an innovative,
promising first step,” said Spong, who wasn’t involved with the
One of the biggest risks for very
young preemies is that their lungs aren’t ready to breathe air, she
explained. Before birth, amniotic fluid flows into their lungs, bringing
growth factors crucial for proper lung development. When they’re born
too soon, doctors hook preemies to ventilators to keep them alive but
risking lifelong lung damage.
Flake’s goal is for the womb-like
system to support the very youngest preemies just for a few weeks, until
their organs are mature enough to better handle regular hospital care
like older preemies who have less risk of death or disability.
The device is simpler than previous
attempts at creating an artificial womb, which haven’t yet panned out.
How the “Biobag” system works:
- The premature lambs were
delivered by C-section and immediately placed into a
temperature-controlled bag filled with a substitute for amniotic fluid
that they swallow and take into their lungs.
“We make gallons of this stuff a
day,” said fetal physiologist Marcus Davey. It’s currently an
electrolyte solution; he’s working to add other factors to make it more
like real amniotic fluid.
- Then the researchers attached the
umbilical cord to a machine that exchanges carbon dioxide in blood with
oxygen, like a placenta normally does.
- The lamb’s heart circulates the
blood, without the need for any other pump.
The researchers tested five lambs
whose biological age was equivalent to 23-week human preemies, and three
more a bit older. All appeared to grow normally, with blood pressure and
other key health measures stable and few complications during the weeks
they were inside the womb-like device.
The study didn’t address long-term
development. Most of the lambs were euthanized for further study that
found normal organ development for their gestational age. One was
bottle-weaned and is now more than a year old, apparently healthy and
living on a farm in Pennsylvania.
Flake stressed that the womb-like
system isn’t intended to support preemies any younger than today’s
limits of viability - not what he calls the more “sensationalistic” idea
of artificially growing embryos.
He acknowledged that parents might
question the approach, but notes that the preemies always could be
whisked into standard care if they fared poorly in the new system. And
while he said further adaptation of the device is needed before it can
begin human testing, he envisioned parents being able to see the baby
and even piping in the sound of mom’s heartbeat.
results published Wednesday, April 26, 2017 by Nature and the New
England Journal of Medicine, researchers have taken an important step
toward better lung cancer treatment. Using experimental tests that
detect bits of DNA that tumors shed into the blood, they were able to
track genetic changes in early-stage cases over time, and to find some
recurrences up to a year before imaging scans could, giving a chance to
try new therapy sooner. (AP Photo/Richard Drew)
Boston (AP) - Researchers
have taken an important step toward better lung cancer treatment by
using blood tests to track genetic changes in tumors as they progress
from their very earliest stages.
With experimental tests that detect
bits of DNA that tumors shed into the blood, they were able to detect
some recurrences of cancer up to a year before imaging scans could,
giving a chance to try new therapy sooner.
It’s the latest development for
tests called liquid biopsies, which analyze cancer using blood rather
than tissue samples. Some doctors use these tests now to guide care for
patients with advanced cancers, mostly in research settings. The new
work is the first time tests like this have been used to monitor the
evolution of lung tumors at an early stage, when there’s a much better
chance of cure.
Only about one third of lung cancer
cases in the United States are found at an early stage, and even fewer
in other parts of the world. But more may be in the future as a result
of screening of longtime smokers at high risk of the disease that
started a few years ago in the U.S.
Early-stage cases are usually
treated with surgery. Many patients get chemotherapy after that, but it
helps relatively few of them.
“We have to treat 20 patients to
cure one. That’s a lot of side effects to cure one patient,” said Dr.
Charles Swanton of the Francis Crick Institute in London.
The new studies he led suggest that
liquid biopsies might help show who would or would not benefit from
chemotherapy, and give an early warning if it’s not working so something
else can be tried.
Cancer Research UK, a charity based
in England, paid for the work, and results were published online by
Nature and the New England Journal of Medicine.
To be clear: This kind of care is
not available yet - the tests used in these studies are experimental and
were customized in a lab to analyze the genes in each patient’s cancer.
But the technology is advancing rapidly.
The company that generated the
tests for the study in Nature - California-based Natera Inc. - plans to
offer the tests for research by universities and drug companies later
this year and hopes to have a version for routine use in cancer care
“This is coming, and it’s coming
fast,” said Dr. David Gandara, a lung specialist at the University of
California, Davis, who had no role in the studies but consults for two
companies developing liquid biopsies. A test that could spare many
people unnecessary treatment “would be huge,” he said.
In the studies, researchers
analyzed tumors from about 100 people with non-small cell lung cancer,
the most common form of the disease. Even in these early-stage cases,
they found big variations in the number of gene flaws, and were able to
trace how the tumors’ genes changed over time.
People with many gene or chromosome
problems were four to five times more likely to have their cancer
return, or to die from their disease within roughly two years.
They also looked at 14 patients
whose cancers recurred after surgery, and compared them to 10 others
whose did not. Blood tests after surgery accurately identified more than
90 percent of them that were destined to relapse, up to a year before
imaging tests showed that had occurred.
The results suggest that using
liquid biopsy tests to help select and adjust treatments is “now
feasible,” at least from a scientific standpoint, the authors write.
A big issue is cost, though. Liquid
biopsies sold now in the U.S. cost nearly $6,000. Tests that more
narrowly track a patient’s particular tumor gene changes, like the one
in these studies, may cost less. They may save money in the long run, by
preventing futile treatment, but this has yet to be shown.
Liquid biopsy video: https:
Lung cancer treatment info: