Citizen Tribune

13 / 17

Tribune Triple E
Tuesday, October 24, 2017 EEE-1
Tribune Expanded Electronic Edition
SILVER SPRING, Md. (AP)
— A potentially groundbreak-
ing treatment for a rare form of
blindness moved one step closer
to U.S. approval Thursday, as fed-
eral health advisers endorsed the
experimental gene therapy for pa-
tients with an inherited condition
that gradually destroys eyesight.
The panel experts to the Food
and Drug Administration voted
unanimously in favor of Spark
Therapeutics' injectable therapy,
which aims to improve vision by
replacing a defective gene needed
to process light.
The vote amounts to a recom-
mendation to approve the therapy.
The FDA has until mid-January
to make its decision and does not
have to follow the panel's recom-
mendation, though it often does.
If approved, Luxturna would be
the first gene therapy in the U.S.
for an inherited disease and the
first in which a corrective gene is
given directly to patients.While the
therapy from Spark Therapeutics
targets a small group of patients —
about 2,000 in the U.S. — experts
say it could pave the way for other
genetic treatments for a variety of
inherited conditions.
Panelists debated several spe-
cifics of the treatment, splitting
on whether to require a minimum
age for treatment; the company
says the drug is intended for those
3 years and older. Panelists con-
cluded that the science behind the
drug — based on research from the
Children's Hospital of Philadelphia
—was remarkably strong with few
serious side effects.
Several patients attended the
meeting to urge panelists to sup-
port the drug, relating experiences
of seeing snow, stars and the moon
for the first time. In most cases, the
patients' travel expenses were paid
for by the drug's developer.
Katelyn Corey, 24, described
her life before the treatment as "a
black and white film."
"Within days of the first treat-
ment I could see vibrant colors
again," Corey said. "I could see the
clock tower of Philadelphia City
Hall at night when mere days be-
fore I thought it was the moon."
Ashley Carper, a mother of two
children with the disorder, said
treatment has allowed her 11-year-
old son Cole to read large-print
books and write his own home-
work. Previously he could only
read with Braille. Cole said the im-
provements extend beyond school-
work.
"I can stay out later when my
friends are outside playing and
now I feel like part of the group,"
he said. "My vision is not perfect,
but what I do have is still really im-
portant to me."
Researchers tested the treat-
ment by recording patients' ability
to complete an obstacle course at
varying levels of light, simulating
real-world conditions like navigat-
ing a dark stairwell. A hallmark of
the inherited disorder is difficulty
seeing at night. Patients with the
mutation generally start losing
their sight before age 18, almost
always progressing to total blind-
ness.
One year after treatment 18 out
of 20 patients who received the
injection showed the maximum
improvement in completing the
obstacle course, and 13 completed
the task at the lowest level of light.
None of the patients in a compari-
son group did.
Doctors deliver the therapy with
an injection in each eye that inserts
a replacement gene into the retina
via a modified virus.
Philadelphia-based Spark Ther-
apeutics hopes to use its technol-
ogy to treat other retinal disorders,
including one called choroider-
emia, which affects about three
times as many people as the condi-
tion currently under FDA review.
The company is also investigating
therapies to treat the blood disor-
der hemophilia and diseases of the
nervous system.
As a potential breakthrough
therapy for a rare disease, much
of the discussion around Luxturna
has focused on its cost. Drugmak-
ers often price similar drugs at
$250,000 or more. Spark has not
given an estimated cost, which
companies usually announce only
after approval. But the company's
stock ticker symbol "ONCE" re-
inforces the company's hope that
this will be a one-time treatment
— and expense.
"It is the aspiration," Spark
Therapeutics CEO Jeff Marrazzo
told The Associated Press prior to
the meeting.
Pharmaceutical analyst Mi-
chael Yee estimates the drug will
be priced between $350,000 and
$450,000 per injection. He expects
sales of $40 million to $50 million
in 2018, growing to as much as
$100 million in 2019.
FDA advisers endorse gene therapy to treat form of blindness
AP
Dr. Barry Byrne listens to testimony concerning the approval of a potentially break-
through drug for a form of blindness during a meeting of the The Cellular, Tissue
and Gene Therapies advisory committee, Thursday, Oct. 12. 2017, at the FDA in
Silver Spring, Md.
NEW YORK (AP) —
America's weight problem
isn't getting any better,
according to new govern-
ment research.
Overall, obesity figures
stayed about the same:
About 40 percent of adults
are obese and 18.5 percent
of children. Those num-
bers are a slight increase
from the last report but the
difference is so small that
it could have occurred by
chance.
Worrisome to experts is
the rate for children and
teenagers, which had hov-
ered around 17 percent for
a decade. The 2-to-5 age
group had the biggest rise.
The years ahead will
show if that's a statistical
blip or marks the start of a
real trend, said the report's
lead author, Dr. Craig
Hales of the U.S. Centers
for Disease Control and
Prevention.
The bad news is that the
numbers didn't go down,
experts say. In recent
years, state and national
health officials have fo-
cused on obesity in kids,
who were the target of the
national Let's Move cam-
paign launched by former
first lady Michelle Obama
in 2010.
The report released Fri-
day covers 2015 and 2016.
"This is quite disappoint-
ing. If we were expecting
the trends to budge, this is
when they would be budg-
ing," said Andrew Stokes,
a Boston University expert
on tracking obesity.
The new figures are
from an annual govern-
ment survey with about
5,000 participants. The
survey is considered the
gold standard for measur-
ing the nation's waistline,
because participants are
put on a scale to verify
their weight.
Obesity means not
merely overweight, but se-
riously overweight, as de-
termined by a calculation
called body mass index .
Until the early 1980s, only
about 1 in 6 adults were
obese. The rate climbed
dramatically to about 1
in 3 around a decade ago,
then seemed to level off for
years.
More details from the
report:
—The 40 percent rate
for adults is statistically
about the same as the near-
ly 38 percent in the 2013-
2014 survey.
—By age, the fattest
adults are in their 40s and
50s. The obesity rate for
that age group is 41 per-
cent for men and 45 per-
cent for women.
—By race and gender,
the problem is still most
common in black and His-
panic women; more than
half are obese.
—Among children, the
rate for the 12-to-19 age
group was the same at
nearly 21 percent. For kids
6 to 11, it rose to 18 per-
cent, from 17 percent.
—But for children ages
2 to 5, the rate jumped to
14 percent from about 9
percent.
US obesity problem is not budging,
new data shows
AP
In this June 26, 2012 file photo, two women converse in New York.
New government figures released Friday, Oct. 13, 2017 showed
small increases that were not considered statistically significant but
were seen by some as a cause for concern. The adult obesity rate
rose from to about 40 percent, from just shy of 38 percent.
Puerto Rico disaster
may cause shortage
of hospital IV bags
(AP) — A key U.S. maker of hospital products said it
expects a temporary shortage of small saline bags because
of the hurricane that hit Puerto Rico.
The hurricane wiped out the island's electrical grid,
shutting down Baxter's three Puerto Rico factories for sev-
eral days. The Deerfield, Illinois-based company is still
ramping up production by using generators.
The Food and Drug Administration said Friday that it's
working with Baxter to limit any shortages, helping the
company get fuel and manufacturing supplies and ship
products. The FDA also is letting Baxter temporarily im-
port its saline "Mini-Bags," as they're called, from the
company's factories in Ireland and Australia.
The IV bags are widely used in hospitals to give patients
fluids and medicines.
Baxter is the biggest maker of small saline bags for
the U.S. market. A couple other companies make similar
products, but since 2014 there have been shortages across
the industry.
"Further shortage of this product could potentially put
U.S. health care at risk," FDA Commissioner Dr. Scott
Gottlieb said in a statement Friday.
Last week, the FDA said it's worried there could be
shortages of about 40 critical medicines because of the
disruptions caused by the hurricane at the dozens of drug
and device factories in Puerto Rico.
Baxter and other companies have said that before Maria
hit, they rushed to ship finished products off the island.
They've also been moving inventory around to try to boost
the supply for the U.S. market.