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Tribune Triple E

Tuesday, October 24, 2017

EEE-1

www.citizentribune.com

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.