% IN
TONIGHT'S LOOK AHEAD
IT
IS
% NOT
A
CURE PER
SE
BUT
PROVING % TO
BE
LIFE
CHANGING.
% A
NEW
BREAKTHROUGH DRUG % COMBINATION
IS
OFFERING
HOPE
TO
% PATIENTS
LIVING
WITH
CYSTIC
% FIBROSIS, A
PULMONARY DISEASE
% IMPACTING 30,000 PEOPLE IN
THE
% UNITED
STATES.
% CYSTIC FIBROSIS
CAN
CAUSE
% SEVERE LUNG
DAMAGE,
DIFFICULTY
% BREATHING,
AND
EARLY
DEATH
DUE % TO
THICK,
STICKY MUCOUS
IN THE
% AIRWAYS.%THERE IS
NO
CURE
FOR
THE
% INHERITED DISEASE BUT
THANKS TO
% TEAMS
OF RESEARCHERS
AT
THE
UW
% AND
ACROSS
THE
COUNTRY, THE
NEW
% TRIAL
DRUG
THERAPY IS TARGETING
% THE
ROOT
CAUSE
OF
THE
DISEASE % AND
SHOWING
AMAZING
RESULTS.% IN A
MOMENT
WE'LL
HEAR
FROM
ONE
% PATIENT SHOWING SUCH %IMPROV
EMENT
IN
HER
CONDITION.
% BUT
FIRST
WE
TURN
TO
DR.
ANDREW % BRAUN
WHO
LED
UW'S
% PARTICIPATION IN THE PHASE
% THREE TRIALS OF
THIS
MEDICATION % PRIOR
TO FDA
APPROVAL.
% THE
DIRECTOR
OF
THE
UW
ADULT % CYSTIC FIBROSIS PROGRAM
AND
% THANK YOU
FOR
BEING
HERE.
% HOW IMPORTANT IS
THIS
% BREAKTHROUGH?
% >>
THIS
IS
A
MOMENTOUS
THERAPY
% FOR PEOPLE
WITH
CYSTIC FIBROSIS.% RIGHT NOW
AND
FOR
THE
LAST 30
% TO
40
YEARS WE'VE
REALLY BEEN
% TREATING THE SYMPTOMS
OF
THE
% DISEASE.%THIS
NEW
MEDICATION
TARGETS
THE
% UNDERLYING
GENETIC
DEFECT
AND
% WE
ARE
ALREADY
SEEING
QUITE
% AMAZING RESULTS
FOR PEOPLE
WHO
% HAVE BEEN
ABLE TO
ACCESS
THE
% MEDICATION.
% >>
WHAT
WAS
YOUR
INITIAL
% REACTION WHEN
YOU
SAW THOSE
% KINDS
OF
RESULTS?
% >> IT
IS
SOMETHING
THAT
WE
HAVE
% NEVER SEEN
IN
CYSTIC
FIBROSIS.
% THIS
MEDICATION
WILL
BE
ADDING
% LIKELY YEARS
OF
LIFE
TO
PEOPLE
% WITH
CF
AND
REALLY
IMPACTING % THEIR
QUALITY
OF
LIFE.
% CURRENTLY MOST
PEOPLE
SPEND
TWO
% TO
THREE
HOURS
A
DAY
DOING
% MULTIPLE
INHALED
RESPIRATORY % THERAPIES.%THIS M
EDICATION
IS
A BILL
% THEY'LL TAKE
ONCE
IN THE
% MORNING AND
ONCE
IN
THE
EVENING
% WILL
LIKELY
IMPACT
THAT QUALITY % OF
LIFE
DRAMATICALLY.
% >>
BECAUSE
IT'S
AN INHERISTED
% DISEASE IT'S TESTED
FOR
IN
% BABIES.
% WHEN
DO
MOST
PEOPLE SHOW % SYMPTOMS
OR
SIGNS?
% >> ALL
CHILDREN
IN
THE
UNITED
% STATES,
ALL
STATES
NOW
SCREEN
% FOR
CYSTIC FIBROSIS
AS
OF
2009.
% THE
UNIVERSITY
OF
WISCONSIN
LED
% THE
EFFORTS
FOR
NEWBORN
% SCREENING
AND
SINCE
THE
MID
% 1980s
THAT
PATIENTS
IN % WISCONSIN
HAVE BEEN
ABLE
TO BE
% DIAGNOSED
AT
BIRTH.
% A
FEW CASES
IN
ADULT
HOOD.
% THEY
WILL
KNOW WITHIN
A
WEEK OR
% TWO
OF LIFE.
% >> LET'S HEAR
FROM
ONE
OF
YOUR
% PATIENTS WHO HAS
RECEIVED THIS % TREATMENT.
% >>
I
THINK
COMPARED
TO
BEFORE
% IT'S
REALLY
HELPED
MY
QUALITY
% OF
LIFE
BEING ABLE
TO
DO
MORE % THINGS
ON
MY
OWN.
% BEING
ABLE
TO
DO
MORE
THINGS
% THAT I
ENJOY
WITH
EASE.
% TO
ME IT
ALMOST
FEELS
AS
CLOSE
% TO
A
CURE
AS
I
EVER
THOUGHT
WAS % POSSIBLE
IN MY
LIFETIME.
% BECAUSE IT'S
KIND
OF
GIVEN ME
% MY
LIFE
BACK.
% >>
SO
SHE
SAYS IT
FEELS
LIKE
AS
% CLOSE
TO
A CURE
AS
SHE
THOUGHT
% SHE
WOULD
EVER
HAVE
IN
HER
% LIFETIME
AND
IT
HAS GIVEN HER
% HER
LIFE BACK.
% HOW MEANINGFUL TO
HEAR
THAT?
% >> IT
IS
REALLY
AMAZING.% WE'VE
NEVER
SEEN
A THERAPY
LIKE
% THIS
BEFORE.
% THE
FACT
THAT
OUR
PATIENTS ARE % THE
NOW GOING
TO
BE
ABLE
TO
% LOOK
TOWARDS A
FUTURE THEY
% DIDN'T
THINK
THEY
WOULD HAVE
A
% QUITE
REMARKABLE.% THIS THERAPY
IS
GOING
TO
BE
% AVAILABLE
FOR
85
TO
90% OF
% PEOPLE
WHO
ARE
LIVING
WITH
CF
% AND
ON
APPROVAL
FOR
ONLY
% PATIENTS WHO
ARE 12
YEARS
AND % OLDER.
% WE
EXPECT CHILDREN TO
BE
ABLE
% TO
BE ACCESSING
THIS
MEDICATION
% HOPEFULLY
WITHIN THE
NEXT
FEW
% YEARS.
% BUT
THERE
ARE
STILL
10% OF
% PATIENTS WE
NEED
TO CONTINUE
TO
% FIGHT
FOR
AND
WILL
NEED
OTHER
% TYPES
OF
GENETIC THERAPIES
TO % HELP TO
DEVELOP
THAT
TYPE
OF % IMPROVEMENT IN
THEIR SYMPTOMS.
% >>
DESCRIBE
THE
IMPROVEMENT
% THAT
YOU
SAW
IN
THAT
PATIENT
OR
% OTHERS
GOING
FROM
PLACE
A
TO
% PLACE B WITH
THE
TREATMENT.
% >>
I
WOULD
CHARACTERIZE THAT
% PATIENT'S SYMPTOMS
AND
LUNG
% FUNCTION TO
BE
STARTED
TO
% DEVELOP
INTO
AN ADVANCED
OR
% SEVERE
STATE.% THIS
THERAPY
FOR
HER
HAS
% IMPROVED HER
BACK INTO
A
MUCH
% DIFFERENT STATE
OF
LUNG
% FUNCTION.
% SHE
IS
NOW
ABLE
TO
RECONSIDER
% INTERACTING WITH
THE THINGS
SHE
% LOVES
TO
DO ON
A
DAILY
BASIS.
% AND
IT
WILL
DRAMATICALLY
BE
% IMPROVING HER
QUALITY OF
LIFE
% GOING FORWARD.%>> ONE
OF
THE
THINGS
THAT
% PEOPLE WITH CYSTIC FIBROSIS %
HAVE TO
BE
CAREFUL
ABOUT
IS
% GETTING, SAY,
A COLD,
RIGHT?
% AND SO
THEY
HAVE
TO
KIND
OF
% LIMIT
WHERE
THEY GO AND
WHO
% THEY
SEE.
% >>
WE'LL
STILL
TELL
OUR
% PATIENTS WHAT
THEY
NEED
TO DO
% TO
STAY
HEALTHY.% STAY
AWAY FROM
PEOPLE
WHO
ARE
% SICK
AND
TAKING
GOOD
HYGIENE
% PRECAUTIONS.
% THIS THERAPY
IS
HIGHLY
% EFFECTIVE.%THEY'LL
BE
AT
RISK FOR
FUTURE
% CONCERNS RELATED TO
THE
DISEASE
% TODAY.%SO IT
IS NOT
A CURE
YET.
% BUT
YES,
IT IS IMPROVING % QUALITY
OF LIFE.
% >>
YOU
SAID
THIS WILL
BE
% AVAILABLE
TO
A
LARGE
NUMBER OF
% CYSTIC FIBROSIS
PATIENTS.% WHAT
ABOUT
RIGHT
NOW?
% >>
SO THE
FDA
APPROVED
IT 2
1/2
% WEEKS
AGO.
% WE'VE
BEEN PUTTING IN
% PRESCRIPTIONS AND
WE
ARE
% WAITING FOR
INSURERS
TO
LOOK
% INTO
AUTHORIZING THE
MEDICATION
% FOR
PATIENTS.
% IT
IS
INITIALLY
APPROVED
FOR
% PEOPLE WITH
A
DIAGNOSIS
OF
% CYSTIC FIBROSIS
WHO
ARE
12 % YEARS
AND
OLDER
AND HAVE
A VERY
% SPECIFIC
GENE
MUTATION.
% THAT
GENE
MUTATION,
WHICH
MANY
% PEOPLE
WITH
CYSTIC
FIBROSIS
% KNOW
THE
NAME,
INCLUDES
85
TO
% 90% OF
PEOPLE
WITH
CF.
% >>
HOW
LONG HAVE
RESEARCHERS
% BEEN WORKING
TOWARDS
THIS
DAY?
% >>
20
TO
25
YEARS OF
% BREAKTHROUGH
SCIENCE
THAT
HAS
% BEEN
FOSTERED
BY
THE
% INVOLVEMENT OF
MANY
PEOPLE
% RAISING MONEY
FOR
CYSTIC
% FIBROSIS,
THE
NATIONAL
CYSTIC
% FIBROSIS FOUNDATION THAT WAS
% CRITICAL IF
FUNDING SOME
OF
% THIS
RESEARCH
IN
THE
BEGINNING
% AND
IT
IS
BOTH
FROM
PATIENTS
% WHO PARTICIPATED IN RESEARCH
% TRIALS
AND
THE
COMMUNITY
THAT
% HAS