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
IMPROVEMENT 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 MEDICATION 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