>> SHE RELAPSED IN OCTOBER 2011 AND AT THAT POINT WE
KNEW IT WASN'T KIND OF THE STANDARD, GARDEN
VARIETY LEUKEMIA.
THEN SHE RELAPSED AGAIN IN FEBRUARY 2012.
AND SO IT WAS AT THAT POINT THAT WE MADE THE DECISION
THAT WE NEEDED TO GO SOMEWHERE ELSE WHERE WE
NEEDED TO TRY SOMETHING NEW AND SOMETHING DIFFERENT.
>> WE WERE TOLD THAT WE WERE DOWN TO 48 HOURS FOR MAKING
A DECISION OR SHE COULD START TO HAVE ORGAN FAILURE.
>> WE KNEW THAT THERE WERE THINGS OFFERED HERE THAT WAS --
THAT WOULD BE DIFFERENT FROM WHAT OUR LOCAL HOSPITAL
COULD OFFER, AND THINGS THAT WERE MORE,
I GUESS, CUTTING EDGE.
NEWER THINGS WERE BEING TRIED THAT WEREN'T BEING
TRIED ANYWHERE ELSE.
>> SO WE'RE REALLY DOWN TO THE CART19 AS WHAT'S LEFT
FOR EMILY TO CURE HER.
SO WE'RE READY TO GO TODAY.
>> RIGHT NOW WE'RE FEELING HOPEFUL,
REALLY HOPEFUL.
IT'S A LITTLE BIT SCARY BECAUSE EMILY IS ONE
OF THE FIRST PATIENTS TO RECEIVE THIS.
SHE'S THE FIRST PEDIATRIC PATIENT.
>> I FELT ALL ALONG THAT SHE'S GOING TO BEAT CANCER
AND TODAY'S OUR DAY WHEN WE'RE GOING TO START DOING IT.
YOU WANT TO SAY ANYTHING ELSE, EM?
WHAT ARE WE GOING TO DO TODAY?
YOU'RE GOING TO GET YOUR T CELLS?
>> UH-HUH.
>> WHAT ARE THEY GOING TO DO?
>> DO YOU REMEMBER?
>> NUH-UH.
>> NUH-UH?
>> WHAT ARE THEY GOING TO TAKE AWAY?
>> CANCER.
>> CANCER, THAT'S RIGHT.
>> SO CART19 IS A TREATMENT FOR PATIENTS WITH FAIRLY
ADVANCED LEUKEMIA.
MOST PATIENTS WHO HAVE THE KIND OF LEUKEMIA THAT CART19
TREATS, WHICH IS THE MOST COMMON KIND OF CHILDHOOD
LEUKEMIA, IT'S CALLED B CELL ALL,
THAT IS A DISEASE WHERE MOST PATIENTS ARE TREATED VERY
SUCCESSFULLY WITH STANDARD CHEMOTHERAPY.
BUT THERE'S A SUBSET OF THOSE PATIENTS,
AND A SUBSTANTIAL NUMBER OF CHILDREN IN
THE UNITED STATES, FOR WHOM CHEMOTHERAPY DOES NOT WORK,
OR IT WORKS TEMPORARILY.
SO FOR THAT GROUP OF PATIENTS WHO HAVE VERY
TREATMENT RESISTANT DISEASE, WE NEED SOMETHING BRAND NEW,
AND CART19 IS FOR THOSE PATIENTS.
THE ESSENTIAL NATURE OF THE CART19 TREATMENT IS THAT WE
HAVE TO GET CELLS FROM THE PATIENT.
THESE CELLS ARE CALLED T CELLS.
THEY'RE CELLS OF THE IMMUNE SYSTEM THAT NEED
TO BE ENGINEERED.
WE CAN ENGINEER THESE CELLS TO GO AFTER CANCER CELLS
AND KILL THEM.
>> SO EMILY HAD HER -- THE CART19 THERAPY APRIL 17,
AND WE WERE TOLD TO EXPECT THAT SHE WOULD POSSIBLY GET
A LITTLE BIT OF A FEVER AND FEEL LIKE SHE HAD THE FLU.
BUT SHE ENDED UP GETTING PRETTY SICK SHORTLY AFTER,
AND SHE WAS PUT ON A VENTILATOR BECAUSE OF HOW
SICK SHE WAS.
>> THEY HAD COME WITH A TEAM OF DOCTORS IN THE PICU EVERY
MORNING AND DID ROUNDS WITH US,
AND WE WERE ALWAYS FIRST.
AND THEY WOULD COME TO US AND KIND OF SHOW US A LINE
ON THE FLOOR AND SAY, "IF THAT'S THE LINE OF SURVIVAL,
EMILY IS ALMOST STEPPED PAST IT."
>> DURING THE FEW DAYS WHEN SHE WAS QUITE SICK,
SHE WAS RECEIVING THE MOST ADVANCED MEDICAL CARE THAT'S
AVAILABLE ON THE PLANET.
SO THE PEOPLE IN THE HOSPITAL WERE TAKING CARE OF
THIS CHILD DURING THE PERIOD SHE WAS QUITE SICK UNTIL
SUCH TIME AS WE LEARNED WHAT WAS WRONG,
HAD A CHANCE TO INTERVENE, AND MADE HER BETTER.
ONE OF THE RESULTS OF HER T CELLS GROWING IN HER BODY
WAS THAT SHE HAD AN INCREASE IN VARIOUS PROTEINS
THAT CONTROLLED THE IMMUNE REACTION.
AND THIS INCREASE IN THOSE PROTEINS WAS ACTUALLY
MAKING HER ILL.
WE ACTUALLY FOUND THAT ONE OF THESE PROTEINS WAS QUITE
ELEVATED, AND AS IT TURNS OUT THERE'S A DRUG THAT
TARGETS THAT EXACT PROTEIN.
WE GAVE HER THAT DRUG AND SHE HAD A DRAMATIC RESPONSE.
THE ICU DOCTOR ON THAT NIGHT TOLD ME THAT HE HAD NEVER
SEEN A PATIENT THAT SICK GET BETTER THAT QUICKLY.
>> WE WEREN'T SURE AT THAT TIME IF THE CART19 CELLS
WERE WORKING, IF THEY WEREN'T WORKING.
>> ABOUT THREE WEEKS AFTER WE GAVE HER HER T CELLS,
WE DID ANOTHER BONE MARROW TEST.
THIS YOUNG LADY HAD NOT BEEN IN A REMISSION FOR A NUMBER
OF MONTHS AND SHE NOW WAS IN A COMPLETE REMISSION.
SO SHE COMPLETELY RESPONDED TO HER T CELL THERAPY.
>> WE WERE ABLE TO GO HOME JUNE 1 AND SINCE THAT TIME
SHE'S BEEN -- SHE'S BEEN GREAT.
SHE'S BEEN HAPPY AND HEALTHY AND RETURNED BACK TO SCHOOL
IN THE FALL.
YOU KNOW, CART19 WAS REALLY THE ONLY OPTION LEFT FOR EMILY.
AND ALTHOUGH WE WEREN'T -- WE WEREN'T SURE HOW IT WAS
GOING TO WORK BECAUSE SHE WAS THE FIRST CHILD,
WE STILL FELT LIKE BY ENROLLING HER IN THE TRIAL,
EVEN IF IT DIDN'T WORK, IT WOULD GIVE THEM A LITTLE BIT
MORE INFORMATION, AND FROM THAT HOPEFULLY THEY WOULD
LEARN SOMETHING AND BE ABLE TO HELP OTHER CHILDREN.
YOU KNOW, BUT WE ENTERED HER INTO THE TRIAL
REALLY HOPEFUL.
SHE'S EXTREMELY SMART.
SHE'S VERY CREATIVE.
SHE'S FUNNY.
SHE MAKES US LAUGH ALL THE TIME.
I MEAN, SHE'S -- SHE NEVER COMPLAINS.
>> SHE HAD TOLD US, YOU KNOW, FROM THE BEGINNING
THAT SHE WOULD CONTINUE TO FIGHT AND DO WHAT
WE ASK AS LONG AS WE WERE THERE WITH HER.
SO WE'VE BOTH BEEN THERE THROUGH THIS ENTIRE FIGHT,
ALL THREE OF US TOGETHER, AND WE JUST STICK TOGETHER
AS A TEAM.
AND SHE'S DEFINITELY OUR HERO.
>> WE CHECKED HER BONE MARROW FOR THE POSSIBILITY
OF DISEASE AT TWO POINTS.
WE CHECKED HER AT THREE MONTHS AND NOW SIX MONTHS
OUT FROM HER TREATMENT.
SHE HAS NO DISEASE WHATSOEVER.
WE NEED TO TREAT A LARGER NUMBER OF PATIENTS BEFORE WE
UNDERSTAND WHAT THE SUCCESS RATE MIGHT ACTUALLY BE.
IT SEEMS LIKE THE CELLS STICK AROUND FOR A LONG TIME;
THAT ACTUALLY MIGHT PROVIDE LONG-TERM DISEASE CONTROL.
THAT IS OF COURSE THE MOST EXCITING POTENTIAL PART.
BUT WE NEED MORE TIME TO KNOW IF THAT'S ACTUALLY
WHERE WE ARE.
TO SEE HER GO FROM LEAVING THE HOSPITAL AND STARTING
TO RECOVER TO BASICALLY GOING TO SCHOOL AND PLAYING SOCCER
AND LOOKING LIKE EVERY OTHER KID IS JUST WONDERFUL.
I THINK IT'S THE BEST THING ABOUT DOING WHAT I DO.