Emily Whitehead First Child Treated in Trial of T Cell Therapy for Acute Lymphoblastic Leukemia

Emily Whitehead First Child Treated in Trial of T Cell Therapy for Acute Lymphoblastic Leukemia


>>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? 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.