>> HERE AT THE CHILDREN'S HOSPITAL OF PHILADELPHIA,
WE ARE ONE OF THE LEADING INSTITUTIONS IN ENROLLMENT
ON EARLY PHASE CLINICAL TRIALS IN THE COUNTRY.
DEVELOPMENTAL THERAPEUTIC SECTION HERE IS A RESEARCH
TEAM THAT REALLY WORKS TOGETHER TO BRING NEW
THERAPIES TO CHILDREN WITH CANCER THROUGH
CLINICAL TRIALS.
OUR TEAM IS COMPOSED OF CLINICIANS,
PHYSICIAN SCIENTISTS, NURSES, SOCIAL WORKERS,
PHARMACISTS, AND A NUMBER OF OTHER STAFF
WHO REALLY HELP US COORDINATE THE CLINICAL
TRIALS SO THAT WE CAN BRING THEM SAFELY TO CHILDREN.
CLINICAL TRIALS ARE RESEARCH STUDIES WHERE WE ARE TRYING
NEW AGENTS IN CHILDREN WITH CANCER TO DETERMINE IF
THEY'RE SAFE, THEY HAVE ACTIVITY,
OR THEY PROVIDE BENEFIT TO CHILDREN WITH CANCER.
FOR MANY OF THE THERAPIES THAT WE USE IN CLINICAL TRIALS,
THEY ARE NOT FDA APPROVED AS SAFE
AND EFFECTIVE DRUGS YET.
SO WE OFTEN REFER TO THEM AS INVESTIGATIONAL DRUGS
OR NEW AGENTS.
>> A LOT OF THESE PATIENTS COME FROM ALL OVER THE COUNTRY
AND ALL OVER THE WORLD TO GET THESE DRUGS.
>> DEVELOPMENTAL THERAPEUTICS, ESPECIALLY IN CHILDREN,
HAS CHANGED DRAMATICALLY IN THE LAST 10 YEARS.
WE ARE NOW BRINGING NEW AGENTS TO CHILDREN MORE
QUICKLY THAN WE HAVE IN THE PAST,
AND MORE CHILDREN ARE RECEIVING NEW AGENTS
THAN EVER BEFORE.
>> SOMETIMES WHEN CHILDREN RELAPSE,
OR THEY RELAPSE A SECOND TIME,
THEIR DOCTORS SAY TO THEM,
"OH, YOU'RE NOT SICK ENOUGH FOR EXPERIMENTAL THERAPY."
BUT EXPERIMENTAL THERAPY IN THIS DAY IN AGE IS NOT JUST
MORE OF THE SAME; IT'S COMPLETELY NEW THERAPY.
YOU NEED EXPERIMENTAL THERAPY TO COME AT IT FROM
A DIFFERENT DIRECTION TO TRY TO ATTACK IT IN A COMPLETELY
DIFFERENT WAY THAN CLASSIC CHEMOTHERAPY
EVER ATTACKED IT.
>> THE KEY HERE, THE INTENT, IS TO PRESERVE THE OPTION
FOR AS MANY TREATMENT POSSIBILITIES FOR ANY GIVEN
PATIENT AS WE CAN.
AND THE EARLIER WE HEAR ABOUT IT, THE MORE WE
CAN ADVISE ABOUT WHAT MAY OR MAY NOT BE AVAILABLE.
>> IF FAMILIES WAIT TOO LONG AND THEY WAIT UNTIL
THE CHILD IS SICK ENOUGH TO RECEIVE EXPERIMENTAL
THERAPY, UNFORTUNATELY, THE CHILD OFTEN HAS ALREADY
HAD TOO MANY COMPLICATIONS TO BE ELIGIBLE TO GET THAT
EXPERIMENTAL THERAPY.
THAT DOCTOR THAT FIRST SAT DOWN WITH YOU AND TOLD YOU
YOU HAD LEUKEMIA IS A DOCTOR THAT'S REALLY SPECIAL,
AND IT'S SOMEBODY WHO YOU TRUST,
AND IT'S SOMEBODY WHO HAS TO BE ON BOARD WITH THIS NEXT STEP,
THIS EXPERIMENTAL THERAPY WITH YOU.
>> SO I THINK THE IDEA IS THAT WE HEAR EARLY ABOUT
PATIENTS WHO HAVE EITHER RECURRENT OR HIGH-RISK DISEASE,
WE HAVE ENTERED INTO A DIALOGUE OF WHAT
MIGHT OR MIGHT NOT BE APPROPRIATE,
WE SEE THE PATIENT IN SECOND OPINION HERE,
AND THEN WE LET THE PATIENT GO BACK TO THEIR TREATING
PHYSICIAN FOR ANY APPROPRIATE CHEMOTHERAPY
AND THEN BRING THEM BACK FOR ANY POTENTIAL EXPERIMENTAL
THERAPY THAT MIGHT BENEFIT THAT PATIENT.
>> WE'RE CONSTANTLY COMMUNICATING,
WHETHER BY PHONE OR BY E-MAIL, WITH YOUR ONCOLOGIST
WHO SENT YOU HERE BECAUSE THEY'RE THE ONES THAT ARE GOING
TO TAKE CARE OF YOU.
WE NEED TO HAVE A GOOD WORKING RELATIONSHIP
WITH THE ENTIRE TEAM TO GET THE CHILD THE BEST
THERAPY POSSIBLE.
>> WHEN SHE RELAPSED THE FIRST TIME WE CAME
TO SEE DR. RHEINGOLD AND SHE SAID THAT WE COULD STAY
AT OUR LOCAL HOSPITAL BECAUSE SHE WOULD RECEIVE THE SAME
TREATMENT THAT THEY WOULD OFFER HERE.
AND SHE DIDN'T FEEL AT THAT POINT THERE WAS ANYTHING
DIFFERENT THAT THEY COULD DO HERE.
SO WE DID STAY AT OUR LOCAL HOSPITAL,
BUT 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.
AND DR. RHEINGOLD AT THAT POINT SAID IT'S TIME TO COME
HERE TO CHOP.
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.
WE JUST HAD A REALLY -- A LOT OF HOPE,
AND FROM THE VERY BEGINNING WE JUST -- WE REALLY HAD
A GOOD FEELING ABOUT IT.
WE SAID, YOU KNOW, IT JUST HAS TO WORK.
IT HAS TO WORK FOR EMILY, AND IT DID.
>> SO WE'RE ALL WORKING TOGETHER TO TRY TO CURE
CHILDHOOD CANCER, BUT IT IS A TOUGH FIGHT.
AND FOR SOME FAMILIES THE BENEFIT COMES FROM KNOWING
THAT THEIR CHILD HAS PARTICIPATED IN THE CLINICAL
TRIAL AND HELPED IN THAT FIGHT.
EVERY CHILD WHO PARTICIPATES IN A CLINICAL STUDY
IS ACTUALLY HELPING TO FIGHT CHILDHOOD CANCER.
BUT IN REALITY, MOST OF THESE PATIENTS ARE NOT CURED
BY NEW THERAPIES.
WE ARE HOWEVER WORKING TO MAKE SAFER,
BETTER DRUGS FOR CHILDREN WITH CANCER.