Developmental Therapeutics at The Children’s Hospital of Philadelphia


>>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.>>IF YOU BELIEVE THAT YOU
HAVE A PATIENT AT HIGH RISK FOR RECURRENCE, IF YOU
HAVE A PATIENT WHOSE DISEASE YOU’RE NOT CONTROLLING WELL,
MEASURING, FOR INSTANCE, MINIMAL RESIDUAL DISEASE AND
IT’S NOT GOING DOWN QUICKLY ENOUGH, OR IF YOU HAVE A
PATIENT WHOSE JUST RELAPSED, WHAT I WOULD SAY IS
CONTACT US EARLY. WE WILL WORK WITH YOU. WE WILL LET YOU KNOW IF
WE DO HAVE ANY TREATMENT OPTIONS FOR YOUR PATIENTS,
EITHER AT THAT MOMENT IN TIME OR AT A LATER POINT.>>YOU DON’T WANT TO WAIT
UNTIL A CHILD HAS BEEN HOSPITALIZED FOR A
LONG PERIOD WITH MULTIPLE INFECTIONS, MAYBE HAVING
KIDNEY OR LIVER PROBLEMS FROM ALL THE COMBINED
CHEMOTHERAPY THEY’VE RECEIVED. THOSE KIND OF THINGS
ARE WHAT KEEPS A CHILD FROM GETTING
EXPERIMENTAL THERAPY.>>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 THE
REFERRING PHYSICIAN ABOUT WHAT MAY OR MAY
NOT BE AVAILABLE.>>WHAT WE TRY TO DO IS
GATHER AS MUCH INFORMATION ABOUT THE CHILD AND ABOUT
THEIR RELAPSED CANCER AS POSSIBLE. AND WE DO THAT SO THAT WE
CAN TRY TO FIND TREATMENTS THAT WILL TARGET THE
CAUSE OF THE RELAPSE, AND TO TRY TO, AS
MUCH AS WE CAN, INDIVIDUALIZE OR TAILOR THE
THERAPY FOR AN INDIVIDUAL CHILD AND HIS OR
HER CIRCUMSTANCES.>>CART19 IS THE FIRST
EXAMPLE OF ENGINEERING CELLS TO ATTACK CANCER THAT WE
FEEL ACTUALLY HAS IN VERY, VERY EARLY RESULTS SHOWN
SOME REAL ABILITY TO REMOVE LARGE AMOUNTS OF DISEASE
AND TO PUT PATIENTS WITH NO OTHER THERAPEUTIC
OPTIONS INTO REMISSION. SO THE ESSENTIAL NATURE
OF THE CART19 TREATMENT IS THAT WE HAVE TO GET
T CELLS FROM THE PATIENT. WE CAN ENGINEER THESE
CELLS SO THAT THEY BASICALLY RESTORE THE ABILITY OF THESE
CELLS TO GO AFTER CANCER CELLS AND KILL THEM. IN ORDER TO MAKE
THESE CELLS, WE HAVE TO HAVE
ACCESS TO THESE CELLS. AND SO IT IS ABSOLUTELY
THE CASE THAT IF IT IS CLEAR THAT YOU HAVE A PATIENT
FOR WHOM THERE’S A HIGH LIKELIHOOD OF THE
DISEASE RETURNING, OR THE DISEASE
HAS JUST RETURNED, THAT IF YOU TREAT THAT
PATIENT WITH INTENSIVE CHEMOTHERAPY MULTIPLE TIMES
AND THEN AT THAT POINT SAY MAYBE IT WOULD BE
APPROPRIATE TO CONSIDER CELL THERAPY IN THAT PATIENT,
WHAT WE’RE LEARNING IS THAT AT THAT POINT YOU ALSO DON’T
HAVE ANY T CELLS TO BE ABLE TO MANUFACTURE. SO I THINK THE REAL
CHANGE IN THINKING AND OUR CHALLENGE IN EDUCATING
FOLKS AND OUR OWN LEARNING ON THIS IS REALLY ABOUT THE
FACT THAT YOU HAVE TO THINK OF COLLECTING THE
CELLS IN ADVANCE. YOU HAVE TO THINK
OF IT IN ORDER. YOU HAVE TO THINK OF THE
COLLECTION OF THE CELLS FIRST, AND THEN YOU HAVE TO
THINK ABOUT GIVING CHEMOTHERAPY SECOND.>>WHEN A PATIENT OR
THEIR PHYSICIAN CONTACTS US, WE REALLY WANT TO
DISCUSS ALL THE OPTIONS. WE WANT TO EDUCATE THEIR
ONCOLOGIST AS WELL SO THAT THE ONCOLOGIST CAN ALSO
TALK WITH THE FAMILY. THAT DOCTOR IS SOMEBODY
WHO HAS TO BE ON BOARD WITH THIS NEXT STEP,
THIS EXPERIMENTAL THERAPY. WE NEED TO HAVE A GOOD
WORKING RELATIONSHIP WITH THE ENTIRE TEAM TO
GET THE CHILD THE BEST THERAPY POSSIBLE.>>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 IF THAT’S SOMETHING THAT ACTUALLY
MAKES SENSE FOR THE FAMILY AND WHAT THE REFERRING
DOC ACTUALLY WANTS TO DO, AND THEN IF WE DO NEED TO
COLLECT CELLS FOR POTENTIAL CELL THERAPY WE GET
THAT DONE QUICKLY. 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. SO IT’S REALLY AN ATTEMPT
TO WORK IN TANDEM WITH THE REFERRING PHYSICIAN,
DO THOSE THINGS THAT THE PATIENT CAN DO AT THEIR
REFERRING INSTITUTIONS, AND REALLY LIMIT OUR ROLE TO
THE THINGS WHERE WE REALLY HAVE THAT AVAILABLE
HERE BUT NOWHERE ELSE.