FDA Approves First Gene Therapy To Successfully Treat Cancer | NBC Nightly News


LITTLE GIRL. REHEMA ELLIS HAS BEEN FOLLOWING HER STORY.>>Reporter: TODAY’S LANDMARK DECISION STARTED WITH A YOUNG PIONEER, EMILY WHITEHEAD, NOW LITERALLY THE TEXTBOOK EXAMPLE OF THIS GROUNDBREAKING THERAPY. LOOK AT THIS FACE. THIS SAYS I’M HAPPY.>>COULDN’T BE MORE PROUD.>>Reporter: WE MET EMILY A YEAR AND A HALF AGO AT AGE 10, STILL CANCER-FREE AFTER BEING DIAGNOSED WITH AN ACUTE FORM OF LEUKEMIA WHEN SHE WAS 5.>>AT ONE POINT THE DOCTOR CAME IN AND SAID, YOU NEED TO CALL YOUR FAMILY IN BECAUSE EMILY’S NOT GOING TO BE HERE TOMORROW.>>Reporter: WHAT DOCTORS AT THE UNIVERSITY OF PENNSYLVANIA AND CHILDREN’S HOSPITAL OF PHILADELPHIA DEVELOPED THE CAR T CELL TREATMENT. EMILY WAS THE FIRST CHILD IN THE WORLD TO RECEIVE IT.>>SHE’S CANCER FREE. AND I COULDN’T BELIEVE IT. I THOUGHT IT WAS A DREAM.>>Reporter: HERE’S HOW IT WORKS. FROM A BLOOD SAMPLE, A PATIENT’S OWN IMMUNE CELLS ARE SEPARATED OUT AND THEN GENETICALLY REPROGRAMMED, TURNING THEM INTO SUPERFIGHTERS THAT HUNT OUT AND DESTROY CANCER CELLS. AT THE FDA HEARING TODAY, FIGHTING BACK TEARS, TOM WHITEHEAD TESTIFIED NEXT TO EMILY.>>IF YOU WANT TO SEE WHAT A CURE LOOKS LIKE, SHE’S STANDING RIGHT BESIDE ME.>>Reporter: THIS FALL THE FULL FDA IS EXPECTED TO APPROVE THIS TREATMENT FOR RELAPSING LEUKEMIA IN CHILDREN AND YOUNG ADULTS. IT’S GRUELING, BUT 83% OF PATIENTS WENT INTO REMISSION. THE ULTIMATE GOAL, TRY IT ON OTHER CANCERS.>>IT’S HARD FOR ME TO EVEN ARTICULATE HOW EXCITING THIS IS. THIS IS — EVERYBODY HAS BEEN WORKING TOWARD THIS FOR SO LONG.>>Reporter: FOR EMILY WHITEHEAD, TODAY’S DECISION IS ABOUT HOPE.>>IT’S KIND OF EXCITING BECAUSE IT WILL GET THE TREATMENT TO A LOT MORE KIDS.>>Reporter: A HISTORIC STEP IN

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