>> GOOD AFTERNOON.
MY NAME IS DR. SHANNA CLARK, THE
ASSOCIATE DIRECTOR OF THE CANCER
FOR CENTER RESEARCH'S OFFICE OF
EQUITY AND INCLUSION AND THE
SENIOR DIVERSITY ADVISOR TO THE
DIRECTOR.
I'M EXCITED TODAY TO INTRODUCE
YOU TO OUR GRAND ROUNDS SPEAKER
WHO I MET AT ASH ABOUT A YEAR
AGO AND WAS SO BLOWN AWAY BY HER
I KNEW WE HAD TO GET HER HERE.
DIRECTOR OF COMMUNITY
ENGAGEMENT, ASSOCIATE DIRECTOR
OF CELL AND GENE THERAPY IN THE
CLINICAL RESEARCH OFFICE AND THE
CHIEF DIVERSITY OFFICER OF THE
CENTER FOR CELL AND GENE THERAPY
AT BAYLOR COLLEGE OF MEDICINE.
THAT'S A LOT OF HATS.
HER RESEARCH AND CLINICAL
INTERESTS FOCUS ON REFRACTARY
MALIGNANCIES SPECIFICALLY HOW TO
HARNESS THE TRANSLATIONAL AND
CLINICAL RESEARCH PROGRAM
CREATING NOVEL EARLY-PHASE CELL
AND GENE THERAPY PRODUCTS AND
TRANSLATING THEM TO
FIRST-IN-HUMAN IMMUNOTHERAPY
TRIALS.
SHE ALSO SERVES AS PROJECT
LEADER FOR PROJECTS ON THE NIH
LYMPHOMA SPORE, LEUKEMIA AND
LYMPHOMA SOCIETY SPECIALIZED
CENTER OF RESEARCH PROGRESS AND
STAND UP TO CANCER, FOR WHICH
SHE RECEIVED A NCI INVESTIGATOR
TEAM LEADERSHIP AWARD.
SHE IS THE DIRECTOR OF THE C I
GT, CLINICAL RESEARCH OPERATIONS
AND THE IMMUNOTHERAPY
FELLOWSHIP.
SHE IS PASSIONATE ABOUT
ENHANCING THE NUMBER AND
DIVERSITY OF YOUNG INVESTIGATORS
IN CANCER RESEARCH AND HAS A
STRONG INTEREST IN HEALTH
DISPARITIES RESEARCH.
AS SUCH, SHE LEADS THE TASK
FORCE FOR PROMOTING DIVERSITY IN
CLINICAL TRIALS WITHIN THE DAN L
DUNK AN COMPREHENSIVE CANCER
CENTER.
SHE LEADS THE DEIA INITIATIVES
AT ASH, ASGST AND ASTCT AND
PRESENTED ON CAR T ACCESS TO
CONGRESS, COMBINING HER
COMPASSIONS FOR BOTH SCIENCE AND
ADVOCACY.
DR.EROUS RECEIVED NUMEROUS
AWARDS INCLUDING THE BAYLOR
COLLEGE OF MEDICINE'S DEPARTMENT
OF PEDIATRICS YOUNG INVESTIGATOR
AWARD, THE OUTSTANDING WOMEN IN
SCIENCE AWARD FROM THE
ASSOCIATION FOR WOMEN IN
SCIENCE, THE BAYLOR COLLEGE OF
MEDICINE MENTORSHIP AWARD AND
THE HOUSTON CHRONICAL HURRICANE
HARVEY HOMETOWN HERO AWARD, TO
NAME A FEW.
WITH THAT, I WILL INTRODUCE
DR. RAYNE ROUCE.
[ APPLAUSE ]
>> THANK YOU FOR NA WONDERFUL
INTRODUCTION.
I'M SO EXCITED TO BE HERE.
I GOT THE PLEASURE OF MEETING A
NUMBER OF THE FELLOWS OVER
LUNCH, WHICH IS ALWAYS AN
AMAZING THING TO DO TO SEE THE
FUTURE OF OUR FIELDS OF THE SO I
CHANGED MY TITLE SLIGHTLY.
I'M GOING TALK ABOUT, IF WE
BUILD IT, THEY WILL COME.
EXCEPT IF THEY CAN'T.
STRATEGIES TO ENHANCE ACCESS TO
GENE-MODIFIED CELL THERAPIES.
HERE ARE MY DISCLOSURES.
ALL OF THESE RELATIONSHIPS HAVE
ENDED.
SO, I'M GOING TO START BY
SUMMARIZING RECENT ADVANCES.
THEY DESCRIBE SOME OF THE UNIQUE
CHALLENGES OF NOVEL CANCER CELL
AND GENE THERAPIES THAT MAKE
PARTICIPATION OF BOTH
UNDERREPRESENTED AND UNDER
RESOURCED PATIENTS CHALLENGING
AND THEN ASSESS STRATEGIES TO
INTENTIONALLY INCREASE ACCESS TO
EARLY PHASE CANCER CELL AND GENE
THERAPY CLINICAL TRIALS THAT ARE
APPLICABLE TO ANY SORT OF CANCER
CLINICAL TRIAL.
IN 2024, THE JOURNEY TO A CANCER
CURE AND THE ROAD BLOCKS ARE NEW
AND UNIQUE.
WE ARE USING MUTATION TARGETED
THERAPY.
WE HAVE THE ABILITY TO SEQUENCE
PATIENT'S TUMORS.
WE ARE USING MONOCLONAL
ANTIBODIES OFTEN ALONE BUT IN
COMBINATION WITH OTHER
THERAPIES.
WITH THE THIS QUEST FOR
PERSONALIZED CANCER CARE, WE SEE
NEW AND UNIQUE ROAD BLOCKS.
MY RESEARCH IN CLINICAL ASPECT
IS FOCUSED ON CART T-CELL
THERAPY.
AND SO I USED TO HAVE TO REALLY
GO INTO A LOT OF DETAIL
EXPLAINING WHAT A CAR-T CELL.
WE ARE ABLE TO EN IN EAR T CELLS
TO EXPRESS ARTIFICIAL RECEPTORS
TO SPECIFICALLY TARGET AN
ANTIGEN ON THE SURFACE OF A
CANCER CELL.
WE USUALLY USE A VIERS THAT
CARRIES THAT NEW GENE INTO THE
T-CELL AND CAUSES ALL OF THE T
CELLS TO EXPRESS THIS ARTIFICIAL
RECEPTOR WHICH CAN ULTIMATELY
FIND ITS TARGET ALMOST LIKE A
GPS TRACKING DEVICE, TARGET THAT
CANCER CELL FOR KILL, BECOME
ACTIVATED AND GO TO, IN MANY
CASES, ERADICATE CANCERS.
IN ORDER TO CREATE THESE CELL
THERAPIES, WE TYPICALLY START
WITH COLLECTING BLOOD FROM THE
PATIENT.
WE CAN DO THIS BY PERIPHERAL
BLOOD, BUT WE CAN ALSO DO THIS
THROUGH LEUKOPHORESIS ISOLATING
OUT THE WHITE BLOOD CELLS AND
THEN ISOLATING THE MONONUCLEAR
CELLS.
THEN FROM THAT, FEEDING WITH
SPECIFIC STIMULANTS THAT T CELLS
LOVE, AND THEN PROVIDING THIS
TRANSDUCTION WHERE WE
ESSENTIALLY PLACE THIS NEW GENE
INTO THE T CELLS AND EXPANDING
THESE CELLS OUT IN CULTURE TO
MEET A CERTAIN DOSE TO GIVE TO
PATIENTS AND WE START WITH THIS
KIND OF REALLY POPULATION OF
LOTS OF DIFFERENT WHITE BLOOD
CELLS.
THEN WE IN FUSE TO A PATIENT.
SO THE STATE OF THE CELL THERAPY
LANDSCAPE IN 2024 IS VERY
DIFFERENT THAN IT WAS WHEN I
STARTED IN THIS SPACE IN ABOUT
2011, 2012.
AND WE REALLY HAVE HAD SO MANY
SCIENTIFIC ADVANCE SYSTEM.
WE HAVE SIX FDA APPROVED CAR-T
CELL AND NOW OVER THE DINNER
TABLE, YOUR PARENT MIGHT KNOW,
YOUR GRANDPARENT MIGHT KNOW WHAT
A CAR-T CELL WAS.
WE SEEN EXPANSION BEYOND BLOOD
CANCERSES AND BEYOND T CELLS.
WE ARE USING NATURAL KILLER
CELLS AND MSCs AND WE RECENTLY
HAVE SEEN CURATIVE GENE THERAPY
APPROVALS FOR
HEMOGLOBINOPATHIES.
WE ARE FANCY NOW.
WE DON'T JUST DO
FIRST-GENERATION STUFF.
WE ARE EDITING AND SNIPPING,
BRINGING OUT OUR T CELLS.
WE CAN DO LOTS OF THINGS TO MAKE
THESE CELLS PERSIST LONGER IN
THE BODY.
WE CAN PUT IN KILL SWITCHES SO
WHEN THEY START TO ACT UP AND
GET OVER EXCITED, WE CAN TURN
THEM OFF.
SO WE ARE ABLE TO DO LOTS OF
THINGS THAT WE RECOGNIZED WE
NEED TO DO FROM BENCH TO BEDSIDE
TRANSLATION AND THEN BACK TO
BENCH.
WE HAVE LOTS EVER BEST PRACTICE
GUIDE LINES NOW ENHANCING
SAFETY.
WHEN I FIRST STARTED IN THIS
FIELD, MY FIRST PATIENT HAD
CYTOKINE RELEASE SYNDROME, IT
WAS BEFORE THE PATIENT HAD
CYTOKINE RELEASE SYNDROME.
WE GAVE A TON OF STEROIDS,
KILLED ALL THE T CELLS AND NOW
WE HAVE ALGORITHMS.
WHEN ANYONE CAN WALK INTO THE
ROOM AND SEE A PATIENT AND
RECOGNIZE THEY ARE HAVING
SPECIFIC ADVERSE EVENT RELATED
TO CAR-T CELLS AND KNOW WHAT TO
DO TO TAKE CARE OF IT.
AND WE ARE LOOKING AT SHARED
CARE MODELS THAT ALLOW TREATMENT
CLOSER TO HOME AND DEVELOPMENT.
BEING HERE AT THE NIH WHERE YOU
ALL ARE LITERALLY LEADING
RESEARCH AND HAVE ACCESS TO SO
MANY GROUNDBREAKING THERAPIES
AND HAVE PEOPLE TRAVEL FROM ALL
OVER THE COUNTRY AND THE WORLD,
IT'S SIMILAR TO HOW WE ARE AT
BAYLOR COLLEGE OF MEDICINE WHERE
A LOT OF OUR TRIALS ARE THE ONLY
ONES LIKE THEM IN THE WORLD.
WE HAVE PEOPLE COMING FROM ALL
OVER.
SO IT IS IMPORTANT FOR US TO
THINK ABOUT HOW WE CAN DO SHARED
CARE MODELS SO WE DON'T REQUIRE
PEOPLE TO UPROOT FOR SIX MONTHS
TO HAVE ACCESS TO THESE
THERAPIES.
HOWEVER, THE REALITY IS, WE
STILL ARE UNABLE TO GET THESE
THERAPIES TO MANY
UNDERREPRESENTED AND UNDER
RESOURCED OR GEOGRAPHICALLY
DISTANT PATIENTS.
SO AT TEXAS CHILDREN'S, WE HAVE
A CENTER FOR CELL AND GENE
THERAPY THAT HAS BEEN AROUND FOR
ABOUT 30 YEARS.
WORKING IN HERPES VIRUSES,
VIRUS-SPECIFIC T-CELLS AND THEN
CAR-T CELLS.
AND WE HAVE ALWAYS HAD A NUMBER
OF ACTIVELY ENROLLING TRIALS.
THE FIRST TIME I LEARNED ABOUT A
CAR-T CELLS WAS TARGETING HER2
ON A BREAST CANCER.
TARGETING GD-2 ON A
NEUROBLASTOMA.
NOT THE CAR-T CELLS THAT HAVE
COME TO THE FOREFRONT TARGETING
CD19.
THIS WAS A REALLY COOL
FORTUITOUS PLACE FOR ME TO BE AT
HOUSTON, AT BAYLOR COLLEGE OF
MEDICINE BECAUSE I HAD THE
OPPORTUNITY TO DO RESEARCH
THERE.
AND THE MAJORITY OF THE TRIALS
THAT WE DO ARE FIRST-IN-HUMAN,
THE MAJORITY ARE INVESTIGATOR
INITIATED.
SO OFTEN COME FROM FELLOWS WHO
HAVE BEEN IN OUR PROGRAM AND
THEN STAY ON AS FACULTY.
AND WE OFTEN ARE ATTEMPTING TO
TARGET CANCERS THAT HAVE PROVEN
TO BE FAR MORE DIFFICULT TO
TARGET THAN SOME OF THE ONES
LIKE B-CELL MALIGNANCIES.
AND WE ARE MOVING BEYOND CAR-T
CELLS AND BEYOND CANCER.
SO WE ALSO HAVE PROTOCOL THAT IS
TARGET AUTOIMMUNE DISEASES AND
WORK WITH A NUMBER OF DIFFERENT
INVESTIGATORS.
AND I'LL SAY THAT I PUT THIS IN
HERE THAT WE HAVE ALL OF THIS
HISTORY WITH THESE STUDIES
BECAUSE I WANT TO POINT OUT THAT
IT TAKES A FULL-BLOWN VILLAGE OR
TWO OR THREE, TO GET SOME OF
THESE TYPES OF THERAPIES FROM
BENCH TO BEDSIDE AND I WAS
FORTUNATE TO WORK LONG SIDE
PEOPLE WHO COULD HELP ME DO
THAT.
SO, ONE OF THE CLINICAL TRIALS
THAT I'M MOST PROUD OF IS A CD5
CAR-T CELL FOR T-CELL
MALIGNANCIES.
ANYONE WHOEVER TAKEN CARE OF A
PATIENT WITH RELAPSED OR
REFRACTARY LYMPHOMA, KNOWS HOW
AGGRESSIVE AND HORRIBLE THE
OUTCOMES ARE IF YOU RELAPSE.
SO IT TOOK A LOT TO GET THIS TO
THE CLINIC.
WE WOULD CREATE THESE CAR-T
CELLS.
THEY WOULD KILL EACH OTHER IN
THE CULTURE BECAUSE THEY ARE
LIKE, YOU EXPRESSED MY ANTIGEN
I'M TARGETING.
I FEEL YOU.
WE COULDN'T EVEN GROW THE CELLS.
WE HAD TO DO ALL KINDS OF
DIFFERENT THINGS TO TRY TO MAKE
THE CELLS GROW.
AND WE FINALLY GOT IT.
AND WHAT WE LEARNED FROM OUR
PRECLINICAL WORK IN MICE IS
THESE CELLS WEREN'T GOING TO
LAST INDEFINITELY THE WAY CD19
CAR-T CELLS LASTED.
AND BECAUSE T-CELL LEUKEMIA AND
LYMPHOMAS ARE SO AGGRESS I WAS,
WE NEEDED TO HAVE A SAFEGUARD
THAT PEOPLE SHOULD GO ON TO AN
ALLOTRANSPLANT IF THEY ACHIEVE
REMISSION.
AND WE INITIALLY REALLY WANTED
TO MAKE SURE THAT WE WERE
TARGETING TUMORS THAT BROADLY
EXPRESSED CD5 AND THE FDA AND US
AGREED THAT THIS WAS THE
FIRST-IN-HUMAN TRIAL WE NEEDED
TO HAVE TRANSPLANT AS A
SAFEGUARD IN CASE OUR CAR-T
CELLS WENT CRAZY AND STARTED
TARGETING NORMAL T CELLS PRONG
LONGED AND CAUSED T-CELL
APLASIA.
SO I POINT THIS OUT BECAUSE IT
IS STRATEGY FROM WHAT WE LEARNED
AT THE BENCH.
WE KNEW WE NEEDED TO WRITE IT
INTO OUR TRIAL TO MAKE SURE IT
WAS SAFE.
AND SO, THIS TRIAL WE NOW
TREATED 26 PATIENTS ON.
WE HAVE BEEN SURPRISED AT HOW
GOOD THE OUTCOMES ARE.
BECAUSE WE OPERATE ALMOST AS
WHAT I LIKE TO CALL A
CENTRALIZED ACADEMIC MODEL WHERE
WE ARE SIMILAR TO THE NIH IN THE
SENSE THAT PEOPLE WILL OFTEN
COME TO US BUT BECAUSE WE CAN
MAKE THESE CELLS WITHOUT THE
PROCESS OF LUKE PHORESIES, WE
CAN SEND A KIT BY FED-EX AND HAS
THE TUBES AND CONSENT AND THIS
WAS SOMETHING THAT WAS REALLY
IMPORTANT FOR US BECAUSE WE
WANTED TO MAKE SURE THAT
PATIENTS DID NOT HAVE TO UPROOT
AND COME TO US TO GET FERRIESED
AND GO BACK AND HAVE TO WAIT FOR
THE CELLS.
WE SEND A KIT.
YOU CAN SEND PERIPHERAL BLOOD
DRAWN FROM A VEIN OR EXISTING IV
OR LINE BACK TO US.
WE MANUFACTURE THE CELLS AND WE
HAVE A COUPLE OF STUD -ES THAT
ARE MULTICENTER OF THE SO PEOPLE
DON'T HAVE TO TRAVEL TO THE HEAT
AND HUMIDITY OF TEXAS IN AUGUST
TO GET THEIR CELLS, WHICH ANYONE
WHOEVER BEEN THERE KNOWS IT'S
REALLY IMPORTANT.
THE BREAKDOWN OF THE TRIAL IS
SIMILAR TO MOST CAR-T CELL
TRIALS.
WE PREP THE BODY, GIVE A COUPLE
OF DAYS OF CHEMO TO GET THE
HOMEOSTATIC CYTOKINES TO HELP
THE CAR-T CELLS GROW AND EXPAND
IN THE BODY.
WE IN FUSE THE CELLS AND DO LOTS
OF CORRELATIVE ANALYSIS AND THEN
WE CHECK RESPONSE.
AND WE HAVE HAD REMARKABLE
RESPONSES.
I'M GOING TO TRY TO USE VERY
CLEAR INSTRUCTIONS OUR AV FOLKS
GAVE ME ABOUT THIS POINTER.
SO YOU CAN SEE PRE CAR T, YOU
DON'T HAVE TO BE A RADIOLOGIST
TO SEE THIS HUGE LIMPOPATHY,
REFRACTARY T-CELL LYMPHOMA FROM
A PATIENT WHO IS VERY HEAVILY
PRE TREATED.
THIS IS PRE-CAR T.
AND JUST 4 WEEKS AFTER
COMPLETELY ERADICATED BY CAR-T
CELLS.
WE SEEN REALLY PROMISING
RESPONSES, ESPECIALLY IN
PATIENTS WITH MATURE T-CELL
LYMPHOMA, A LITTLE BIT OF A
DIFFERENT BIOLOGY THAN IMMATURE
AND ALSO BENCH TO BEDSIDE
CHANGED OUR MANUFACTURE BECAUSE
WE FOUND THAT WHEN WE LEFT THESE
CELLS IN CULTURE EVEN JUST FOR A
WEEK AFTER TRANSDUCING WITH THE
CAR, THEY WERE BECOMING OVERLY
EXHAUSTED.
THEY WERE TOO EXHAUSTED, HAVING
PARTIES, GETTING TIRED AND WE
PUT THEM IN THE BODY AND THEY'RE
LIKE, WE'LL JUST TAKE A NAP.
WE HAD TO SHORTEN THE
MANUFACTURE.
WE ADDED CHEMICAL IN HI
ABOUTITION TO STOP THEM FROM
GETTING OVER EXCITED WITHOUT
THEIR TARGET.
SO WE ALSO HAVE IA NUMBER OF
STUDIES TARGETING OTHER
MALIGNANCIES THAT ARE
REFRACTARY.
WE HAVE A CD30 CAR-T CELL TRIAL.
YOU ALSO HAVE THAT HERE AT THE
NIH.
WE HAVE BOTH HAD VERY PROMISING
RESULTS WHERE WE ARE SEEING
PATIENTS THAT HAVE EVIDENCE OF
DISEASE THAT IS COMPLETELY
ERADICATED.
AND, WHAT HAS BEEN REALLY
CHALLENGING, ANYONE WHO IS
INTERESTED IN CELL AND GENE
THERAPIES, WHICH I KNOW A COUPLE
OF FELLOWS ARE, YOU KNOW HOW HOW
CHALLENGING IT IS TO USE THESE
THERAPIES TO TARGETS SOLID
TUMORS AND BRAIN TUMORS.
LEUKEMIA IS JUST FLOATING AROUND
IN THE BLOOD, HANGING AROUND IN
THE BONE MARROW.
MY SOLID TUMORS ARE LIKE, YOU DO
LEUKEMIA.
THERE IS SO MUCH STUDY.
EVEN IF I HAVE AMAZING OUTCOMES,
THEY ARE LIKE, WELL -- TOUCHE,
OKAY?
BECAUSE CAR-T CELLS AND CELL
INDUCED THERAPIES TO GET INTO
THE SOLID TUMOR, YOU HAVE TO
DEAL WITH A EXTREMELY HOSTILE
ENVIRONMENT.
THERE ARE SECURITY OUTSIDE,
THERE IS GANG MEMBERS TRYING TO
TAKE YOU OUT.
IT'S REALLY HARD.
WE SEEN PROMISING RESULTS IN
SOLID TUMORS WHERE THIS IS A
PATIENT WITH GLIOBLASTOMA WHICH
WE KNOW HOW TERRIBLE IT IS.
AND JUST SEEING STABLE DISEASE
AND SEEING STRINGAGE CAN OFTEN
BE A WIN.
AND WE SEEN THIS IN
NEUROBLASTOMA AND RIBOMY
SARCOMA.
A LOT OF THE FIELD SAYING WE CAN
TARGET B-CELL MALIGNANCIES BUT
WE HAVE TO THINK ABOUT THESE
OTHER TUMORS AND WE HAVE TO
THINK ABOUT WAYS WE CAN MAKE OUR
CAR-T CELLS MORE POWERFUL, WAYS
WE CAN COMBINE THE THERAPY WITH
RADIATION, WHICH CHECKPOINT
INHIBITION TO PRIME THE SPACE.
WE ALSO HAVE TO DO THE OFF THE
SHELF -- DO YOU HAVE COSTCO?
SAMS?
OKAY.
THE COSTCO EDITION WHERE YOU CAN
SAY, MY PATIENT HAS THIS, I NEED
THIS AND THAT.
SO WE HAVE A LOT OF OFF THE
SHELF PROTOCOLS.
ONE OF THE FIRST I DID WAS OFF
THE SHELF.
WE USED PARTIAL HLA MATCHING BUT
WE DO VIRUS-SPECIFIC T CELLS
BECAUSE THAT NATIVE T-CELL
RECEPTOR WILL BE TRAINED TO
TARGET VIRUS.
SO SHOULD NOT CAUSE
GRAFT-VERSUS-HOST DISEASE.
AND THEN YOU CAN EQUIP THE CELL
WITH VIRUS SPECIFIC T CELLS THAT
HAVE A CAR ON THEM.
WE CAN PUT THEM INTO THE BODY
AND HAVE GOOD RESPONSES.
THE COOL THING ABOUT THIS IS
THAT IT'S NOT AS PERSONALIZED.
YOU CAN SCALE IT UP.
YOU DO REPEAT DOSING AND YOU CAN
LITERALLY DERIVE UNIVERSAL
PRODUCTS FROM SINGLE OR FEW
HEALTHY DONORS.
THERETO IS IT A LOT OF CRISPR
EDILATING TO EDIT OUT THE TCR
AND EQUIP YOUR CELLS WITH THE
ABILITY TO NOT CAUSE
GRAFT-VERSUS-HOST DISEASE IN A
PATIENT, BUT ALSO TO NOT GET
MEDICINALLY REJECTED BY
SOMEONE'S IMMUNE SYSTEM.
AND SO AT OUR CENTER, ONE OF THE
BIG PUSHES AND THINGS WE DO IS
TRY TO MAKE THESE CELLS FROM
PERIPHERAL BLOOD AS MUCH AS
POSSIBLE BECAUSE HOW MANY OF
Y'ALL IN HERE HAVE KIDS?
A FEW PEOPLE.
I DON'T HAVE CHILDREN BUT I'M
LIKE A CHILD.
SO ME HAVING TO GET UP AND GET
ON THE PLANE, MAKE IT TO THE
AIRPORT ON TIME, GO TO GET A
FREEZEIS, IT'S A LOT.
WE TRY TO THINK ABOUT WHAT THE
PATIENT AND FAMILY NEEDS AND THE
FACT THESE PATIENTS REFERRED TO
US ARE VERY WELL TAKEN CARE OF
AT THEIR HOME INSTITUTION BY
THEIR HOME PROVIDERS AND THEY
ARE PERFECTLY CAPABLE OF BEING
ABLE TO SEND THAT PERIPHERAL
BLOOD TO US.
SO THIS ALLOWS US TO SEND THESE
KITS, GET THE BLOOD SENT BACK TO
US, MANUFACTURE AND WE STILL ARE
IN THAT CENTRALIZED ACADEMIC
MANUFACTURE WHERE YOU STILL HAVE
TO TRAVEL TO US FOR THE PHASE I
TRIALINGS, WHICH IS PROBABLY
APPROPRIATE.
BUT IT ALLOWS US TO TARGET A
BUNCH OF DIFFERENT TYPES OF
CANCER.
WE HAVE A HUGE CENTER WITH LOTS
OF FOLKS WORKING ON THESE.
SO, I WANTED TO -- LOOKING AT
ONE STUDY AND LOOKING AT A YEAR
IN HOW FAR PATIENTS COME FROM.
SO TEXAS IS OBVIOUSLY QUITE
LARGE.
THE DARKER THE BLUE THE MORE
PATIENTS THAT COME FROM THERE.
WHILE MOST OF THE PATIENTS MAY
COME FROM HOUSTON, 21% CAME FROM
THE REST OF TEXAS BUT 28% FROM
22 OTHER STATES.
IT'S NOT BECAUSE THEY WANTED TO
COME TO TEXAS IN AUGUST.
IT'S BECAUSE THEY LITERALLY HAD
NO OTHER TREATMENT OPTIONS THAT
WERE VIABLE.
AND SO, IT ALWAYS STRUCK ME THAT
WE'RE ABLE TO DO THIS REALLY
FANCY SCIENCE BUT WE STILL ARE
LITERALLY UPROOTING PEOPLE FROM
THEIR LIVES TO COME AND TO
RECEIVE THESE THERAPIES.
AND SO PATIENT ACCESS TO CELL
THERAPIES INCLUDING CAR-T CELLS,
IS TRULY AN UPHILL BATTLE AND
THERE ARE SO MANY ROAD BLOCKS
THAT ARE ANTICIPATED AND
UNANTICIPATED AND NOT JUST FOR
THE PATIENTS, BUT ALSO FOR THE
PROVIDERS.
AND THEN TRICKLE DOWN TO THE
RESEARCHERS.
SO I'M CONSTANTLY TALKING WITH
MY BASIC SCIENCE COLLEAGUES
ABOUT THE FACT THAT WE ARE
READY.
WE ARE OPEN.
WE ARE READY TO GET THE TRIAL
GOING.
AND THEN THEY ARE LIKE, WHY
HAVEN'T WE ACCRUED?
WHY HAVEN'T WE HAD THIS?
WE HAVE TO TALK ABOUT SOME OF
THESE BARRIERS SO IT MATTERS TO
EVERYONE.
SO I THINK EVERYONE IS FAMILIAR
WITH THE JOURNEY THAT THE CELLS
TAKE.
WE COLLECT THE CELLS, WE SEND
THEM TO A MANUFACTURING
FACILITY.
IF IT'S OUTSIDE MANUFACTURE FOR
ONES WE ARE MANUFACTURING
OURSELVES.
WE PUT THE CAR GENE IN THERE.
WE GROW THE CELLS AND GIVE THEM
BACK.
BUT, I WANT TO TALK A LITTLE BIT
ABOUT WHAT HAPPENS TO THE
PATIENT AND THE PROVIDERS.
SO FIRST THE PREPARATION.
SO IF YOU EVER HAD A REFRACTARY
PATIENT THAT IS ELIGIBLE FOR A
CLINICAL TRIAL THAT IS NOT
AVAILABLE AT YOUR SITE, YOU KNOW
THAT YOU AND YOUR NURSE
COORDINATOR AND YOUR ATTENDING
AND YOUR FELLOW AND YOUR SOCIAL
WORKER AND YOUR FINANCIAL
COUNCILOR HAVE A LOT OF STUFF TO
DO TO WORK WITH THAT FAMILY TO
TRY TO IDENTIFY EVEN A TREATMENT
SITE.
YOU KNOW THERE MAY NOT BE A SPOT
AVAILABLE ON A CLINICAL TRIAL
ESPECIALLY IN EARLY PHASE I.
THEN YOU HAVE TO COORDINATE THE
COLLECTION WHETHER IT'S
HAPPENING AT YOUR SITE OR
SOMEWHERE ELSE.
AND YOU OFTEN HAVE TO GET
INSURANCE APPROVAL.
SO EVEN THOUGH I RUN CLINICAL
TRIALS THAT ARE QUALIFIED
CLINICAL TRIALS THAT THE ACTUAL
RESEARCH IS COVERED BY THE
CLINICAL TRIAL, YOU STILL OFTEN
HAVE TO GET COVERAGE FOR ROUTINE
CLINICAL COSTS ASSOCIATED WITH
THAT CLINICAL TRIAL.
FOR A PATIENT THAT IS COMING
FROM OUT OF STATE AND OUT OF
NETWORK, ALMOST UNIVERSALLY THE
FIRST ANSWER IS, ABSOLUTELY NOT.
AND Y'ALL ARE AT THE NIH WHERE
YOU COVER A LOT OF DIFFERENT
THINGS BUT I'M GOING TO TALK
ABOUT THAT TOO BECAUSE IT'S NOT
AS OPEN AND SHUT.
BUT THE POINT IS THE TREATING
PROVIDERS, PATIENTS AND
CAREGIVERS, THEY HAVE VERY
LIMITED CONTROL IN THIS ASPECT
OF THE PROCESS.
THEN, YOU'RE LIKE, OKAY, WE MADE
IT.
WE COLLECTED THE CELLS.
OKAY.
NOW WE'RE IN THE MANUFACTURE
PERIOD, RIGHT?
AND ANYONE WHO HAS REFERRED
SOMEONE FOR A CELL THERAPY
CLINICAL TRIALS KNOW SYSTEM THE
AMOUNT OF TIME QUOTED TO YOU CAN
VARY GREATLY.
WELL, IT TAKES A WEEK AND A HALF
TO MAKE THE CELLS BUT THEN WE
HAVE TO DO A CULTURE FOR
AUTONOMOUS GROWTH, THREE WEEKS,
QUALITY ASSURANCE THAT WILL TAKE
THIS MUCH TIME.
SO IT OFTEN WILL TAKE 4-12
WEEKS.
THESE ARE REFRACTARY PATIENTS.
SO WE KNOW WHAT IS HAPPENING
DURING THAT TIME POINT.
BUT IMPORTANTLY, THE PRIORITY IS
OFTEN DEPENDENT ON THE ORDER OF
REFERRAL NOT THE CLINICAL NEED.
SO EVEN IF IT'S AN
INVESTIGATIONAL TRIAL AND YOU'RE
TALKING TO THAT INVESTIGATOR, IT
IS VERY COMMON TO SAY HOW MANY
PATIENTS ARE ON THE WAIT LIST
FOR THIS PHASE I TRIAL TO TREAT
UP TO 26 PATIENTS?
WELL, WE HAVE 9 PATIENTS ON THE
WAIT LIST.
FOR YOUR PATIENT AT THAT MOMENT,
THAT MAY SEEM LIKE IT'S NOT
REALLY AN OPTION.
AND THE MORE COMPLEX, WHEN WE
GET FANCY IN THE LAB AND WE'RE
EDITING AND SOUPING UP THE CAR-T
CELLS, THE MORE REGULATORY
COMPLEXITY THERE IS.
AND THEN YOU'RE IN THIS
BALANCING ACT WHERE WHILE YOU'RE
AWAITING THESE PERSONALIZED
CELLS TO BE MANUFACTURED FOR THE
PATIENT, YOU'RE TRYING TO KEEP
THEIR DISEASE UNDER CONTROL BUT
ALSO TRYING TO NOT GO OVERBOARD
AND PUT THEM AT RISK FOR ALL OF
THESE CO-MORBIDITIES WHEN THE
CELLS ARE READY, THEY SAY,
ACTUALLY, HE HAS AN INVASIVE
FUNGAL INFECTION?
THAT'S NOT GOING TO WORK.
SO IT'S A FINE BALANCING ACT
THAT TAKES A LOT OF
COMMUNICATION AND VERY
CHALLENGING FOR THE PATIENT THAT
IS BETWEEN MULTIPLE CENTERS AND
MULTIPLE PROVIDERS.
AND THEN THE PLANNING.
SO I HAVE THREE PATIENTS THAT
ARE COMING WITHIN THE NEXT WEEK
FROM ALL OVER THE COUNTRY AND
THE AMOUNT OF TIME AND EFFORT
AND NUMBER OF PEOPLE TO TRY TO
GET EVERYTHING TOGETHER FOR A
RELAPSE REFRACTARY PATIENT TO
TRAVEL ACROSS THE COUNTRY, TO
RECEIVE CELLS THAT ARE OTHERWISE
GOING TO BE THROWN AWAY, OR USED
FOR RESEARCH, NOT USED FOR
ANYONE ELSE IF THEY DON'T GET
THEM, IT'S SUBSTANTIAL.
SO THIS IS OBVIOUSLY NOT SIMPLE.
AND THE ISSUE IS, WHEN YOU EVEN
DISCUSS THIS WITH PEOPLE, THEY
OFTEN, THE ROAD WILL BE CLOSED
UPFRONT.
THEY ARE LIKE, I DON'T THINK MY
PATIENT WILL BE ABLE TO DO THAT.
AND SO I'M GOING TO GO THROUGH A
FEW COMMON SCENARIOS.
THESE ARE PEDE 8 RICK.
I KNOW MOST OF YOU --
PEDIATRIC -- I KNOW MOST OF YOU
STEAK CARE OF ADULTS.
-- TAKE CARE OF ADULTS.
YOU WILL THINK ABOUT SCENARIOS
YOU MAY HAVE SEEN THAT MAY BE
SIMILAR.
THESE ARE MADE UP NAMES.
ELI IS 9.
HE HAS REFRACTARY TALL LIVES IN
MISSOURI WITH MOM AND SINCE.
ELIGIBLE FOR TRANSPLANT BUT
RESIDUAL DISEASE AND HAS
NUMEROUS TAPERRIES IN THE PAST
AND REFERRING ONCOLOGIST HEARD
ABOUT THE SUCCESS OF YOUR CD5
CAR-T CELL TRIAL AND SAYS MAYBE
THIS WILL BE GREAT FOR HIM BUT
REALLY WORRIED ABOUT TRAVEL,
INSURANCE, HE KNOWS THEY ARE OUT
OF NETWORK AND HOW THEIR FAMILY
WILL HANDLED BEING UPROOTED TO
ANOTHER CITY.
SHE MENTIONS THE TRIAL TO THE
FAMILY BUT RECOMMENDS
ALTERNATIVE TREATMENT.
CONSIDERING THAT WILL BE LESS
DISRUPTIVE.
FOR EXAMPLE, IT IS IN HOUSTON
TEXAS BUT ATS A LOT.
AND I'M NOT SURE THAT YOU ALL
ARE GOING TO BE UP FOR IT.
SO WE ARE NOT GOING TO GO INTO
DETAIL BUT I DO HAVE THESE ORAL
CHEMOS THAT ARE PROBABLY GOING
TO BE MORE PALLIATIVE.
SO THIS IS A VERY COMMON
SCENARIO THAT HAPPENS.
HODGKIN LYMPHOMA GOT LOTS OF
PAST THERAPIES AND RESIDUAL
DISEASE.
EL JILL FOR TRIAL BUT UNINSURED
AND UNDOCUMENTED.
DAD WORKS FULL-TIME, MOM IS AT
HOME, HER MOM MENTIONED IN THE
PAST THAT THE TREATMENT IS IN
GOD'S HANDS.
SO PRIMARY ONCOLOGIST DETERMINES
THAT DESPITE THE PROMISING
RESULTS, TOO MANY LOGISTICAL
BARRIERS AND MENTIONS THAT I
THINK THEY MIGHT BE HESITANT TO
PARTICIPATE IN GENE THERAPY
RESEARCH ANYWAY BECAUSE OF A LOT
OF EXPERIMENTATION AND THINGS
HAPPENED IN THE PAST.
I KNOW THEY ARE SPIRITUAL AND
HUT THINGS IN GOD'S HANDS SO I'M
HESITANT TO MENTION IT TO THEM.
DAVID HAS A TERRIBLE BRAIN
TUMOR.
LIMITED TREATMENT OPTIONS,
ELIGIBLE FOR A TRIAL.
BECAUSE OF THE CLINICAL TEAM'S
EXPERIENCE WITH HAVING
DIFFICULTIES RECEIVING INSURANCE
APPROVAL FOR OTHER THERAPIES AND
BECAUSE HE MISSED SEVERAL
APPOINTMENTS DUE TO DAD'S WORK
SCHEDULE AND THE STRICT CONFINES
OF THE STUDY, THEY SAY YOU'LL
HAVE TO COMEBA THREE DAYS A
WEEK, FOUR DAYS A WEEK.
THE DOCTOR E-MAILS YOU AND SAYS
I'M NOT SURE WHETHER WE SHOULD
MENTION THIS STUDY.
I THINK IT WILL ADD MORE STRESS.
IF YOU DON'T BELIEVE THAT THESE
ARE REAL SCENARIOS, I CAN TELL
YOU THESE ARE SCENARIOS I DEAL
WITH EVERY SINGLE DAY.
USUALLY BY VERY WELL MEANING
PROVIDERS WHO HAVE A
RELATIONSHIP WITH THE PATIENT
AND A FAMILY, HAVE KNOWN THEM
FOR SEVERAL YEARS.
I'VE NEVER MET THEM, RIGHT?
BUT THEY REACHUTE TO ME AND SAY,
BASED ON WHAT I FEEL ABOUT THIS
PATIENT'S FAMILY, I'M NOT SURE
THIS IS SOMETHING WE SHOULD EVEN
EXPLORE.
SHOULD WE SET UP A CALL?
I'M HAPPY TO DO A TELEVISIT AND
REALLY JUST TALK ABOUT IT AND
SEE IF IT IS SOMETHING THEY ARE
INTERESTED IN.
LET ME TALK TO THEM, I'M NOT
SURE.
THE POINT IS, THAT A LOT OF
TIMES WHEN I GO TO ACADEMIC
MEETINGS TALKING ABOUT CAR-T
CELLS, WE ARE ALWAYS TALKING
ABOUT HOW TO MAKE THE
MANUFACTURE BETTER, FASTER,
STRONGER, MAKE THE CELLS BETTER,
BUT WE'RE OFTEN NOT TALKING
ABOUT THE ROAD BLOCKS THAT ARE
DISPROPORTIONATELY, ESPECIALLY
EFFECTING CERTAIN POPULATIONS.
IF YOU NOTICE IN THOSE
SCENARIOS, I DID NOT MENTION
RACE OR ETHNICITY.
UNFORTUNATELY, A LOT OF
UNDERREPRESENTED PATIENTS HAPPEN
TO HAVE SOME OF THE SAME SOCIAL
DETERMINANTS OF HEALTH AND
ISSUES THAT ARE THERE, BUT THIS
COULD BE ANY PATIENT.
IT COULD BE ANY PATIENT.
I THINK THAT IS THE MOST
IMPORTANT THING TO THINK ABOUT.
SO THERE ARE INSURANCE BARRIERS,
COVERAGE DELAYS AND DENIALS.
DENIALS CAN BE JUST -- DELAYS
COULD BE JUST AS SIGNIFICANT AS
DENIALS BECAUSE YOU'RE MORE
LIKELY TO GET A PATIENT AT AN
ADVANCED STAGE AFTER THEY HAD
MORE TREATMENTS THAT MAY NOT
NECESSARILY BE BENEFICIAL TO THE
PATIENT.
LO GEST CALL ISSUES RELATED TO
THE STUDY REQUIREMENTS.
THESE ARE THINGS THAT OFTEN
DISPROPORTIONATELY AFFECT
UNDERREPRESENTED PATIENTS,
PATIENTS WHO ARE OF LOWER
SOCIOECONOMIC STATUS,
GEOGRAPHICALLY RESTRICTED.
IF YOU LIVE IN BETHESDA, IF YOU
LIVE IN DC, IF YOU HAVE A
TWO-PARENT HOME, IF YOU HAVE A
LARGE SOCIAL NETWORK, YOU MAY BE
ABLE TO COME THREE DAYS A WEEK
AND BRING THAT CHILD FOR
PHARMACOKINETICS AND DYNAMICS
TESTING.
IF YOU DO NOT, IF YOU DON'T FIT
ANY OF THOSE CATEGORIES, IT CAN
BE A GAME CHANGER.
WE WILL WRITE IN THE TRIAL, THEY
HAVE TO COME OFF OF STUDY --
THIS IS REQUIRED BY THE STUDY.
WE HAVE THE ABILITY TO BE MORE
FLEXIBLE.
WE HAVE THE ABILITY TO SAY YES,
I WOULD LOVE TO GET THAT BLOOD
THREE TIMES A WEEK.
I CAN'T WAIT TO GET TO THE LAB
AND RUN THAT LUMINEX.
SEE WHAT THE CYTOKINES ARE
DOING.
HOWEVER, IN THE REAL WORLD, WE
HAVE TO RECOGNIZE THAT CAN
LITERALLY BE A LIMITATION THAT
CAUSES SOME PATIENTS TO NOT
RECEIVE THE THERAPY.
AND THEN, WE - WHO DOESN'T HAVE
UNCONSCIOUS BIAS IN HERE?
I HAVE A LOT.
WE ALL DO.
AND I RECOGNIZE IT, RIGHT?
I THINK ABOUT IT ALL THE TIME
BECAUSE I RECOGNIZE THAT THE
BIASES THAT CAN COME OUT IN HIGH
STRESS SITUATIONS, AND I KNOW
Y'ALL ARE NEVER IN HIGH STRESS
SITUATIONS, RIGHT?
SO, BECAUSE OF THAT, WE OFTEN
ARE THINKING THAT WE'RE DOING
WHAT IS IN OUR PATIENT'S BEST
INTEREST BY REMOVING CERTAIN
OPTIONS OR DISCUSSING CERTAIN
OPTIONS ABOVE OTHERS BECAUSE WE
THINK WE ARE DOING WHAT IS BEST
FOR THEM.
RESEARCH OVERWHELMINGLY SHOWS
THAT PATIENTS WANT TO KNOW ABOUT
ALL OF THE OPTIONS.
THEY WANT TO BE INVOLVED IN THAT
DECISION-MAKING.
SO WE KNOW HOW TRUSTED OUR
RELATIONSHIP IS WITH OUR
PATIENTS AND ONCOLOGY TOW TAKE
CARE OF THEM FOR SO LONG AND WE
SEE THEM SO FREQUENTLY.
SO WE HAVE TO BE REALLY ATTUNED
TO THE FACT THAT WE MAY BE
REMOVING OPTIONS AND MAKING THEM
SOUND LESS PALATABLE BECAUSE OF
A PRE CONCEPTION IT WILL BE MORE
CHALLENGING FOR THE FAMILY.
COMING FROM A GOOD PLACE,
THOUGH.
UNLIKE MEDICINES THAT ARE
LITERALLY OFF THE SHELF, PEOPLE
FEEL LIKE THEY HAVE TO HAVE ALL
OF THIS STUFF FIGURED OUT BEFORE
THEY OFFER SOMETHING TO A
PATIENT OR A FAMILY.
SO THERE ARE OTHER HARSH WELL
REALITIES EVEN WHEN YOU'RE IN A
PLACE WHERE YOU HAVE FINANCIAL
ASSISTANCE WHERE THE TRIAL IS
PAID FOR, HOUSING, LODGING,
ET CETERA, WHERE JUST HAVING TO
UPROOT TO TRAVEL TO AND REMAIN
FOR AN EXTENDED PERIOD TO
PARTICIPATE IN A TRIAL, IS A
LOT.
EVEN IF YOU SAY, BUT WE COVER
HOUSING.
WE'LL BE OKAY.
WE GIVE FOOD VOUCHERS.
IT IS REALLY A PRIVILEGE THAT
MANY OF US CAN PROBABLY DO BUT A
LOT OF PEOPLE JUST CANNOT DO
THAT.
AND THEN, WE HAVE TO LOOK AT
REFERRAL PATTERNS.
I STARTED DOING SOME STUDIES
WHERE WE TALKED TO REFERRING
DOCTORS, TALKED TO PATIENTS,
TALKED TO DOCTORS AT CENTERS
DOING THE TRIALS.
AND YOU START TO SEE THESE
REFERRING PATTERNS THAT REALLY
DO INDICATE THAT THERE MAY BE
SOME BIASES.
AND ONE THING THAT WE CAN CHANGE
AS MUCH AS WE WERE TALKING ABOUT
EARLIER, HOW YOU HAVE A TRIAL --
WHY IS THIS A PART OF THE
ELIGIBILITY CRITERIA?
DO YOU KNOW IF IT'S ALWAYS BEEN
LIKE THAT.
WHY IS IT AST ABOVE THE 5 TIMES
UPPER LIMIT -- WHY IS THIS A
PART?
EVERY TIME YOU HAVE TO WRITE A
SINGLE PATIENT PROTOCOL AND TALK
ABOUT COMPASSIONATE USE,
VARIATION, YOU SHOULD THINK, IS
OUR TRIAL CRITERIA TOO
STRINGENT?
AND A LOT OF THESE THINGS, BMI
IS NOT -- IS BASED ON A EUROPEAN
VERSION OF BMI.
IF YOUD HAVE OVERLY RESTRICTIVE
CRITERIA BASED ON BMI, PATIENTS
THAT ARE HIV PROCESS POSITIVE
WHEN THERE IS NO CLINICAL
RATIONAL TO EXCLUDE THEM FROM A
STUDY, PEOPLE WITH DIABETES THAT
IS CONTROLLED, HYPERTENSION THAT
IS CONTROLLED, WHY FOR MANY OF
OUR STUDIES WOULD THOSE BE
EXCLUSIVE?
WE ALL DO IT.
THE NUMBER OF TIMES I LOOKED AT
STUDIES, I'M LIKE WHAT IS IN
THIS CRITERIA?
WE HAVE TO THINK ABOUT THAT.
IT'S NOT JUST GETTING ON THE
TRIAL.
IT'S STAYING ON THE TRIAL.
IT'S NOT JUST ABOUT ENROLLMENT,
IT'S RETENTION.
SO WE KNOW THAT IN THE
CAR-T-CELL THERAPY SPACE, THERE
ARE SIGNIFICANT SOCIOECONOMIC
AND RACIAL DISPARITIES.
THE NUMBER OF AFRICAN-AMERICAN
PATIENTS, DESPITE EVEN IN
DISEASES LIKE MULTIPLE MYELOMA,
WHERE AFRICAN-AMERICANS AND
BLACKS ARE OVERREPRESENTED, THE
NUMBER OF PATIENTS ENROLLED ON
THESE TRIALS, 71% IS WHITE
PATIENTS.
THESE ARE PIVOTAL TRIALS THAT
LED TO FDA APPROVAL.
AND IT DISEASE LIKE MULTIPLE
MYELOMA.
THAT TELLS US IF WE ARE DOING A
THERAPY WE BELIEVE IS BASED
SCIENTIFICALLY, IT SHOULD WORK
BASED ON EXPRESSION OF A MARKER,
NOT ON ANYTHING ELSE.
AND THIS DISEASE IS OVER RENTED
IN A PATIENT POPULATION AND YET,
YOU ONLY HAVE 5.6% OF THE
PATIENT POPULATION THAT IS THAT.
THEN THAT IS AN ISSUE, RIGHT?
AND YOU SHOULDN'T RECOGNIZE THAT
AT THE VERY END BUT WE ALL DO
IT.
SAME THING ON CAR-T CELL TRIALS
AND LYMPHOMA.
SAME PATTERNS THAT WE SEE.
AND OVERWHELMINGLY IF YOU TALK
TO DISPARITIES RESEARCHERS, THEY
WILL SAY, ACCESS NOT WILLINGNESS
IS WHERE THE DIFFICULTY LIES IN
RECRUITING AND MAINTAINING
MINORITY RESEARCH PARTICIPANTS.
IT'S NOT THAT BECAUSE OF
HISTORICAL MISTRUST, IT'S
IMPOSSIBLE TO ENROLL SOMEONE.
MANY PATIENTS WILL SAY, IF YOU
LOOK AT SURVEYS OF PATIENTS,
THERE ARE SO MANY PUBLISHED
WHERE THEY SAY I WASN'T AWARE
THAT WAS AN OPTION FOR ME.
IF YOU SPENT AN HOUR TALKING
ABOUT AN OPTION AND YOU LEFT THE
ROOM AND THEN SOMEONE CAME BACK,
WHOEVER HAD THIS?
THE PATIENT SAID YOU DIDN'T
MENTION AND YOU'RE LIKE, WHAT?
I SAID -- LIKE IT HAPPENS TO ALL
OF US, RIGHT?
I THINK THAT WE HAVE TO REFRAME
IT IN THAT YES, WE MAY HAVE
MENTIONED THE OPTION.
YES WE MAY HAVE DISCUSSED IT THE
WAY WE WOULD LIKE TO HEAR IT.
PERHAPS THE WAY THAT WE
DISCUSSED IT WAS NOT IN A WAY
THAT THE PATIENT FELT LIKE IT
WAS TRULY A VIABLE OPTION FOR
THEM.
AND THERE IS WORK WE CAN
DO - THAT.
SO I BECAME VERY DEDICATED TO
EXPANDING ACCESS OF CLINICAL
TRIALS TO NOVEL AGENTS BECAUSE
THERE IS A LOT OF EVIDENCE IN
NON CANCER.
A LOT OF EVIDENCE IN CANCER,
LESS EVIDENCE IN EARLY PHASE
TRIALS.
AND WHAT IS CLEAR IS YOU HAVE TO
CONSIDER THE BARRIERS UPFRONT
AND THE THROUGHOUT AND CONSIDER
THEM AT THE END.
AND ACCESSIBILITY IS A BIG THING
THAT WE CAN ACTUALLY HELP WITH.
WHEN YOU THINK ABOUT ELIGIBILITY
CRITERIA, LACK OF KNOWLEDGE, THE
WAY YOUR CONSENT FORM IS
WRITTEN, STUDY LOGISTICS AND
THEN ADDRESS THINGS LIKE LACK OF
TRUST AND DIFFERENT ATTITUDES
AND BELIEFS THAT SOMETIMES MAY
BE CULTURALLY BASED.
BUT, WE HAVE TO ASSESS THEM
EARLY AND WE HAVE TO ASSESS THEM
FREQUENTLY AND INVOLVE OTHER
PEOPLE.
WHO IS A SOCIAL WORKER MONDAY,
WEDNESDAY, FRIDAYS?
I AM.
I'M A FINANCIAL COUNSELOR ON
TUESDAY, A PSYCHIATRIST ON
THURSDAY.
HOWEVER, IF I'M TRYING TO DO ALL
OF THOSE JOBS, I'M NOT ENGAGING
THE PEOPLE WHO LITERALLY THAT IS
THEIR EXPERTISE.
SO ONE THING WE HAVE TO REALIZE
IS THAT WE MAY NOT KNOW ABOUT A
RESOURCE BUT THERE IS SOMEONE
WHO KNOWS ABOUT IT AND WE HAVE
TO BRING THOSE PEOPLE IN EARLY
WHEN WE'RE DESIGNING TRIALS.
SO AS I WAS THINKING ABOUT THIS,
I DREW THESE DIFFERENT.
I MADE THE TABLE A LITTLE MORE
DIVERSE BECAUSE I FOUND THIS
PICTURE AND EDITED IT AND IT WAS
PRETTY AND I EDIT MYSELF IN
THERE.
SO AS I WAS THINKING ABOUT THESE
THINGS, I HAVE AMAZING
MENTORSHIP AND I WAS ENCOURAGED
TO STAY IN MY LANE AND PEOPLE
WOULD SAY, IT WILL BE
CHALLENGING FOR YOU BECAUSE
YOU'RE NOT A HEALTH EQUITY
RESEARCHER.
YOU'RE NOT GOING TO BE ABLE DO
DO THIS.
OR, AND WE HEAR THIS A LAYOUT.
IT'S REALLY CHALLENGING TO TAKE
RACE AND ETHNICITY INTO ACCOUNT
IF YOU'RE IN A PHASE I TRIAL
WHERE YOU'RE ONLY ENROLLING 23
PATIENTS.
ALL OF US GET CONCERNED LIKE WE
WANT TO EXPAND DIVERSITY BUT WE
THINK ABOUT PATIENT POPULATIONS
AND WE'RE LIKE, HOW ARE WE GOING
TO GUARANTEE THAT ON A VERY
SMALL TRIAL?
OR YOU'LL HAVE TO CHOOSE WHETHER
YOU FOCUS ON THE SCIENCE OR YOU
FOCUS ON THE DISPARITIES.
YOU'RE NOT GOING TO BE ABLE DO
DO BOTH AND WE NEED BIGGER
CHANGE AND THERE HAS TO BE
POLICY CHANGE AND SCIENTISTS
CAN'T DO THIS.
I'M LIKE THIS IS MY LANE.
I WEAR ALL THESE HATS AND LEAD
ALL THESE DEI INITIATIVES AND
I'M A CLINICAL RESEARCHER AND I
HAVE BEEN IN THE LAB AND I'M A
TRANSLATIONAL RESEARCHER AND I
DO ALL OF THESE THINGS.
SO WHY WOULD I NOT?
IF I'M IDENTIFYING A PROBLEM
WITH MY CLINICAL TRIAL, WHY
WOULD I NOT BE ABLE TO IMPACT
SOME OF THESE THINGS?
AND SO, I SAY THIS ALL THE TIME.
IF WE CAN BUILD IT, WE CAN
ADVOCATE FOR ACCESS.
I CANNOT TELL Y'ALL THESE
SCIENCES -- THE NUMBER OF THINGS
THEY CAN PUT IN A T-CELL.
I'M LIKE, IS THIS THE FUTURE?
LIKE LITERALLY YOU CAN ALMOST DO
ANYTHING BUT IF YOU CAN'T GET
THAT TO THE PATIENT IT'S A
PROBLEM AND YOU HAVE TO THINK
ABOUT IT THROUGHOUT.
IT REALLY IS EVERYONE'S LANE
BECAUSE WE ALL PLAY A ROLE.
SO, I STARTED BY ESTABLISHING
THIS WILL DIVERSITY COUNCIL
WITHIN OUR COMPREHENSIVE CANCER
CENTER.
ACTIVITY BENCH SCIENTISTS,
TRAINEES, PATIENTS, ADVOCATES,
RESEARCH COORDINATORS, COMMUNITY
MEMBERS, AND THEN I REALIZED
THAT MOST PATIENT ADVOCACY
GROUPS AND PATIENT ADVOCACY
BOARDS ARE MADE UP OF PEOPLE WHO
ARE OFTEN HAVE THE ABILITY TO GO
AROUND ADVOCATING AND THAT IS
OFTEN UPPER MIDDLE-CLASS, HIGH
HEALTH LITERACY, NOT NECESSARILY
REFLECTIVE OF THE PATIENT
POPULATION I SERVE.
SO WE HAVE A VERY DIVERSE BOARD
AND I WANTED TO HAVE A CANCER
CELL THERAPY SPECIFIC PATIENT
AND CAREGIVER ADVISORY BOARD WHO
HAD BEEN ON OUR TRIALS AND
STRUGGLED WITH THINGS TO REALLY
BE ABLE TO LOOK AT OUR TRIALS
FROM UPFRONT.
LOOK AT WHAT MAY BE AN ISSUE.
HAVE A TRUE VOICE AND THEN TALK
ABOUT WHAT IS IMPORTANT TO THE
COMMUNITY AND REVIEW ALL OF OUR
RESOURCES.
AND ONE THING THAT IS CLEAR, IS
YOU HAVE TO UNDERSTAND THE
STATUS QUO BEFORE YOU CAN
IMPROVE.
AND SO, I WANTED TO LOOK AT THE
RACE AND ETHNICITY OF PATIENTS
WHO WERE GETTING COMMERCIAL FDA
APPROVED CAR-T CELLS WHO WERE
ENROLLING ON OUR IEC,
IMMUNOEFFECTIVE CELL TRIALS AND
LOOKING AT THE CANCER CASES IN
OUR DEMOGRAPHIC AREA AND SEE
WHERE WE WOO CAN IMPROVE.
WE HAVE LOTS OF HISPANIC AND
LATINO PATIENTS IN OUR AREA,
ESPECIALLY IN PEDIATRICS.
AND WE HAVE A GOOD NUMBER OF
AFRICAN-AMERICANS AND BLACKS.
WHILE WE MAY BE DOING WELL IN
SOME AREAS, I LOOK AT WHERE WE
HAVE TO IMPROVE AND TALK TO THE
PATIENTS AND CAREGIVERS TO SEE
HOW WE CAN DO THAT.
AND SO WORKING WITH OUR
COMMUNITY AND PATIENT CAREGIVER
ADVISORY BOARD AND OUR TASK
FORCE, WE IDENTIFIED THESE
DOMAINS.
NONE OF THESE ARE GOING TO SEEM
LIKE THIS TO YOU.
YOU SEEN THEM BEFORE.
THAT TELLS US THAT WE ARE SEEING
THE SAME PATTERNS OVER AND OVER.
SO WHEN YOU'RE THINKING ABOUT
ENHANCING ACCESS, YOU'RE
THINKING ABOUT RECRUITMENT,
RECRUEL AND RETENTION.
THE NUMBER AND FREQUENCY OF
VISITS.
THE UNBELIEVABLE OF PEOPLE WHO
SAY IF I HAVE TO COME TWICE A
WEEK, IT'S A NO.
IF I HAVE TO COME 3 TIMES A
WEEK, I STILL HAVE TO WORK AND
STILL HAVE TO DO THIS AND THAT.
IF YOU KNEW THAT WAS GOING TO BE
THE DECIDING FACTOR IN WHETHER
SOMEONE ENROLLED ON YOUR TRIAL,
I WOULD PROBABLY SAY, WE WILL
MAKE THE SECOND VISIT OPTIONAL
AND MAYBE 60% OF THE PATIENTS
WILL COME ON THAT VISIT.
IF IT'S JUST FOR LABS.
OBVIOUSLY FOR SAFETY, WE HAVE TO
BE SAFE.
BUT ALSO LOOKING AT WHETHER YOU
ALLOW LOCAL VISITS CLOSER TO
HOME FOR CERTAIN THINGS.
WHETHER YOU ARE TRACKING YOUR
RURAL BY RACE, ETHNICITY, BY
PEOPLE OUTSIDE OF YOUR IMMEDIATE
AREA, BY PEOPLE FROM DIFFERENT
GEOGRAPHIC REGIONS.
FOR YOU ALL, THE NIH, BY PEOPLE
WHO HAVE PRIVATE VERSUS PUBLIC
HEALTH INSURANCE, EVEN THOUGH IT
MAY NOT BE A FACTOR HERE BECAUSE
IT LEADS YOU TO OTHER CLUES
ABOUT OTHER ASPECTS.
LOOKING AT FINANCIAL BURDEN, WE
ALL KNOW IF WE EVER BEEN TO ANY
MEDICAL CENTER IN THE WORLD AND
WE DO NOT WORK AT AND WE HAD TO
PAY, THAT WOULD SET US BACK.
THAT 14 DOLLARS FOR THAT 3 HOURS
SET US BACK.
INCLUSION AND EXCLUSION
CRITERIA.
WE CAN CONTROL MAKING THE
BROADEST POSSIBLE INCLUSION
CRITERIA THAT IS SCIENTIFICALLY
AND CLINICALLY SOUND.
AND REALLY STANDING UP AND
SAYING, IS THERE A REASON THAT
WE HAVE REALLY USED THIS SAME
CRITERIA FOR YEARS AND YEARS?
AND THEN CULTURAL AND LINGUISTIC
COMPETENCE.
TAILORING RECRUITMENT MATERIALS
FOR DIFFERENT POPULATIONS.
WE WORK WITH OUR PATIENT
ADVOCATES AND WHO WANTS TO READ
A 16 PAGE CONSENT FORM?
NOBODY AT ALL.
OKAY?
NOT EVEN US.
AND SO BEING ABLE TO HAVE A
SIMPLIFIED VERSION.
YOU STILL HAVE YOUR REGULAR
INFORMED CONSENT THAT HAS THE
BOILERPLATE IRB LANGUAGE BUT YOU
ALSO HAVE ADDITIONAL.
AND WE CREATED THIS GUIDANCE FOR
ASSESSING BARRIERS TO ACCESS,
ENROLLMENT AND RETENTION IN
SPECIAL POPULATIONS AND WE DID
THIS AND GO AROUND TO DIFFERENT
DIVISIONS WITHIN OUR CANCER
CENTER AND IT WAS ALSO RECENTLY
ADOPTED BY THE BONE MARROW
TRANSPLANT AND CLINICAL TRIALS
NETWORK, A NATIONAL NETWORK.
SO WE WILL BE DOING THIS FOR
THEM AND BY CHILDREN'S ONCOLOGY
GROUP.
IT ALLOWS PEOPLE FROM STUDY
CONCEPTUALIZATION OR IF A
SPONSORED PROTOCOL IS COMING TO
YOU TO HAVE THIS LIST OF THINGS
THAT YOU'RE JUST LOOKING, YOU'RE
ASSESSING.
WILL THIS BE A PROBLEM?
DO WE HAVE A WAY TO FIX THIS?
SHOULD WE INVOLVE SOCIAL WORK
EARLY?
SHOULD WE ASK FOR THIS?
DO WE NEED SPECIFIC EFFORTS FOR
A NAVIGATOR?
AND IT'S NOT MEANT TO BE
PUNITIVE AND IT'S BEEN VERY
HELPFUL.
AND THEN JUST A FEW MORE
ADDITIONAL CONSIDERATIONS ON
STUDY DESIGN.
IT IS REALLY IMPORTANT TO LOOK
AT SCREENING AND ENROLLMENT
FAILURES AND WHETHER IT'S A TRUE
CLINICAL REASON SOMEONE DID NOT
MEET THE ELIGIBILITY CRITERIA,
INVESTIGATOR DISCRETION, WHETHER
THE PATIENT DID NOT PROVIDE A
REASON.
BECAUSE IT CAN GIVE YOU SOME
CLUES.
AND THEN REASONS FOR COMING OFF
TREATMENT OR STUDY BECAUSE THE
PERCEPTION IS A LOT OF THESE ARE
CLINICAL.
THE REALITY IS THAT MOST OF
THESE ARE NOT CLINICAL.
SO THAT HELPS YOU SEE WHY DID
YOU COME OFF STUDY?
WELL, IT JUST GOT REALLY HARD
FOR ME.
SO SOMEONE COMING OFF STUDY WHEN
THEY HAVE BEEN ON THE STUDY FOR
TWO MONTHS OF A DRUG THEY ARE
GOING TO GET FOR THREE MONTHS,
IS SUBSTANTIAL.
BECAUSE YOU WERE ABLE TO ENROLL
THEM BUT NOT NECESSARILY TO
RETAIN THEM.
WHAT IS KNOWN OF THE BIOLOGICAL
IMPORTANCE OF RACE OR GENDER TO
THE DISEASE?
YOU OFTEN WILL HAVE NO IDEA.
THE INITIAL OF TIMES THAT I'M IN
A CONSENT DISCUSSION AND SOMEONE
WHO LOOKS LIKE ME SAYS, HAS THIS
BEEN STUDIED IN BLACK PEOPLE?
AND I HAVE TO SAY, ACTUALLY, NO.
AND THIS FIRST-IN-HUMAN TRIAL WE
HAVE NOT ENROLLED A BLACK PERSON
YET.
HOWEVER, BASED ON HOW THESE
CELLS WORK, I WOULD NOT THINK
THAT THERE IS A SCIENTIFIC
REASON FOR THIS THERAPY TO HAVE
A DIFFERENT SAFETY PROFILE OR
DIFFERENT EFFICACY.
BUT YOU KNOW WHAT?
IT'S REALLY IMPORTANT TO KNOW.
AND IT'S UNACCEPTABLE NOT TO
KNOW.
SO, I DO THINK REPRESENTATION IS
KEY.
AND BEING COMFORTABLE ANSWERING
QUESTIONS ABOUT THAT WHEN
PATIENTS ASK YOU.
IS IT APPROPRIATE TO TEST
HETEROGENEITY OF EFFECT?
DEMOGRAPHIC DATA THERE, WE ALL
NONE 2024, THESE SHOULD BE SELF
REPORTED.
WE CAN'T GO DOWN LIKE, THAT
PERSON LOOKS LIKE -- CHECK THIS
OFF.
BUT WE ALSO HAVE TO RECOGNIZE
THAT THOSE -- HAS ANYONE HAD A
PATIENT BE CONFUSED WHEN YOU
HAND THEM A RACE AND ETHNICITY
FORM?
OUR PATIENTS CONFUSED ALL THE
TIME.
I DO NOT IDENTIFY AS ANY OF
THESE.
THIS IS AN AMERICAN THING,
RIGHT?
WE HAVE PATIENTS FROM ALL OVER
THE WORLD AND ANCESTRY ALL OVER
THE WORLD.
PEOPLE ARE OFTEN CONFUSED AND
SAY I DON'T IDENTIFY AS WHITE.
I IDENTIFY AS HISPANIC/LATINO.
THAT'S ETHNICITY.
BUT I DON'T IDENTIFY AS WHITE.
SO IT'S REALLY SOMETHING THAT
THERE ARE EXPANDED CATEGORIES
THAT PEOPLE ARE TRYING TO START
USING THAT CAPTURE THAT GRANULAR
DATA BUT HAVE EXPANDED
CATEGORIES WHERE PEOPLE CAN
LITERALLY TELL WHAT THEY
IDENTIFY AS AND THEY ARE A LOT
EASIER TO USE.
SHORTENING CONSENT FORMS.
INCORPORATING BRIEF OVERVIEW.
ANY TIME I DO A CONSENT
DISCUSSION WHETHER ABOUT A
SINGLING MEDICATION OR A COMPLEX
STUDY, I SAY, LOOK, STUFF
HAPPENS IN AMERICA THAT WAS
UNACCEPTABLE IN THE PAST WHEN IT
RELATED TO NOT DOING INFORMED
CONSENT FOR RESEARCH.
AND BECAUSE OF ALL OF THAT
HORRIBLE STUFF, WE HAVE 1000
STAFF GUARDS IN PLACE TO MAKE
SURE IT NEVER HAPPENS AGAIN.
SO I TELL EVERY PATIENT THAT
BECAUSE I WOULDN'T WANT ANY OF
THAT.
AND TALK ABOUT THE STANDARD OF
CARE VERSUS THE RESEARCH.
AND THEN OFFERING OTHER FORMATS.
EVERYONE ISN'T DOWN FOR
HOUR-AND-A-HALF SHUT DOWN
FACE-TO-FACE AND MULTIPLE PACKS
OF PAPER.
WE HAVE TO THINK, ESPECIALLY A
TRIAL WE'RE LEADING OR A
PHARMACEUTICAL COMPANY SAYING WE
WANT YOU TO WORK ON THIS TRIAL,
ABOUT HAVING THINGS THAT ARE
EASIER TO UNDERSTAND.
AND THEN FOR ME IN TEXAS, AND I
KNOW IF ARE YOU TOO, WE HAVE TO
THINK ABOUT BARRIERS TO
ENROLLMENT FOR ASYLUM SEEKERS
AND REFUGEES AND IMMIGRANTS AND
CONCERNS ABOUT PROVIDING
DOCUMENTATION.
WE WERE GOVERNMENT FUNDED,
THAT'S A BADGE OF HONOR.
MY STUDY IS GOVERNMENT FUNDED.
WHEN YOU ASK PEOPLE WHAT THAT
MEANS, THE NUMBER OF PEOPLE THAT
BELIEVE THAT THAT MEANS YOU'RE
AUTOMATICALLY SHARING LOTS OF
THEIR INFORMATION WITH THE
GOVERNMENT, THE SPOOKY
GOVERNMENT, TO COME AFTER
THEM -- WE HAVE TO REMEMBER THAT
WE ARE EXPLAINING WHAT THAT
MEANS WHEN WE'RE SAYING IT.
CULTURALLY COMPETENT
COMMUNICATION.
WE HEAR ABOUT THIS A LOT.
I DO A LOT OF TRAININGS ON THIS.
IT INCLUDES ADDRESSING
HISTORICAL EVENTS THAT ARE TOUGH
TO TALK ABOUT.
WE KNOW THAT THERE IS EVIDENCE
THAT SAYS THAT IT CAN HELP IF A
TEAM MEMBER OR PROVIDER HAS A
SHARED CULTURAL BACKGROUND WITH
SOMEONE.
WE KNOW THAT THAT IS NOT THE
REALITY OF TODAY.
BUT, THE POINT IS THAT IF ANY OF
US, IF SOMEONE SAYS, WHAT ABOUT
THIS?
I'M WORRIED ABOUT
EXPERIMENTATION, WE KNOW WHAT WE
KNOW.
WE KNOW HOW LONG IT TOOK TO GET
THAT STUDY THROUGH THE I ARE.
B.
I KNOW HOW MANY CORRESPOND
UNDERSTAND I HAVE WITH THE FDA.
I KNOW HOW LONG THAT DATA SAFETY
MONITORING REPORT IS THAT I HAVE
TO DO.
BEING ABLE TO SAY TO PEOPLE, I
GET IT THE MISTRUST.
LET ME TELL YOU ALL THE
SAFEGUARDS IN PLACE FOR YOU.
AND THEN RECOGNIZING OUR BIASES
ARE REALLY IMPORTANT.
AND SO I DO ENCOURAGE Y'ALL TO
USE YOUR SCIENTIFIC AND CLINICAL
EXPERTISE TO ADVOCATE.
IT MAY NOT BE SPECIFICALLY FOR
YOU ALL OR NIH OR POLICY CHANGE,
BUT YOU'RE ADVOCATE NOTHING
OTHER WAYS.
ONE THING THAT IS IMPORTANT TO
ME IS EVERY TIME I PUBLISH
SOMETHING ABOUT RESEARCH, I'M
GOING TO PUBLISH SOMETHING ABOUT
HOW ACCESS CAN OFFSET POVERTY.
HOW WE NEED A MULTIPRONGED
EFFECT TO GET ACCESS TO THESE
CAR-T CELLS.
I GO TO CONGRESS A LOT TO TALK
ABOUT THESE EXCITING THERAPIES
AND THEN TALK ABOUT ALL OF THE
LIMITATIONS OF BRINGING THESE
OUT AND HAVE WORKED WITH THE
BIDEN AND HARRIS ADMINISTRATION
ON THE CANCER GENE THERAPY AND
SICKLE CELL GENE THERAPY.
THE COOL THING IS THAT PEOPLE
RECOGNIZE HOW EXCITING OUR
SCIENCE IS AND OFTEN, IT'S
COMMON KNOWLEDGE TO US.
IT'S HARD TO GET TO THIS THERAPY
BUT IT'S NOT COMMON KNOWLEDGE TO
PEOPLE, ESPECIALLY IN POSITIONS.
AND IT OFTEN TAKES GETTING THE
RIGHT PEOPLE AT THE TABLE.
SO I DO A LOT OF POLICY SUMMIT
THINGS WITH DIFFERENT ADVOCACY
ORGANIZATIONS.
THIS IS LIKE IN THE MIDDLE OF
COVID AND I'M LIKE, I DON'T KNOW
Y'ALL, I WILL HAVE MY MASK ON.
AND THEN ONE OTHER THING,
SOMETIMES YOU HAVE TO BRING THE
RESEARCH TO THE PEOPLE.
SO ONE OF THE BIG THINGS WE DONE
AT BAYLOR, WE HAVE SAFETY NET
HOSPITALS THAT ARE LARGELY
UNINSURED, UNDERREPRESENTED
POPULATIONS AND WE ARE STARTING
A CELL THERAPY PROGRAM THERE.
WE MAKE THE CELLS IN OUR CENTER
AND WE CAN ABOUT I THINK THEM
OVER AND WE HAVE TO START WITH
NON GENE MODIFIED CELLS AND THE
GOAL IS TO EXPAND THEM.
AND IT TAKES A VILLAGE.
YOU CAN PARTNER.
I JUST GOT A P20 GRANT FROM NIH,
A PARTNERSHIP OF A COMPREHENSIVE
CANCER CENTER WITH PREDOMINANTLY
UNDERREPRESENTED SERVING
INSTITUTIONS.
SO I WORK WITH RESEARCH AND
CANCER RESEARCH EDUCATION,
OUTREACH, ALL KINDS OF COOL
STUFF.
SO, WHERE DO WE GO FROM HERE?
SO I WANT TO ENCOURAGE YOU WHEN
YOU'RE PRESENTING TUBES TO
PARTICIPATE IN RESEARCH,
REMEMBER THAT A LACK OF INTEREST
MAY BE DUE TO LACK OF ADEQUATE
AWARENESS FOR THAT PATIENT.
AND OFTEN SAYING IS THERE
SOMETHING THAT CONCERNS YOU?
DO YOU WANT TO THINK ABOUT THIS?
IS IT SOMETHING YOU WANT TO TALK
ABOUT WITH YOUR FAMILY MEMBERS?
I'M HAPPY TO ANSWER.
REMEMBER PRAYER AND SPIRIT AT
THEY CAN COEXIST.
THERE IS A LOT OF DATA ON PEOPLE
WHO PROCLAIM THEY ARE RELIGIOUS
OR SPIRITUAL, I'M LEAVING THIS
UP TO GOD OR ALLAH OR -- WILL
SAY THAT AND THEN THE PROVIDERS
WILL INTERPRET THAT THIS MEANS
THEY ARE NOT GOING TO BE AS
INTERESTED IN THE SCIENCE AND
NOT INTERESTED IN THIS
INVESTIGATIONAL TRIAL.
AND RELYING ON AND USING THE
LIVED EXPERIENCES, YOU ALL HAVE
AMAZING FOLKS THAT WORK IN
HEALTH EQUITY, WORK IN COMMUNITY
OUTREACH AND ENGAGEMENT AND
OFTEN YOU MAY HAVE A TRIAL, YOU
MAY HAVE A STUDY, YOU MAY HAVE A
TREATMENT THAT YOU'RE HAVING
TROUBLE WITH AND YOU CAN REACH
OUT TO THOSE FOLKS TO PROVIDE
CONSULTATIONS.
AND THEN JUST SPEAKING ABOUT THE
FACT THAT WE HAVE TO CLEARLY
CONSIDER THESE BARRIERS AT EVERY
ASPECT EVEN BEFORE WE GET TO THE
CLINIC BUT THEN AFTER.
I AM THE EPIDEMY OF TEAM SCIENCE
AND HAVE BENEFITED SO MUCH FROM
SO MANY WONDERFUL PEOPLE THAT I
WORK WITH AND ESPECIALLY
PATIENTS AND FAMILIES AND MY
FUNDING SOURCES AND ALL OF YOU.
AND I'M HAPPY TO TAKE ANY
QUESTIONS.
THANK YOU.
[ APPLAUSE ]
>> THANK YOU SO MUCH.
IT'S A FANTASTIC TALK.
I'M ROSIE KAPLAN AT THE ONCOLOGY
BRANCH.
THIS IS A TOPIC VERY NEAR TO MY
HEART BECAUSE WE DO A LOT OF
CELL THERAPY HERE ALSO AND I GET
A LOT OF THE QUESTIONS, A LOT OF
THE TIME YOU BROUGHT UP.
WE SEE RARE POPULATIONS OF
PATIENTS, MAYBE WE DON'T HAVE
THE DIVERSITY THAT WE NEED AND
THAT'S WHY WE DON'T HAVE
DIVERSITY IN OUR TRIALS.
AND WHAT WE FIND HERE IS HARD
AND I LOVE YOUR THOUGHTS ABOUT
THAT, WE DON'T EVEN GET TO THE
REFERRAL STAGE.
THEY ARE NOT EVEN COMING DO US
TO KNOW THEY ARE MISSING.
>> IT WAS SO HUGE.
WE WERE TALKING ABOUT THIS
EARLIER.
WHAT WE HAVE IS A VAST UNDER
ESTIMATION.
LIKE WE DO NOT EVEN CAPTURE THE
INFORMATION ON ALL OF THE
PATIENTS WHO DON'T EVEN MAKE IT.
IT TAKES A LEVEL OF NAVIGATIONAL
HEALTH LITERACY.
YOU HAVE TO EVEN GET TO THAT
POINT.
AND A LOT OF WHAT WE DO IN OUR
COMMUNITY OUTREACH AND
ENGAGEMENT OFFICE AND WHAT I'M
DOING WITH THE CLINICAL TRIALS
NETWORK IS LITERALLY ALSO
TALKING TO REFERRING PROVIDERS
AND -- BECAUSE WE HAVE TO GET
THERE.
AND WE WERE JUST TALKING ABOUT A
PROJECT EARLIER WHERE KNOWING
WITH REFERRING PROVIDERS WHAT --
BECAUSE YOU KNOW THIS TREATMENT
OPTION IS HERE.
YOU PROBABLY HAD MENTIONED IT.
YOU DIDN'T QUITE E-MAIL.
YOU DIDN'T NECESSARILY REACH OUT
BECAUSE YOU THOUGHT THE DOOR
WOULD BE SHUT.
AND SO, WE HAVE TO MOVE BEYOND
JUST US IN OUR BUBBLES OF THESE
HUGE ACADEMIC CENTERS AND WORK
WITH REFERRING PROVIDERS.
AND ONE THING WE DID AT OUR
CENTER, I LOOKED AT ALL OF THE
PEOPLE THAT HAVE REFERRED FOR
TRANSPLANT, PEOPLE HAVE
REFERRED, REFRACTARY PATIENTS.
NOT NECESSARILY FOR A TRIAL.
THOSE REFERRING PROVIDERS.
PUT TOGETHER A PROTOCOL WHERE
I'M ACTUALLY REACHING OUT TO
THOSE PROVIDERS, DOING A SURVEY,
WORKING WITH AN EPIDEMIOLOGIST
TO DO INTERVIEWS WITH THEM AND
THEN CAPTURING FROM THE PATIENTS
AND THEY CAN ENROLL PATIENTS
THAT THEY PRESENT THE OPTION TO
FOR US TO FOLLOW-UP WITH.
SO, A LOT OF IT, I THINK THAT
WE'RE TAKING THE EASY WAY BUT WE
ALL -- WE ARE ALL LIKE, THIS IS
A RARE POPULATION.
WE GET WHAT WE GET.
BUT, IF WE CAN TRY TO MAKE SURE
THAT WE ENSURE THAT EVERY SINGLE
PATIENT THAT HAS A BARRIER,
REGARDLESS OF RACE AND
ETHNICITY, IF THERE IS A
MECHANISM FOR US TO AT LEAST
THINK ABOUT HOW WE CAN COUNTER
ACT THAT, WE UNDOUBTEDLY WILL
START TO SEE INCREASES IN THE
NUMBER OF DIVERSE PATIENTS.
AND WE DON'T CAPTURE NON
REFERRALS AND WE DON'T CAPTURE
SCREEN FAILURES WELL.
AS WELL AS WE SHOULD.
SO I THINK IT DOES TAKE A
REFERRING PROVIDER-TYPE NETWORK.
AND I'D LOVE TO TALK MORE ABOUT
IT BECAUSE IT'S SOMETHING THAT I
THINK WE ALL RECOGNIZE.
LIKE THE ONES THAT DON'T GET
THERE, ARE THE PROBLEM, RIGHT?
>> AND MY SECOND QUESTION IS
ABOUT, WE ARE VERY INTERESTED --
MY LABORATORIY IS FOCUSED ON
TUMOR MICROENVIRONMENT.
I'M INTERESTED IN THE SOCIAL
DETERMINANTS OF HEALTH IMPACT ON
TUMOR MICROENVIRONMENT RESPONSE
TO THERAPIES, IMMUNE --
>> AND THERE IS A SCIENTIFIC
RATIONAL THAT WE KNOW THAT
EPIGENETICS, TUMOR
MICROENVIRONMENT.
WE KNOW ALL OF THAT IMPACTED BY
SOCIAL DETERMINANTS OF HEALTH
COULD POTENTIALLY BE A FACTOR IN
WHY A CERTAIN POPULATION MAY OR
MAY NOT RESPOND.
AND I THINK THAT WE -- THIS IS
AN EXAMPLE OF SOMEONE WHO IS A
SCIENTIST HAS A LABORATORY
SAYING, WAIT, WHAT ABOUT THIS?
AND I THINK THAT IT'S SO
IMPORTANT.
WE DON'T HAVE ENOUGH INFORMATION
ABOUT IT RIGHT NOW BUT I'M SO
EXCITED WHEN PEOPLE TALK ABOUT
IT BECAUSE IT TAKES PARTNERING
WITH PEOPLE WHO HAVE DIFFERENT
EXPERIENCES AND THAT IS EXACTLY
WHAT I DID.
I WAS LIKE WAIT A MINUTE, BUT I
ALSO DIDN'T KNOW HOW TO START,
RIGHT?
SO I WAS LIKE, I'M GOING TO HAVE
TO REACH OUT TO SOME TRUE
DISPARITIES RESEARCHERS AND WORK
WITH EPIDEMIOLOGISTS AND FOLKS
AND SO I ENCOURAGE AVERAGE TO DO
THAT.
IF YOU HAVE THE -- EVERYONE TO
DO THAT.
IF YOU HAVE THE QUESTION, THESE
FOLKS WILL KNOW HOW TO HELP YOU.
THAT'S REALLY GRATIFYING.
>> I REALLY APPRECIATE YOUR
ADVOCACY FOR IT BECAUSE I ALSO
THINK LIKE LARGER GROUPS LIKE
COJ -- THEY NEED TO HELP PARTNER
WITH US BECAUSE WE ARE JUST ONE
SITE AND WE REALLY NEED --
>> SO WE ARE STARTING -- I
PRESENTED AT COD SCIENTIFIC
CHAIRS LAST YEAR AND WE ARE
STARTING SOME DIFFERENT PROJECTS
SPECIFICALLY TO TRY TO ADDRESS
THAT BECAUSE OFTEN YOU LOOK AT
YOUR DATA AND YOU'RE LIKE, WE'RE
DOING FINE.
BUT WE'RE NOT DOING FINE, RIGHT?
LIKE WE ALL CAN DO BETTER.
I WOULD LOVE TO FOLLOW-UP WITH
YOU.
>> THANK YOU VERY MUCH.
>> THANK YOU.
>> HI, THANK YOU FOR A VERY
INSPIRING AND INFORMATIVE TALK.
>> THANK YOU DR. PREWIT.
SEE I REMEMBERED YOUR NAME.
>> YOU DID, THANK YOU.
THIS PAST WEEK I MET WITH A
YOUNG PARENT WHO SEEMED TO HAVE
JUST PROBLEM AFTER PROBLEM IN
HIS SOCIAL LIFE.
AND YOU TOUCHED ON OR TALKED
ABOUT CONSIDERATIONS WE CAN TAKE
WITH WRITING A CLINICAL TRIAL IN
TERMS OF TRYING TO HELP WITH
RETENTION.
WHAT IS YOUR ADVICE FOR HELPING
THE PATIENT OVERCOME BARRIERS IN
THEIR LIVES THAT IMPACT
RETENTION?
>> YES, SO SUCH AN EXCELLENT
QUESTION.
YOU KNOW, OUR PATIENTS -- IF YOU
THINK ABOUT A YOUNG PATIENT
HOWEVER OLD THIS PATIENT WAS AND
JUST LIFE IN GENERAL AND THEN
THROWING CANCER ON TOP OF THAT
AND THEN THROWING WE ARE GOING
TO DO ALL OF THIS.
IT'S SO COMPLEX THAT I THINK IF
ANY OF OF US FELT -- AND WE'RE
ALL SUPER PEOPLE, RIGHT?
BUT IF ANY OF US FELT WE HAD THE
ABILITY TO HELP THEM NAVIGATE
THROUGH ALL OF THAT, WE WOULD BE
REALLY MISTAKEN.
I THINK ONE OF THE MOST
IMPORTANT THINGS THAT I TRY TOW
DO WITH EVERY SINGLE ONE OF MY
PATIENTS IS HELP THEM UNDERSTAND
THAT THIS IS NOT NORMAL.
THE SITUATION YOU'RE IN IS NOT
NORMAL.
I DON'T CARE WHAT YOUR SOCIAL
SUPPORT IS, THIS IS NOT NORMAL.
SO WHEN THEY SAY, I DON'T NEED
THE SOCIAL WORKER, I DON'T NEED
THE FINANCIAL -- I REALLY TRY TO
ENCOURAGE THEM.
WE HAVE THEM MEET WITH EVERYONE.
I THINK IT WILL BE REALLY
IMPORTANT.
THIS IS ABOUT TO BE REALLY
DISRUPTIVE AND WE WANT YOU TO BE
ABLE TO HAVE THE OPPORTUNITY TO
DO AS WELL AS POSSIBLE AND PART
OF DOING AS WELL AS POSSIBLE MAY
INVOLVE BEING ON A CLINICAL
TRIAL.
AND PART OF THAT WILL
UNDOUBTEDLY -- CANCER EFFECTS
EVERY ASPECT OF YOUR LIFE.
WE ALL KNOW THAT.
I THINK NORMALIZING IT AND
NORMALIZING THE FACT THAT EVEN A
HIGH-INCOME ENGINEER -- ENGINEER
WHO IS VERY WELL READ AND CAN
TELL YOU ABOUT THE LATEST PAPER
THAT JUST CAME OUT, MAY BENEFIT
FROM HAVING SOME SOCIAL WORK
SUPPORT.
MAY BENEFIT FROM SEEING
PSYCHOLOGY.
AND SO I THINK THAT ALSO THAT
SELF-EFFICACY, ONE THING THAT WE
DON'T DO AS WELL, AND I'M A
PEDIATRICIAN SO I TREAT
35-YEAR-OLDS, LIKE LET ME HELP
YOU.
AND WILL MY SOCIAL WORK
COLLEAGUES ALWAYS HAVE TO HEP
ME.
SELF-EFFICACY.
WE OFTEN ARE TRYING TO FIX THE
PROBLEM FOR THEM RATHER THAN
HELPING THEM GETTING THE TOOLS
TO FIX IT.
AND WHAT WE HAVE TO REALIZE IS
THOSE PROBLEMS AREN'T GOING TO
GO AWAY WHEN THEY ARE DONE WITH
THEIR TREATMENT.
IF WE ARE DOING A LOT OF THINGS
BUT NOT BUILDING SELF-EFFICACY,
IT CAN BE CHALLENGING.
SO I HAVE WATCHED SOCIAL WORKERS
BUILD SO MUCH SELF-EFFICACY WITH
PEOPLE AND SAY, WE CAN DO THIS
BUT WE'RE NOT ABLE TO DO THAT.
BUT LET ME SHOW YOU THESE
RESOURCES AND HERE IS YOUR TASK
FOR TOMORROW.
HERE IS YOUR TASK FOR FRIDAY.
I WANT YOU TO BE ABLE TO DO
THIS.
BECAUSE IT IS HARD.
AND I DON'T THINK THERE IS A
SINGLE ANSWER BUT I TRY TO NOT
ASSUME THAT I HAVE ALL THE
ANSWERS ABOUT SUPPORT AND TRY TO
BRING THE EXPERTS IN.
BUT THEN FOLLOW-UP WITH THEM.
BECAUSE OFTEN WE DO
CONSULTATIONS AND PUT IN THE
CHART, YOU HAVE BEEN CONSULTED
AND THEN YOU GAVE ADVICE AND
THEY DID NOT WHERE THE GLOVES
YOU ASKED THEM TO.
WE DO THAT TOO.
WE BRING PEOPLE IN AND NEVER
FOLLOW-UP TO KNOW IF THERE IS
SOMETHING ADDITIONAL WE CAN DO
TO HELP.
>> THANK YOU.
>> YOU'RE WELCOME.
>> HI, MY NAME IS LIZ.
ONE OF THE FELLOWS HERE AND I
JUST -- THIS IS A REALLY
INSPIRING TALK AND I DID WORK
PREVIOUSLY WITH EXPANDED ACCESS
AND IN SOME OF THE CONVERSATIONS
THAT I'VE HAD PARTICULARLY WITH
PHARMACEUTICAL COMPANIES, THEY
HAVE DIVIDES THIS THOUGHT OF,
OKAY, INDIVIDUALS WHO DON'T MEET
THE ENTRY CRY TOOIA, IF WE
CREATE A COHORT THAT WILL ALLOW
FOR REAL-WORLD DATA BUT THEY ARE
NOT INCLUDED IN THE DATA POINTS,
AND IT SEEMS LIKE SUCH A
DIVERSITY OF DICHOTOMY AND I'M
JUST CURIOUS JUST BASED ON YOUR
EXPERIENCE HOW WE SHOULD GO
ABOUT PROCESSING THESE
DECISIONS?
>> I WOULD SAY THAT -- SO WE DID
THIS -- THERE IS A CUBA EXPANDED
ACCESS THAT YOU'RE PROBABLY
FAMILIAR WITH.
I WENT TO A TALK IT WAS ON A
PANEL A FEW MONTHS AGO AND IT
WAS AT NYU AND THEY HAD LOTS OF
PHARMACEUTICAL FOLKS THERE AND
IT WAS ALL ABOUT EXPANDED ACCESS
FOR RARE DISEASES.
WHICH ESSENTIALLY EVERYTHING WE
DO IS A RARE DISEASE.
AND YOU'RE ABSOLUTELY RIGHT.
AND THEY ARE VERY -- SOME PEOPLE
WERE -- IT WAS SPECIFICALLY
ABOUT GENE THERAPIES AND SOME
PEOPLE ARE ABSOLUTELY NOT.
WE HAVE TO HAVE THIS AND THAT.
SO, THEY ARE IN NEED OF YOUR
PATIENT POPULATIONS AND THEY ARE
IN NEED OF YOU.
SO PEOPLE OFTEN THINK THAT YOU
CAN'T PUSH A PHARMACEUTICAL
COMPANY.
BUT I WILL SAY UPFRONT, IT'S
GOING TO BE VERY CHALLENGING.
THIS EXCLUSION CRITERIA, IS
GOING TO BE VERY CHALLENGING FOR
MY PATIENT POPULATION.
LET ME TELL YOU WHY.
AND THEY SAY WELL, WE HAVE TO
HAVE IT.
MAYBE WHY CAN DO A SINGLE
PATIENT.
I SAY OKAY, BUT I'M NOT WRITING
THE SINGLE PATIENT.
YOU'RE GOING TO HAVE TO DO A
MANAGE ACCESS PROGRAM BECAUSE
I'M NOT DOING THAT.
BECAUSE THAT'S NOT SOMETHING
THAT I SHOULD HAVE TO DO.
AND IF PEOPLE ARE REPEATEDLY
TELLING YOU THIS IS A PROBLEM,
YOU NEED TO LOOK AT IT.
I DO THINK NOW PHARMACEUTICAL
COMPANIES ARE NOT -- ARE
UNLIKELY TO SPEARHEAD REAL WORLD
APPROACHES BUT IF THERE IS A
WHOLE REAL-WORLD CONSORT OF
PATIENTS WHO DID NOT MEET
CRITERIA BUT GOT EXPANDED
ACCESS, I DO THINK WITHIN
ACADEMIA, WE HAVE THE ABILITY TO
PARTNER WITH INSTITUTIONS AND
SAY, WE'D LIKE TO STUDY THIS
REAL-WORLD CONSORT.
THE PHARMACEUTICAL COMPANIES
WILL SEE THAT AND WILL OFTEN
PROVIDE GRANTS TO DO THAT, MONTH
KNOWY TO DO THAT, SUPPORTS,
STATISTICAL SUPPORT.
SO I THINK THAT WHEN YOU
RECOGNIZE A NEED THAT THERE IS
AN EXPANDED, EXTENDED
COMPASSIONATE USE INDICATION
THAT YOU'RE REPEATING A NUMBER
OF TIMES -- LIKE WHENEVER YOU
SEE THAT YOU HAVE DONE IT MORE
THAN TWICE, THE MOST LIKELY
THING IS OTHER PEOPLE HAVE DONE
IT MORE THAN TWICE.
SO IN THE SAME SPACES WHERE
YOU'RE WITH YOUR COLLEAGUES,
TALKING ABOUT WE DON'T HAVE A
LOT OF DATA ON THESE PATIENTS.
YOU GO THE ONES I HAD DID WELL
DESPITE THE FACT THAT THEY
DIDN'T MEET THIS INCLUSION
CRITERIA.
AND THAT WILL OFTEN LEAD TO MORE
STUDIES AND POTENTIALLY LEAD
THEM AT SHOULD WE CHANGE THE
LABEL?
THEIR BOTTOM LINE IS A LITTLE
DIFFERENT THAN OURS.
BUT I DO THINK THAT THERE IS A
WAY TO, IN ACADEMIA, APPROACH
THE COMPANY AND SAY, HEY, WE
KNOW THAT YOU MAY NOT BE
INTERESTED IN REPORTING ON THOSE
PATIENTS BUT WE AS THE REAL
WORLD, TAKING CARE OF THESE
PATIENTS, WOULD LIKE TO DO A
REAL-WORLD STUDY.
AND IT CAN BE A RETROSPECTIVE
STUDY, GATHER DATA AND THEN
POTENTIALLY DO SOMETHING
PROSPECTIVE.
>> THANK YOU.
>> YOU'RE WELCOME.
>> I THINK WE'RE AT TIME.
>> THANK YOU ALL SO MUCH.
I REALLY APPRECIATE YOU INVITING
ME.
IT'S ALWAYS WONDERFUL TO COME TO
THE NIH DESPITE THE GETTING INTO
THE FORTRESS.
BUT, I ALWAYS LOVE COMING HERE
AND I REALLY APPRECIATE Y'ALL'S
ATTENTION AND Y'ALL ARE ALL
DOING GREAT AND,A MAZING THINGS
AND YOU MAKE ME EXCITED ABOUT
THE FUTURE!
THANK YOU!
[ APPLAUSE ]