Welcome to the Clinical Center Grand Rounds,
a weekly series of educational lectures for physicians and
health care professionals broadcast from the Clinical
Center at the National Institutes of Health in
Bethesda, MD.
The NIH Clinical Center is the world's largest hospital totally
dedicated to investigational research and leads the global
effort in training today's investigators and discovering
tomorrow's cures.
Learn more by visiting us online at http://clinicalcenter.nih.gov
WE ARE HONORED TO HAVE
DR. FRANCIS COLLINS, A SENIOR
INVESTIGATOR OF THE INTRAMURAL
PROGRAM OF THE NATIONAL HUMAN
GENOME RESEARCH INSTITUTE AND
FORMALLY THE 16th DIRECTOR OF
THE NATIONAL INTUITYS OF HEALTH.
DR. COLLYNN SYSTEM A PHYSICIAN
GENETICIST NOTED FOR HIS
LANDMARK DISCOVERY OF
DISEASE-RELEASED GENES AND
PREVIOUS LEADERSHIP OF THE
NATIONAL HUMAN GENOME PROJECT
WHICH CULMINATED IN 2003 WITH
THE IDENTIFICATION MAPPING AND
SEQUENCING ALL OF THE GENINGS OF
THE HUMAN GENOME.
APPOINTED AS DIRECTOR OF THE NIH
HE SERVED UNDER 3 PRESIDENTS
UNTIL STEPPING DUN IN 2021.
HE WENT ON TO SERVE THE BIDEN
ADMINISTRATION AS ACTING ADVISOR
AND CONTINUED HIS WHITE HOUSE
SERVICE AS A SPECIAL ADVISOR TO
THE PRESIDENT FOR SPECIAL
PROJECTS, LEADING TO A PROGRAM
TO ELIMINATE HEPATITIS C IN THE
UNITED STATES.
DR. COLLINS CURRENTLY SERVES AS
A SENIOR INVESTIGATOR IN THE
INTRAMURAL PROGRAM OF NHGRI
PURSUING GENOMICS RESEARCH ON
TYPE 2 DIABETES AND
PROGERIATRICKIZATIONIA, THE
DISORDER OF PREMATURE AGING.
DR. COLLINS RECEIVED HIS
BACHELOR OF SCIENCE AND
CHEMISTRY AT THE UNIVERSITY OF
VIRGINIA AND MASTERS IN
PHILOSOPHY IN Ph.D. AND
PHYSICAL CHEMIST RADIOY FROM
YALE.
HE THEN EARNED HIS MEDICAL
DEGREE WITH HONORS FROM THE
UNIVERSITY OF NORTH CAROLINA AND
COMPLETED INTERNAL MEDICINE
RESIDENCY AT MEMORIAL HOSPITAL
AND FELLOWSHIP IN HUMAN GENETICS
AND PEDIATRICS AT YALE
UNIVERSITY SCHOOL OF MEDICINE.
DR. COLLINS IS AN ELECTED MEMBER
OF BOTH THE NATION WILL ACADEMY
OF MEDICINE AND THE NATIONAL
ACADEMY OF SCIENCES.
HE IS A RECIPIENT OF THE
PRESIDENTIAL MEDAL OF FREEDOM
AND THE NATIONAL MEDAL OF
SCIENCE.
IN 2020 HE WAS ELECTED AS A
FOREIGN MEMBER OF THE ROYAL
SOCIETY AND WAS ALSO THE 50th
WINNER OF THEATISMLE TON PRIZE
WHICH CELEBRATES SCIENTIFIC AND
SPIRITUAL CURIOSITY.
NOW, PLEASE JOIN ME IN WELCOMING
TODAY'S GRAND ROUND SPEAKER
DR. COLLINS FOR HIS
PRESENTATION, HIS SECOND DECADES
AT THE FOREFRONT OF MEDICAL
RESEARCH: THE NIH CLINICAL
CENTER.
[ APPLAUSE ]
>>THANK YOU VERY MUCH, JIM.
GOOD AFTERNOON TO EVERYBODY HERE
IN LIPSETT AUDITORIUM AND ALL
THE PEOPLE WATCHING REMOTELY.
IT IS GREAT THAT SOME OF YOU ARE
HERE BECAUSE IT'S BEEN A LONG
TIME SINCE WE'VE BEEN ABLE TO
ASSEMBLE GRAND ROUNDS IN QUITE
THIS NUMBER OF FOLKS GIVEN THE
COVID CHALLENGES THAT WE'VE ALL
BEEN GOING THROUGH WHICH ARE NOT
ENTIRELY OVER BUT WE ARE
DEFINITELY IN A BETTER PLACE
THAN WE WERE IN A FEW 3 YEARS
PAST.
IT WILL BE CHALLENGING TO TAKE
ON THE REQUEST THAT WAS PUT
FORWARD TO ME TO SUMMARIZE 7
DECADES OF MEDICAL RESEARCH IN
45 MINUTES SO LET ME RIGHT UP
FRONT SAY IF I LEAVE OUT AN AREA
THAT YOU THINK WAS REALLY,
REALLY IMPORTANT, PLEASE FORGIVE
ME BECAUSE THE COMP LICKERMENTS
OF THESE 70 YEARS, ARE TRULY
BREATH TAKING AS YOU WILL SEE.
AND YET, I COULD NO WAY, NO HOW,
POSSIBLY COVER ALL THE THINGS
THAT MAY HAVE DESERVED TO BE
MENTIONED IN A TALK LIKE THIS.
FIRST OF ALL FINANCIAL
DISCLOSURES.
SOMEDAY I HOPE TO HAVE SOMETHING
ON THIS SLIDE, BUT AS A FEDERAL
EMPLOYEE ON THE MOMENT NOTHING
TO DISCLOSE, BUT I DO HAVE
GRATITUDE TO DISCLOSE BECAUSE I
DID LEAN ON SOME PEOPLE TO HELP
ME WITH THIS PRESENTATION.
PARTICULARLY JOHN GALLEN WHO
GAVE A TALK LIKE THIS LAST FALL
TO THE RESEARCH BOARD WHERE I
BORROWED LIBERALLY FROM HIS
IDEAS ABOUT WHICH THINGS TO TRY
TO INCLUDE.
AND KIM PALACE WHO RUNS THE
HISTORY OFFICE FOR NIH WAS ALSO
HUGELY HELPFUL TO ME AS WAS
DEVON VALERA AND DONNIE BLISS,
AND A FEW IMAGES FROM PEOPLE YOU
SEE LISTED HERE.
I'M SUPPOSED TO GIVE YOU
LEARNING OBJECTIVES, SO FIRST OF
ALL TO APPRECIATE WHAT HAS BEEN
DONE OVER THE LAST SEIVET YEARS
AND SECOND, WE'LL GET TO THAT,
TO THINK ABOUT POSSIBLE FUTURE
ADVANCES FOR WHICH THIS CENTER
IS UNIQUELY POSITIONS: THIS IS
THE HOUSE OF HOPE, THE WORLD'S
LARGEST CLINICAL RESEARCH
CENTER.
ADMITTING PATIENTS ONLY ON
PROTOCOLS.
MANY OF THEM COMING HERE AS SORT
OF THEIR BEST HOPE AND LAST
RESORT AFTER THE REST OF MEDICAL
CARE HAS NOT REALLY BEEN ABLE TO
COME UP WITH ANSWERS.
SOME SELF-REFERED, SOMETHING BY
PHYSICIAN, SOMETHING LIKE 1500
CLINICAL STUDIES IN PROGRESS AND
RECOGNIZED IN MANY WAYS BUT I
THINK A PARTICULAR 1 TO
REMEMBER, 12 YEARS AGO, THE
LASKER BLOOMBERG PUBLIC SERVICE
AWARD GIVEN TO AN INDIVIDUAL, IN
THAT CASE, GIVEN TO THE NIH
CLINICAL CENTER AND YES, GOTTEN
SOME ATTENTION OVER THE YEARS.
MANY OF THE THINGS THAT HAPPEN
HERE DO INTEREST THE PUBLIC AND
THE PRESS.
THIS PARTICULAR DOCUMENTARY
CALLED FIRST IN HUMAN I THINK
WAS A WONDERFUL REPRESENTATION
OF WHAT THE REAL EXPERIENCE IS
LIKE IN THIS RESEARCH HOSPITAL
WHERE PATIENTS COME HERE,
PUTTING THEIR TRUST IN US AND WE
DON'T KNOW NOR DO THEY WHETHER
THE EXPERIMENTAL APPROACH WE'RE
GOING TO TAKE IS NECESSARILY
GOING TO WORK OR NOT BUT IT'S A
NOBLE ENTERPRISE AND YOU CAN SEE
THAT IN THAT DOCUMENTARY.
WELL, LET'S TURN BACK THE CLOCK,
BECAUSE THAT'S WHAT WE'RE
SUPPOSED TO DO WHEN WE DO A 70
YEAR ANNIVERSARY.
CHECK THAT OUT.
THAT BIG PILE OF DIRT IN THE
BACK WAS GOING TO BECOME
BUILDING 10.
I THINK THE BUILDING IN THE
BACKGROUND MUST BE THE CLOIOF
THER BECAUSE I THINK THAT'S WHAT
WOULD BE THERE AT THAT POINT AND
WELL, BUILDING 1 WAS HERE SINCE
THE MIDDLE AGES SO THAT'S
OBVIOUSLY THERE AND ALWAYS WILL
BE.
SO THAT WAS THE BEGINNING OF
PUTTING THAT TOGETHER, JUMP
FORWARD A YEAR OR 2, THE
DEDICATION OF BUILDING 10.
A LOT OF PEOPLE CROWDED OUT
THERE, YOU CAN SEE THE ENORMOUS
BRICK BUILDING.
FIRST TIME I CAME TO THE NIH
CAMPUS I WAS IN BETWEEN Ph D
IN PHYSICAL CHEMIST RADIOY AND
THINKING ABOUT GOING TO MEDICAL
SCHOOL AND I HAD THE OPPORTUNITY
TO COME AND VISIT WITH IRA
PASTON AND HIS LAB WHICH WAS
REALLY WONDERFUL, HE OPENED HIS
LAB MEETING WHO KNEW VERY LITTLE
ABOUT BIOLOGICAL SCIENCE BECAUSE
I WAS A CHEMIST AND WALKING
AFTER I HAD VISITED WITH HIM UP
IN BUILDING 37, WALKED BA PAST
THIS AND LOOKED UP AT THIS
BUILDING AND SAID THAT IS THE
BIGGEST TARN BUILDING I'VE EVER
SEEN IN MY LIFE.
AND IN MANY WAYS IT STILL IS,
BUT WHAT IN A PHENOMENAL
LOCATION, WHAT A PHENOMENAL
BUILDING IT IS FOR THINGS THAT
HAVE HAPPENED OVER THESE 70
YEARS, BEGINNING WITH THE VERY
FIRST PATIENT.
THIS IS A FARMER WHO CAME TO NIH
AS THE FIRST PATIENT WITH A
PROBLEM WITH PROSTATE CANCER.
SO STEPPING FORWARD FROM THERE,
I WANT TO WALK YOU THROUGH IN
VARIOUS AREAS, VARIOUS THEMES
JUST SOME OF THE MILESTONES THAT
HAVE OCCURRED HERE OVER THESE 7
DECADES AND AGAIN, IT'S GOING TO
BE A LITTLE BIT PERSONAL IN
TERMS OF WHAT I PICKED AND WHAT
I DIDN'T BECAUSE I CAN'T
POSSIBLY DO EVERYTHING AND
AGAIN, I WAS BENEFITED A LOT BY
JOHN GALLENS WHOSE KNOWLEDGE OF
THIS IS MUCH GREATER THAN MINE
TRYING TO MAKE SURE WE DIDN'T
LEAVE OUT THINGS THAT WERE
REALLY QUITE IMPRESSIVE
LANDMARKS BUT THERE ARE TOO MANY
EVEN OF THOSE.
SO START WITH CANCER; AN AWFUL
LOT OF CANCER ADVANCES HAVE
HAPPENED IN THIS BUILDING.
THE VERY FIRST CURE OF A SOLID
TUMOR CARIO CARCINOMA BY MEX
TREKSATE BY LEE AND HURTZ, WE
OFTEN SAY WHEN WE TALK ABOUT 1
OF THE MOST SIGNIFICANT ADVANCES
IN CANCER RESEARCH, IT'S THE
CURE OF CHILDHOOD A. L. L.
THAT WAS FIRST DONE HERE IN
1962, FREI AND FREIREICH, AND
THEN THE CONCEPT OF CHEMO
THERAPY.
COMING FORWARD WITH THE NAMES
HERE ISSUES THESE WERE MOSTLY
MEN, WE WILL SEE WOMEN LATER AND
I'M GLAD TO SEE THAT TOO BUT THE
REALITY OF 1965 IS THAT THESE
WERE MOSTLY MEN LEADING THE
RESEARCH EFFORTS HERE.
AND THEN COMING FORWARD FROM
CHEMO THERAPY TO IMMUNOTHERAPY,
I HAVE TO SAY A WORD ABOUT STEVE
ROSENBERG AND THE WAY IN WHICH
HIS VISION ABOUT HOW TO ACTIVATE
THE IMMUNE SYSTEM GAVE US A
WHOLE NEW SET OF TOOLS FOR
APPROACHING CANCER WHICH ARE
TRULY INSPIRING A LOT OF THE
CURRENT ACTIVITY TODAY.
SO I WILL FOCUS BRIEFLY ON THAT
1.
STEVE CAME IN THIS 1974,
STRAIGHT OUT OF HIS TRAINING AND
IMMEDIATELY TO BECOME A BRANCH
CHIEF AND PURSUING THIS IDEA OF
ACTIVATING THE IMMUNE RESPONSE,
INITIALLY WITH A LOT OF
FRUSTRATIONS AND FAILURES, BUT
THEN SOME SUCCESSES WITH
MELANOMA, AND MORE RECENTLY,
MOVING THAT FORWARD, WITH CAR
T-CELLS, USING TILL CELLS, TUMOR
INFILTRATING LYMPHOCYTES TO
TARGET MUTATIONS TO THE POINT
WHERE WHERE THERE ARE SUCCESSES
STARTING TO,A PEER IN BREAST
CANCER, PANCREATIC CANCER, COLON
CANCER AND SO ON.
AND I THINK STEVE WOULD SAY THIS
IS THE KIND OF FACILITY THAT
MADE IT POSSIBLE OVER THIS
STRETCH OF TIME WITH MANY HIGH
RISK AND MANY PROTOCOLS THAT DID
NOT SUCCEED TO KEEP PUSHING
FORWARD, THOSE BOUNDARIES WHICH
MIGHT HAVE BEEN VERY DIFFICULT
TO DO IN ANY OTHER PLACE AND NOW
HAS UNLEASHED AN ENTIRE FIELD OF
CANCER IMMUNOTHERAPY, MANY
PEOPLE LOOK AT THAT AS SOMETHING
THAT STEVE DESERVES TO BE
CONSIDERED THE REAL FATHER OF
THIS FIELD.
CONTINUING WITH CANCER, MARSTEN,
LANE, THE REMARKABLE WORK THAT'S
BEEN DONE HERE OVER MANY YEARS
DISCOVERING THE GENETIC BASIS
AND COMING UP WITH THERAPEUTICS
AS A RESILT FOR HEREDITARY
KIDNEY CANCER.
ROBERT PASTON AND ROBERT
[INDISCERNIBLE] WITH AN APPROACH
TO LEUKEMIA USING IMMUNO TOXICS
AND VERY COOL MOLECULAR BIOLOGY
AND ALSO A VERY COMMON DISEASE
WHICH HAS REALLY BEEN
TRANSFORMED BY PEOPLE RIGHT HERE
IN THIS BUILDING, PETER, BRAD
WOOD, WHO IN THEIR EFFORTS TO
REALLY FIGURE OUT HOW DO YOU
MANAGE A DISEASE THAT IS
EXTREMELY FREQUENT, BUT OFTEN
INDOLENT AND OFTEN TO THE POINT
WHERE IT DOESN'T REQUIRE THE
MOST AGGRESSIVE INTERVENTION,
HOW DO YOU KNOW WHEN TO ACT AND
WHEN NOT TO?
AND THIS APPROACH OF COMBINING
MRI IMAGING AND TARGETED
BIOPSYING HAS STOATALLY CHANGED
THE WAY WE APPROACH THIS
CONDITION, TO THE BENEFIT OF
MILLIONS OF MEN.
OKAY, JUMPING FROM CANCER, THESE
ARE IN NO PARTICULAR ORDER BUT
SICKLE CELL ANEMIA, A DISORDER
THAT ANY OF US WHO CARE ABOUT
MEN DEALIAN DISORDERS HAVE
STUDIED FROM THE VERY BEGINNING
1 OF THE FIRST, IN FACT THE
FIRST MOLECULAR DISEASE
UNDERSTANDING WHAT THE ACTUAL
MUTATION IS, A SINGLE LETTER
MISSPELLED IN THE BETA GLOBIN
GENE.
TO VERY SIGNIFICANT THINGS I CAN
PUT ON THIS, 1 IS GRIF ROGERS
RESEARCH WITH COLLEAGUES, TO
SHOW THAT HYDROXY UREA COULD IN
FACT PROVIDE CLINICAL BENEFIT TO
INDIVIDUALS WITH SICKLE CELL
DISEASE AND THAT IS STANDARD OF
CARE IN MANY PLACES INCLUDING
NOW IN SOME PLACES IN AFRICA.
BUT THE EXCITEMENT YOU SEE
WRITTEN ABOUT PARTICULARLY NOW,
ESPECIALLY THIS YEAR IS FDA IS
CONSIDERING WHETHER TO APPROACH
GENE THERAPY FOR SICKLE CELL
DISEASE, MUCH OF IT COMING RIGHT
HERE FROM THE WORK OF JOHN
TISDALE AND COURTNEY FITS HUGH
WORKING WITH BLUE BIRD, A
COLLABORATOR COMPANY TO TRY TO
SEE WHETHER YOU COULD IN FACT
TAKE BONE MARROW CELLS OUT OF
THE PATIENT, PROVIDE A GENE
THERAPY VECTOR THAT WOULD ALLOW
THE PRODECLARATION OF A LOT OF
NONSICKLE HEMOGLOBIN AND HAVE
SOMEBODY WHOSE HAD SICKLE
DISEASE FOR THEIR WHOLE LIFE, AS
YOU SEE HERE IN THE EXAMPLE OF
JANELLE STEVENSON, ESSENTIALLY
IT SEEMS, NOT JUST BENEFITED BUT
POTENTIALLY CURES FOR YEARS,
STILL SOMETHING TO LOOK AT IF
SOMETHING HAPPENS DOWN STREAM
BUT AS THE YEARS GO BY 1 AND 1
COULD USE THE C-WORD.
SIMILAR EFFORTS USING CRSPR, BY
OTHER APPROACHES ALSO GOING
FORWARD. THIS IS A
TRANSFORMATIVE MOMENT FOR THIS
FIRST MOLECULAR DISEASE AND WELL
IT IS TIME FOR THAT.
TALK ABOUT INFECTIOUS DISEASES.
I WAS NOTE HERE IN THE EARLY
1980S BUT AN AWFUL LOT OF WHAT
WENT ON TO THE CLINICAL CENTER
AND RIGHTLY SO WAS DEVOTE TOTD
HIV/AIDS LED BY TONY FAUCI AND
CLIFF LANE AND MANY OTHER
COLLEAGUES TRYING TO FIGURE OUT
WHAT IS THE CAUSE OF THIS,
DISCOVERING THE VIRAL CAUSE
COMING UP WITH IDEAS ABOUT
THERAPY.
THE VERY FIRST TREATMENT FOR
HIV, AZT, DEVELOPED BY SAM
BURGER WHO WENT ON TO BECOME THE
HEAD OF THE NATIONAL CANCER
INSTITUTE.
BUT OF COURSE OTHER INFECTIOUS
DISEASE VS HAD A RATHER
INTERESTING AND SIGNIFICANT
COURSE HERE IN BUILDING 10 AND
IN A FACILITIES NEARBY, EBOLA,
THE VACCINE FOR THIS DEVELOPED
OVER AT THE VACCINE RESEARCH
CENTER IN COLLABORATION WITH A
COMPANY BY NANCY SULLIVAN AND OF
COURSE, 1 OF THE MOST DRAMATIC
MOMENTS SINCE I'VE BEEN HERE IN
30 YEARS WHEN WE WERE CALLED
UPON, NOT ONLY TO DO RESEARCH ON
DEVELOPING A TREATMENT BUT
ACTUALLY TO TAKE CARE OF SOME OF
THE SICKEST PATIENTS YOU CAN
IMAGINE WHO WERE INFECT WIDE
THIS REALLY FRIGHTENING VIRUS
AND THAT INCLUDED BRINGING 2 US
A NURSE FROM DALLAS WHO WAS
QUITE ILL AND BEING CARED FOR IN
OUR XECIAL CLINICAL UNIT BY NONE
OTHER THAN TONY FAUCI HIMSELF
AND A NUMBER OF HIS OTHER
COLLEAGUES, WHICH THEN LED US TO
A PRETTY OUTCOME.
FIRST OF ALL THOUGH, THE VACCINE
EFFORTS AS YOU CAN SEE COMING
FORWARD WITH WHAT TURNS OUT TO
BE A VACCINE THAT IS SAFE AND
GOT MUCH DEBATED AND DISCUSSED
IN PLACES LIKE THE WHITE HOUSE
AS YOU CAN SEE WITH
PRESIDENT OBAMA THERE AND THEN
HERE THIS PICTURE COMING OUT OF
THE CLINICAL CENTER AFTER THE
ANNOUNCEMENT THAT NINNA PH AM
WAS NO LONGER INFECT WIDE THE
EBOLA VIRUS, TONY FAUCI, WITH
HIS ARM AROUND HER SHOULDER,
SOME OF HER FAMILY BEHIND HER,
THIS WAS QUITE A DAY, I MUST SAY
AND IT MADE YOU FEEL LIKE, WOW,
THIS PLACE NOT ONLY HAS THE MOST
AMAZING BASIC SCIENCE TO BE
BUILDING A VACCINE BUT ALSO TO
TAKE CARE OF SOME OF THE SICKEST
PATIENTS CAN YOU POSSIBLY
IMAGINE AND GET THEM WELL AGAIN.
CONTINUING WITH INFECTIOUS
DISEASE, AND I'M JUST REALIZING
SOMEHOW WHEN I MADE THIS SLIDE 1
THING GOT DROPPED OUT SO LET ME
SAY WHAT SOMEHOW GOT LOST, I
WANT TO TALK ABOUT HEPATITIS C.
AS YOU HEARD, I AM COMPLETELY
OBSESSED ABOUT THE IDEA THAT WE
MIGHT ACTUALLY ELIMINATE THIS
DISEASE THAT'S KILLING 15,000
PEOPLE IN THE UNITED STATES FOR
WHICH WE HAVE A HIGHER SAFE AND
EFFECTIVE ORAL CURE WHERE 1 PILL
A DAY FOR 12 WEEKS HAS A 98%
CURE RATE AND YET PEOPLE ARE NOT
GETTING IT AND THE REASON WE
KNOW ABOUT HEPATITIS C, A LOT OF
IT IS, BECAUSE OF HARVEY ARTHUR
WHO IS SITTING HERE AND HIS
COLLEAGUES IDENTIFYING THE VIRUS
AND COMING UP WITH A STRATEGY
THAT INDUSTRY THEN JUMPED UPON
TO COME UP WITH A THERAPEUTIC, A
HUGE CONTRIBUTION AND NOW WE
JUST HAVE TO MAKE SURE THAT
THOSE 2 EXPW HALF MILLION PEOPLE
WHO ARE CURRENTLY INFECTED
ACTUALLY GET ACCESS TO THE DRUG
DESPITE SOME OF THE LIMITATIONS
OF OUR HEALTHCARE SYSTEM AND THE
COST, AND THAT'S WHAT I'M
SPENDING AN AWFUL LOT OF MY TIME
ON RIGHT NOW.
ANOTHER THING THAT HAPPENED
HERE, ANOTHER PERSON SITTING IN
THE AUDIENCE, JOHN OSHAY AND
WARREN LEONARD IS WORKINGA A
PATHWAY THAT IS TURNING OUT TO
BE 1 OF THE MOST IMPORTANT FOR
AUTOIMMUNE DISEASES THAT BEING
THE JACK STAT 3, AND THE WHOLE
CLASS OF JACK INIBs, JACK
INHIBITORS WHO HAVE TURNED UTR
TO BE A GOD SEND FOR PEOPLE WHO
SUFFER FROM RHEUMATOID ARTHRITIS
AND LUPUS.
AND OF COURSE INFECT
YOURCANDIDATES DISEASE, IF YOU
WERE IN TWEBT 11 YOU MIGHT HAVE
BEEN AWARE AS MANY OF US WERE
THAT OUR CLINICAL CENTER WAS
FACING A CIRCUMSTANCE AND BEING
VERY PUBLIC ABOUT IT AND MANY
HOSPITALS OF FACING AND NOT
BEING PUBLIC ABOUT WHICH IS THE
RISK OF HAVING A HIGHLY
RESISTANT ORGANISM IN OUR CASE,
THE [INDISCERNIBLE], AND BEING
HERE WITH PATIENTS THAT ARE
IMMUNO SUPPRESSED THIS WAS A BIG
DEAL SO HOW DO YOU FIGURE THIS
OUT?
THE USUAL APPROACH DIDN'T WORK
SO WELL IN TERMS OF TRYING TO
UNDERSTAND EXACTLY WHAT THE
TRANSMISSIONS WERE JUST BY
PHYSICAL PROXIMITY.
SO JULIE AND TARA AND DAVID GOT
TOGETHER HERE AND BASICALLY USED
GENOME SEQUENCING TO UNDERSTAND
HOW ISOLATIONS OF THIS
PARTICULAR ORGANISM FROM
MULTIPLE PATIENTS WERE RELATED
TO EACH OTHER AND THAT WAS THE
MOST RELIABLE WAY TO SORT OUT
EXACTLY HOW THIS HAD PASSED
AROUND.
THIS ENDED UP IN THE NEW ENGLAND
JOURNAL AS WELL AS WALL STREET
JOURNAL AND THE ANSWER WAS THAT
UNEXPECTED HIDING PLACE, FAUCETS
SO A LOT OF REPLUMBING HAD TO
GET DONE TO TAKE CARE OF THIS.
THIS SET A STANDARD FOR THE WAY
IN WHICH THIS HOSPITAL
EPIDEMIOLOGY FOR RESISTANT
ORGANISMS NEEDS TO BE DONE IF
YOU'RE REALLY GOING TO FIND OUT
THE ANSWERS THAT YOU NEED.
ANOTHER CONTRIBUTION OF OUR
CLINICAL CENTER, NOT A HAPPY
SITUATION, BUT A GREAT
OPPORTUNITY FOR RESEARCH AND TO
FIND A SOLUTION.
VACCINES OF COURSE, WE HAVE TO
TALK ABOUT BECAUSE THIS HAS BEEN
ANOTHER PHENOMINALLY IMPORTANT
CONTRIBUTION OF NIH AND ITS
CLINICAL CENTER.
NEELSZON AND ROBBINS
PARTICULARLY FOR THE H-FLU
VACCINE WHICH IS TRANSFORMED THE
EXPERIENCE OF VERY YOUNG
CHILDREN BY MAKING THIS NO
LONGER THE TERRIBLE CAUSE OF
MENINGITIS AND OFTEN PREMATURE
DEATH THAT IT USED TO BE,
HEPATITIS A, B, AND ROTO VIRUS
VACCINES WORKED ON BY THE TRIO
YOU SEE LISTED HERE AND MANY
OTHERS.
MY LAB'S IN BUILDING 50, THOSE
IN BUILDING 50, YOU SEE THAT
PARTICULAR ELECTRON MICROSCOPE
IN THE LOB THAT'S WAS USED BY
ALBERT KAPIKIAN, THAT WAS THE
WAY IN WHICH MANY OF THESE
VIRUSES WERE FIRST IDENTIFIED TO
BE ABLE TO DEVELOP THE VACCINE
STRATEGIES.
HPV VACCINE TECHNOLOGY, NOW A
SEEN BEING USED--VACCINE BEING
USED ACROSS THE WORLD AS A WAY
OF PRESENTING CERVICAL CANCER
AND OF COURSE MOST RECENTLY OUR
HEROS FROM THE VACCINE RESEARCH
CENTER, AND THEIR TEAMS AND I
WILL COME BACK TO THAT IN A
MOMENT.
RIGHT NOW I WILL HIGHLIGHT THIS
1 BECAUSE THIS WAS ALSO A REALLY
GOOD EXAMPLE OF 2 SUPERB
MOLECULAR BIOLOGISTS WHO WOULD
NOT HAVE CALLED THEMSELVES
VACCINE EXPERTS OR
VACCINOLOGYISTS BUT JUST GOT
INTERESTED IN A WAY THAT YOU
MIGHT BE ABLE TO TAKE THIS
PARTICULAR VIRUS AND MAKE
SOMETHING IMMUNOGENIC THAT WOULD
BE PROTECTIVE AND PUBLISHED
PAPERS LIKE THIS AND THEN WITHIN
A FEW YEARS WHAT DO YOU KNOW,
FDA APPROVE THANKSGIVING AND AS
A RESULT OF THAT, THE LASKER
AWARD GOING TO DOUG AND JOHN A
FEW YEARS AGO.
A GREAT STORY OF BASIC
SCIENTISTS TURNING OUT TO BECOME
CLINICAL SCIENTISTS IN A HEROIC
WAY.
MENTAL ILLNESS, ANOTHER OBVIOUS
PARTICULARLY DEEP CONCERN OF ALL
OF US AS WE SEE AROUND US THE
STRUGGLES THAT HAVE BEEN
HAPPENING, THEY WERE THERE
BEFORE BUT COVID HAS CERTAINLY
ACCENTUATED THOSE.
NIH HAS BEEN IN THAT SPACE ALSO
IN A BIG WAY.
ALONG THE FIRST CLINICAL TRIALS
OF LITHIUM WAY BACK IN 1969
WHICH IS NOW STANDARD OF CARE
FOR PEOPLE WITH BIPOLAR DISEASE.
THE FIRST TREATMENT OF CHILDHOOD
SCHIZOPHRENIA BY JUDY RAPPA
PORT.
AND RECENTLY KETAMINE IN
SUICIDAL DEPRESSION, COMING UP
WITH A BETTER WAY OF PREVENTING
SUICIDE.
LET ME SAY A WORD ABOUT CARLOS'
WORK.
I'VE HAD THE CHANCE TO MEET WITH
HIM WHEN HE TREATS A PATIENT AND
HEAR THE INTERVIEW OF WHAT THE
PATIENT IS GOING THROUGH AND FOR
SOME OF THESE FOLKS THAT HAVE
HAD THE MOST RESISTANT FORM OF
CLINICAL DEPRESSION FOR DECADES
SOME OF THE STORIES YOU HEAR
FROM REALLY DRAMATIC, FROM
DESPAIR TO HOPE AFTER HOURS OF
AN INFUSION OF JUST THE RIGHT
DOSE OF INTRAVENOUS KETAMINE,
THIS MOLECULE NOT OF COURSE A
PANACEA FOR EVERYTHING BUT
CLEARLY FOR SOME OF THESE
INDIVIDUALS IN A WAY THAT IS
TRANSFORMED OUR UNDERSTANDING OF
HOW DEPRESSION WORKS AND GIVEN A
LOT OF PEOPLE AN OPPORTUNITY TO
HAVE THE CLOUDS LIFTED THAT HAVE
BEEN THERE FOR THEIR ENTIRE
LIVES.
AND THEN RARE DISEASES, A LOT OF
WHAT WE DO INVOLVES THE STUDY OF
RARE DISEASES, MANY OF THEM IN
CHILDREN, WE'RE FORTUNATE TO
HAVE THE CHILDREN'S INN DOWN TE
HILL TO MAKE IT POSSIBLE FOR
THESE KIDS AND THEIR PARENTS TO
COME AND BE INVOLVED IN RESEARCH
STUDY.
SOME OF THEM START OUT AS
NATURAL HISTORY STUDIES AND THEN
MOVE FORWARD TO THE IDEA OF
POSSIBLE THERAPEUTICS.
MY OWN LAB'S HAD THAT
EXPERIENCE, WITH PROGERIA, THIS
RAREST FORM OF PREMATURE AGING.
FOR A WHILE WE HAD A TRIAL GOING
ON HERE, SUBSEQUENT TO THAT
PROGERIATRICKIZATIONIA HAS AN
APPROVED DRUG AND WE'RE IN THE
PROCESS OF SETTING UP THE
POSSIBILITY OF A REALLY BOLD
APPROACH TO DO A CURE USING IN
VIVO GENE EDITING THAT TRIAL IF
WE CAN GET IT STARTED WILL BE
RUN AT BOSTON CHILDREN'S BUT SO
MUCH OF THE EVIDENCE WAS
ORIGINALLY DERIVED HERE IN THAT
INITIAL NATURAL HISTORY STUDY.
SO THERE'S A LOT THAT HAPPENED
HERE OVER TIME, PEOPLE MAY HAVE
FORGOTTEN THE VERY FIRST HUMAN
GENE THERAPY FOR ADHD AND THERE
WAS A LOT OF BUZZ BACK THEN BUT
NOW IT SEEMS THERE'S A NEW TRIAL
EVERY WEEK BUT THIS WAS THE VERY
FIRST 1.
DAN CASTENER'S WORK STARTING
WITH THE MEDITERRANEAN FEVER BUT
THEN DEFINING A WHOLE NEW CLASS
OF AUTOIMFLA AMATORY DISEASES
HAS TURNED INTO AN UNDERSTANDING
ABOUT A CATEGORY OF ILLNESS THAT
WE JUST DIDN'T HAVE BEFORE.
AND THEN I HAVE TO MENTION THE
UNDIAGNOSED DISEASES PROGRAM
FOUNDED BY BILL AND HIS TEAM,
OVER TIME DOZENS CLINICIANS
SEEKING TO HELP OUT PEOPLE WHO
HAVE HAD A DIAGNOSE NEVER REALLY
MADE AVAILABLE DESPITE MANY
EFFORTS TO DO SO.
THE MEMBERS ARE PRETTY
STAGGERING HERE, I GOT THESE
FROM BILL, A NUMBER OF MEDICAL
RECORDS THAT HAVE BEEN SCANNED
OVER 5000, OVER 1500 HAVE BEEN
ADMITTED AND EVALUATED OVER THIS
14 YEAR PERIOD.
FORTY% CHILDREN AND YOU CAN SEE
NEUROLOGICAL IS NOR THAN HALF,
MOST OF THESE POKE WHO IS GET
EXOHMS OR WHOLE GENOMES AND A
LOT OF DIAGNOSIS GET MADE AND
PUBLICATIONS.
AS A RESULT YOU KEY WHERE THOSE
ARE, A LOT OF IMPACT, A LOT OF
NEW DISEASES HAVE BEEN
IDENTIFIED AND THIS IS ALSO A
TERRIFIC TEACHING OPPORTUNITY,
THE WEEKLY PATIENT ROUNDS
ATTRACT LOTS OF YOUNG TRAINEES,
THE POST BACKS IN MY LAB ALL
LOVE GOING TO UDP, PATIENT
ROUNDS BECAUSE YOU SEE HOW
EXPERIENCED CLINICIAN TRY TO
THINK THROUGH A CHALLENGING
PROBLEM, SORTING THROUGH ALL THE
DATA, WHAT DO WE KNOW ABOUT
PHYSICAL FINDING, ABOUT THE
MEDICAL HISTORY, LAB RESULTS AND
WHAT SHOULD WE DO NEXT TO TRY TO
SORT THIS OUT.
AND THIS HAS BECOME NOT JUST AN
NIH CLINICAL CENTER EFFORT, IT
SPREAD OUT TO BECOME A NATIONAL
NETWORK, THE UNDIAGNOSED DECS
NETWORK AND NOW INTERNATIONALLY
THE UDNI, ALL OF THAT BASICALLY
TRIGGERING OFF WHAT BEGAN HERE
WITH DEDICATION OF BILL AND MANY
OTHERS.
SO THAT'S A WHOMP THROUGH JUST A
FEW THINGS, I HOPE YOU'RE
LOOKING AT THAT BEING PRETTY
MUCH IN AWE, BECAUSE I HAD, I
WAS PUTTING THIS TALK TOGETHER,
I KNEW OF SOME OF THESE AND
VAGUELY AWARE OF SOME OF THE
OTHERS ASK TO SEE THIS TRACK
RECORD OF THIS NUMBER OF MAJOR
ADVANCES THAT CHANGED THE COURSE
OF MEDICINE OF ALL ALL COMES
FROM THIS CLINICAL CENTER, THIS
BUILDING IS PRETTY DARN
PHENOMENAL, NO PLACE ELSE ON
EARTH THAT COMES CLOSE TO THAT
LEVEL OF ACHIEVEMENT.
SO WE SHOULD SAY CONGRATULATIONS
TO THE CLINICAL CENTER AND THEN
WE SHOULD THINK ABOUT, OKAY, NOW
WHAT?
SO ARE YOU READY TO GO ON TO
THAT PART?
WHAT ABOUT THE NEXT 70 YEARS?
WELL, OKAY, I CAN'T POSSIBLY TRY
TO MAKE PREDICTIONS OVER 70
YEARS.
I MIGHT BE ABLE TO TRY TO GUESS
OVER 5 OR MAYBE 10.
WE'RE IN THIS EXPONENTIAL PHASE
OF PROGRESS IN BIOMEDICAL
RESEARCH, MUCH OF IT DRIVEN BY
TECHNOLOGY AND SO, 1 HAS TO BE A
LITTLE FOOL HARDY TO IMAGINE YOU
CAN SEE MUCH BEYOND THE NEXT
HILL.
BUT LET'S TRY AND SAY A LITTLE
BIT ABOUT WHERE ARE SOME AREAS,
ANYWAY, THAT THE CLINICAL CENTER
IS PARTICULARLY WELL POSITIONED
TO TAKE ON, AND MOST OF THESE
YOU WILL SEE HAS ALREADY BEGUN
TO POSITION ITSELF BY STRONG
LEADERSHIP AND VISION ON THE
PART OF THE CLINICAL CENTER
GOVERNING BOARD, ON THE PART OF
OUR REMARKABLE CEO JIM GILLMAN,
ON ALL THE OTHERS WHO LEAD THIS
EFFORT, JOHN GALLEN AND ROLE AS
CHIEF SCIENTIST, EVERYBODY WHO
SEES THE OPPORTUNITY AT THE
CLINICAL CENTER TENDS TO HAVE
IDEAS ABOUT WHERE IT SHOULD GO
NEXT.
SO I HAVE SOME, TOO.
FIRST OF ALL, WE WILL HAVE TO BE
SURE THAT THE FACILITIES
MAINTAIN THE CUTTING EDGE
CAPABILITIES TO LET THINGS
HAPPEN THAT OTHERWISE ARE HARD
TO DO.
AND I THINK WE WOULD ALL AGREE
AND THAT'S NOT EASY BECAUSE
CONGRESS DOESN'T REALLY ENJOY
THE IDEA OF GIVING A LOT OF
MONEY TO MAINTENANCE AND WE HAVE
OUR OWN ISSUES OF MAINTINANCY
WITH OUR OWN CURRENT PHYSICAL
PLAN BUT WE ARE SEEING SOME
EXPANSION OF THAT CAPABILITY AND
I OF COURSE HAVE TO MENTION THAT
BIG CONSTRUCTION THAT YOU SEE
OVER THERE, GETTING GOING FOR
THE RLM, THE SURGERY RADIOLOGY
MEDICINE BUILDING LABORATORY
THAT,'S A PICTURE FROM THE
GROUND BREAKING, THAT'S ANDREA
PALM, DEPUTY SECRETARY OF HHS
AND SOME OTHER PEOPLE YOU KNOW
QUITE WELL, AND WHAT'S THIS
GOING TO LOOK LIKE?
RIGHT NOW IT LOOKS LIKE A LOT OF
DIRT ON THE GROUND BUT THIS MAY
BE THE EASIEST WAY IN THE MODEL
YOU CAN SEE AT THE ATRIUM OF THE
CLINICAL CENTER, ALL THE THINGS
OVER THERE WITH THE ORANGE ROOFS
THAT WILL BE THIS NEW FACILITY,
THE SRLM, SUBSTANTIAL ADDITION
IN TERMS OF OUR FOOTPRINT AND
OUR CAPABILITYINGS OF BEING ABLE
TO DO RESEARCH OFFERING REALLY
IMPORTANT THINGS LIKE OPERATING
ROOMS, GIVEN THAT OUR CURRENT
OPERATING ROOMS ARE REALLY QUITE
DATED AND MANY OTHER THINGS AS
WELL IN RADIOLOGY AND LAB
MEDICINE.
IT TOOK US QUITE A FEW YEARS OF
ADVOCATING FOR THE FUNDS FOR
THIS TO DO, BECAUSE YOU CAN'T
BUILD A BUILDING LIKE THIS,
UNLESS CONGRESS SPECIFICALLY
APPROPRIATES THE MONEY FOR IT
AND AGAIN, CONGRESS IS OFTEN
TIMES MORE INTERESTED IN SAYING
WE'RE GOING TO GIVE YOU MONEY TO
CURE CANCER AND WE WILL GIVE YOU
MONEY TO BUILD A BUILDING BUT
YOU CAN'T BUILD THE--YOU CAN'T
CURE THE CANCER WITHOUT THE
FACILITIES.
ULTIMATELY, WITH A LOT OF
CAJOLES AND ARM TWISTING THIS
BECAME POSSIBLE AND IT WILL BE
HAPPENING.
SO THAT'S GOING TO HAVE A LOT OF
CONSEQUENCES FOR MANY OF THE
PROTOCOLS THAT WE ARE ABLE TO
RUN HERE.
CELL THERAPY, IS AN AREA THAT
CLEARLY IS JUST BURSTING WITH
POTENTIAL, I MENTIONED SOME OF
STEVE ROSENBERG'S WORK AND SOME
OF JOHN TISDALE'S WORK SO FOR
THINGS LIKE HEMATOPOIETIC STEM
CELLS AND CAR T-CELLS AND OTHER
CREATE OF CANCER APPROACHES WHAT
YOU NEED IS A FACILITY THAT CAN
IN A GMP WAY MANAGE THAT SORT
AND TO BE HIGHLY EFFICIENT AND
SAFE.
AND A LOT OF INVESTMENT HAS BEEN
MADE HERE AT NIH TO BE ON THE
PATH AND WE'RE GETTING CLOSE TO
HAVING THE KIND OF SCALE THAT WE
THINK WILL BE NEEDED FOR ALL THE
PEOPLE WHO ARE REALLY ANXIOUS TO
BEGIN TO USE THOSE FACILITIES SO
I WILL SHOW YOU 2 OF THOSE NEW
FACILITIES THAT ARE GETTING
CLOSE.
THIS IS T-10 B WHICH WAS
BASICALLY BUILT IN WHAT USED TO
BE THE PATIO ON THE EAST SIDE OF
BUILDING 10 AND YOU CAN SEE THE
FLOOR PLAN THERE, IT MEETS GMP
SPECIFICATIONS, IT IS SCHEDULED
TO OPEN, VERY SOON IN THE NEXT
FEW MONTHS.
IT HAS 4 CULTURE ROOMS AND ALL
THE THINGS YOU WOULD WANT TO DO
TO BE ABLE TO DO THIS IN A VERY
HIGHLY WELL CONTROLLED FASHION.
AND THEN THERE'S THIS OTHER NEW
BUILDING WHICH IS TO BE
CONSTRUCTED BY THE FALL AND TO
OPEN IN SUMMER OF NEXT YEAR
WHICH YOU PROBABLY ALSO HAVE
SEEN UNDER CONSTRUCTION.
BETWEEN THE 2 OF THESE WE WILL
HAVE PROBABLY THE BEST
CAPABILITIES OF ANY CENTER
OUTSIDE OF INDUSTRY TO BE ABLE
TO DO THESE KINDS OF PROTOCOLS
SO THAT ALSO REFLECTS AN AWFUL
LOT OF THINKING OR PLANNING OVER
THE COURSE OF ABOUT 10 YEARS TO
GET US THERE.
ONE OF THE THINGS THAT THE CELL
THERAPY CENTERS MIGHT DO, IS
GOING TO BE IN THE AREA OF GENE
THERAPY, BECAUSE SOME OF THE
GENE THERAPIES WILL INVOLVE
SIMILAR APPROACHES.
HERE'S RAW --WHERE I WANT TO MAA
PARTICULAR POINT THAT I'M NOT
QUITE SURE HOW WE'RE GOING TO
HAVE AN EASY ANSWER TO AND THAT
IS THAT A LOT OF THE GENE
THERAPIES THAT ARE GOING TO BE
COMING FORWARD TO BE MOST
SUCCESSFUL WILL NEED TO BE DONE
AT THE EARLIEST MOMENT IN THE
LIFETIME OF THE INDIVIDUAL AND
PARTICULARLY IF YOU'RE TALKING
ABOUT A RARE GENETIC DISEASE,
THOSE OFTEN HAPPEN TO NEED THEIR
INTERVENTIONS EARLY.
AN EXAMPLE, AN EXAMPLE WHICH HAS
INSPIRED MANY OF US BECAUSE IN
MANY WAYS THIS WAS SORT OF THE
MOMENT WHERE YOU BEGAN TO SEE
THAT GENE THERAPY COULD BE
ALMOST MIRACULOUS, THAT'S SPINAL
MUSCULAR ATROPHY, A DEC WHICH IS
AUTOSOMAL RECESSIVE AND KIDS
BORN WITH THIS ARE FLOPPY AND
BASICALLY, THEY HAVE WHAT YOU
MIGHT CALL AN INFANTILE VERSION
OF ALS WITH THE MOTOR NEURONS
BASICALLY DYING OFF AND IF NO
INTERVENTION HAPPENS AND UNTIL
RECENTLY THERE WAS NO
INTERVENTION THAT WORKED THESE
CHILDREN WOULD DIE ESSENTIALLY
OF SUFFOCATION BY ABOUT A YEAR
OR YEAR AND A HALF.
YOU CAN'T IMAGINE ANYTHING MUCH
MORE HEART BREAKING THAN THIS
AND IT'S NOT THAT RARE A
DISEASE.
BUT THIS IS 1 OF THOSE WHERE A
LOT OF RISK TAKING WAS TAKEN
BOTH BY RESEARCHERS AND BY
FAMILIES TO SEE WHETHER IT MIGHT
BE POSSIBLE, WITH THE GENE
THERAPY TO DO SOMETHING ABOUT
THE DYSFUNCTIONING GENE THAT IS
NEEDING TO BE FIXED IN THE
SPINAL CORD.
THAT SOUNDS REALLY HARD.
IT WAS REALLY HARD BUT WITH AN
AAV 9 GENE THERAPY VECTOR
APPROPRIATELY LOADED, WITH THE
RIGHT P A YLOAD, THIS WAS THEN
BEGUN FOR A FEW KIDS.
I HAVE TO SHOW YOU A PICTURE OF
1 OF THEM.
THIS IS MATEO, MATEO HAD A
SISTER WHO HAD SMA AND DIED AT
ABOUT A YEAR SO THE FAMILY KNEW
THAT THEIR KIDS WERE AT RISK.
SO HE WAS DIAGNOSED WITHIN A
COUPLE WEEKS AT BIRTH AND GOT
THE GENE THERAPY AS I REMEMBER
WHEN HE WAS ONLY A MONTH AND A
HALF OLD AND THERE HE IS.
THIS IS ABOUT AGE 2 AND A HALF.
OKAY, GET THAT ELEVATOR BUTTON.
OKAY, MATEO, I THINK, TIME TO GO
TO WORK AND LOOK AT THIS ...
THAT'S WITH HIS DAD, I ALWAYS
GET CHOKED UP WATCHING THAT SO
THAT IS A LITTLE BOY WHO WITHOUT
THIS INTERVENTION WOULD SIMPLY
NOT HAVE HAD A CHANCE OF BEING
WITH US.
AND THAT HAS HAPPENED NOW WITH
THOSE THAT GET TREATED AS EARLY
AS POSSIBLE IN REMARKABLE WAYS.
THE LONGER YOU WEIGHT, THE LESS
RESULT YOU GET BECAUSE ONCE THE
ANTERIOR HORN CELL HAS DIED YOU
CAN BE THE BRING IT BACK.
I BRING THIS UP PARTICULARLY
BECAUSE I AM SOMEBODY WHO CARES
A LOT ABOUT GENETIC DISEASES,
AND A LOT OF THE RARE DISEASES
THAT WE WILL WANT TO TREAT, WE
WILL WANT TO TREAT THEM REALLY
EARLY AND THEREFORE I AM WORRIED
THAT WE ARE GOING TO HAVE TO
COME UP WITH SOME KIND OF A
SOLUTION FOR THE PROBLEM THAT WE
CURRENTLY HAVE IN TERMS OF
PEDEIATE RICKS FOR KIDS THAT ARE
YOUNGER THAN AGE 2 ASK THAT'S A
HARD PROBLEM AND I DON'T HAVE A
GOOD SOLUTION TO IT BUT I WANT
TO PUT IT OUT THERE AS PART OF
THIS LOOKING FORWARD.
BECAUSE I WOULDN'T WANT THE NIH
CLINICAL CENTER NOT TO BE IN THE
PLACE TO BE ABLE TO ACT UPON
SOME OF THOSE OPPORTUNITIES AND
THEY'RE GOING TO BE COMING.
VACCINES IS WHAT WE CAN DO AND
HAVE DONE IN OUR REPUTATION HERE
IS ASTOUNDING, AND I THINK
ALMOST THE WHOLE WORLD NOTICED
THAT IN THE CONTEXT OF COVID.
HERE IS THAT VISIT FROM
PRESIDENT BIDEN WHO CAME TO NIH
WITHIN 2 WEEKS OF BEING
INAUGURATED TO HEAR ABOUT WHAT
WAS HAPPENING AND HOW THE
VACCINE HAD BEEN PUT TOGETHER
AND YOU SEE BARNY GRAHAM ON THE
LEFT AND YOU SEE [INDISCERNIBLE]
ON THE RIGHT AND TONY FAUCI IS
THERE AND I'M THERE AND THE GUY
IN THE DARK SUIT THAT'S JEFF
[INDISCERNIBLE] WHO IS CURRENTLY
THE PRESIDENT'S CHIEF OF STAFF
AND IT WAS A DRAMATIC MOMENT TO
BE SURE AND A TESTIMONY TO THE
VISION THAT WENT INTO THE
FOUNDING OF THAT BRC WAY BACK
DURING THE CLINTON
ADMINISTRATION AND THE WAY IT
HAS EMERGED AS SUCH A POWER
HOUSE OF SCIENTIFIC INNOVATION.
BUT THEY'RE ALSO IN NEED OF MORE
CAPABILITIES SO I WAS GLAD TO
SEE AND I DON'T QUITE THINK I
KNEW WHAT THIS CONSTRUCTION WAS
ALL ABOUT, THAT'S THE NEW NORTH
TOWER VACCINE RESEARCH CENTER,
THIS IS AN ARTIST CONCEPTION,
ONCE ITS BUILT WILL GREATLY
EXPAND THEIR CAPAC IT--DIGITAL
TO TAKE ON EVEN MORE OF THE
CHALLENGES THAT YOU KNOW WILL BE
ASKED TO ADDRESS.
ANOTHER AREA THAT WE I THINK
HAVE MADE A SIGNIFICANT
STATEMENT ABOUT BECAUSE OF ITS
REALLY SERIOUS IMPACT ON
INDIVIDUALS AND FAMILY SYSTEM
ALZHEIMER'S DISEASE.
MUCH OF ALZHEIMER'S DISEASE
RESEARCH HAS BEEN GOING ON IN
OUR GRANTEE COMMUNITY AND
CERTAINLY IN INDUSTRY AND WE'RE
FINALLY AT THE POINT NOW WHERE
WE HAVE THERAPEUTICS WHICH
THEY'RE FAR FROM PERFECT BUT IF
ADMINISTERED TO PEOPLE VERY
EARLY DO SEEM CAPABLE OF SLOWING
DOWN THE DISEASE, WE WANT TO SEE
THAT EVEN BETTER AND SO AGAIN,
IN A VISIONARY WAY, THE BUILDING
THAT WAS JUST DEDICATED ABOUT A
YEAR AGO, IS NOW POPULATED WITH
PEOPLE WHO ARE GOING TO BE
TAKING PARTICULARLY INTERESTING
AND CREATIVE APPROACHES BOTH TO
ALZHEIMER AND TO RELATED
DEMENTIAS, THIS IS AT THE
DEDICATION OF THAT BUILDING
WHICH YOU CAN SEE THE SIGN IS
THE ROY BLUNT CENTER AND THAT IS
SENATOR BLUNT AND STANDING NEXT
TO HIM CONGRESSMAN TOM COAL.
I HAVE TO STOP FOR A MINUTE AND
SAY THOSE 2 INDIVIDUALS HAVE
BEEN OVER THE COURSE OF SOME 7
OR 8 YEARS 1 OF THE MOST
SIGNIFICANT REASONS WHY NIH'S
BUDGET HAS GROWN BY 57% SINCE
2015.
INCREDIBLY DEDICATED TO WHAT WE
DO HERE AND SO, IT WAS WONDERFUL
TO BE ABLE TO RECOGNIZE SENATOR
BLUNT IN THAT REGARD BY PUTTING
TOGETHER OF THIS CENTER WITH ALL
THAT IT CAN OFFER AND AGAIN
SENATOR BLUNT AND HIS WIFE
ABBY IS THERE.
SO MUCH POSSIBILITY THERE.
PRECISION MEDICINE, ANOTHER
FAVORITE TOPIC OF MINE.
YEAH, I'M BEING A LITTLE BIT
PERSONAL IN TERMS OF THE THINGS
THAT I PICK HERE.
PRECISION MEDICINE BEING THE
OPPORTUNITY TO TRY TO
INTORPERATE INTO THE WAYS THAT
WE PREVENT AND TREAT DISEASE,
EVERYTHING ABOUT THE INDIVIDUAL,
THE OPPOSITE OF
ONE-SIZE-FITS-ALL.
AND 1 WAY TO DO THAT OF COURSE
IS TO HAVE GENOME SEQUENCES ON
EVERYBODY AND ANOTHER WAY TO DO
IT IS TO HAVE REALLY RIGOROUS
DATA ON ENVIRONMENTAL EXPOSURES,
BEHAVIORS, SOCIOECONOMIC STATUS,
SOCIAL DETERMINANTS OF HEALTH,
EVERYTHING YOU CAN THINK OF THAT
PLAYS OUT AND IN ORDER TO MAKE
SENSE OF THAT AND REALLY
UNDERSTAND THE RELATIONSHIPS TO
MEDICAL OUTCOMES YOU NEED A VERY
LARGE STUDY, SO THAT YOU CAN
ACTUALLY BEGIN TO TELL WHAT ARE
THE DRIVERS OF WHETHER SOMEBODY
IS HEALTHY OR IS BEGINNING TO BE
FALLING INTO A CHRONIC ILLNESS.
SO WE ARE AT A VERY CRITICAL
JUNCTURE AND AN INTERESTING 1 IN
THIS COUNTRY BECAUSE WE HAVE
SUCH A STUDY.
ALL OF US STARTED 4 OR 5 YEARS
AGO IS NOW UP TO 600 AND
SOMETHING THOUSAND ENROLLEES.
I HOPE MANY OF YOU ARE ALREADY
INVOLVED IN ALL OF US BUT IT'S
NOT TOO LATE BECAUSE WE'RE GOING
FOR A MILLION.
IF YOU WANT TO JOIN, JOIN
ALLOFUS.ORG.
I'M IN IT I HAVE MY WHOLE GENOME
SEQUENCE BACK AND THERE'S LOTS
OF INTERESTING STUFF IN THERE.
AND ALL OF US IS ALSO UNIQUE IN
SEVERAL OTHER WAYS BESIDES THE
FACT THAT IT'S PROVIDING GENOME
SEQUENCES, IT IS GIVING ALL THE
DATA BACK TO PEOPLE SO YOU KNOW
IF YOU'RE PART OF THIS, WHAT
THEY KNOW AND IT IS ALSO HIGHLY
DIVERSE IN THE WAY IN WHICH THE
DESIGN WAS DONE.
YOU CAN SEE FROM THE PIE CHART
JUST ABOUT HALF, BUT ONLY ABOUT
HALF ARE WHITE AND THEN THERE IS
A STRONG REPRESENTATION OF RACE
AND ETHNICITY AND ALSO OVER ON
THE RIGHT YOU WILL SEE OTHER
KINDS OF DIVERSITY IN TERMS OF
INCOME, EDUCATIONAL ATTAINMENT,
SEXUAL ORIENTATION, GEOGRAPHY,
DISABILITY AND SO ON.
NO OTHER COHORT STUDY I THINK IN
THE U.S. OR MAYBE ANYWHERE HAS
MADE THIS KIND OF AN EFFORT TO,A
CHIEF SUCH A DIVERSE POPULATION
OF PARTICIPANTS.
AND YEAH, IF YOU ADD IT ALL UP,
80% IF YOU COUNT THE THINGS ON
THE RIGHT ARE UNDERREPRESENTED.
ALL THESE FOLKS ARE CONSENTED TO
MAKE THEIR MEDICAL RECORDS
AVAILABLE, TO HAVE THEIR GENOME
SEQUENCED TO HAVE LOTS AND LOTS
OF QUESTIONNAIRES THAT THEY FILL
OUT ABOUT THEIR OWN HEALTH
BEHAVIORS AND MEDICAL EXPERIENCE
APPROXIMATES, AND THIS IS NOW
AVAILABLE TO RESEARCHERS AND THE
RESEARCHER WORK BENCH IN A
FASHION THAT THE RESEARCHERS
PROMISE NOT TO TRY TO IDENTIFY
INDIVIDUALS AND THE DATA IS
ESSENTIALLY ANONYMIZED SO YOU
CAPTAIN TELL WHO IS WHO BUT YOU
CAN DO A HUGE AMOUNT OF
INTERESTING RESEARCH HERE AND
THAT IS REALLY STARTING TO KICK
UP IN A BIG WAY.
BUT I PUT THIS IN FRONT OF YOU
BECAUSE I THINK THERE IS A
REMARKABLE OPPORTUNITY HERE
INVOLVING THE CLINICAL CENTER,
WHICH IS NOT HAPPENED QUITE YET,
BUT IT'S BEGINNING TO BE
DISCUSSED.
AFTER ALL, 603,000 PARTICIPANTS.
245,000 OF THEM ALREADY HAVE
HIGH GENOME ACCESS SEQUENCES,
ALREADY ACTIVE ACROSS THE
GENOME.
IMAGINE THAT YOU COULD LOOK
THROUGH 245,000 GENOME
SEQUENCES.
YOU ARE GOING TO FIND SOME
INTERESTING AND SURPRISING
THINGS.
YOU WILL FIND PEOPLE WHO SEEM TO
HAVE LOST BOTH FUNCTIONAL COPIES
OF A GENE WE THOUGHT WAS
IMPORTANT AND YET THEY'RE
ENROLLED IN THIS STUDY SO THERE
MUST BE SOME WAY THAT DOING OKAY
ANYWAY.
WE SPENT A LOT OF TIME LOOKING,
TRY TO UNDERSTAND GENE JUNCTIONS
BY STUDYING MICE, HERE WE HAVE A
CHANCE TO STUDY PEOPLE BECAUSE
NATURE HAS ALREADY DON FOR US
THE HUMAN KNOCK OUT PROJECT WE
HAVE TO GO AND DISCOVERY IT AND
THERE WILL BE PEOPLE WHO HAVE A
GENO TYPE THAT WILL TELL YOU
THEY SHOULD HAVE A DISEASE THIS
IS A DISEASE, IT'S ALREADY
STARTING TO POP UP BUT THEY
DON'T SEEM TO SO THIS IS AN
OPPORTUNITY AND UNBI ASSED WAY
TO FIGURE OUT WHAT PEN TRANCE IS
REALLY IS LIKE.
SO YOU COULD CALL THIS AND THIS
IS SOMETHING LES BEESEEKER HAS
DONE IN ANOTHER STUDY CALLED
CLINSEQ, WHICH IS BASICALLY GENO
TYPE FIRST.
YOU WILL NOT PICK TO TORT OF
APPROACH SOMEBODY FOR RESEARCH
BECAUSE OF THEIR PHENOTYPE,
BECAUSE OF THEIR GENO TYPE.
THE PATIENTS WHO ARE PART OF ALL
OF US HAVE ALL BEEN CONSENTED
FOR RECONTACT, SO THIS IS
ETHICALLY ALLOWABLE AND THE
QUESTION IS, WHERE DO YOU START?
WHICH GENO TYPES WOULD BE MOST
APPROPRIATE TO BEGIN TO EXPLORE?
AND THEN HOW DO YOU DECIDE HOW
TO DO SOMETHING TO FIGURE OUT
WHAT THE PHENOTYPE IS, WHEN YOU
DON'T REALLY QUITE NOW KNOW WHAT
YOU'RE LOOKING FOR.
I DON'T KNOW ANY PLACE ON THE
PLANET THAT'S BETTER SITUATED TO
DO THAT TYPE OF GENO TYPE FIRST
PHENOTYPING THAN THE NIH
CLINICAL CENTER.
IF WE HAVE THE APPROPRIATE
COMBINATIONS OF CLINICIANS
BECAUSE THIS SOUNDS LIKE THE
UNDIAGNOSED DISEASES PROGRAM,
DOESN'T IT?
EXCEPT THERE YOU'RE STARTING
WITH A PUZZLING PHENOTYPE, HERE
JIEWR STARTING WITH A PUZZLING
GENO TYPE AND TRYING TO SORT
THAT OUT.
IT DOES SEEM THAT THE CLINICAL
CENTER IS THE KIND OF PLACE THAT
WAS SORT OF DESIGNED TO DO THIS.
THIS COULD BE A PILOT, IF IT
LOOKS LIKE THIS IS USEFUL
ACTIVITIES, MANY OTHER ACADEMIC
CENTERS MAY WANT TO DO THINGS
THAT ARE SIMILAR BUT FOR US TO
BEGIN TO START IT OFF AND SEE
WHAT WE CAN LEARN, I THINK WOULD
BE A VERY INTERESTING AND
EXCITING OPPORTUNITY AND I
SUSPECT A LOT OF CLINICIANS
WOULD KIND OF GET AN INTERESTING
EXPERIENCE OUT OF BEING PART OF
SUCH A TEAM TO SORT OUT WHAT ARE
THE RIGHT THINGS YOU WANT TO DO
WITH YOUR PARTICIPANT PATIENT IN
TERMS OF FIGURING OUT WHAT ARE
THE CONSEQUENCES OF A PARTICULAR
SURPRISING DNA GENO TYPE.
SO WE'LL SEE WHERE THAT GOES,
THIS IS ACCOUNTIVELY BEING
DISCUSSED BY A FEW OF US RIGHT
NOW ANDY WOO WILL SEE WHERE IT
GOES.
COMING CLOSE TO WINDING UP HERE,
BUT 2 OTHER POINTS, I WANT TO
MAKE ABOUT CHALLENGES FOR THE
NEXT 70 YEARS, AND THIS IS 1
THAT TENDS NOT TO BE ON
EVERYBODY'S MINDS BUT IT SHOULD
BE.
I CAN CERTAINLY TELL YOU THAT
BACK IN 2015 WHEN WE HAD AN
ISSUE IN THE PHARMACY WHERE
THERE WERE CONCERNS ABOUT SAFETY
OF SOME OF THE COMPOUNDS THAT
WERE BEING PREPARED, THIS BECAME
VERY MUCH OF INTEREST TO THE
U.S. CONGRESS, TO THE POINT
WHERE WE WERE REALLY QUITE
ALARMED THAT THIS WAS SUCH A
CONCERN THAT IT MIGHT REALLY
DAMAGE OVERALL CERTAINTY OF OUR
ENTIRE ENTERPRISE AND THAT WAS A
CONCERN BUT OF COURSE MORE
IMPORTANTLY THIS, IS NOT
SOMETHING YOU SHOULD JUST DECIDE
OH WELL, MISTAKES HAPPEN.
THESE ARE PATIENTS THAT WE'RE
TAKING CARE OF, THEY COME HERE
TRUSTING US TO DO EVERYTHING
THAT WE CAN TO GIVE THEM THE
BEST CARE WE CAN AND THAT MEANS
WE HAVE TO BE TUNED IN TO ALL OF
THE SAFETY ISSUES AS MAYBE THE
MOST IMPORTANT THING THAT WE'RE
WE'RE DEDICATED TOO.
THAT REQUIRED A CHANGE IN THE
CULTURE, BACK IN THOSE DIFFICULT
DAYS, WE HAD A RED TEAM THAT
CAME AND HELPED US WITH THAT, WE
HAD EXPERT LIKE PAUL O'NEIL AND
PETER [INDISCERNIBLE] WHO CAME
AND GIVE US INFORMATION ABOUT
WHAT OTHER INSTITUTIONS THAT HAD
FACED UP TO THIS HAD DONE.
WE HIRED JIM GILMAN AND WE
BROUGHT TOGETHER A WHOLE TEAM
THAT WOULD CHANGE THE WAY IN
WHICH WE APPROACH PATIENT
SAFETY.
WE KNEW THIS WAS HARD.
THIS STUDY WAS DONE 25 YEARS AGO
BY THE NATIONAL ACADEMY OF
MEDICINE, BASICALLY TO ERR IS
HUMAN, ESTIMATE THAD SOMETHING
IN THE NEIGHBORHOOD OF 50,000,
MAYBE A HUNDRED THOUSAND DEATHS
WERE HAPPENING EVERY YEAR
BECAUSE OF MEDICAL ERRORS AND
MANY OF THOSE ARE PREVENTIBLE,
AND IF YOU THINK WE MUST HAVE
FIXED THAT BY NOW.
HERE'S A STUDY JUST PUBLISHED A
YEAR AGO BUT IT WAS BASED UPON
DATA BACK IN OCTOBER OF 2018.
A QUARTER OF MEDICARE PATIENTS
EXPERIENCED HARMS, HALF OF THOSE
WERE PREVENTIBLE IN
OCTOBER 2018, THERE'S A MORE
RECENT NEW ENGLAND JOURNAL
ARTICLE THAT HAS SIMILAR RESUMS
SO WE HAVE TO TAKE THIS
SERIOUSLY.
THIS IS SUCH AN IMPORTANT ISSUE
AND MANY PEOPLE WOULD POINT OUT
WE WOULD NEVER TOLERATE THIS IN
MUCH OF ANY OTHER INDUSTRY,
CERTAINLY NOT THE AIRLINE
INDUSTRY WHERE MISTAKES ARE LIKE
OH, WELL, WE HAVE TO HAVE THE
SAME ATTITUDE AND WE HAVE TO BE
WILLING TO SAY THIS IF YOU
DISCOVER SOMETHING HAS GONE
WRONG, THAT YOUR A HERO IF YOU
TALK ABOUT IT AS OPPOSE TO NOT
TALKING ABOUT IT.
A BIG ADVANCE THAT HAPPENED THAT
I GOT TO BE PART OF THIS, QUITE
A FEW TIMES, IS THE PATIENT
HUDDLE, EVERY DAY, BRINGING
TOGETHER THERE IN THE MEDICAL
BOARD ROOM ALL THE PEOPLE MOST
INVOLVED IN PATIENT CARE SAYING
OKAY, IN THE LAST 24 HOURS, IS
THERE ANYTHING THAT UPON HAD
THAT WE NEED TO LEARN FROM THAT
WAS A CLOSE CALL OR EVEN
SOMETHING THAT WENT BEYOND A
CLOSE CALL AND THEN HAVING THE
METRICS AND KEEPING TRACK OF ALL
THAT, TO KNOW HOW WE ARE DOING
IN THIS SPACE, IS REALLY
CRITICAL.
IF WE'RE GOING TO SAY, WE ARE,
THE LARGEST, AND MOST EFFECTIVE
RESEARCH HOSPITAL IN THE WORLD,
WE HAVE TO BE EFFECTIVE AT THIS,
TOO.
AND FINALLY, I DO WANT TO SAY
JUST A WORD ABOUT THIS CRITICAL
SET OF NEW INITTIAIVE ITS, AND
NEW ATTITUDES THAT WE HAVE
DEVELOPED HERE PRETTY MUCH IN
THE COURSE OF THE LAST 2 OR 3
YEARS BUT CERTAINLY GOING BACK
FURTHER ABOUT YOU WE BROUGHT IT
MUCH MORE TO THE FRONT AND THAT
HAS TO RELATE TO HOW IMPORTANT
IT IS THAT WE WORRY ABOUT OUR
REPRESENTATION IN EVERY WAY.
THAT WE RECOGNIZE AS AN
INSTITUTION, WE ARE AN
INSTITUTION THAT HAS BEEN
BROUGHT THROUGH A CULTURE THAT
HAS STRUCTURAL RACISM BUILT INTO
IT, AND WE CAN'T SAY THAT THAT'S
NOT ALSO PART OF OUR HISTORY AND
WE HAVE TO COME UP WITH WAYS TO
FOCUS ON THAT MORE EXPLICITLY
AND THAT MEANS FOCUSING IN AN
INTENTIONAL WAY ON HEALTH
DISPARITIES NOT JUST TO ADMIRE
THE PROBLEM BUT TO COME UP WITH
SOLUTIONS TO FOCUS ON OUR
INTERNAL WORKFORCE AND
PARTICULARLY ON THINGS LIKE
DISCRIMINATION AND HARASS, AS
WELL AS OUR EXTERNAL WORKFORCE
AND THIS IS WHAT THE UNITE
INITIATIVE IS DOING IN A REALLY
REMARKABLE WAY INVOLVING PEOPLE
AT BOTH LEVELS AT BOTH
IMPOSSIBLE TO BUILD RAMURAL AND
EXTRAMURAL AND I WANT TO SAY HOW
IMPRESSED I HAVE BEEN WITH THE
ACCOMPLISHMENTS THAT HAVE BEEN
MADE SO FAR AND THE DETERMINE
NATION THAT DOES NOT SEEM TO BE
WANING AT ALL AND YOU CAN READ
MORE ABOUT THIS IN THE PROGRESS
REPORT PRACTICES MA RIBERNARD
AND OTHER COLLEAGUES.
SO THINK ABOUT THOSE THINGS AS
SORT OF THINGS ABOUT THE FUTURE,
WE DID THE PAST, WE DID THE
FUTURE, FINALLY, I THINK JUST
COMING BA BEING TO WHAT A
PRIVILEGE IT IS, SPEAKING FOR
MYSELF BUT I HOPE I'M SPEAKING
FOR ALL OF YOU TO BE ABLE TO BE
PART OF THIS HOUSE OF HOPE, TO
THINK ABOUT WHAT IT HAS DONE
OVER THESE 70 YEARS TO RECOGNIZE
THE PEOPLE THAT WE ARE MOST TO
BE GRATEFUL TOO ARE THE PATIENT
WHO IS HAVE COME HERE AND PUT
THEIR TRUST IN US AND NOT ALL OF
THEM HAVE HAD THE HAPPY OUTCOME
AND YET, THEY WERE WILLING, EVEN
IF IT WASN'T GOING TO HELP THEM,
TO KNOW THAT WE WERE GOING TO
LEARN SOMETHING THAT MIGHT HELP
THE NEXT PERSON.
IT IS THE HOUSE OF HOPE AND IT
IS OUR 70th BIRTHDAY, SO I
GUESS WE SHOULD CELEBRATE THAT
BUT ALSO IF IT'S ABOUT HOPE,
RECOGNIZE THAT HOPE IN MANY WAYS
IS SOMETHING YOU CAN'T JUST
THROW OUT THERE LIKE A NICE
IDEA, IT HAS CONSEQUENCES, HOPE
IN EVERY SPHERE OF LIFE IS A
PRIVILEGE.
WE ARE PRIVILEGED TO BE IN THE
HOUSE OF HOPE AND IT ATTACHES TO
ACTION.
THIS CLINICAL CENTER IS A HOUSE
OF HOPE BUT IT'S A HOUSE OF
ACTION AND I'M PROUD TO BE ABLE
TO TELL BUT IT AND TO HAVE SPENT
MY LAST 30 YEARS HERE TO SEE THE
WAY IN WHICH THIS HAS EVOLVED
AND WILL CONTINUE TO EVOLVE IN A
WAY THAT WILL SAVE MANY LIVES
AND REDUCE LOTS OF SUFFERING BUT
ONLY BECAUSE OF ALL OF YOU AND
YOUR COMMITMENT TO KEEPING THAT
DREAM GOING IN A WAY THAT HAS
NEVER HAD A BETTER CHANCE THAN
RIGHT NOW BUT IT'S GOING TO TAKE
EVERY BIT OF ENERGY AND
CREATIVITY AND RISK TAKING THAT
HAS CHARACTERIZED THIS CLINICAL
CENTER FROM THE VERY BEGINNING
BACK IN 1953.
THANK YOU ALL SO MUCH.
[ APPLAUSE ]
NTHANK YOU FRANCIS.
SO THANKS FOR A WONDERFUL
PRESENTATION.
WE DO HAVE TIME FOR QUESTIONS,
THERE IS AN OPPORTUNITY FOR
THOSE OF YOU ON VIDEOCAST TO
SEND QUESTIONS DR. BURKLOW WILL
FIELD THOSE AND PASS THEM ALONG
TO US FROM 1 OF THE MICROPHONES
BUT IF YOU HAVE A QUESTION, IT
NOW WOULD BE THE TIME TO HEAD TO
THE MICROPHONE.
CLEARLY YOU ANSWERED EVERYTHING
THAT WAS ASKED AND THANK YOU
VERY MUCH FOR A WONDERFUL
PRESENTATION AND I JUST WANT TO
SAY OUR PATIENTS ARE OUR HEROES.
I DID HAVE A FEW THINGS THAT
CAME IN FROM VIDEOCAST.
YOU DID A GREAT JOB OF
ADVERTISING THE ALL OF US STUDY,
I DID GET A QUESTION, HOW DO
PEOPLE SIGN UP FOR THE ALL OF US
STUDY?
THAT WOULD BE GOOD TO REPEAT
THAT.
>>THANK YOU FOR WHOEVER ASKED
THAT QUESTION.
YEAH, WE ARE STILL LOOKING FOR,
YOU KNOW ANOTHER 380,000 PEOPLE.
IT'S VERY EASY.
JOINALLOFUS.ORG, GO TO THAT AND
YOU WILL SEE THE STEPS, YOU WILL
BE ASKED GO THROUGH MATERIAL AND
TELL YOU ABOUT THE STUDY AND
SOMETHING ABOUT THE WHAT THE
CONSENT WILL INVOLVE AND THE
FACT THAT THERE WILL NEED TO BE
PHYSICAL MEASUREMENTS AND A
BLOOD SAMPLE BUT IT'S PRETTY
SIMPLE.
THAT'S THE PLACE TO START.
AND I THINK IF YOU JOIN, YOU
WILL LEARN A LOT ABOUT YOURSELF
THAT YOU MIGHT OTHERWISE NOT BE
GIVEN THE CHANCE TO DO.
>>THANK YOU DR. COLLINS.
ANOTHER QUESTION, MORE OF A
COMMENT, THANK YOU VERY
HIGHLIGHTING THE WORK WITHIN THE
CLINICAL CENTER INVESTIGATORS.
ONE COMMENTER DID WANT TO SAY
THAT OF COURSE OUTSIDE THE NIH,
WE'RE [INDISCERNIBLE] WAS THE
SIGNIFICANT CONTRIBUTION OF NIH
BIOLOGICAL AND MEDICAL SCIENCE
OF THE 20th CENTURY, I KNOW
YOU HIGHLIGHT CLINICAL CENTER
INVESTIGATORS.
>>YEAH, AND I STRUGGLE WIDE
THAT A BIT BECAUSE WALKING IN
HERE TODAY WENT PAST THE DISPLAY
FOR MARSHAL NUREMBERG LIKE A
PHENOMENAL CONTRIBUTION THAT WAS
MADE RIGHT HERE AT NIH.
I THOUGHT MY TASK WAS TO TALK
MORE ABOUT THE CLINICAL CENTER
THEREFORE THE CLINICAL
APPLICATIONS BUT I COULD HAVE
EASILY SPENT MORE TIME ON SOME
OF THE OTHER BASIC SCIENCE
ACHIEVEMENTS WHICH ARE EQUALLY
STUNNING.
>>THANK YOU AGAIN DR. COLLINS
FOR SUCH A GREAT TALK.
I'M DR. JOYCE CHUNG AT THE
CLINICAL CENTER I WANTED TO ASK
IF YOU COULD COMMENTOT
INCREDIBLE OPPORTUNITIES FOR
TRAINING HERE, WE HAVE A LOT OF
POST BACKS, WE HAVE A TREMENDOUS
NUMBER OF PEOPLE WHO COME HERE
TO LEARN AND LAUNCH THEIR
CAREERS SO I WAS WONDERING IF
YOU COULD TALK ABOUT THAT
CONTRIBUTION AS WELL.
>>I WOULD LOVE TO.
AS A TRAINING OPPORTUNITY, IT'S
HARD TO OVERSTATE HOW REMARKABLE
IT IS TO BE IN THIS PLACE AND OF
COURSE THAT'S PARTLY BECAUSE OF
THE CLINICAL CENTER AND THE
ACTUAL PROTOCOLS BUT IT'S ALSO
BECAUSE OF THE AMAZING BASIC
SCIENCE THAT SURROUNDS US ON
THIS CAMPUS AND ALSO UP IN
BALTIMORE AND IN NORTH CAROLINA
AND A COUPLE OTHER PLACES.
ALMOST ANYTHING YOU WANT TO KNOW
ABOUT, THERE'S NUMBER AT THE NIH
WHO'S IN THE SORT OF TOP 10 OR
15 EXPERS IN THE WORLD AND THE
PLACE IS ALSO QUITE OPEN TO
ACCEPTING PEOPLE TO JUST WALK IN
AND ASK QUESTIONS.
I WILL PARTICULARLY SAY A WORD
ABOUT THE POST BACK PROGRAM
BECAUSE MY LAB HAS BENEFITED
FROM THAT 4 POST BACKS IN MY
LAB, WE WILL HAVE 5 BY NEXT
WEEK.
THESE ARE FOLK WHO IS COME HERE
WITH INCREDIBLE TALENT AFTER
THEIR UNDERGRADUATE TRAINING
TRYING TO FIGURE OUT WHETHER
RESEARCH IS REALLY THEIR CALLING
OR WILL THEY GO TO MEDICAL
SCHOOL OR MD-Ph.D. OR Ph.D.
OR NONE OF THE ABOVE AND THEY
BRING THAT SKILL AND CREATIVITY
AND THAT DRIVE AND THEY MAKE AN
ENVIRONMENT JUST THAT MUCH MORE
EXCITING BY THEIR PRESENCE.
AND I THINK WE GIVE THEM A
PRETTY GOOD CHANCE, ALSO TO GET
THEIR ANSWERS TO WHAT IT IS THAT
THEY MIGHT WANT TO DO NEXT.
SO THAT'S THE POST BACKS,
CERTAINLY ALSO THE POST DOCKS,
ALTHOUGH RIGHT NOW WE HAVE SORT
OF A NATIONAL SHORTAGE OF POST
DOCKS AND THAT'S CERTAINLY
HAPPENING A BIT HERE AT NIH AS
WELL.
THE POST DOCS ARE JUST ANENTIOUS
SENTIAL PART OF WHAT WE GET TO
DO AND GRADUATE STUDENTS LET ME
ALSO SAY THAT WE DON'T HAVE A
GRADUATE PROGRAM WE CAN GIVE
DEGREES AND WE HAVE A LOT OF
PEOPLE DO THEIR GRADUATE WORK
HERE AND GET THEIR DEGREES AT
OTHER INSTITUTIONS AS PART OF
THAT PARTNERSHIP PROGRAM.
THIS PLACE WOULD NOT FUNCTION
WITHOUT THE TRAINEES AND
CERTAINLY WOULD NONAPOPTOTIC THE
BE ANY FUN SO I'M GLAD YOU ASK
THE QUESTION AND I THINK IF
ANYBODY'S LISTENING WHO'S THINK
BEING WHERE THEY MIGHT WANT TO
DO FUTURE TRAINING, CHECK US
OUT, WE'RE ON THE WEB.
[LAUGHTER]
>>THANK YOU VERY MUCH IT'S
REALLY HUMBLING TO SEE IT AND BE
PART OF IT.
CAN YOU COMMENTOT ROLE
TECHNOLOGY PLAYS BROADLY IN
THESE INNOVATIONS AND HOW WE
SHOULD TALK ABOUT THE
INNOVATIONS IN THE CLINICAL
CENTER?
>>YEAH, I THINK IT PLAYS A HUGE
ROLE.
I SHOULD HAVE HAD SOMETHING
ABOUT THE TRANSFUSION
CAPABILITIES HERE IN THE
CLINICAL CENTER AS A TECHNOLOGY
THAT HAS MADE A HUGE MEMBER OF
THINGS POSSIBLE BUT IT'S
CERTAINLY ALSO WHAT I'M TALKING
ABOUT SELLING GENE THERAPY,
THOSE ARE ALL TECHNOLOGY DRIVEN,
THE KINDS OF THINGS THAT ARE
DONE IN INTERVENTIONAL RADIOLOGY
BY SOMEBODY LIKE BRAD WOOD ARE
ALSO TRANSFORMATIVE IN THE
ABILITY TO TAKE CARE OF PATIENTS
AND I'M SURE THE CHANCES OF
DOING EVEN MORE OF THAT WITH THE
NEW BUILDING WILL GO UP.
SO IT'S EVERYTHING FROM THE FACT
THAT WE CAN DO SINGLE IS CELL
BIOLOGY RUE TEENLY WHICH I WOULD
BT HAVE SAID WE COULD DO 5 YEARS
AGO, TO DEALING WITH STEM CELLS,
THE VERY FIRST HUMAN TRIAL OF
AUTOOUS IPS DEDRIVED CELLS FOR
THE RETINA, BEING DONE HERE BY
CAMILLE [INDISCERNIBLE] AND THAT
ALSO INCLUDES ARTIFICIAL
INTELLIGENCE MACHINE LEARNING
WHICH WE ALL WOULD AGREE IS AN
OPPORTUNITY FOR MEDICINE THAT IS
REALLY JUST BEGINNING TO GET
GOING AND I WOULD HOPE THAT IN
THE COMING YEARS WE WOULD GET
EVEN STRONGENER THAT SPACE AS
WELL.
SO I'M KIND OF A TECHY AS CAN
YOU CAN TELL BUT IF YOU LOOK AT
WHAT'S HAPPENING AT THE
FOUNDATION OF BREAK THROUGHS IN
BIOMEDICAL RESEARCH IT'S OFTEN A
TECHNOLOGY THAT'S MAKING IT
HAPPEN.
>>THANK YOU AGAIN FOR YOUR TALK
DR. COLLINS I'M GINA MOODY FROM
NIH, I WAS CURIOUS IF YOU WOULD
HAVE ADVICE FOR PEOPLE EMERGING
AS CLINICAL INVESTIGATORS AND
HOW WE HAVE GOOD STEWARDSHIP OF
THE OPPORTUNITY WE'VE BEEN
GIFTED WITH MY BEING HERE?
>>WELL WE WANT MORE AND MORE OF
THOSE GIFTED CLIENICAL
INVESTIGATORS TO COME HERE
BECAUSE THE CLINICAL CENTER
REALLY DOESN'T WORK UNLESS WE
HAVE THOSE FOLKS WITH A DREAM OF
SOME CLINICAL PROGRAM THAT THEY
WANT TO START OF SOME KIND OF A
CLINICAL PROTOCOL THEY WANT TO
WRITE AND SO I HOPE THEY WILL
SEE THIS AS A TRULY UNIQUE
OPPORTUNITY TO LIVE OUT THAT
KIND OF A DREAM.
MAYBE YOUR QUESTION IS ALSO
ASKING ARE WE MAKING SURE THOUGH
THAT WE'RE USING THE RESOURCES
AS RESPONSIBLY AS POSSIBLE
BECAUSE THIS IS BOTH AN EXPENSE
AND ALSO YOU'RE ASKING PATIENTS
TO TAKE PART IN A PROTOCOL AND
YOU WANT TO BE SURE IT'S BEEN
WELL DESIGNED TO HAVE THE
MAXIMUM LIKELY BENEFIT AND
REDUCING THE RISKS AS MUCH AS
POSSIBLE.
BETWEEN THE IRB SYSTEM AND THE
FACT THAT INSTITUTES HAVE THEIR
SCIENTIFIC REVIEW, PROCESS FOR
CLINICAL PROTOCOLS, WE'RE IN A
PRETTY GOOD PLACE THERE IS I
HOPE THAT'S SOMETHING THAT
PEOPLE UNDERSTAND THAT THIS
ISN'T JUST SORT OF RANDOM IDEA
OF SOMEBODY STARTING A PROTOCOL
WITHOUT SOME OVERSIGHT.
THE OVERSIGHT'S IMPORTANT.
WE DON'T WANT IT TO BE TOO RISK
AVERS, HOWEVER BECAUSE AN AWFUL
LOT OF THINGS THAT TURN OUT TO
BE, THE BIG BREAKTHROUGHS, YOU
DON'T KNOW IF THEY WILL WORK AND
YOU HAVE TO FIGURE OUT HOW TO
EXPLAIN THAT TO A PARTICIPANT IN
A WAY NATHEY KNOW WHAT THEY'RE
GETTING INTO, AND IF THEY WANT
TO TAKE THE RISK THEN WE SHOULD
NOT BE SO PATERNALISTIC AS TO
DENY THAT.
>>DRO COLLINS, I'M WITH THE
CLINICAL CENTER, AND WONDERING
IN ALL OF THIS NEW TERRITORY
WHAT YOU'RE THINKING ABOUT
BIOETHICS CONCERNS, THE MEANING
AND PURPOSE QUESTIONS IN THE
2-SECONDS WE HAVE LEFT?
[LAUGHTER]
>>YEAH, BIOETHICS IN 2-SECONDS
THAT WILL NOT BE EASY BUT I DO
APPRECIATE YOUR RAISING IT
BEFORE WE WALK OUT OF THIS ROOM
BECAUSE THAT HAS TO BE PART OF
EVERYTHING THAT WE'RE DOING.
WE ARE WORKING AT THE EDGE OF
THE POSSIBLE.
WE ARE WORKING SOMETIMES AT THE
EDGE OF WHAT IT MEANS TO BE
HUMAN IF WE'RE TALKING ABOUT
INTERVENTIONS WITH THE BRAIN FOR
INSTANCE WHICH INCREASINGLY
WE'RE GOING TO BE DOING OR
MODIFYING THE GENOME.
SO I THINK FAT THACT WE HAVE A
VERY STRONG BIOETHICS UNIT HERE,
WHICH A LOT OF PLACES DON'T
NECESSARILY HAVE WITH DR. GRADY
IS SOMETHING WE SHOULD BE
TAPPING INTO WITH THE STRENGTH
THAT'S THERE AND HAVING OUR EYES
WIDE OPEN AND TRYING TO
ANTICIPATE BEFORE IT'S SUDDENLY
AN OH MY GOSH, WHY DID WE DO
THAT?
WHERE ARE WE GOING?
WHAT SHOULD WE BE THINKING ABOUT
AS FAR AS THE GUARD RAILS AND
THE GUIDELINES.
>>THANK YOU.
>>FRANCIS THANKS AGAIN FOR A
GREAT TALK AND THANKS FOR
EVERYBODY THAT HAD THE COURAGE
TO ASK A QUESTION AND THANK YOU
ALL FOR ATTENDING AND HAVE A
GREAT DAY.
THANKS.
[ APPLAUSE ]