ALL RIGHT GOOD MORNING EVERYONE.
WELCOME TO THE DC LUPUS
CONSORTIUM AND 30th
ANNIVERSARY OF THE LUPUS
CLINICAL RESEARCH.
MY NAME IS JULIA ONYECHI, I AM A
PHYSICIAN ASSISTANT WITH NIAMS
AND I AM ALSO THE NIAMS LEAD
FIGURE THE PROJECT HOPE CENTER.
THIS WILL HIGHLIGHT THE
OUTSTANDING PROGRESS MADE IN
LUPUS BY THE NIH IN THE LAST 30
YEARS, FEATURING TESTIMONIALS
FROM RESEARCH PARTICIPANTS,
UPDATES ON CURRENT LUPUS
RESEARCH ACTIVITIES AT NIAMS AND
DISCUSSION ON FUTURE DIRECTIONS
OF RESEARCH AT THE NIH.
THIS EVENT SEEKS TO BUILD ON THE
SUCCESS OF THE DC LUPUS
CONSORTIUM WHICH IS A PLATFORM
TO COLLABORATE BETWEEN NE H
SCIENTISTS, PHYSICIANS OUTSIDE
OF THE NIH AND PATIENT ADVOCACY
GROUPS INTERESTED IN THE LUPUS
CLINICAL RESEARCH.
WE HAVE A VERY PACKED ARK GENDA
FOR YOU GUYS SO WE WILL GET
STARTED RIGHT,A WAY.
FIRST UP AND DR. LINDE CRISWELL,
SHE IS THE DIRECTOR OF NIAMS AND
SHE WILL GIVE AN INTRODUCTION ON
THE DC LUPUS CONSORTIUM.
PLEASE GIVE HER A WARM WELCOME.
KD--SALLY ARK PLAWZ
--[ APPLAUSE ]
>> SO GOOD MORNING, EVERYONE AND
WELCOME TO THE ANNUAL MEETING OF
THE DC LUPUS CONSORTIUM, THIS
YEAR'S MEETING IS A SPECIAL 1 OF
COURSE BECAUSE WE'RE CELEBRATING
30 YEARS OF LUPUS RESEARCH
WITHIN THE LUPUS NATURAL HISTORY
PROTOCOL HERE AT NIH AND THE
FIRST TIME WE'VE MET IN PERSON
SINCE THE COVID-19 PANDEMIC AND
MY FIRST OPPORTUNITY TO ATTEND
THIS MEETING.
SO BEFORE WE START THOUGH, I
WANT TO ACKNOWLEDGE MISS ELAINE
PONCIEO, AND HER COLLEAGUES FOR
THEIR TREMENDOUS EFFORTS IN
ORGANIZING THIS MEETING.
WE ARE HERE TO CELEBRATE AND
RECOGNIZE THE PATIENTS, THE
PROVIDERS, THE RESEARCHERS, AND
THE ODORS VOCATES WHO SUPPORTED
LUPUS RESEARCH AT THE CLINICAL
CENTER FOR OVER 30IERS AND IN
PARTICULAR WE ACKNOWLEDGE THOSE
OF YOU WHO PARTICIPATED IN THE
LUPUS NATURAL HISTORY PROTOCOL
WHICH GOING ON ITS 30th YEAR.
THIS EFFORT HAS BEEN 1 OF THE
WORLD'S LARMINGEST LUPUS
CLINICAL RESEARCH DATA SETS, AND
ITS BECOME A RESOURCE TO BETTER
UNDERSTAND AND CHARACTERIZE THE
HETEROGENEITY OF LUPUS AND ALSO
TO PROVIDE BIOLOGICAL SPECIMENS
AND CLINICAL PHENOTYPES
SUPPORTING LUPUS RESEARCH, SO I
CONGRATULATE THE CLINICAL
RESEARCH AUTOPSY SERIES FOR
THEIR DEDICATION AND THEIR
DILIGENT WORK OVER THE PAST
SEVERAL YEARS AND TO EXPAND AND
FURTHER STRENGTHEN THIS PROGRAM.
FOR THOSE OF YOU NOT FAMILIAR, I
WANT TO PROVIDE CONTEXT FOR
WHERE THE LUPUS CLINICAL TRIALS
UNIT FITS AMONG THE MANY LUPUS
RESEARCH EFFORTS ACROSS NIH.
THE NIH IS MADE UP OF 27
INSTITUTES AND CENTERS AND IT'S
THE LARGEST MEDICAL RESEARCH
FUNDING AGENCY IN THE WORLD.
AND ALTHOUGH NIAMS IS THE NIH
LEAD FOR LUPUS RESEARCH, SEVERAL
OTHER INSTITUTES AND CENTERS
ALSO SUPPORT MEDICAL RESEARCH ON
LUPUS AND OTHER AUTOIMMUNE
DISEASES AND MANY RESEARCHERS
FROM THOSE OTHER UNITS
COLLABORATE WITH NIAMS
INVESTIGATORS TO STUDY LUPUS AND
TO CARE FOR LUPUS PATIENTS AT
THE NIH CLINICAL CENTER.
NIAMS IS LUPUS CLINICAL TRIALS
UNIT SHOWN ON MY LEFT SIDE
IMPLEMENTS PROTOCOLS IN SUPPORT
OF EMERGING RESEARCH TOPICS AND
THE DC LUPUS CONSORTIUM WAS
ESTABLISHED BY THE LCTU ALMOST
10 YEARS AGO TO BETTER CONNECT
WITH LOCAL AND REGIONAL
PHYSICIANS PATIENT ADVOCACY
GROUPS AND ACADEMIC COMMUNITIES
IN HOPES OF INCREASING AWARENESS
OF IMPORTANT STUDIES AT THE NIH
CLINICAL STUDY SUCH AS THE
NATURAL HISTORY PROTOCOL AS WELL
AS TO PROMOTE PARTNERSHIPS AND
REFERRALS TO THE NIERK AMS LUPUS
CLINICAL RESEARCH PROGRAM.
SUCH EFFORTS ARE MUCH
APPRECIATED AND WELL RESPECTED
IN OUR COMMUNITIES AS MONTGOMERY
COUNCIL MEMBER WILL
[INDISCERNIBLE] RECOGNIZED THE
WORK DONE BY NIH'S LCTU AND THE
LUPUS CLINICAL RESEARCH PROGRAM
IN HIS LUPUS AWARENESS MONTH
PROCLAMATION LAST WEEK AND
THERE'S A BEAUTIFUL PHOTO OF THE
TEAM REPRESENTED ON THE RIGHT
HAND SIDE OF THE SLIDE.
BUT YOUR WORK IN THE FIELD HAS
HAD A TREMENDOUS IMPACT, DECADES
AGO, MANY PEOPLE WITH LUPUS DIED
WITHIN 5 YEARS OF THEIR
DIAGNOSIS, BUT TODAY THANKS TO
COLLABORATIVE MEDICAL RESEARCH
SUPPORTED BY THE NIH AND OTHERS,
PEOPLE WITH LUPUS ARE LIVING
LONGER MORE ACTIVE AND
PRODUCTIVE LIVES.
THE PAST 2 DECADES OF RESEARCH
HAVE YIELDED A WEALTH OF NEW
INFORMATION AND GROWTH IN
IMPROVING OUR UNDERSTANDING OF
LUPUS AND ITS CO-MORBIDITIES AND
I WANT TO TAKE JUST A MOMENT TO
HIGHLIGHT A FEW OF THESE
IMPORTANT SUCCESSES.
FOR EXAMPLE, WE NOW KNOW THAT
MOST WOMEN WHO HAVE LUPUS CAN
EXPECT A GOOD PREGNANCY IF THEIR
DISEASE IS INACTIVE, WE CAN ALSO
NOW IDENTIFY LUPUS PATIENTS WHO
ARE AT THE HIGHEST RISK OF
CARDIAC ISSUES AND WE ALSO HAVE
NEW INSIGHT INTOS WHY WOMEN ARE
AT SUCH GREATER RISK OF LUPUS
THAN MEN, AND OF COURSE,
IMPORTANTLY, WE HAVE NEWER DRUGS
TO TREAT LUPUS, INCRUDING THE
B-LYMPHOCYTE INHIBITOR, THE
ANTAGONIST AND A CALCIUM
[INDISCERNIBLE], SO DISCOVERIES
LIKE THESE ARE A RESULT OF
COLLABORATIONS AND NIH
INVESTMENTS INCREASING OUR
UNDERSTANDING OF THE MANY
COMPLICATIONS OF LUPUS AND
POTENTIALLY NEW WAYS TO TREAT
THEM.
NIAMS, APPRECIATES THE MANY
STRONG PARTNERSHIPS WE HAVE IN
THE LUPUS RESEARCH FIELD, FIRST
AND FOREMOST WITHOUT THE SUPPORT
OF OUR PATIENTS AND CAREGIVERS
THOUGH, OUR EXPLORATIONS TO
BETTER UNDERSTAND LUPUS WOULDN'T
BE POSSIBLE SO YOU ARE AT THE
HEART OF WHY WE DO WHAT WE DO.
NIH ALSO PARTNERS WITH OTHER
FEDERAL AGENCIES, PATIENT
ADVOCACY GROUPS, PROFESSIONAL
ORGANIZATIONS, ACADEMIA, PHARMA
AND BIOCHECK INDUSTRIES TO TAKE
A MULTIDISCIPLINARY APPROACH TO
BETTER UNDERSTAND COMPLICATED
DISEASES LIKE LUPUS.
ONE EXAMPLE OF A VERY SUCCESSFUL
AND WELL DESIGNED AND GOVERNED
PUBLIC-PRIVATE PARTNERSHIP IS
THE ACCELERATING MEDICINES
PARTNERSHIP PROGRAMS OR AMP.
THIS INVOLVES THE NIH, THE FDA,
INDUSTRY, NONPROFIT GROUPS AND
OTHER ORGANIZATIONS TO TACKLE
COMPLICATED BIOMEDICAL RESEARCH
QUESTIONS AND VERY FORTUNATELY,
RHEUMATOID ARTHRITIS AND LUPUS
WERE SELECTED AS THE FOCUS OF 1
OF THE 4 ORIGINAL AMP PROGRAMS
BACK IN 2014.
THIS PROGRAM HAS YIELDED VERY
EXCITING RESULTS THAT PROVIDE US
WITH A BETTER UNDERSTANDING OF
THE UNDERLYING PROCESSES, THAT
CONTRIBUTE TO THESE DISEASES,
FACILITATING THE IDENTIFICATION
OF POTENTIAL NEW TARGETS AND
DEVELOPMENT OF MORE EFFECTIVE
TREATMENTS.
NOW BECAUSE THAT PROGRAM HAS
BEEN SO SUCCESSFUL, THE
ROIMATOID ARTHRITIS LUPUS
PROGRAM HAS NOW BEEN EXPANDED A
COUPLE OF YEARS AGO TO INCLUDE
SIR EYA SIS, SORTING
SIGNALSIATIC AGERRITIS AND
SOJGREN'S DISEASE EMPLOY ALSO
WHAT WE LEARNED AND HOW
IMPORTANT COLLABORATION, TEAM
CONSCIENCE AND TRAINING THE NEXT
GENERATION OF TEAM SCIENCE
LEADERS ARE THOSE HAVE BEEN VERY
WELL ACCELERATED IN THIS
MEDICINE'S PARTNERSHIP PROGRAM.
I WANT TO MENTION 1 OTHER
IMPORTANT PARTNERSHIP YOU MAY
NOT HAVE HEARD ABOUT YET BECAUSE
IT'S RELATIVELY NEW, THE NEW
OFFICE THEA NIH, THE OFFICE OF
IMMUNE DISEASE RESEARCH AND THE
INAUGURAL PROGRAM LAUNCHED OUT
OF THIS NEW OFFICE IS CALLED THE
EXPOETICSOME IN AUTOIMMUNE
DISEASE COLLABORATING TEAMS,
XACT, AND THE GOAL OF THIS
EXCITING NEW EFFORT IS
DISCOVERED ENVIRONMENTAL
EXPOSURES THAT INFLUENCE DISEASE
SUSCEPTIBILITY, ONSET AND
OUTCOMES AND OUT OF THE INITIAL
6, XACT REWARD RECIPIENTS OF 2
OF THEM FOCUS ON LUPUS, SO
THAT'S EXCITING NEWS FOR THIS
COMMUNITY.
BUT NETWORK IAMS ALSO
COORDINATES THE LUPUS WORKING
GROUP WHICH FACILITATES
COLLABORATION AMONG NIH
INSTITUTE ANDS CENTERS, OTHER
AGENCIES, VOLUNTARY AND
PROFESSIONAL ORGANIZATIONS AND
INDUSTRY GROUP WHO IS SHARE AN
INTEREST IN LUPUS.
NOW WE ALSO PARTNER WITH
PROFESSIONAL AND VOLUNTARY
ORGANIZATIONS THROUGH A GROUP
CALLED THE NIAMS COALITION WHICH
IS HAN INDEPENDENT CONSORTIUM OF
ORGANIZATIONS, WE HAVE OVER 90
ORGANIZATIONS CURRENTLY THAT ARE
NATIONAL IN SCOPE AND THROUGH
THE COALITION, WE INVOLVE THE
COMMUNITY IN SCIENTIFIC
MEETINGS, ROUND TABLE DISCUSSION
AND LISTENING SESSIONS THAT
INFORM OUR ACTIVITIES.
MEMBERS INCLUDE MANY OF THE
ORGANIZATIONS THAT ARE HERE
TODAY, SO THANK YOU SO MUCH FOR
YOUR INVOLVEMENT.
SO PARTNERSHIPS ARE POWERFUL AND
BY COMBINING THE RESOURCES AND
TALENTS OF GOVERNMENT, INDUSTRY,
NONPROFITS AND ACADEME IMRA, WE
CAN CONTINUE MAKING GREAT
STRIDES IN LUPUS RESEARCH.
SO I WOULD LIKE TO EMPHASIZE THE
SIGNIFICANCE OF RESEARCH FOR
ENABLING LONGER, HEALTHIER, AND
MORE PRODUCTIVE LIVES FOR LUPUS
PATIENTS AND REALLY ALL
AMERICANS, THAT'S WHY WE'RE HERE
AND WE RECOGNIZE OF COURSE,
THOUGH THAT THERE ARE MANY MORE
IMPORTANT QUESTIONS THAT NEED TO
BE ANSWERED SO WE'RE COMMITTED
TO CONTINUING TO SEEK THOSE
ANSWERS AS YOU HEAR FROM THE
SPEAKERS THIS MORNING, REMEMBER
THAT WE'RE ALL WORKING TOGETHER
TO ADVANCE LUPUS RESEARCH,
EDUCATION AND AWARENESS AND THE
DC LUPUS CONSORTIUM IS A VERY
IMPORTANT CATALYST FOR LUPUS
RESEARCH AND WE THANK YOU FOR
YOUR IMPORTANT ROLE IN EXPANDING
OUR LUPUS RESEARCH EFFORTS.
SO THANK YOU VERY MUCH.
I HOPE YOU ENJOY THE REST OF THE
DAY.
[ APPLAUSE ]
>> THANK YOU DR. CRISWELL, NEXT
WE HAVE DR. [INDISCERNIBLE]
DIRECTOR OF CLINICAL RESEARCH AT
NIAMS, HE WILL BE PRESENTING ON
LUPUS RESEARCH TURNING 30 AT THE
NIH.
[ APPLAUSE ]
>> THANK YOU SO MUCH JULIE AND
GOOD MORNING EVERYONE.
OKAY, SO FIRST OF ALL THANK YOU
FOR COMING IN TODAY AND JOINING
US AND I HOPE COMING TO THE NIH
FOR THOSE WHO DON'T ON WORK HERE
WON'T BE--WASN'T A BIG
CHALLENGE, SOMETIMES IT CAN BE.
SO I JUST WANT TO TALK A LITTLE
BIT ABOUT THE POSTER THAT WE
DESIGNED AND I THINK ALMOST ALL
OF YOU SITTING IN THE AUDIENCE
CAN ACKNOWLEDGE OR APPRECIATE
THAT LUPUS IS A VERY
HETEROGENEOUS DISEASE AND IT
COMES IN ALL SHAPES AND FORMS,
AND NOT ONLY THAT, THE PATIENTS,
EACH AND EVERY PATIENT IS
DIFFERENT.
SO WE CHOSE THE BUTTERFLY WHICH
USUALLY REPRESENT LUPUS BUT WE
CHOOSE THE BUTTERFLY IN ALL
DIFFERENT SHAPES AND SIZES, TO
EMPHASIZE THE POINT THAT EACH
AND EVERY PATIENT IS
INDIVIDUALLY DIFFERENT AND THE
DEC HAS SO MUCH HETEROGENEITY,
SO THAT'S A BIT OF BACK GROWBD
ABOUT WHAT THE POSTER IS ALL
ABOUT.
SO LET ME TAKE YOU BACK TO THE
YEAR 1994.
SOME OF YOU IN THE AUDIENCE LIKE
ME, MAYBE OLD ENOUGH TO REMEMBER
19, SOME MAY NOT BE.
SO FOR THOSE WHO STILL REMEMBER,
THEY WILL REMEMBER SOME OF THE
THINGS THAT HAPPENED IN 19 SO
YOU GOT MAIL, THAT WAS STARTED
IN 19, [LAUGHTER]
, AND THEN A POPULAR SERIES THAT
WE STILL ENJOY WAS THERE, AND
THEN THE WORLD ARENA NELSON
MANDELA WAS ABLE TO CAST HIS
VOTE FOR THE VERY FESTER TIME IN
19.
SO SIGNIFICANT YEAR, A LOT OF
IMPORTANT THINGS HAPPENED AND
THE NOBEL PRIZE WAS AWARDED FOR
DISCOVERING THE G-PROTEIN WHICH
LED TO SEVERAL SIGNIFICANT
DISCOVERIES AND THERAPEUTIC
TARGETS LATER ON BUT RIGHT HERE
IN BETHESDA, SOMETHING ALSO VERY
IMPORTANT AND INTERESTING
HAPPENED IN 1994 AND THAT'S WHY
WE ARE ALL HERE.
SO THE STUDIES OF THE
PATHOGENESIS AND NATURAL HISTORY
OF SLE WAS ESTABLISHED IN 19 AT
NIH AND THIS WAS THE STUDY THAT
FORMALIZED THE LONG-TERM FOLLOW
UP OF LUPUS PATIENTS AT THE NIH
SO I DO RECOGNIZE THAT LUPUS
RESEARCH HAS BEEN GOING ON AT
THE NIH FOR MANY DECADES, EVEN
BEFORE BACK TO 1994 BUT WE HAVE
CHOSEN THIS YEAR, 19 AS A
LANDMARK BECAUSE THIS IS THE
LONGEST STUDY THAT HAS BEEN
ONGOING CONTINUOUSLY FOR THE
LAST 30 YEARS AND WE'VE BEEN
FOLLOWING PATIENT AND SOME OF
THE PATIENTS ARE HERE IN
AUDIENCE WHO HAVE BEEN COMING
HERE FOR THE LAST 30 YEARS SO
THAT'S WHY WE SORT OF DECIDED TO
USE THAT AS A LANDMARK WITH
KEEPING IN MIND THAT IF THIS
RESEARCH HAS BEEN GOING ON FOR
EVEN SEVERAL DECK'SS BEFORE
THAT.
SO YOU KNOW, WHEN I STAND HERE,
I UNDERSTAND THAT I STAND HERE
IN TRYING TO FILL SHOES OF BIG
GIANTS WHO FOLLOWED BEFORE ME,
THE STUDY FIRST PI WAS
DR. DIMITRI [INDISCERNIBLE] WHO
WAS MOVED TO EUROPE, AND
[INDISCERNIBLE] WAS THERE AND
MARK [INDISCERNIBLE] WAS ALSO 1
OF THE PIs AND I HAVE BEEN THE
PI OF THIS STUDY FOR ALMOST
12-13 YEARS NOW.
SO, OVER THE NEXT 30 YEARS, WE
HAVE DONE A LOT OF RESEARCH, WE
HAVE TAKEN CARE OF PATIENTS, WE
HAVE DONE ADVOCACY, WE HAVE DONE
MULTIPLE THINGS AND MULTIPLE
REALMS, AND WE HAVE MANY
SUCCESSES, BUT AT THE SAME TIME
WE HAVE SUCCESSES, WE ALSO HAVE
SOME CHALLENGES AND SOME OF THE
PARTNERS, SOME OF THE COLLEAGUES
THAT STARTED THE JOURNEY WITH US
ARE NOT HERE WITH US ANYMORE.
SO SOME OF THE LUPUS RESEARCHERS
FOR EXAMPLE, DR. GCORRELATED
RLEY, WHO IS NOT WITH US ANYMORE
AND WE HAVE LOST SOME OF THE
PATIENTS, SO EVEN THOUGH WE
CELEBRATE, WE ALSO WANT TO
REMEMBER AND RECOGNIZE THOSE WHO
HAVE LOST THE BATTLE THROUGH OUR
THIS 30 YEARS AND IN HONOR OF
THAT I WOULD REQUEST ALL OF YOU
TO PLEASE STAND UP FOR A FEW
SECONDS TO OBSERVE A MOMENT OF
SILENCE FOR THOSE WHO ARE NOT
WITH US ANYMORE AND HAVE LOST
THE JOURNEY.
THANK YOU.
SO A LITTLE BIT ABOUT THE LUPUS
RESEARCH AT NIH AND WHAT WE HAVE
DONE AND WHAT WE HAVE
ACCOMPLISHED.
SO FAR OVER THE LAST 30 YEARS WE
HAVE RECRUITED 1254 PATIENTS AND
THE RECRUITMENT IS ONGOING AS
DR. CRISWELL MENTIONED, THIS IS
THE LONGEST COHORT OF FOLLOW UP
PATES GOING ON FOR THE LONGEST
TIME AROUND THE WORLD.
WE PUBLISHED CLOSE TO 180
STUDIES.
WE HAVE DONE MULTIPLE CLINICAL
TRIALS.
SOME OF THEM ARE ONGOING, SOME
OF THEM HAVE BEEN PUBLISHED AND
THEN YOU CAN DO A PUB MED SEARCH
TO LOOK THEM UP BUT WE HAVE MADE
MAJOR CONTRIBUTIONS NOT ONLY TO
PATIENT CARE, BUT ALSO TO
ADVANCING THE SCIENCE AND
BRIDGING THE GAP IN KNOWLEDGE OF
UNDERSTANDING THE LUPUS.
AT THE PRESENT OUR CURRENT
COHORT OF LUPUS IS VERY ROBUST
AND MULTIETHNIC, AND WE HAVE
REPRESENTATIVE OF MULTILE
ETHNICITIES, WE ARE TRYING TO
MAKE IT MORE ROBUST AND MORE
PROPORTIONATE IN TERMS OF
PRESENTATION, NOT ONLY IS THAT
IT'S RACIALLY ROBUST AND
DIVERSIFIED AND WE ALSO HAVE GIO
GRAPHICALLY DIVERSE POPULATIONS,
SO WE HAVE PATIENTS COMING FROM
MOSTLY FROM THE DMV AREA, BUT
FROM MULTIPLE AREAS OF PARTS OF
THE UNITED STATES AS WELL AS A
LOT OF INTERNATIONAL PATIENT WHO
IS COME AND SEEK SECOND OPINION
OR PARTICIPATE IN RESEARCH AND
THEN YOU COME HERE ONCE A YEAR
OR SO AND A LOT OF THEM HAVE
BEEN COMING HERE FOR A LONG TIME
EMPLOY SO LUPUS RESEARCHERS AT
THE NIH, WE DO A LOT OF
COMPLICATED LUPUS PATIENT
REFERENCE WE GET FROM THE
CONSORTIUM OUR COLLEAGUES SEND
THEM TO US FOR A SECOND OPINION,
WE ALSO PLAN AND CONDUCT MAJOR
TRIALS BASED ON DISCOVERIES IN
THE EPITHELIAL RAMIEWR AND
EXTRAMURAL PROGRAM AND WE HAVE A
LOT OF COLLABORATIVE PROJECTS
AND THE DC CONSORTIUM WE
ESTABLISHED 30 YEARS AGO AND WE
COME TOGETHER ONCE AIER TO
DISCUSS WHAT SHOULD WE DO NEXT
IN LUPUS RESEARCH AND WE ALSO
INFORM WHAT WE HAVE DONE, WHAT
WE HAVE LEARNED ABOUT LUPUS TO
OUR PATIENTS TO OUR
COLLABORATORS AND SO FORTH AND
IT HAS BEEN A VERY SUCCESSFUL
ENDEAVOR.
IN ADDITION TO THAT WE ARE ALSO
ACTIVELY INVOLVED IN PATIENT
EDUCATION IN TERMS OF WE ALL DO
TRAINING AND MENTORSHIP
ACTIVITIES AND A LOT OF TRAINING
GOING ON HERE AND OVERALL IT HAS
FROM A RATHER SMALLER SET UP TO
A VERY HUGE SET UP TO
MULTIDIMENSIONALKIVITYS SO I
WOULD SAY THAT THERE ARE BRIGHT
FUTURES AHEAD AND WE ARE ALL
HOPEFUL THAT WE WILL MAKE MORE
DISCOVERIES WITH THE
CONTRIBUTIONS FROM OUR PATIENTS
AND WE WILL LEARN MORE ABOUT
LUPUS AND WE WILL 1 DAY HOPE TO
SOLVE THIS CRUEL MYSTERY.
SO, THIS IS OUR LUPUS CLINICAL
TRIALS UNIT AND YOU WILL SEE
MULTIPLE PEOPLE OF OUR UNIT
WORKING AROUND HERE, SOME ARE
HERE IN THE AUDIENCE, SOME ARE
OUTSIDE HELPING OUT AND I DO
WANT TO GIVE A SHOUT OUT TO ALL
THE VOLUNTEERS SO TO SET UP THIS
EVENT IT TOOK AN ENORMOUS EFFORT
FROM MULTIPLE, MULTIPLE PEOPLE
AND A LOT OF PEOPLE WORKED
BEHIND THE SCENES TO GET THIS
EVENT DONE AT THIS HUGE LEVEL
AND I CANNOT NAME EACH AND EVERY
1 BECAUSE I MAY FORGET
SOMEBODY'S NAME AND I MAY GET IN
TROUBLE BUT I DO WANT TO GIVE A
SHOUT OUT TO 2 PEOPLE WHO HAVE
BEEN VERY INSTRUMENTAL IN THIS,
SO [INDISCERNIBLE] AND
[INDISCERNIBLE] AND THEY ARE
BOTH HERE AND THANK YOU SO MUCH.
AND AND THEN I WOULD SAY, THANK
YOU FOR EVERYONE FOR COMING
HERE, THANK YOU ESPECIALLY TO
ALL THE PATIENTS WHO HAVE
CONTRIBUTED SO MUCH AND WITHOUT
OUR PATIENTS, OBVIOUSLY WE WILL
NOT BE ABLE TO DO ANYTHING.
SO THANK YOU FOR BEING SO
ALTRUISTIC, CONTRIBUTING TO
RESEARCH HERE, DOING THANK YOU
TO ALL THE FAMILIES AND FRIEND
WHO IS SUPPORT THE PATIENTS AND
ALL THE VOLUNTEERS AND ALL THE
COLLEAGUES WHO ARE HERE, AND I
HOPE THAT YOU'LL ENJOY THE EVENT
TODAY, THANK YOU.
[ APPLAUSE ]
>> THANK YOU DOCTOR, NEXT UP WE
HAVE DR. COLBERT, HE IS THE
DIRECTOR OF NIARK MS AND HE WILL
GIVE AN NIAMS OVERVIEW, GIVE HIM
A WARM WELCOME.
>> THANK YOU JULIE.
WELCOME EVERYBODY.
IT'S NICE TO SEE SUCH A PACKED
AUDITORIUM.
SO, AS JULIE MENTIONED,
DR. [INDISCERNIBLE] ASKED IF I
COULD GIVE A BRIEF OVERVIEW OF
THE NIAMS INTRAMURAL RESEARCH
PROGRAM AND ALSO TO FOCUS A
LINGS BIT ON JUST THE SUPPORT
THAT THE NIAMS SUPPORT THAT IT
PUTS INTO THE RESEARCH.
SO THE 1 THING YOU ARE AT THAT
TIME SUPPOSED TO DO TO--THAT YOU
ARE NOT SUPPOSED TO START WITH
IS A ORGCHART BUT I WILL MAKE A
POINT.
FIRST I WILL TART WITH THE FACT
THAT WE ARE A MEDIUM SIZED
EN--STRATEGIESITUTE, WE HAVE A
MEDIUM SIZED IMPOSSIBLE TO BUILD
RAMURAL PROGRAM AND CLINICAL
PROGRAM BUT I THINK WE DO QUITE
A BIT AND I'M REALLY PROUD OF
THAT SO WHAT THIS SLIDE SHOWS
YOU IS HOW WE'RE DIVIDED INTO
DIFFERENT LABS AND SECTIONS AND
UNITS, ET CETERA, YOU DON'T NEED
TO LOOK AT ALL THOSE DETAILS.
WHAT I HIGHLIGHTED IN PURPLE, IS
VARIOUS EFFORTS THAT ARE
INVOLVED THIS CLINICAL RESEARCH
AND THEN EACH SECTION THAT HAS
AN ASTERISK HAS SOMETHING TO DO
WITH LUPUS RESEARCH AND I WILL
GO INTO A FEW OF THOSE AREAS.
WE HAVE A LITTLE OVER 40
CLINICAL AND RESEARCH FACULTY OF
SENIOR INVESTIGATORS TO EARLY
CAREER INVESTIGATORS AND A
COUPLE OF ADJUNCT INVESTIGATORS
FROM OTHER INSTITUTES.
SO THIS IS A DIFFERENT VIEW NOW
OF THE CLINICAL PROGRAM, TELLING
YOU THAT WE NOT ONLY DO LUPUS
RESEARCH BUT WE ACTUALLY DO
RESEARCH IN SEVERAL OTHER
DISEASES AND A SIGNIFICANT
PROPORTION OF RESOURCES GO TO
DERMATOLOGY RESEARCH, SO YOU CAN
SEE IN THE BLUE, THAT'S THE
UPPER LEFT, WE BASIC SKIN
IMMUNOLOGY RESEARCH, MICROBIOME
RESEARCH, CAR SIN O GENESIS
RESEARCH, WE HAVE A SIGNIFICANT
VASC LIGHT ISOTOPE RESEARCH
PROGRAM AND WE ALSO DO
SPONDYLOROSIS AND ARTHRITIS
RESEARCH.
WE DO CONSULT SERVICES FOR
RHEUMATOLOGY IN ARK DULT AND
PEDIATRIC BUT THE REASON O
REASON WE'RE HERE TODAY IS TO
TALK ABOUT WAWE DO IN LUPUS
RELATED RESEARCH WHICH IS SHOWN
IN THE ORANGE OVALS IN THE
BOTTOM.
SO OVERALL ABOUT 25% OF THE
CLINICAL PROGRAM RESOURCES GO TO
LUPUS AND SYSTEMIC AUTOIMMUNITY.
ANOTHER--LET'S SEE IF I CAN MAKE
IT WORK.
ANOTHER 30% GOES TO FELLOWSHIP
AND EARLY CAREER INVESTIGATOR
PATHWAYS, THAT'S SCHOLARS AND
CLINICAL ASSISTANT INVESTIGATORS
IN ADDITION TO OUR RHEUMATOLOGY
FELLOWS, ABOUT 15% TOWARDS
SYSTEMIC VASC LIGHTIS RESEARCH
AND THEN THE REST IS DIVIDED
AMONGST THE OTHER DISEASE AREAS
THAT I MENTIONED TO YOU.
SO I WANT TO TAKE A COUPLE
MINUTES AND HIGHLIGHT SOME OF
THE INDIVIDUALS WHO YOU'VE HEARD
ABOUT AND YOU WILL HEAR FROM IN
THE REST OF THE PROGRAM AND JUST
TO SAY THAT WHILE THIS IS
ABSOLUTELY A TEAM EFFORT, THE
TEAM IS LED BY DR. MARRIANA
KAPLAN WHO IS A SENIOR
INVESTIGATOR AND CHIEF OF THE
SYSTEMIC AUTOIMMUNITY BRANCH AND
MARRIANA IS AN NIH DISTINGUISHED
INVESTIGATOR.
SHE WILL BE TALKING SHORTLY IN
THE PROGRAM.
IN ADDITION, YOU'VE OBVIOUSLY
HEARD FROM DR. HASNI, HE'S DONE
A WONDERFUL YOB CORPS OF
ORGANIZING THE TEAM AND THE
PROGRAM.
HE'S THE DIRECTOR OF THE
CLINICAL TRIALS LUPUS UNIT.
AND ANOTHER STAFF CLINICIAN WHO
WILL YOU HEAR FROM LATER
DR. GUPTA IS INTERESTED IN HOW
WE CAN LEARN FROM RARE GENETIC
DISEASES ABOUT PATHWAYS THAT
BECOME ACTIVATED IN SYSTEMIC
LUPUS, DR. FRANCO IS A TENURED
TRACK INVESTIGATOR WHO IS
INTERESTED IN UNDERSTANDING MORE
ABOUT HOW GLUE CO CORTICOIDS
WORK, A TREMENDOUS AREA FOR
LUPUS AND OTHER RHEUM TO LOGIC
DISEASES.
YOU WILL HEAR ALSO FROM
DR. LAURA LEWANDOWSKI, SHE'S A
CLINICAL RATHER AND HE'S
INTERESTED THIS THE GENETICS OF
LUPUS FROM A GLOBAL PERSPECTIVE.
WE HAVE 2 SCHOLARS IN
TRANSLATIONAL RESEARCH WHO WORK
IN LUPUS RELATED AREAS, WILL
AMBLER WHO IS IN DR. KAPLAN'S
LAB AND HE'S INTERESTED IN
UNDERSTANDING MORE ABOUT HOW SEX
DIFFERENCES INFLUENCE THE IMMUNE
RESPONSE WHICH IS VERY IMPORTANT
IN MANY OF OUR AUTOIMMUNE
DISEASES AND DR. MEGAN NELSON,
DR. NELSON WORKS WITH
DR. CRISWELL, WHO WE HEARD FROM
SHE'S INTERESTED IN THE GENETICS
OF LUPUS IN LUPUS ASIAN
POPULATIONS.
SO THE LUPUS TEAM HAS 2
OUTSTANDING PROVIDERS, UNIAND
MICHAEL, JUNE WILL TELL YOU
ABOUT THE QUALITY INDICATOR
PROJECTS SHE'S BEEN WORKING ON
WITH THE LUPUS TEAM AND MICHAEL
WILL UPDATE YOU ON THE
ANTIFROLIMAB STUDY, THERE ARE 3
RESEARCH COORDINATORS IN THE
PROGRAM, ELAINE, AND LUPNA, AND
YOU WILL HEAR FROM ELAINE AND
YENEALEM, AND THEY INTERVIEW
SOME OF THE PATIENT WHO IS ARE
HERE TODAY AND LAST BUT NOT
LEAST WE HAVE THE VOTER
IMPORTANT PATIENT CARE
COORDINATORS.
ISOTOPE BELLA HAS BEEN
INTRODUCED TO YOU AND EMILY
JONES, EMILY WE NEED A PICTURE
OF YOU FOR THE WEBSITE.
THE LUPUS TEAM ALSO HAS CLINICAL
RESEARCH SPECIALIST DR. NAQI AND
DR. MANNA WHO ARE HERE TODAY AS
WELL.
SO I WANT TO FINISH UP WITH A
PICTURE YOU'VE SEEN BEFORE,
EMILY DID GET CAPTURED BY THE
PHOTOGRAPHER HERE, AND ALSO I
WANTED TO MENTION DR. CHRISTINA
[INDISCERNIBLE] CHRISTINA IS
ALSO A MEMBER OF DR. CRISWELL'S
RESEARCH PROGRAM.
SHE'S A THROW A FOOTBALL
CLINICIAN AND SHE'S DOES LUPUS
RESEARCH AND HELPS OUT
SIGNIFICANTLY IN THE LUPUS
CLINIC SO THANK YOU FOR YOUR
EXPOSURE TO RADIATIONS AND THANK
YOU TO THE ENTIRE LUPUS TEAM.
YOU MAKE US VERY PROUD, BUT MORE
IMPORTANTLY, YOU DO A TREMENDOUS
AMOUNT FOR OUR PATIENTS, PUSHING
THE NEEDLE FORWARD IN TERMS OF
RESEARCH BUT ALSO THE
OUTSTANDING CARE THAT YOU
DELIVER.
SO THANK YOU VERY MUCH.
[ APPLAUSE ]
>> THANK YOU DOCTOR, NEXT WE
WOULD LIKE TO TAKE THIS MOMENT
TO RECOGNIZE DR. GENE
[INDISCERNIBLE].
HE IS FORMER CLINICAL DIRECTOR
OF NIDDK TO SAY A FEW WORDS.
OKAY, THANK YOU AGAIN, JULIE.
SO AGAIN, DOING RESEARCH IN
LUPUS IS NOT POSSIBLE WITHOUT
COLLABORATION AND COLLABORATIVE
EFFORT AND I THINK DR. BALO,
MANY OF THE IN THE AUDIENCE
RECOGNIZE HIM VERY WELL FOR THE
CONTRIBUTIONS HE HAS MADE AND
HAVE MADE A SIGNIFICANT IMPACT
IN CLINICAL RESEARCH WHICH HAS
CHANGED THE LIFE OF MANY
PATIENTS AROUND THE WORLD, NOT
ONLY IN THE UNITED STATES, SO
DR. BALO CAME TO THE NIH IN
1972, AS A CLINICAL SOCIETY
STUDYING IMIEWNLOLOGY, HE
DEVELOPED THE FIRST INHOUSE
NEPHROLOGY SERVICE FOR PATIENTS
AT THE NIH CLINICAL CENTER ASK
CLINICAL NEPHROLOGY.
DR. BALO'S CLINICAL SERVICE GREW
EXPONENTIALLY AND AS HIS
REPUTATION AFTER SEVERAL YEARS
HE'S BEEN THERE, THEN HE STARTED
AS A PHYSICIAN AT THE NATIONAL
INSTITUTES OF ARTHRITIS AND
METABOLIC DISEASE WHICH WAS THE
PREDECESSOR INSTITUTE OF NIAMS
AND NIDDK.
AFTER JOINING IN 1997, HE
DEVELOPED A WORLD CLASS CLINICAL
RESEARCH PROGRAM ON
IMMUNOLOGICALLY MEDIATED
[INDISCERNIBLE] DISEASE AND HE
QUICKLY ROSE THROUGH THE RANKS
OF THE NIH, BECAME THE CHIEF OF
THE NEVERROLOGY SERVICES, AND
THEN AFTER THAT HE JOINED THE
NIDDK'S CLINICAL DIRECTOR IN
1988 AND THIS IS A POSITION HE
HELD FOR 31 YEARS WHO BECAME THE
LONGEST SERVING CLINICAL
DIRECTOR AT THE NIH AND AFTER
THIS LONG ILLUSTRIOUS CAREER, HE
RETIRED FROM THE CLINICAL
DIRECTOR POSITION IN 2022 AND
AFTER THAT HE SERVED AS AN
ACTING CHIEF FOR SINCE
JULY 2022.
HE'S WIDELY RECOGNIZED AN EXPERT
IN GLUE MERRIAL DISEASES
PARTICULARLY IN FOR LUPUS
NEPHRITIS AND HAS BEEN INVOLVED
IN THE PIVOTAL TRIALS WHICH HAS
LED TO THE SO CALLED NIH
PROTOCOL FOR TREATMENT OF LUPUS
NEPHRITIS USING PSYCHOFOSTER
NURSED FOCUSED ON MADE WHICH HAS
BEEN USED ALL OVER THE WORLD.
HE HAS PUBLISHED OVER 250 PAPES
AND CHAPTERS AND HE ALSO HELPED
US TREAT A LOT OF PATIENTS
THROUGH THE RENAL CONSULTATION
SERVICE.
HE HAS TAUGHT OWL OF US
INCLUDING ME ABOUT HOW TO LOOK
AT KIDNEY PATHOLOGY AND LOOKING
AT THE MICROSCOPE TO UNDERSTANDS
WHAT IS GOING ON AND HE IS VERY
GOOD IN TERMS OF CORRELATING
THAT WITH THE CLINICAL SYMPTOMS
THAT HAS BEEN GOING ON WITH THE
PATIENT.
HE HAS RECEIVED SEVERAL AWARDS
INCLUDING THE NIH CLINICAL
DIRECTORS AWARD, PUBLIC SERVICE
AWARD, AWARD FROM VARIOUS OTHER
OUTSIDE INSTITUTIONS, NIH
DIRECTOR'S AWARD, AND THEN
DR. BALO CONTINUES TO
PARTICIPATE IN PROVIDING
GUIDANCE AND MENTORSHIP TO
TRAINERS AND INVESTIGATORS IN
THE KIDNEY DISEASE BRANCH.
HE WILL CONTINUE TO ADVISE IN
CLINICAL CONSULTATIVE MATTERS
GIVEN HIS YEARS OF EXPERTISE IN
CLINICAL NEPHROLOGY, SO IT'S A
BRIEF INTRODUCTION BUT I WOULD
LIKE JIM TO PLEASE--HERE.
[ APPLAUSE ]
>> WELL, THANK YOU SO FOR FOR
YOUR GENEROUS COMMENT.
I DIDN'T KNOW WHO YOU WERE
TALKING ABOUT AT FESTER BUT I
DID CATCH THE DRIFT SOONER OR
LATER.
I CAME IN 1972 AS WAS MENTIONED
AND I CAME TO WORK IN NIAID, I
WAS 1 OF TONY FAUCI'S FIRST
FELLOWS AND BEING TRAINED IN
NEPHROLOGY, I ACTUALLY HELPED
DEVELOP THE PROGRAMS THROUGHOUT
THE CLINICAL CENTER, BUT I SAW
THE GREATEST OPPORTUNITY TO
REALLY INTERACT IN DEEP
COLLABORATION WITH RHEUMATOLOGY
FACULTY.
MY GREATEST MENTOR IN THE LUPUS
AREA WAS JOHN DECKER WHO IS NO
LONGER WITH US.
OTHERS LIKE PAUL CLAUS, FRED,
JACK, AND THEN ORIGEINALLY I
ENVISIONED THAT--ORIGEINALLILY I
ENVISIONED THAT PEOPLE LIKE
WILLIAM [INDISCERNIBLE] WERE
CONFUSED ABOUT LUPUS.
YOU KNOW HE BASICALLY CONSIDERED
THERE WAS A SKIN DISEASE THAT
WAS RELATED TO ARATHEMA GROUP OF
SKIN DISEASES AND IT DID INCLUDE
LUPUS AND I ENVISIONED IF WE
COULD TEASE ALL THAT APART, WE
WOULD FIND 1 SINGLE DISEASE THAT
WAS REALLY BENEFITING LUPUS AS
OPPOSE TO A MISHMASH OF LOTS AND
LOTS OF DIFFERENT THINGS
ENCLUEDING GLUE MERRIAL
NEPHRITIS, ENDOCARTITEIS,
RHEUMATOID ARTHRITIS, EVERYTHING
SEEM TO HAVE BEEN A MIX OF
SYNDROMES BACK IN THOSE EARLY
DAYS BUT NAIVE ETICSY OVER THE
YEARS, LUPUS ISN'T JUST 1
DISEASE, IT'S MOSTLY A SYNDROME
COMPOSED OF DIFFERENT
COMPONENTS.
THE KIDNEY DISEASE WHICH IS MY
PARTICULAR PROFESSIONAL LOVE
BEING A VERY PROMINENT FACTOR IN
THE PROGNOSIS OF LUPUS AND I'M
JUST SO GRATIFIED TO HAVE MET A
FEW PATIENTS WHO CAME UP TO ME
THIS MORNING.
IT REALLY WAS HEART WARMING TO
SEE YOU AND TO SEE HOW WELL YOU
HAVE DONE OVER THE YEARS.
BUT, AGAIN, AS [INDISCERNIBLE]
HAS SHOWN US, THE SUCCESSES HAVE
BEEN VERY REWARDING BUT THEY'RE
NOT ALWAYS SUCCESSFUL SO WE HAVE
TO KEEP ON WITH THE BATTLE.
I'M VERY HUMBLED BY THE AWARD
AND THIS RECOGNITION, BUT I'M
REALLY DEEPLY TOUCHED.
SO THANK YOU ALL FOR WHAT YOU'VE
GIVEN TO ME OVER THE YEARS
BECAUSE I'VE LEARNED SO MUCH AND
I HOPE I'VE GIVEN JUST A LITTLE
BACK.
THANK YOU.
[ APPLAUSE ]
>> WE HAD A LITTLE SOMETHING FOR
YOU BUT IT'S ON ITS WAY.
SO EVERYTHING--IF IT MAYBES IT
IN TIME, WE WILL ASK YOU AGAIN
BUT YOU KNOW THANK YOU VERY
MUCH.
SORRY.
[ APPLAUSE ]
>> THANK YOU DR. HASNI, THANK
YOU DR. BALOW, OUR NEXT SPEAKER
IS DR. MEGAN MOCKWOOD, SHE IS
ASSISTANT PROFESSOR OF MEDICINE
AT GEORGETOWN UNIVERSITY.
SHE'S THE DIRECTOR OF LUPUS
PROGRAM AT GEORGETOWN UNIVERSITY
HOSPITAL AND SHE WILL BE
SPEAKING ON IMPROVING LUPUS
OUTCOMES THROUGH PARTICIPATION
IN CLINICAL RESEARCH, PLEASE
GIVE HER A WARM WELCOME.
>> GOOD MORNING.
I'M SO DELIGHTED TO BE HERE AND
THANK YOU VERY MUCH FOR THE
INVITATION TO SPEAK ABOUT A
TOPIC THAT I THINK IS IMPORTANT
TO ALL OF US IN THIS ROOM.
I WANT TO TART BY SAYING THAT
THE ACCURATE REPRESENTATION OF
PATIENT FIST CLINICAL TRIALS NOT
ONLY HELPS TO FIGHT HEALTH
DISPARITIES AND HEALTH
INIQUITIES BUT IT'S JUST GOOD
SCIENCE.
AND THAT'S WHY IT'S CRITICAL
THAT PARTICIPANTS ACCURATELY
REPRESENT THE POPULATIONS THAT
POTENTIAL MEDICATIONS ARE
DESIGNED TO TREAT AND DESPITE A
GREATER PREVALENCE OF LUPUS
AMONG RACIAL AND ETHNIC
MINORITIES, MARKET GAPS EXIST
BETWEEN POPULATIONS AFFECTED BY
LUPUS AND THOSE ENROLL INDEED
CLINICAL TRIALS.
SO THE CHART ON THE LEFT
REPRESENTS THE PREVALENCE OF
LUPUS, YOU CAN SEE THAT BLACK
PEOPLE IN PURPLE MAKE UP A
PREVALENT 43% OF LUPUS CASES,
THE CHARTOT RIGHT, SHOWS THE
NUMBER OF PATIENTS REPRESENTED
IN CLINICAL TRIALS, AND YOU CAN
SEE THAT BLACK PEOPLE ONLY MAKE
UP 14% OF PATES IN CLINICAL
LUPUS TRIALS.
IN CONTRAST, GOING BACK TO THE
CHART ON THE LEFT, YOU CAN SEE
HERE THAT 33% OF PREVALENT LUPUS
CASES ARE IN WHITE PEOPLE BUT
THEY'RE VASTLY OVERREPRESENTED
IN LUPUS CLINICAL TRIALS, MAKING
UP OVER 50% OF THE PARTICIPANTS
IN LUPUS CLINICAL TRIALS.
AND WHAT WE'RE HERE TODAY TO
TALK ABOUT LUPUS, THIS IS REALLY
NOT JUST A PROBLEM IN LUPUS,
THIS IS A PROBLEM IN ALL
CLINICAL TRIALS AND ALL
DISEASES, THE CHART ON THE LEFT
DISPLAYS DATA FROM THE U.S.
CENSUS, THAT SHOWS THAT 60% OF
THE PEOPLE IN THIS COUNTRY ARE
WHITE AND THE LIGHT BLUE, BUT
COMPARING THIS TO THE U.S. TRIAL
STATISTICS AS REPRESENTED IN THE
CHART ON THE RIGHT, CAN YOU
AGAIN SEE THAT WHITE PATIENTS
ARE VASTLY OVERREPRESENTED IN
CLINICAL TRIALS AT 80% SO THE
POINT HERE IS NOT THAT WE WANT
TO DISCOURAGE WHITE PATIENTS
FROM PARTICIPATE NOTHING
CLINICAL TRIALS BUT RATHER WE
WANT TO ENCOURAGE THE
PARTICIPATION OF EVERYBODY ELSE.
AND THIS HAS REALLY LED TO A
DATA GAP IN LUPUS, AND I WOULD
ARGUE THAT THIS IS NOT JUST A
RESEARCH PROGRAM BUT A SOCIAL
JUSTICE PROGRAM AND WHEN GROUPS
OF PEOPLE ARE NOT ACCURATELY
REPRESENTED IN CLINICAL TRIALS
IT CAN CONTRIBUTE TO A WIDENING
OF HEALTH INIQUITIES.
WE KNOW THAT ALL INDIVIDUALS DO
NOT EXPERIENCE DEC OR RESPOND TO
THERAPIES IN THE SAME WAY.
AND WHEN DISEASES AND MEDICAL
TREATMENTS ARE NOT STUDIED IN
DIRVERSE POPULATIONS,
RESEARCHERS AND SCIENTISTS MAY
NOT HAVE THE INFORMATION THEY
NEED TO UNDERSTAND HOW CERTAIN
GROUPS ARE AFFECTED.
AND THE DATA GAP CAN REALLY
IMPACT THE DEVELOPMENT OF
MEDICAL TREATMENTS AND MAY LEAD
TO DISEASES GOING UNDER
DIAGNOSED, UNTREATED, OR TREATED
WITH MEDICINES THAT MAY NOT WORK
AS WELL FOR CERTAIN PEOPLE
EMPLOY SO THEREFORE DIVERSE
PARTICIPATION IS NECESSARY FOR
THE APPROVAL OF NEW, SAFE, AND
EFFECTIVE THERAPIES FOR ALL
PATIENTS.
AND THIS IS PARTICULARLY
IMPORTANT IN LUPUS BECAUSE
SIGNIFICANT RACIAL AND ETHNIC
DISPARITIES EXIST IN LUPUS, WE
ALL KNOW THAT LUPUS
DISPROPORTIONATELY AFFECTS OUR
BLACK AND LATINO PATIENT WHO IS
HAVE BOTH THE GREATER MORBIDITY
AND GREATER MORTALITY FROM THE
DISEASE.
BLACK AFRICAN ANCESTRY IN
PARTICULAR IS ASSOCIATE WIDE A
HIGHER PREVALENCE OF LUPUS,
GREATER DEC SEVERE MITRALIT AND
INCREASED RISK OF CARDIOVASCULAR
EVENTS AS WELL AS MORE AND/OR
ORGAN DAMAGE AND MORTALITY RATES
COMPARED TO WHITE PAIBTS WITH
LUPUS AND WE ALSO KNOW THAT
BLACK PEOPLE WITH LUPUS HAVE
LOWER LEVELS OF SOCIAL SUPPORT
COMPARED TO WHITE PEOPLE WITH
LUPUS.
AND THE CHALLENGES AND
CONSEQUENCES OF
UNDERREPRESENTATION AND CLINICAL
TRIAL RECRUITMENT AND
PARTICIPATION ARE REALLY
EXEMPLIFIED BY BELIMUMAB, AND IT
WAS EFFICACIOUS AND THE NUMBER
OF PEOPLE IN THE TRIAL WAS SMALL
AND MOST GROUP ANALYSIS GAVE
RESULS REGARDING EFFICACY IN THE
BLACK POPULATION EMPLOY SO AS A
RESULT THE FDA ADD A CAUTIONARY
STATEMENT REGARDING BELIMUMAB IN
RELATING TO THE AFRICAN AMERICAN
ANCESTRY AND YOU CAN IMAGINE
THESE WARNINGS LED TO
SIGNIFICANT CONFUSION ABOUT
PRESCRIBING BELIMUMAB LIKELY
RESULTING IN DELAYED TREATMENT
FOR MANY PATIENTS WITH LUPUS WHO
WE KNOW INCUR THE GREATEST
DISEASE BURDEN.
AND THE FDA THEN MANDATED A POST
MARKETING STUDY TO ALLEVIATE THE
CONCERNS ABOUT THE SAFETY AND
EFFICACY OF BELLE IN BLACK
PATIENTS CALLED EMBRACE AND
EMBRACE RECRUITED ONLY PATIENT
WHO IS SELF-IDENTIFIED AS BEING
A BLACK RACE AND ALTHOUGH THE
STUDY DID NOT MEET THE PRIMARY
END POINT, IT DID PROVIDE ENOUGH
EVIDENCE OF THE EFFICACY OF
BELIMUMAB FOR THE FDA TO REMOVE
THE CAUTIONARY STATEMENT FROM
THE PRODUCT LABEL.
SO I THINK WE COULD SPEAK AT
LENGTH ABOUT THE LESSONS LEARNED
FROM THE STORY FOR THE SAKE OF
TIME, I WILL JUST COMMENT THAT
IF PARTICIPANTS AND RECRUITMENT
IN THESE INITIAL PHASE 2 AND
PHASE 3 RANDOMIZED CONTROL
TRIALS HAD ADEQUATELY REFLECTED
THE LUPUS POPULATION, THIS
EMBRACE STUDY MIGHT NOT BE
NECESSARY AND THESE DELAYS IN
TREATMENT LIKELY WOULD NOT HAVE
OCCURRED.
SO IN OUR LAST MINUTE, I WOULD
LIKE TO TAKE THE COMMUNITY TO
SPEAK DIRECTLY TO THE PATIENTS
THAT ARE HERE TODAY, COMMUNITY
LEADERS AND PROVIDERS LIKE
MYSELF IN THE COMMUNITY WHO MAY
OR MAY NOT BE INVOLVED IN
CLINICAL RESEARCH, YOU REALLY DO
HAVE THE POWER TO CONTRIBUTE TO
THE HEALTH OF YOUR COMMUNITY.
DIVERSITY AND CLINICAL TRIALS
NOT ONLY HELP TO FIGHT HEALTH
INEQUALITYYS BUT IT COULD LEAD
TO KNOWLEDGE ABOUT DISEASE ASKS
POTENTIAL TREATMENTS FOR ALL,
THAT'S WHY WHEN YOU TAKE PART IN
A CLINICAL TRIAL, YOU REALLY ARE
SUPPORTING YOUR FAMILY, YOUR
FRIENDS, YOUR ENTIRE COMMUNITY
AND YOUR PARTICIPATION PROVIDES
UNIQUE AND IMPORTANT INFORMATION
THAT CAN HELP RESEARCHERS AND
SCIENTISTS LIKE THOSE HERE AT
THE NIH TAKE STEPS TOWARDS
DEVELOPING A HEALTHIER WORLD.
SO WITH THAT I WILL SAY THANK
YOU VERY MUCH.
[ APPLAUSE ]
>> THANK YOU DR. LOCKWOOD.
NEXT WE HAVE COMING UP ELAINE
PONSIEO.
HE WILL INTERVIEWING
MRS. JENNIFER FAULK.
GIVE THEM A WARM WELCOME.
>> OKAY, GOOD MORNING EVERYONE.
GOOD MORNING MRS. FAULK.
FIRST THANK YOU SO MUCH FOR
TRAVELING ALL THIS WAY TO SHARE
YOUR STORY.
I KNOW WE'VE TALKED NUMEROUS
TIMES BUT I THINK THIS AUDIENCE
WILL BE VERY INTERESTED IN WHAT
YOU HAVE TO SHARE WITH US.
AND THEN ALSO, YOU ACTUALLY HAVE
PICTURES FOR US FOR THE AUDIENCE
TO SEE AS WELL SO YOU CAN TAKE
ACROSS YOUR JOURNEY WITH LUPUS
AND HOW IT HAS IMPACTED YOU.
SO FIRST MRS. FAULK, I WILL
START WITH THE INITIAL QUESTION,
COULD YOU PLEAS TELL US ABOUT
WHEN YOU'RE FIRST DIAGNOSED WITH
LUPUS?
>> ABSOLUTELY, SO I FESTER WANT
TO ALSO SAY THANK TO YOU
EVERYONE FOR BEING HERE, THIS IS
QUITE A SHOCK FOR SOMEONE WHO'S
40 YEARS IN THE PROGRAM
DR. BALOW, I'M PART OF YOUR CREW
AND I'M STILL STANDING.
SO THIS PICTURE IS ACTUALLY
AFTER I WAS DIAGNOSED WITH LUPUS
AT 16 IN 1981, I WENT TO THE
LIBRARY, THERE WAS NO GOOGLE
YET.
MY NIECE KIDS ME THAN THIS THAT
I'M OLDER THAN GOOGLE BUT THE
ONLY THING IT SAID WAS FATAL
BLOOD DISEASE, SO 16 YEAR-OLD
WALKING OUT OF THE LIBRARY AND
BY 18, I WAS--HAD THE RENAL
FUNCTION INVOLVEMENT, AND I WENT
TO A RENAL DOCTOR WHO
ESSENTIALLY TOLD ME THAT I WOULD
NOT LIVE TO SEE 20 AND THAT I
HAD TO DECIDE BETWEEN 2 DRUGS,
[INDISCERNIBLE] THAT I HAD TO
DECIDE WHICH I ALSO HAD VERY BIG
INTEREST IN CHEMISTRY AT THE
TIME AND I KNEW THAT CAN'T BE
RIGHT.
NOW CAN I DOCTOR ASK A PATE TO
CHOOSE.
WELL, WE ENDED UP HAVING
IRONICALLY MET A YOUNG LADY WHO
HAD WORK WIDE
DR. [INDISCERNIBLE] AND HAD A
PHONE NUMBER AND CALLED HIM AND
SO WE DREEF HERE THAT YEAR.
THIS ACTUALLY IN 1983, I HAD
JUST SIGNED ON AS A PATIENT, MY
KIDNEYS WERE BAD ENOUGH, GO
JENNIFER THAT I WAS ABLE GET IN
THE PROGRAM, SO THIS IS MY FIRST
DAY OF COLLEGE WHICH I WAS TOLD
WHY TO OR WHY WOULD I EVER WANT
TO CONSIDER GOING TO COLLEGE
BECAUSE I WASN'T GOING TO LIVE
TO BE 20 EMPLOY SO IT WAS QUITE
A GREAT DAY, THIS IS MY MOM AND
DAD.
SO THE NEXT PICTURE IS THE
DAMAGE AND WHAT HAPPENS WITH ...
SORRY.
>> IT'S OKAY.
>> I'M SURE--THANK YOU.
[ APPLAUSE ]
>> I ENCOURAGE YOU TO TAKE
PICTURES TO LOOK BACK BECAUSE
LOOKING BACK IS JUST AS
IMPORTANT AND IT WREAKS HAVOC,
THE DRUGS SOMETIMES ARE WORSE
THAN THE DISEASE, I LOST ALL MY
HAIR, I GAINED 60-POUNDS, I WAS
REALLY NOT DOING WELL AND BY
1988 I HAD COME BACK BECAUSE
WITH THE PROGRAM I PULLED
[INDISCERNIBLE] SO I WAS TAKING
THE [INDISCERNIBLE] BUT WITH THE
PREDNISONE, THIS IS WHAT
HAPPENED AND THEN I OF COURSE
LOST MY HAIR SHORTLY THEREAFTER.
BUT I DID MAKE IT THROUGH
COLLEGE, I DROPPED OUT 4 TIMES,
BACHELOR OF BUSINESS WITH A
MINOR IN CHEMISTRY WHICH I THINK
PROBABLY ANNOYED MY DOCTORS EVEN
MORE BECAUSE I ASK QUESTIONS
CONSTANTLY.
THANK YOU.
I DID WANT TO SAY WHEN I CAME
AND SIGNED ON, I HAD TO DECIDE
WHEN I WAS AN 18 YEAR-OLD TO
GIVE MY BODY TO SCIENCE WHILE I
WAS ALIVE.
I WAS WANDERING THE HALLS, AND
LISTENING TO A WALKMAN, AND YES,
YOU MAY NOT KNOW WHAT THAT WAS,
AND IT I FOUND A BIBLE VERSE
THAT SAYS THERE A'S POOL OF
WATER WHERE THE ANGELS GATHER
AND IT SAYS WHEN THE SICK PERSON
STIRS THE WATER, THE SICK PERSON
IS HEALED.
SO I HAD THE FAITH AND NIH
PROVIDED THE HOPE AND YOU DID
BECAUSE HERE I SIT TODAY.
SO WHEN I JOINED, IF YOU CAN GO
TO THE NEXT PICTURE, THIS WAS
WHEN I WAS AT MY WORST AND AGAIN
TAKE PICTURES AS HORRIBLE AS
THAT LOOKS AND AS HORRIBLE IS TO
LOOK BACK AT IT, IT'S REALLY
IMPORTANT TO REFLECT AND THIS
WAS THIS IS WHEN YOU SWITCHED ME
FROM THE SOLUMEDEROL TO CYTOXAN,
I LOST THIRD POUNDS WATER WEIGHT
AND MY KIDNEYS KICKED AND BY THE
FIFTH TREATMENT I WAS STABLE AND
I AM 53 NOW.
SO THIS WAS MY FIRST DOSE,
FRIENDS AND MY FAMILY MEMBERS TO
GET ME THROUGH THAT FIRST DOSE.
THIS WAS MY LAST TIME I DROPPED
OUT OF COLLEGE BUT THIS IS WHEN
I RETURNED AND DID ACTUALLY
FINISH, SO THIS WAS A GREAT,
AGAIN, THIS WAS ON THE 90S.
SO THERE WAS IN-PATIENT THEN,
TOO, SO THE NEXT--
>> YEAH AND THEN MRS. FAULK WHEN
WE WERE SPEAKING AND THE YOU
KNOW THE STUDY YOU PARTICIPATED
IN OVER 30 YEARS AGO NOW.
>> FORTY.
>> FORTY YEARS AGO, NOW THIS IS
OUR STANDARD OF TREATMENT NOW WE
PROVIDE OUR PATIENTS FLOABALLY.
SO IT DOES PUT IT INTO
PERSPECTIVE WHAT RESEARCH CAN DO
AND HELP FUTURE PATIENTS.
SO AS A RESEARCHER, YOU KNOW I
THANK YOU AS A VOLUNTEER FOR
DOING THIS BECAUSE WITHOUT YOUR
PARTICIPATION THEN, MAYBE YOU
WOULDN'T HAVE THIS TREATMENT
TODAY, SO I DO THANK YOU EMPLOY.
>> THANK YOU NAND THEN WE'LL GO
TO THE NEXT PICTURE.
>> SO 1999 I DID GET MARRIED,
ANY OPPORTUNITY TO GET YOUR
WEDDING PICTURE IN SOMETHING,
BECAUSE YOU NEVER PULL THEM OUT
AGAIN, SO HERE'S MY WEDDING
PICTURE WITH MY HUSBAND GLEN,
HERE TODAY, IT'S LIKE A LIFE
THAT HE WASN'T AFTER THE
ACCIDENT WARE OF IN TERMS OF
LIKE, IT WAS THIS NIGHTMARE THAT
I CAME THROUGH AND THEN TO LOOK
BACK AT THIS NOW.
SO I THANK YOU GLEN FOR BEING
HERE.
I WAS REMISSION, 10 YEARS AT
THIS POINT AND THEN THIS IS MOST
RECENTLY BUT I WANTED TO YOU
KNOW TO THAT IF PARTICIPATING IN
NIH STUDY CAN SAVE A LIFE, IT'S
WORTH IT BECAUSE THAT 1 LIFE
MIGHT ACTUALLY BE YOUR OWN SO
THANK YOU.
>> I DO HAVE JUST A FEW
QUESTIONS, THANK YOU SO MUCH.
I DO THANK YOU FOR REACHING OUT
TO US BECAUSE WE STARTED TOWKING
BACK IN 2021 AND YOU'RE
INTERESTED IN TELLING YOUR STORY
AND I'M SO GLAD NOW THAT WE HAVE
PLATFORM FOR YOU TO BE ABLE TO
SHARE YOUR MESSAGE.
IS THERE ANYTHING ELSE THAT YOU
MIGHT WANT TO SHARE WITH
EVERYONE HERE.
>> YES, LET'S LOOK AT LONG-TERM
SIDE EVENS.
THAT'S PROBABLY OBVIOUSLY WITH
ALL THE DRUGS LOOKINGA THE IT
AND IF MYSELF AND I KNOW MANY OF
YOU PATIENTS SITTING IN THE ROOM
TODAY, THAT'S A CONCERN, IT'S A
CONCERN OBVIOUSLY BECAUSE BACK
40 YEARS AGO WHEN I TOOK THE
DRUG, THE ONLY INFORMATION THAT
EVEN LOOKING AT WHAT IT WAS AND
WHAT COULD POSSIBLY HAPPEN, IT
WAS A LIFE-DEATH CHOICE.
YOU HAD TO BUT THINK OF IT
FLOABALLY, WHAT THIS CAN DO FOR
GENERATIONS, FOR WHAT THIS CAN
CHANGE AND SO, THAT FOR ME, FOR
ENCOURAGING ALL OF YOU, IT
UPSETS ME ABOUT THE DISPARITIES
IN THE ETHNICITY OF PEOPLE
SIGNING UP AND WE NEED TO WORK
ON THAT, WE NEED TO DO BETTER ON
THAT, WE MUST DO BETTER ON THAT
BECAUSE THAT'S THE ONLY WAY WE
WILL REFLECT OUR COMMUNITY AS A
WHOLE SO I DO FEEL VERY STRONGLY
ABOUT THAT BUT AGAIN, I JUST
WOULD LIKE TO SAY THANK YOU TO
THE RESEARCHERS, TO YOU, ELAINE,
TO DR. BALOW, DR. GUPTA, THINK
ABOUT 40 YEARS AGO THAT I READ
AT 16 YEARS OLD, THAT WAS IT.
SO TODAY IT'S A NEW REALITY,
IT'S A NEW REALITY FOR YOUNG
PATIENTS GETTING DIAGNOSED AND
I'M GRATEFUL BUT I SEE
GENERATIONS BEING GRATEFUL TOO.
>> THANK YOU SO MUCH.
WITH THAT MRS. FAULK, WE JUST
WANT TO DO 1 OR 2 MINUTES OF ANY
AUDIENCE QUESTIONS.
YOU'RE WELCOME TO PLEASE GO TO
THE MICROPHONE AND IF YOU HAVE A
QUESTION FOR MRS. FAULK, PLEASE
FEEL FREE.
>> UH OH.
>> SO I'M YOU KNOW KIND OF
CURIOUS THAT YOU CAME TO NIH FOR
SO MANY YEARS AND COMING BACK
NOW AFTER 30, 40IERS HOW DO YOU
THINK THINGS HAVE CHANGED AND IN
TERMS OF RESEARCH, WE LIKE TO
HEAR TRUSTEES PATIENTS WHO
SHOULD BE ON OUR AGENDA SO
ANYTHING FROM THE PATIENTS
PERSPECTIVE, 1 ABOUT THE NIH HOW
THINGS HAVE CHANGED AND WHAT SHE
WE BE THINKING ABOUT IN
EMERGINGS OF NEXT IN RESEARCH.
NFIRST TO COME BACK, I HAVEN'T
BEEN BACK IN 24 YEARS I AM
HUMBLED BY ALL THE RESEARCH THAT
IS HAPPENING RIGHT NONAPOPTOTIC
AND SEEING THE SLIDES.
THAT WASN'T GOING ON, THAT
WASN'T GOING ON 40 YEARS AGO AT
ALL.
SO THAT WARMS MY HEART AND FILLS
ME, I THINK THE GENETICS AND
THAT'S HUGELY IMPORTANT, THE
GENOME STUDY AND KNOWING AND
MAPPING AND FIGURE IT OUT.
THE PROBLEM IS IT'S WHACK A
MOLE, EVERYTHING'S BLAME OFFICE
OF DIVERSITY SYMPTOMS OF LUPUS
AND EVERY SINGLE THING THAT
HAPPENED TO ME AND I'M SURE MANY
OF YOU PATIENTS CAN SPEAK TO
THIS, OH THAT'S LUPUS, THAT'S
THE LUPUS, OH COME ON PEOPLE.
AND THERE HAS TO BE A
QUARTERBACK, THAT'S THE OTHER
THING I WANTED TO MENTION.
YOU CAN'T FARM OUT A PERSON'S
BODY TO DIFFERENT PEOPLE AND NOT
HAVE 1 PERSON IN CHARGE.
SO PATIENTS PLEASE BE ACTIVE,
TAKE THE ACTION IN WHAT YOU'RE
DOING IN THAT RESPECT AND USING
THE PATIENT TO BE A PART OF THAT
ROLE SO THAT THEY HAVE CONTROL.
BECAUSE YOU HAVE NO CONTROL OVER
WHAT'S HAPPENING TO YOUR BODY,
IT'S LIKE THIS OUT OF THE WAY,
OUT OF CONTROL FREIGHT TRAIN
GOING DOWN THE TRACKS SO HAVING
THE PATIENT WORK ON THAT, I
WOULD LIKE TO SEE IT, TOO,
BECAUSE WITH BACK WHEN I FIRST
TOOK THAT CYTOXIN, THE
[INDISCERNIBLE] AND CYTOXIN,
SOMEONE HAD ME SPEAK TO AN ASIAN
DOCTOR WHO WORK WIDE ME ON
MEDITATION AND I WAS GETTING SO
SICK AND I WAS SO STRESSED OUT
ABOUT TAKING THIS AND THEY
HELPED ME WORK ON BREATHING,
DIDN'T WORK TOO WELL, EARLIER
BUT WORKED ON BREATHING, WORKED
ON VISUALIZATION, ON BUILDING MY
WHITE COUNT, BUILDING MY ARMY
EVERY TIME I CLOSE MY EYES SO I
WOULD LOVE TO SEE CROSS OVER,
GIVING PEASHTS WHOLISTIC AS WELL
AS THE SCIENCE BECAUSE--THANK
YOU.
[ APPLAUSE ]
TAKE CHARGE, UNDERSTAND WHAT
THESE TEFS ME BUT HELPING YOUR
PATIENTS TO UNDERSTAND IT, TALK
OVER THEIR HEADS, TALK TO THEM
TO UNDERSTAND WHAT'S HAPPENING
AS BEST YOU CAN, IT'S SO
UNBELIEVABLY COMPLICATED BUT
THAT WHOLE WHACK A MOLE GAME IS
SO DIFFICULT, IT'S SO DIFFICULT
BECAUSE EVERYTHING'S POPPING UP
DIFFERENTLY FOR EACH PERSON SO I
APPLAUD ALL OF YOU PATIENTS TOO,
BECAUSE GET ACTIVE, GET INVOLVED
BUT ALSO TRY TO LOOK AT NEW
THINGS THAT ARE HAPPENING THAT
CAN HELP YOU BECAUSE YOU HAVE TO
BE YOUR QUARTERBACK.
[ APPLAUSE ]
>> CAN I ASK YOU SOMETHING?
>> SURE.
>> LIKE YOU I'M A PATIENT.
ONE OF THE ISSUES THAT AFFECT ME
THE MOST IS THE HAIR LOSS BUT I
HAVE PERMANENT HAIR LOSS.
I SEE THAT WHEN YOU IT'S BEEN
DIFFERENT, CAN YOU EXPLAIN I
MEAN?
>> SO I THINK THIS IS ALSO PLACE
TWHO AND I'M SURE A DOCTOR CAN
SPEAK TO THIS AS WELL BUT I
THINK FOR EACH PERSON IT'S
DIFFERENT.
I DID LOSE ALL MY HAIR, IT I DID
LOSE IT, IT COMES BACK BABY FINE
AND THAT WAS SOMETHING THAT TOOK
TIME BUT I--THIS IS GOING TO
SOUND--TALK TO YOUR HAIR DRESSER
BUT WHAT CAN YOU DO, THE LESS
YOU TOUCH IT, THE LESS YOU DO
THINGS TO IT USING--WASHING IT,
USE MORE CONDITIONER, I THINK
THAT'S PROBABLY SIMPLISTIC SOME
OF YOU DON'T CARE ABOUT THAT BUT
FROM PATIENT TO PATIENT, I WOULD
LOVE TO TALK TO YOU AFTERWARDS
AS WELL EMPLOY THANK YOU.
THANK YOU AGAIN, SO MUCH
KD--SALLY A.
>> THANK YOU MRS. JENNIFER FAULK
FOR SHARING YOUR STORY.
NEXT WE HAVE COMING UP TO THE
STAGE DR. MARRIANA KAPLAN, SHE
IS THE CHIEF OF THE SYSTEMIC
AUTOIMMUNITY BRANCH AND SHE'S
THE DEPUTY SCIENTIFIC DIRECTOR
OF NETWORK IAMS, SHE WILL SPEAK
ON CARDIOVASCULAR DISEASE IN
LUPUS, AND RESEARCH DISCOVERIES
HERE AT NIAMS, IMIF HER A WARM
WELCOME.
NTHANK YOU VERY MUCH, GOOD
MORNING EVERYONE.
IT'S WONDER EVERL TO SEE
PATIENTS AND COLLEAGUES HERE AS
WE TALK ABOUT SOME OF THE
DISCOVERIES THAT HAVE BEEN MADE
AT NIAMS OVER MANY DECADES AND I
WANT TO FOCUS TODAY ON 1
COMPLICATION THAT IMPACTS LUPUS
SIGNIFICANTLY WHICH IS
CARDIOVASCULAR COMPLICATIONS BUT
ALSO TO EMPHASIZE THE IMPORTANCE
OF TRANSLATIONAL RESEARCH AND
HOW CAN WE LEARN AND BRING
THINGS THAT WE LEARN AT THE
BENCH TO THE BEDSIDE AND THEN
BACK FROM THE DISCOVERIES WE
MAKE THROUGH CLINICAL TRIALS TO
MORE BENCH AND REDIRECT SOME OF
THE BENCH RESEARCH AS WE DO
EVERY DAY, SO, JUST WANT TO
EMPHASIZE THAT ALTHOUGH WE HAVE
MADE AMAZING PROGRESS IN
DIAGNOSING AND TREATING LUPUS
THERE ARE TILL SOME AREAS THAT
ARE LAGGING IN UNDERSTANDING AND
PREVENTION AND TREATMENT AND 1
OF THEM IS CARDIOVASCULAR
DISEASE AS MANY OF YOU KNOW
VASCULAR EVENTS ARE IMPORTANT
CONTRIBUTORS TO MORBIDITY AND
MORTALITY IN LUPUS, IN YOUNG BEM
THERE IS TO GET A HEART ATTACK
OR STREAK IS ABOUT 50 FOLD
GREATER THAN A WOMAN WHO DOESN'T
HAVE LUPUS, AND CONTRARY TO WHAT
WAS CONSIDERED MANY DECADES AGO,
THIS IS DUE TO ACCELERATED
ABNORMAL GLUCOSE TOLERANCELER O
CLEROSEIS, WHAT PEOPLE MUCH
OLDER MAY GET AS OUR ARTERIES
AGE AND THIS HAPPENS IN LUPUS
MUCH EARLIER.
AND WE CANNOT EXPLAIN THESE BY
THE TYPICAL RISK FACTORS OF
CARDIOVASCULAR DISEASE, SMOKING,
DIABETES, NOT THAT THEY DON'T
PLAY A ROLE AND THEY SHOULDN'T
BE CONTROLLING LUPUS BUT THERE'S
SOMETHING MORE GOING ON IN LUPUS
PATIENTS AND I HAVE SPENT
SEVERAL DECADES OF MY RESEARCH
AT TRYING TO UNDERSTAND HOW THE
IMMUNE SYSTEM IN LUPUS PATIENTS
MAY CONTRIBUTE TO THE DEVELOPING
PREMATURE VASCULAR DEC.
AND THE DAMAGE TO THE BLOOD
VESSELS NOT ONLY IMPORTANT FOR
THE RACE FOR HEART ATTACKS CAN
STROKE BUT THE THING THAT
CONTRIBUTES TO SOME OF THE
COMPLICATIONS THAT AFFECT WOMEN
WITH LUPUS, THE KIDNEYS, THE
BRAIN, EVEN COMPLICATIONS DURING
PREGNANCY AND JUST TO MENTION
THIS IS--THIS IS HAPPENING ALSO
IN KIDS THAT DEVELOP LUPUS EARLY
ON.
SO THIS IS SOMETHING UNUSUAL
ABOUT THIS BECAUSE AS I
MENTIONED WOMEN TYPICALLY HAVE A
LOWER RISK FOR HEART DISEASE AND
DISEASE THAT PRIMARILY STRIKES
YOUNG WOMEN ALTHOUGH IT CAN
AFFECT AT ANY AGE AND MANY OF
THE REASONS BY WHICH WE EXPLAIN
CARDIO VASCULARY DEC OUTSIDE OF
LUPUS REALLY DO NOT APPLY IN
THIS DISEASE AND IN FACT SOME OF
THE TRIALS OF DRUGS OF EFFECTIVE
IN PREVENTING CVD, HAVE BEEN IN
LUPUS SO THERE'S A BIG NEED TO
FURTHER LANE WHAT'S GOING ON AND
COME UP WITH DRUGS THAT CAN
PREVENT AND DO EARLY TREATMENT
OF THIS COMPLICATION.
AND SO, ABOUT OVER 10 YEARS AGO,
SHORTLY AFTER I JOINED NIAMS, WE
ESTABLISHED WITHIN THE NATURAL
HISTORY, LUPUS PROTOCOL, ARDIO
VASCULAR NATURAL HISTORY
PROTOCOL, THAT HAS BEEN
INVALUABLE IN TRYING TO FURTHER
UNDERSTAND WHY WOMEN OR PATIENTS
WITH LUPUS DEVELOP
CARDIOVASCULAR DOZE AND ALSO
START TO TRY TO IDENTIFY DRUGS
THAT MAY HELP US PREVENT OR
TARGET THIS COMPLICATION.
AND JUST BROADLY SPEAKING, WE
ARE BRINGING THE PEASHTS TO THE
NIH CLINICAL CENTER ASK WE'RE
DOING A VERY COMPREHENSIVE
ASSESSMENT OF WHAT'S HAPPENING
TO THE BLOOD VESSELS SO WE CAN
IDENTIFY EARLY ON BEFORE END
ORGAN DAMAGE OCCURS HOW THE
ARTERIES MAY BE SUFFERING, WHY
IS THIS HAPPENING, CAN WE
IDENTIFY BY O MARKERS THAT WILL
ALLOW US TO DETECT THOSE
PATIENTS AT HIGHER RISK TO
DEVELOP CARDIO VASC LOOR DISEASE
AND ALSO TO START TESTING SOME
DRUGS THAT WE THINK MAY BE
IMPORTANT OR PLAY AN IMPORTANT
ROLE IN PREVENTING CARDIO VASC
LOOR DISEASE AND SOME OF THESE
DRUGS ACTUALLY WE HAVE
REPURPOSED, THEY HAVE BEEN
APPROVED FOR OTHER CONDITIONS
BUT WE THOUGHT BASED ON OUR
RESEARCH AND THE RESEARCH OF
OTHERS IT MAY BE USEFUL TO
PREVENT BLOOD CELL DAMAGE IN
PATIENTS WITH LUPUS, SOME DRUGS
ARE APPROVE INDEED LUPUS, USED
IN LUPUS BUT THEY HAVEN'T BEEN
STUDIED FOR CARDIOVASCULAR
DISEASE AND WE ARE HOPING ALSO
TO IN THE BEST CASE SCENARIO TO
USE DRUGS THAT WON'T SUPPRESS
THE IMMUNE SYSTEM OF LUPUS
PATIENTS BUT MAY HAVE AN EFFECT
IN MODULATING HOW THEIR IMMUNE
SYSTEM IS AFFECTING THE BLOOD
VESSEL SO IT'S STILL EARLY BUT
WE ARE MAKING PROGRESS AND I
WOULD LIKE TO EMPHASIZE JUST A
COUPLE OF TRIALS WE HAVE
FINISHED A COUPLE THAT ARE
ONGOING OR MAYBE ABOUT TO START
AND WHAT I CAN SAY IS ALL OF
THIS CAME FROM RESEARCH THAT WE
DID IN THE LAB AND OBSERVES WE
DID THAT LED HYPOTHESIZE THAT
THIS MAY BE A WAY TO GO.
SO 1 OF THEM IS A DRUG THAT IS
ACTUALLY USED TO TREAT TYPE 2
DIABETES, [INDISCERNIBLE]
AGONIST AND THEY HAVE BEEN
PROPOSED TO HAVE PROTECTION OF
THE CARDIOVASCULAR SYSTEM JUST
BEYOND CONTROLLING GLUCOSE THIS
DIABETICS AND THEY HAVE BEEN
TESTED IN OTHER CONDITIONS SO WE
COMPLETED A TRIAL SEVERAL YEARS
AGO TO TEST WHETHER 1 OF THESE
DRUGS WOULD IMPROVE FUNCTION IN
PATIENTS WITH LUPUS HERE AT THE
NIH CLINICAL CENTER AND I'M
SORRY TO SHOW GRAPHS BUT IN A
SHORT PERIOD OF TIME, WHEN WE
TREAT THESE PATIENTS WITH THE
DRUG [INDISCERNIBLE] COMPARED TO
PLACEBO WE SAW VERY STRIKING
EFFECT -S WHEN THE PATIENTS WERE
ON THE DRUG THAT WERE NOT
PRESENT WHEN THEY WERE ON THE
PLACEBO AND THIS INCLUDED
IMPROVING HOW THEIR BLOOD
VESSELS FUNCTION WHICH IS VERY
IMPORTANT IN PREDICKING WHETHER
A PATIENT MAY DEVELOP CARDIO
VASC LOOR DISEASE BUT ALSO AT
THE LEVEL OF THINGS THAT MAY GO,
MAY BE ABNORMAL IN LUPUS
PATIENTS THAT MAY DEVELOP
CARDIOVASCULAR DISEASE SUCH AS
HAVING ABNORMAL LIPIDS WE KNOW
IS A RISK FACTOR IN MANY DISEE E
DISEASES, WE FOUND A VERY
TRIKING IMPROVEMENT FROM JUST
THESE DRUGS, SO JUST IN DRUGS
THAT TREAT INSULIN RESISTANCE
MAY EFFECTS IN PEOPLE WITH
INFLAMMATION, WITH INFLAMMATORY
DISEASE SUCH AS LUPUS AND WE'RE
HOPING TO EXPAND SIMILAR STUDIES
IN THE FUTURE.
MY LAB HAS BEEN VERY INTERESTING
IN UNDERSTANDING HOW CERTAIN
COMPONENTS OF THE IMMUNE SYSTEM,
THE INNATE WHAT WE CALL THE
INNATE IMMUNE SYSTEM, MAY
CONTRIBUTE TO PREMATURE CARDIO
CARDIOVASCULAR DISEASE IN LUPUS
IN GENERAL.
SO WHAT WE HAVE CONE OVER THE
YEARS IS WE HAVE HYPOTHESIZED
THAT THESE COMPONENTS OF THE
INNATE IMMUNE SYSTEM, ARE
DRIVING CARDIO VASC LOOR
DISEASE, SO SOME OF THE
COMPONENTS WE THINK ARE
IMPORTANT CAN NOW BE TARGETED
THROUGH DRUGS THAT WERE RECENTLY
APPROVED EITHER IN LUPUS OR IN
OTHER AUTOIMMUNE DECS AND I JUST
WANT TO SHOW YOU THAT 1 OF THEM
WHICH WAS THE JACK INHIBITORS
THAT THERE HAVE BEEN A FEW
CLINICAL TRIALS IN LUPUS
SUGGESTING THIS DRUG MAY BE
USEFUL EVEN IF THEY HAVEN'T BEEN
APROWF PROVED FOR LUPUS YET BUT
WE HAVE ALSO TESTED THESE DRUGS
TOFACITINIB, AND FOUND IT
IMPROVED FUNCTIONS IN PATIENTS
AND SOME PARAMETERS THAT MIGHT
EXPOSE PATIENTS TO
CARDIOVASCULAR DISEASE EMPLOY SO
NOW WE'RE MOVING FORWARD WITH
ANOTHER DRUG AND THE STUDY IS
ABOUT TO START, THE TOFACITINIB
TRIAL AND WE'RE HOPING TO DO
INHIBITION OF A COUPLE PATHWAYS
THAT ARE NORMALLY IN LUPUS AND
AS 1 OF THE READ OUTS WE WILL
LOOK AT THE VASCULAR DISEASE OF
THIS PATIENT.
AND THE OTHER DRUG THAT WE ARE
ALREADY ONGOING CLINICAL TRIAL
BASED ON PREVIOUS STUDIES WE DID
IS WHAT HAPPENS WHEN WE BLOCK
THIS COMPOUND OF TYPE 1
INTERFERONS WHICH WE THINK ARE
VERY IMPORTANT IN PROMOTING
LUPUS BUT WE HAVE ALSO
HYPOTHESIZED THEY PLAY A ROLE IN
CARDIO VASC LOOR DISEASE, AND
WHEN WE HAVE SEEN STUDIES THAT
BLOCK THIS DRUG, WE SEE SIGNALS
APPROXIMATE AND WE MAY BE ABLE
TO IMPROVE VASCULAR FUNCTION AND
DEC IN THESE PATIENTS.
SO YOU WILL HEAR MORE ABOUT THIS
STUDY FROM MICHAEL DAVIS ABOUT
YOU THIS IS A STUDY THAT I AM
VERY HAPPY TO HAVE EMBARKED IN
BECAUSE IT REFLEBS MANY YEARS OF
RESEARCH AND TESTING THE
HYPOTHESIS WHETHER IF WE BLOCK
THESE MOLECULES WE WILL BE ABLE
TO PREVENT OR MITIGATE
CARDIOVASCULAR DISEASE
DEVELOPMENT.
AND FINALLY, I TOLD YOU ABOUT
ARE THERE WAYS WE ARE MAY BE
ABLE TO MODULATE CARDIOVASCULAR
DISEASE WITHOUT CAUSING
SIGNIFICANT SUPPRESSION OF THE
IMMUNE SYSTEM AND ALSO TO
EMPHASIZE THE COLLABORATIVE
NATURE OF OUR WORK, THIS IS
ACTUALLY A CLINICAL TRIAL THAT
IS GOING TO BE STARTED BY NHLBI,
BY DR. MICHAEL [INDISCERNIBLE]
AN INVESTIGATOR AT NHLBI IN
COLLABORATION WITH THE LCTU WITH
US AND THIS WILL TEST ACTUALLY
IF WE IMPROVE THE METABOLISM OF
IMMUNE CELLS IN LUPUS THROUGH
SUPPLEMENT CALLED
[INDISCERNIBLE], IF NOT ONLY IT
WILL IMPROVE THE IMMUNE
FUNCTIONS OF THESE PATIENTS OR
HOW DISREGULATED THE IMMUNE
SYSTEM BUT ALSO ARE WE GOING TO
HAVE AN IMPACT ON THE
CARDIOVASCULAR DISEASE.
SO THESE ARE HIGHLIGHTS I WANT
TO EMPHASIZE BECAUSE WE'RE
MAKING SIGNATURES NEFF CANT
PROGRESS AND ALTHOUGH IT WILL
TAKE TIME TO DEFINE WHICH DRUG
MAY BE THE BEST FOR CARDIO VASC
LOOR DISEASE PREVENTION AND WE
MAY NEED MORE THAN 1 SO I THINK
THE LAST IMMENSE PROGRESS IN
BETTER BEDDING THIS IMPORTANT
COMPLICATION AND I JUST WANT TO
THANK ALL THE SUPPORT FROM NIAMS
AND THE CLINICAL PROGRAM FROM
DR. GOLGBERG AND EVERYONE WHO
MADE THIS POSSIBLE AND THE
PEASHTS WITH WHOM THESE HAVE
BEEN--YOU ARE THE 1S THAT
INSPIRE US AND WE'RE HOPING TO
MAKE IN CONTRIBUTIONS IN THE
NEXT FEW YEARS TO BETTER
UNDERSTAND THIS AND OTHER
COMPLICATIONS IN THE DEC.
THANK YOU VERY MUCH.
>> THANK YOU DR. KAPLAN.
NEXT I WOULD LIKE TO INVITE BACK
TO THE STAGE PLSES JENNIFER
FAULK, DR. HASNI, AND
DR. CRISWELL, AND DR. BALLOW, WE
CAUGHT HIM JUST IN TIME.
>> WE HAVE A QUICK PRESENTATION
THIS IS FOR DR. BALOW FOR ALL
HIS WORK HE HAS DONE.
[ APPLAUSE ]
>> OUR NEXT SPEAKER IS DR. LAURA
LEWANDOWSKI.
SHE IS THE CLINICAL INVESTIGATOR
AT NIAMS.
HER PRESENTATION WILL BE ON
YEENS AND LUPUS, UNDERSTANDING
THE GENETICS OF LUPUS IN GLOBAL
POPULATIONS.
GIVE HER A WARM WELCOME.
[ APPLAUSE ]
>> HI, EVERYONE.
IT'S SO WONDERFUL TO BE HERE
WITH ALL OF YOU TODAY.
IT'S GREAT TO LOOK OUT AND SEE
SO MANY FAMILIAR FACES FROM SO
MANY DIFFERENT PLACES, COMMUNITY
ORGANIZERS, ADVOCATES AND
PATIENTS SO THANK YOU FOR
ALLOWING ME TO BE HERE WITH YOU
TODAY, YOU KNOW I WANT TO
ACKNOWLEDGE ALL THE INCREDIBLE
PEOPLE THAT ARE A PART OF THE
WORK WE DO HERE INCLUDING THE
YOU ISSUE THE AMAZING PATIENTS
BECAUSE WITHOUT YOU THE PATIENTS
PARTICIPATE NOTHING OUR
RESEARCH, ABSOLUTELY NONE OF
THIS WOULD BE POSSIBLE SEE A BIG
SHOUT OUT TO YOU EMPLOY SO WE
HEARD A LOT TODAY ABOUT THE
IMPACT OF LUPUS AND WE ALL KNOW
THAT IT IS A DEVASTATING DISEASE
THAT CAN AFFECT ANY ORGAN
SYSTEM, THE FLARES CAN BE
UNPREDICTABLE AND THAT CAN MAKE
LIFE WITH LUPUS REALLY, REALLY
DIFFICULT.
I'M A PEDIATRIC RHEUMATOLOGIST
SO I'M REALLY INTERESTED IN
LUPUS THAT STARTS IN CHILDHOOD.
AND ACTUALLY 1 OUT OF EVERY 5
LUPUS CASES IS DIAGNOSED BEFORE
AGE 18 AND WE HEARD A LITTLE
ABOUT THAT IN THE PATIENT STORY
EARLIER THIS MORNING WITH
MRS. FAULK.
KIDSWITH LUPUS HAVE A VERY
SEVERE DISEASE AND LOTS OF
DIFFERENT WAYS SO THEY HAVE MORE
DISEASE ACTIVITY, THEY HAVE MORE
OF THE SEVERE ORGAN SYSTEM
INVOLVEMENT LIKE THEIR KIDNEYS
OR THEIR BRAINS AND THEY
ACTUALLY GET MORE ORGAN DAMAGE
DESPITE HAVING YOUNGER HEALTHIER
BODIES THAN ADULTS.
I BECAME REALLY INTERESTED IN
LUPUS IN TBLOABAL POPULATIONS
DURING MY TRAINING EMPLOY
BECAUSE WE HAVE HEARD EARLIER
TODAY THAT LUPUS IS MORE SEVERE
AND PREVALENT IN NONWHITE
POPPULESS AND YOU KNOW JUST
PRESENTED HOW LUPUS IS MORE
SEVERE IN CHILDREN AND I WAS
ASKING THE QUESTION, WELL WHAT
ABOUT CHILDREN IN AFRICA?
AND THERE WAS VERY LITTLE DATA
AT THE TIME, SO THERE WAS 1
PAPER OF 30 PATIENTS FROM THE
1990S FROM SOUTH AFRICA AND THAT
WAS ABOUT IT.
AND SO WITH THIGH MENTORS AT
DUKE WHICH IS WHERE I TRAINED, I
DEVELOPED A COLLABORATION WITH
AN AMAZING PERSON, DR. CHRIS COT
AND WENT TO SOUTH AFRICA TO HELP
HIM CHARACTERIZE THOSE LUPUS
PATIENTS BECAUSE WHEN I
CONTACTED HIM, I SAID, YOU KNOW
I'M NOT SEEING A LOT ABOUT THIS
IN THE LITERATURE, DO YOU HAVE
ANY PATIENTS AND HE SAID, I HAVE
PATIENTS COMING OUT OF MY EARS
AND THEY'RE SUPER SICK BUT I'M
HERE TAKING CARE OF THEM AND I
JUST DON'T REALLY HAVE THE TIME
AND THE RESOURCES TO REPORT THEM
IN THE LITERATURE AND SO WE
WORKED TOGETHER TO CREATE THE
LARGEST REGISTRY OF LUPUS
PATIENTS IN SUB-SAHARAN AFRICA
AND DESCRIBE THEM.
SO LIKE PEDIATRIC PATIENTS IN
THE USES, THEY WERE MORE SEVERE
THAN ADULTS IN THE U.S., BUT
WITH WE COMPARE CHILDREN TO
CHILDREN, CHILDREN WITH LUPUS IN
AFRICA HAD MORE ORGAN DISEASE,
MORE DAMAGE AND HIGHER MORTALITY
AND I BECAME REALLY CONVINCED
THAT STUDYING CHILDHOOD IN ONSET
LUPUS IN DIVERSE POPULATIONS
WORLD WIDE IS CRITICAL.
WE HEARD A BIT TODAY ABOUT SOME
OF THE HEALTH DISPARITIES THAT
EXIST IN RESEARCH AND I JUST
WANT TO KIND OF PUT AN EYE TO
THE GLOBAL DISPARITIES THAT ALSO
EXIST.
SO, IF YOU LOOK AT WHERE MOST OF
THE WORLD'S CHILDREN LIVE,
FIGURE A IS A MAP OF THE
PEDIATRIC POPULATION IN THE
WORLD, AND MOST OF THE WORLD'S
CHILDREN LIVE IN LESS RESOURCE
NATIONS, SO, THE DARKER PINK
COLORS ASIA SUB-SAHARAN AFRICA,
SOUTHEAST ASIA IS WHERE MOST OF
THE WORLD'S CHILDREN LIVE, AND
IF YOU LOOK AT FIGURE B, IT'S AN
ALMOST INVERTED MAP WHERE
THEY'RE LOCATED IN NORTH
AMERICA, SO VERY FEW CHILDREN
HAVE ACCESS TO CLINICAL CARE AND
FURTHER MORE EVEN FEWER HAVE
ACCESS TO CLINICAL RESEARCH.
AND THAT REALLY SKEWS OUR
UNDERSTANDING OF THESE
POPULATIONS AND ALSO WHAT ARE
THE BEST--OUR UNDERSTANDING OF
WHAT ARE THE BEST MEDICINES TO
TREAT THEM AND BECAUSE I'M A
PURPOSE WHO THINKS A LOT ABOUT
GENETICS RESEARCH, THERE ARE
ALSO A LOT OF FLOABAL
DISPARITIES AND GENETICS
RESEARCH THAT HAVE RESULTED FROM
THESE DISPARITIES AND SO IF YOU
LOOK AT ALL OF THE PATIENTS IN
OUR GWAS CATALOG, 80% OF THEM
ARE OF EUROPEAN ANCESTRY AND THE
REST OF THE WORLD'S POPULATION
MAKES UP 20%.
NOW THERE'S LOTS OF PEOPLE WHO
REALIZED THIS IS A PROBLEM, NOT
JUST ME AND THERE'S ASH MAZING
TEAMS OF PEOPLE WITH GREAT
EFFORTS LIKE ALL OF US AND MANY
OTHER THINGS WORKING TO ADDRESS
THIS BUT WE'VE GOT A LONG WAY TO
GO SO WE HAVE A WHILE TO GET
THERE.
FOURTH
--NOW WE KNOW THAT THERE IS SOME
PART OF LUPUS THIS AS HAS A
GENETIC COMPONENT AND ALTHOUGH
WE DON'T FULLY UNDERSTAND THE
CAUSES OF LUPUS WE THINK PEOPLE
HAVE A CERTAIN GENETIC
SUSCEPTIBILITY ASK THERE ARE
ENVIRONMENTAL EVENTS THAT
TRIGGER YOU INTO CLINICAL
AUTOIMMUNITY AND IF YOU HAVE A
TWIN WITH LUPUS, AN IDENTICAL
TWIN YOUR CHANCE OF HAVING LUPUS
IS SOMEWHERE FROM 25-50%, SO
GENETICS ARE INVOLVED AND YOU
CAN HAVE RARE VARIANTS WHICH ARE
VARIANTS THAT OCCUR IN THE
PROTEIN CODING REGION OF THE
DNA, THEY USUALLY CAUSE THE
PROTEIN TO ACT IN A DIFFERENT
WAY AND ARE MORE LIKELY TO CAUSE
A PROBLEM AND THEN THERE ARE
COMMON VARIANTS SO THOSE ARE
USUALLY LESS DAMAGING BECAUSE
THEY OCCUR COMMONLY IN THE
POPULATION AND YOU MIGHT NEED
ACCUMULATION OF THOSE AND
ENVIRONMENTAL EVENT, TO KIND OF
CAUSE A PROBLEM, BUT WE REALLY
HAVEN'T LOOKED AT THIS IN A VERY
COMMON VARIANT IN THE ADUTS WE
WE DON'T FULLY UNDERSTAND THE
GENETIC ARCHITECTURE OF WILL YOU
PLEASEUS.
SO OUR LAB WANTED TO GETTA AT
THAT TO SEQUENCE LARGE MEMBERS
OF PEDEIAT AND I CAN ADULT
PATIENTS AND REALLY UNDERSTAND
OF THAT GENETIC COMPONENT TO
LUPUS, LET'S LOOK UNDER THE
HOOD, LET'S LOOK AT KIDS WHO ARE
MORE LIKELY TO HAVE GENETIC
KRIEEVERS OF THESE DISEASE
BECAUSE OF EARLY AND SEVERE
ONSET DISEASE AND LET'S FIGURE
IT OUT OF RARE VARIANTS,
ENVIRONMENTAL EVENTS.
AND SO WE CREATED THE GLOBAL
COLLABORATION FOR ADVANCING
GENETICS RESEARCH.
WE HAVE,AISE MAZING PATIENTING
HERE AT THE NIH AND WE HAVE MANY
WONDERFUL COLLABORATORS AROUND
THE WORLD INCLUDING CARA, A
PEDIATRIC ORGANIZATION TO GET
PATIENTS FROM ALL OVER NORTH
AMERICA AND COLLABORATORS WITH
CANADA, MEXICO, SOUTH AFFRIC
ATHE UK AND PAKISTAN TO INCREASE
DIVERSITY OF OUR POPULATIONS AND
I'M THOT GOING TO GO TOO MUCH
INTO THE WERE SIDE OF IT, BUT
JUST TO SAY, YOU KNOW WE HAD A
PAPER COME OUT LAST YEAR, IT
KIND OF STARTING TO LOOK AT
THESE POPULATIONS, THESE DIVERSE
POPULATIONS AROUND THE EXPORLD
WOO AND WE DID A TODAY OF TRIOS,
HAD IS 1 OF THE OTHER ADVANTAGES
OF USING PEDIATRIC POPULATIONS,
CAN YOU COMPARE THEM TO THE
PARENTS AND THAT CAN MAKE THE
INFORMATION MORE EFFECTIVE AND
WE LOOK AT HOW OFTEN ARE THOSE
TRANSMIT FRIDAY PARENT TO CHILD.
AND WHEN WE LOOKED WE FOUND, WE
DIDN'T FIND ANYTHING IN THOSE
COMMON VARIANTS, WE HAD A VERY
SMALL POPULATION, ONLY 83
PATIENTS SO THAT'S NOT SUPER
PRIZING BUT WHAT WAS REALLY
STRIKING WAS HOW ENRICHED THIS
POPULATION WAS IN RARE VARIANTS,
SO THOSE VARIANTS IN PROTEINS
CAUSING A PROBLEM AND WE FOUND
SOME PRETTY INTERESTING 1S, SOME
OF WHICH WERE GOING TO LOOK A
BIT FURTHER INTO DOWN THE ROAD
BUT THIS KIND OF JUST CONFIRMED
TO US THAT THIS IS AN IMPORTANT
POPULATION IN WHICH TO STUDY THE
GENETICS OF THIS DISEASE.
SO IN CONCLUSION, IT'S IMPORTANT
TO STUDY THE GENETICS OF LUPUS,
IT'S IMPORTANT TO DO THIS IN A
WAY THAT'S ENCLUESIVE AND
INCLUDES A DIVERSE GROUP OF
PATES BOTH AGE, GEOGRAPHIC
LOCATION AND ANCESTRY, AND THE
MORE THAT WE LEARN ABOUT THIS,
HOPEFULLY WE CAN THAT I CAN THIS
FROM THIS BROAD CLINICAL
EMBRELLAA OF LUPUS WE HEARD
ABOUT EARLIER TODAY TO MORE
PRECISE IN LECULAR DIAGNOSIS, SO
WE CAN BETTER TARGET TREATMENTS
REDUCE SIDE EFFECTS AND IMPROVE
OUTCOMES FOR PATIENTS.
AND THE MORE WE LEARN ABOUT
LUPUS PATIENTS EVERYWHERE, THE
MORE WE ANYWHERE, THE MORE WE
LEARN ABOUT LUPUS PATIENTS
EVERYWHERE.
AND WE HAVE AN ACTIVE GENETICS
STUDY HERE AT NIAMS.
SO IF YOU'RE INTERESTED REACH
OUT TO US AND LET US KNOW WITH
THAT I WISH YOU ALL HAPPY LUPUS
AWARENESS MONTH AND THANK YOU SO
UPON FOR ALL YOU DO TO HELP US
WITH OUR RESEARCH.
[ APPLAUSE ]
>> THANK YOU DR. LEWANDOWSKI,
NEXT WE HAVE DR. GUPTA, DIRECTOR
OF PATHWAY DRICH THERAPEUTICS
AND LUPUS DISEASES IN NIAMS AND
HE WILL SPEAK ON RARE DISEASE
SCOEVERYS IN LUPUS.
--DISCOVERIES IN LUPUS.
>> HI, WELL, THANK YOU JULIE FOR
THAT KIND INTRODUCTION AND THANK
YOU ALL FOR I --JOINING US FORE
CELEBRATION OF 30 YEARS OF
CLINICAL RESEARCH IN LUPUS AT
NIH AND THAT SOMEONE WHO'S BEEN
PART OF THE GROUP FOR NOW 13 OUT
OF THOSE 30 YEARS, IT'S REALLY
BEEN AN AMAZING JOURNEY, I CAN
TELL YOU THAT AND I WANTED TO
TAKE THIS OPPORTUNITY TO TELL
YOU A STORY, DURING THIS JOURNEY
AND HOW WE ARE LEVERAGING THIS
UNIQUE INSTITUTION, THE
INTRAMURAL NIH, AND THE CLINICAL
CENTER WHERE YOU ARE SEATED NOW.
AND THESE UNIQUE AND RARE
PATIENTS WHO WE SEE HAVE THESE
RARE DISEASES AND HOW WE ARE
ABLE TO HELP THOSE PATIENTS WITH
THOSE RARE DISEASES FROM WHAT WE
LEARNED IN STUDIES IN LUPUS, AS
WELL AS THOSE OF OUR LUPUS
PATIENTS.
SO WE TALKED A LOT ABOUT HOW
LUPUS IS SO HIDE ROG NOWS, FOR
SO MANY YEARS WE'VE BEEN
CLASSIFYING OUR DEC BASED ON
CLINICAL PHENOTYPE BUT WE ALL
KNOW, I MEAN, OUR LUPUS PATIENTS
ARE VERY HED ROMG NOWS.
BUT WHAT WE REALLY NEED TO START
DOING IS IDENTIFYING PATIENT
WHEN IS WE TALK TO THEM AND LOOK
FURTHER AND MORE DETAILED INTO
WHAT ARE THE PATHWAYS THAT ARE
DISREGULATED IN THESE PATIENTS
AT THAT GIVEN PERIOD OF TIME AND
WOPS WE SAW DOING THAT WE WILL
FIND PERHAPS A DISEASE THAT'S
DRIVING AT 1 TIME IN NAPATIENT
MAY BE NOT THAT UPREGULATED AND
DRIVING THE DISEASE IN ANOTHER
PATIENT AND THAT OTHER PATIENT
MY NEED A DIFFERENT THERAPY THAN
WHAT THE DEC CALLED NICK C AND
WHEN WE DO START LOOKING AT THIS
IN THIS WAY OUR HOPE IS THAT WE
WILL ACTUALLY START FINDING
PATIENTS WHO MAY HAVE A
COMPLETELY DIFFERENT DISEASE
DRIVEN BY THEIR OWN UNIQUE
PATHWAYS HIDDEN WITHIN THIS
GROUP THAT WE'VE GIVEN THE NAME
OF LUPUS.
SO WE'VE APPROACHED THIS BY
STUDYING AS DR. KAPLAN MENTIONED
BY FOCUSING STUDIES ON INNATE
IMMUNE SYSTEM WHICH IS 1 ARM OF
THE IMMUNE SYSTEM, IT'S YOUR
FIRST LINE OF DEFENSE AGAINST
INFECTION AND AS PART AS OF THE
NEUTROPHILS, AND WE KNOW THESE
ARE MOLECULES THAT HELP FIGHT
INFECTIONS BUT WE ALSO KNOW THEY
ARE VERY HIGH LE UPREGULATED IN
LUPUS.
AND WE ALSO STUDY THE
INTERFERONS BY STUDYING THE
SIGNAL THE OF JAKSTAT PATHWAY,
SO WHAT HAPPEN WHEN IS THEY BIND
ON CELLS?
THEY ACTIVATE THESE CHANNELS
KINASES WHICH ARE ENZYMES BOUND
TO THAT RECEPTOR, THERE'S 4 OF
THEM, WHICH IN TURN ACTIVATE THE
TRANSCRIPTION MOLECULES CALLED
STATS THERE ARE 7 OF THEM, WHICH
EVENT WHY ULTIMATELILY LEADS TO
A CASCADE OF BASICALLY LEADING
TO INFLAMMATION.
AND WE KNOW AS I MENTIONED
INTERFERON SIGNALING AND
PATHOGENIC IN OUR PATIENTS AND
STUDIES DONE HERE BY DR. OSHAY
AND OTHERS, WE NOW HAVE DRUGS
THAT CAN TARGET THESE KINASES
AND THEY'RE APPROVED FOR OTHER
DISEASES LIKE RHEUMATOID
ARTHRITIS, HOWEVER, WHEN WE AND
OTHERS LOOK AT USING THIS DRUG
IN LUPUS, THE RESULTS HAVE BEEN
MIXED.
WE CLEARLY KNOW THERE'S A SUBSET
OF PATIENT WHO IS DO RESPOND
BETTER AND WE NEED MORE STUDIES
TO ELECTRIC AT IT MORE FURTHER.
ANOTHER APPROACH THAT I'VE TAKEN
IS LOOKING AT THESE PATIENTS WE
SEER HERE AT NIH WHICH HAVE
GENETIC DETECTS IN THIS CRITICAL
MOLECULES CALLED STATS, AS PART
OF THE JAKSTAT PATHWAY AND 1 OF
THESE RARE MONOGENIC DEC IS
CALLED JOB SYNDROME.
NOW IT'S A FAIRLY RARE DEC,
THERE'S ABOUT 300 REPORTED CASES
WORLD WIDE.
AND LIKE THE BIBLICAL CHARACTER
JOB UNFORTUNATE PATIENTS GET
WIDE SPREAD SKIN INFECTIONS,
BOILS, STEF LO CAUCUS AND CANDID
A INFECTIONS AND THE MUTATION
THEY HAVE SIGNIFY LOSS OF
FUNCTION MUTATION IN THEIR STAT
3 MOLECULE.
A FEW YEARS AGO WE WERE
CONSULTED TO SEE A JOB'S PATIENT
WHO ALSO HAD LUPUS, THIS WAS
VERY INTRIGUING TO US BECAUSE
WHEN PEOPLE HAVE LOOKED AT STAT
3, THIS MOLECULE IN LUPUS, IT'S
ACTUALLY OVEREXPRESSED AND THERE
ARE TALKS OF TARGETING THIS IN
SOME WAY AND BLOCKING IT SO THAT
WE CAN HELP LUPUS PATIENTS BUT
HERE WE HAVE AIAN JOB SYNDROME
PATIENT WHO IS DOES NOT HAVE A
FUNCTIONING STAT 3 BUT ALSO HAS
LUPUS.
SO WE WERE VERY INTRIGUED, WE
COLLABORATED WITH THE NIAID
COLLEAGUES WHO AGAIN SEE THESE
RARE PATIENTS AND THEY HAVE THE
LARGEST COHORT IN THE WORLD
ABOUT 160 PATIENTS HERE WITH
JOB'S AND YES INDEED, 10 OF
THESE PATIENTS HAD LUPUS LIKE
MEN FESTATIONS, NOW THAT
ABSOLUTE NUMBER LOOKS LOW BUT
WHEN YOU LOOK AT THE PREVALENCE,
6.3% IN JOB'S WHEN YOU LOOK AT
THE REGULAR PREVALENCE OF LUPUS
IN THE POPULATION, THAT'S A 21
FOLD INCREASE IN PREVALENCE OF
LUPUS IN JOB, SO THEY'RE ENRICH
INDEED IN POPULATION, MOST OF
THESE PATIENTS ARE WOMEN, NEARLY
ALL OF THEM HAD TYPICAL LUPUS
ANTIBODIES, AND 50% OF THEM HAD
A SEVERE KIND OF LUPUS THAT
INFECTED THEIR KIDNEY LUPUS
NEPHRITIS.
AND WHEN WE GOT BLOOD AT THEM
AND LOOKED AT IT IN OUR LABS WE
FOUND AN ENACTIVATED IMMUNE
SYSTEM WITH ACTIVATION OF THE
NUTRIFILLS THEY WERE MAKING
ANTIBODIES AGAINST THESE
NEUTROPHILIC CONDUCTS AND
ACTIVATED THE PATHWAY.
NOW WHAT WAS REALLY INTRIGUING
NOW THAT THIS ACTIVATION OF THIS
INNATE IMMUNE PATHWAY WAS NOT
JUST RESTRICT OF THROUGH
PATIENTS WITH JOB'S AND LUPUS,
IT WAS IN ALL JOB'S PATIENTS,
SO, WHAT'S GOING ON?
TURNS OUT WHEN YOU ACTIVATE YOUR
STAT 3, YOU END UP PRODUCING
THIS MOLECULE CALLED SOX3, WHICH
IS ABLE TO BLOCK THE SIGNALING
OF INTERFERONS BUT BECAUSE THESE
PATIENTS DO NOT HAVE AN
ACTIVATION, DO NOT HAVE A
FUNCTION AT STAT 3, THEY DO NOT
PRODUCE ENOUGH SOX 3 AND THUS
THE PATHWAYS UPREGULATED LEADING
TO INFLAMMATION.
SO WHAT DO WE DO WITH THESE
PATIENTS WHO HAVE THESE IMMUNO
DEFICIENCY, THEY HAVE INFECTIONS
AND THEY HAVE SEVERE LUPUS IN
ADDITION THEY HAVEN'T ACTIVATED
IMMUNE--INNATE IMMUNE SYSTEM.
NOW I MENTIONED WE HAVE A DRUG
NOW THAT IS ABLE TO BLOCK THE
ACTIVATED INNATE IMMUNE SYSTEM
THAT CLASS OF DRUG IS CALLED
JACK INHIBITORS AND HERE WE
HAVE, A FAIRLY HOMOGENOUS GROUP
OF PATIENTS WITH THE SINGLE GENE
MUTATION THAT IS WITH LUPUS AND
WE HAVE A TARGETED DRUG THAT CAN
TARGET THE PATHWAY THAT'S
AFFECTED IN THESE PATIENTS AND
WE'VE STUDIED THIS DRUG IN OUR
PATIENTS WITH LUPUS, SO NOW WE
HAVE A CLINICAL PROTOCOL GOING
WITH OUR COLLABORATORS AT NAID,
WHERE WE WILL BE TREATING
PATIENTS WITH JOB'S SYNDROME AD
JACK INHIBITORS AND IN
COLLABORATION WITH DERMATOLOGY
COLLEAGUES WE WILL LOOK AT HOW
THEY DO CLINICALLY AS WELL AS
DOING EXTENSIVE MECHANISTIC
STUDIESAs WELL AS LOOKING AT
TISSUE LEVEL AND WHAT'S GOING ON
IN THEIR SKIN .
SO LASTLY, WITH THIS
COLLABORATION WITH NIAID, NOW
WHAT WE DO IS NOT JUST LOOK AT
THE STAT 3 LOSS OF FUNCTION
PATIENTS WITH JOBS, WE NOW HAVE
ACCESS TO RARE ERDISEASES LIKE
PATIENT WITH STAT 3 GAIN OF
FUNCTION EMPLOY WE HAVE PATIENTS
WITH STAT 1 GAIN OF FUNCTION,
ANOTHER RARE DISEASE AND A AT
THAT TIME 1 LOSS OF FUNCTION AND
BY STUDYING THESE PATIENTS WITH
THESE RARE MONOGENIC DISEASES,
WHAT WE'RE REALLY TRYING TO
SUNDAY HOW DOES THE JACK STAT
PATHWAY WORK?
HOW DO STAT 1 AND 3, THESE 2
MOLECULES TALK TO EACH OTHER?
AND WHAT CAN WE LEARN ABOUT OUR
DISEASE WITH THAT?
AND LASTLY, I WOULD BE REMISS BY
NOT MENTIONING THAT THIS AMAZING
COLLABORATION, ANOTHER AMAZING
COLLABORATION FROM SO MANY
PEOPLE HERE BETWEEN NIAMS, AND
WE ARE ABLE TO DO WHOLE GENOME
SEQUENCING ON OUR LUPUS PATIENTS
AND WE ARE FINDING VARIANTS IN
THE JACK STAT PATHWAY IN SOME OF
OUR PATIENTS, WHAT THIS MEAN
SYSTEM PERHAPS WE MAY BE ABLE TO
IDENTIFY PATIENTS WHO WOULD
RESPOND BETTER TO THESE TARGETED
THERAPIES THAT WE HAVE.
AND THE GOAL OF THE IDEA HERE IS
EARLY IDENT IBZ OF LUPUS SO WE
CAN ACT SOONER AS WELL AS
DEVELOPING TARGETED THERAPY FOR
ALL OUR PATIENTS AND WITH WITH
THAT I WILL LIKE TO THANK YOU
FOR YOUR ATTENTION.
[ APPLAUSE ]
>> PLEASE WELCOME OUR NEXT
SPEAKER.
>> THANK YOU SO MUCH AND THANK
YOU FOR THE INVITATION TO SPEAK
TODAY SO I HAVE THE PLEASURE OF
SERVING AS THE VICE PRESIDENT OF
RESEARCH AT THE LUPUS FOUNDATION
OF AMERICA AND TODAY I WILL TALK
ABOUT HEALTH DISPARITIES AND
LUPUS IN THE ROLE OF SOCIAL
DETERMINANTS OF HEALTH.
THESE ARE MY DISCLOSURES, AGAIN,
THE OBJECTIVES FOR TODAY ARE
HERE AND SO WE KNOW THERE'S BEEN
SOME CONTROVERSY AS RELATES TO
THE EPIDEMIOLOGY OF LUPUS BUT
BASED ON THE LATEST DATA ISSUE
THE CDC SAID THAT ABOUT 200,000
PEOPLE WITHIN THE U.S.
POPULATION ARE LIVING WITH
SYSTEMIC LUPUS, THERE ARE
SEVERAL STUDIES THAT MIGHT
SUGGEST OTHERWISE BUT REGARLESS
WE KNOW THAT THERE IS A HIGHER
PREVALENCE OF LUPUS IN CERTAIN
POPULATIONS SPECIFICALLY IN
AMERICAN INDIAN AND ALASKA
NATIVE POPULATIONS AS WELL AS
HISPANIC POPULATIONS THERE'S 2-3
TIMES GREATER USE
PREVENTIVEALATION, AND OTHERS
TODAY THAT MINORITIES TOAPPED
HAVE A HIGHER BURDEN OF LUPUS
AND MORE SEVERE DISEASE, BUT WE
REALLY DON'T KNOW WHY THAT
OCCURS.
AND JUDGE UOF THE TO TAKE A
DEEPER DIVE INTO THAT, WHEN
LOOKING AT LUPUS NEPHRITIS, WE
KNOW THAT AMONG U.S. MEDICAID
RECIPIENTS LEAPUS NEPHRITIS IS 4
TIMES MORE COMMON IN BLACK THAN
WHITE PEOPLE LIVING WITH LUPUS,
HISPANIC PATIENTS IN THE
CALIFORNIA REGISTRY WERE SHOWN
TO HAVE 3.5 HIGHER FOLD KIDNEY
INVOLVEMENT COMPARE WIDE WHITE
PATIENTS AND IN THE GEORGIA
LUPUS REG TREE, NEWLY DIAGNOSED
BLACK PATIENTS HAVE A HIGHER
INSDEN OF DEVELOPING END STAGE
RENAL DEC THAN WHITE PATIENTS.
AND OVER ALL END STAGE RENAL
DISEASE BECAUSE OF LUPUS
NEPHRITIS HAS BEEN MUCH GREATER
IN BLACK, AMERICAN INDIAN,
ALASKAINATE AND I HAVE HIS
HISPANIC POPULATION COMPARED TO
WHITE AND ASIAN POPULATIONS.
IN ADDITION TO THAT WHEN WE
ELECTRIC AT OVER AWE
TRANSPLANTATION, BLACK HISPANIC
AND MEDICAID PATIENTS WITH END
STAGE RENAL DISEASE ARE LESS
LIKELY TO BE PLACED ON A KIDNEY
TRANSPLANT WAIT LIST, SO THAT'S
NOT JUST ALL FOR LUPUS, THAT'S
ALL STAGE RENAL DISEASE AND SO
WHEN WE LOOK AT PARAMETERS AND
DATA AS IT RELATES TO GET RENAL
TRANSPLANTATION WE LOOK AT
MEDICAID DATA AND WHAT IT SHOWED
WAS WHEN WE THINK ABOUT
INCOMPLETE EVALUATIONS AND WHO'S
BEING EVALUATED FOR RENAL
TRANSPLANT ISSUES THE
DIFFERENCES ARE STRIKING BETWEEN
BLACK AND WHITE POPULATIONS IN
ADDITION TO THAT, THERE'S A
LOWER POPULATION OF REFERRALS
AND AS WELL AS PATIENTS ON THE
TRANSPLANT LIST AND THERE'S A
HUGE DIFFERENCE IN WHO'S
ACTUALLY ABLE TO UNDERGO RENAL
TRANSPLABTAATION IN BLACK AND
WHITE POPULATIONS SO THIS IS A
HUGE ISSUE WITH YOU KNOW MANY
DIFFERENT CAUSES THAT WE CAN'T
GET IN TODAY DUE TO TIME.
THE MOST SHOCKING INFORMATION IS
LOOKING AT DEATH RATES, SO WE
SEE THAT BLACKS ARE 4 TIMES MORE
LIKELY TO DIE FROM LUPUS THAN
WHITES AND WE ALSO KNOW THAT
LUPUS IS A TOP FIFTH OR SIXTH
CAUSE OF DEATH IN BLACK AND
HISPANIC POPULATIONS WHO ARE
AGES 15-44 AND IN ADDITION TO
THAT THE GEORGIA LUPUS REGISTRY
HAS SHOWN THAT BLACK WOMEN
LIVING WITH LUPUS DIE 13 YEARS
EARLIER THAN WHITE WOMEN LIVING
WITH THE SAME DEC EMPLOY I
SUBMIT TO YOU THAT HOUSTON OR
NIH, WE HAVE A PROBLEM.
SO WHEN WE THINK ABOUT SOLVING
THIS ISSUE, THE LUPUS FOUNDATION
OF AMERICA HAS COLLABORATED AND
FORMED A GROUP OF EXPERTS UNDER
THE GUISE OF A PROJECT CALLED
LUPUS AIM WHERE WE'RE REALLY
STUDYING THIS OUT AND LOOKING AT
HOW WE MIGHT PUT FORTH A
SOLUTION TO THE COMMUNITY TO
HELP SOLVE THIS PROBLEM.
SO THE FIRST THING WE HAD TO DO
WAS DEVELOP A FRAMEWORK IN WHICH
TO LOOK AT THIS AND SO WE
ADAPTED A FRAMEWORK THAT WAS
ORIGINALLY DEVELOPED BY THE
WORLD HEALTH ORGANIZATION.
AND SO WHEN WE THINK ABOUT THIS
ISSUE, WE LOOK AT STRUCTURAL
DETERMINANTS OF HEALTH, I WANT
TO HEIGHT LIGHT THAT RACIAL
DISCRIMINATION AND REALLY HUGE
FOR THOSE WHO ARE MARGINALIZED
IN COMMUNITIES WITHIN THE U.S.
AND IT HAS BEEN SHOWN THERE ARE
LINKAGES BETWEEN RACISM AND
HEALTH OUTCOMES WITHIN THE
UNITED STATES EMPLOY STUDY VS
SHOWN THAT RACISM ADVERSELY
IMPACTS LUPUS ACTIVITY ASK ORGAN
DAMAGE IN GEORGIA IN WOMEN WITH
LUPUS AND ANTICIPATORY RACISM
THAT YOU THINK YOU WILL
ENCOUNTER, JUST ANTICIPATING IT
HAS BEEN LINKED TO GREATER LUPUS
DISEASE ACTIVITY AS WELL.
IN ADDITION TO THAT AND I
APOLOGIZE BECAUSE THIS IS NOT
SHOWING UP APPROPRIATELY ON
SCREEN BUT ECONOMIC INSTABILITY
IS ALSO LINKED TO RACIAL ETHNIC
DISPARITIES, NO SURPRISE THERE,
RIGHT IN SO WE KNOW THAT LOWER
SOCIOECONOMIC STATUS OR
FUNCTIONING OR HIGHY DEPRESSION
RATES IN LUPUS, THE GOAL STUDY
SHOWED THAT LUPUS PATIENTS FOR
MINORITY GROUPS WITH THE LOWER
POVERTY INCOME RATIO FOR
PHYSICAL HEALTH AND BLACK PEOPLE
WITH LUPUS ARE TWICE AS LIKELY
TO EXPERIENCE JOB LOSS COMPARED
TO WHITES WHICH CAN LEAD TO
ECONOMIC INSTABILITY AND AS WELL
AS POVERTY AND STUDY VS ALSO
SHOWN THAT POVERTY INCREASES
DAMAGE ACCUMULATION IN SLE,
IRRESPECTIVE OF RACE/ETHNICITY.
IF YOU TURN YOUR ATTENTION TO
THE GRAPH ON THE RIGHT, THIS
SHOWS DIFFERENCES IN POPERTY
RATES IN U.S.
SO BASED ON THAT WE KNOW THE
NUMBER OF PEOPLE LIVING IN
POVERTY HAS DRASTICALLY DECLINED
BUT BLACK PEOPLE STILL TEND TO
HAVE THE HIGHEST RATES OF
POVERTY AND THIS IS LIKELY 1
CONTRIBUTOR TO DISPARITIES THAT
WE SEE IN THE U.S.
IN ADDITION TO THAT, WHEN WE
LOOK AT OVERALL NET WORTH WITHIN
THE UNITED STATES, WE SEE THAT
THE OTHER NONHISPANIC GROUP IN
RED HERE, AND I DON'T KNOW WHERE
THE POINTER IS BUT TYPICALLY
WHEN WE SEE THAT IN THE LITTURE
THAT'S THE POPULATION SO THAT
FAR EBS SEEDS EVERYONE ELSE
WITHIN THE UNITED STATES AND THE
BLUE LINE IS WHITES, THE GREEN
LINE IS HISPANIC LATINO
POPULATIONS AND AGAIN BLACK
POPULATIONS IS IN ORANGE.
AND THEN WHEN WE LOOK AT THE
OVERALL WELT EQUALITY COENTIOUS
FICIENT HERE WHAT WE SEE IS THAT
TYPICALLY IF THERE WERE SIMILAR
INCOMES ACROSS DIFFERENT
POPULATIONS THAT LINE WOULD BE
FLAT BUT HERE WE SEE THAT IT HAS
CONSISTENTLY GONE UP OVER THE
YEARS FROM 1989 TO 2019, AND SO
THIS TELLS US THAT THERE'S A
HUGE WEALTH GAP IN THE UNITED
STATES WHERE ABOUT .1% OF THE
POPULATION OWNS ABOUT 90% OF THE
WEALTH THAT THE REST OF THE
POPULATION HAS.
IN ADDITION TO THAT WE KNOW THAT
PLACE MATTERS SO PEOPLE WHO LIVE
IN AREAS WHERE THERE ARE HIGH
LEVELS OF POVERTY TEND TO HAVE
POOR OUTCOMES AND THERE IS TRUE
FOR LUPUS, THERE ARE STUDIES
DONE BY ED YELLEN THAT HAVE
SHOWN THAT PEOPLE WHO
[INDISCERNIBLE] LUPUS AND HAVE
LESS WORSENING OF THEIR DEC AS
THEY EXIT POVERTY SO, THIS IS
IMPORTANT TO THINK ABOUT WHEN WE
THINK ABOUT WAYS TO ELIMINATE
HEGHT DISPARITIES.
IN ARK DITION TO THAT WE KNOW
THERE ARE HUGE ISSUES AROUND
HEALTHCARE ACCESS AND QUALITY AS
WELL AS YOU KNOW ISSUES AROUND
BEING ABLE TO AFFORD AND YOU
KNOW UTILIZE THE HEALTHCARE
SYSTEM IN AN INADEQUATE WAY, LOW
SOCIOECONOMIC STATUS IS
ASSOCIATE WIDE HIGHER OVERALL
HEALTHCARE COSTS, THERE IS
VARIABILITY IN HEALTHCARE COST
DEPENDING ON THE TYPE OF
DEMOCRACY THE PERSON HAS
AND--DIAGNOSE THE PERSON HAS AND
INSURANCE THEY HAVE, TOTAL COSTS
ARE NOTABLILY HIGHER IN MAID
CADE AND PATES WITH LUPUS ARE
TWICE AS LIKELY TO REPORT COST
RELATED PRESCRIPTION
NONADHERENCE AS A STRATEGY TO
ECONOMIZE SO THEY'RE TRYING TO
SAVE MONEY BECAUSE THEIR
MEDICATIONS COST SO MUCH.
STUDIES HAVE ALSO DEMONSTRATE
THAD THOSE WITH LUPUS NEPHRITIS
ESPECIALLY FROM THE ETHNIC
MINORITY GROUPS ARE MORE LIKELY
TO RECEIVE INADEQUATE END STAGE
RENAL DEC WHICH COULD
POTENTIALLY LEAD TO WORSENING
OUTCOMES.
AND AGAIN, THERE ARE ISSUES
AROUND ACCESSIBILITY TO CARE AS
WELL.
AND OTHERS HAVE ALREADY
MENTIONED THIS BUT LUPUS
CLINICAL TRIAL PARTICIPATION IS
A HUGE ISSUE.
SIMPLY BECAUSE IF THERE ARE
MEDICATIONS BEING DEVELOPED THAT
DON'T NECESSARILY TREAT ALL OF
THE POPULATION, THEN WE'LL
CONTINUE TO SEE HEALTH
DISPARITIES AND I WON'T GO
THROUGH THIS BECAUSE IT'S
ALREADY BEEN MENTIONED BUT THE
POINT IS, WE NEED MORE DIVERSITY
IN CLINICAL TRIALS IN ORDER TO
LEVEL THE PLAYING FIELD.
SO ANYTHING BACK TO THIS
FRAMEWORK, AND LOOKING AT
ADDRESSING THESE ISSUES WE LOOK
AT SOCIAL DETERMINANTS OF HEALTH
AND SO WE PULL TOGETHER A TEAM
OF PEOPLE WHO HAVE EXPERTISE IN
THIS AREA, WE ALSO INTERVIEWED
PATIENTS WITH LUPUS AS WELL AS A
LEADERSHIP TEAM WITHIN THE LUPUS
FOUNDATION OF AMERICA TO REALLY
UNDERSTAND THEIR OPINIONS ABOUT
WHAT NEEDS TO BE DONE IN ORDER
TO ELIMINATE HEALTH DISPARITIES
IN LUPUS AND SO WE ENGAGE IN A
NUMBER OF DELPH I EXERCISES TO
GET AT THE CRUX OF THIS ISSUE
AND THIS IS JUST A SAMPLE OF THE
DATA WE COLLECTED THIS FAR BUT
THE MAIN THING I WANT TO POINT
OUT HERE IS THAT'S FIZZLED UP TO
THE TOP IS FINANCIAL RESOURCES.
BOTH PATIENTS, LEADERSHIP WITHIN
THE LFA AND EXPERTS WITHIN THE
FIELD AGREE THAT YOU KNOW, A BIG
PIECE OF THE PROBLEM IS FINANCE
OR A LACK THEREOF.
IN ADDITION TO THAT, LOOKING AT
THIS ISSUE MORE BROADLY,
THINKING ABOUT STRUCTURALLY
YOUAL DETERMINE NABTS OF HEALTH
AND WHAT CAN BE DONE, POLICIES
HAVE TO CHANGE, RIGHT IN SO
POLICIES NEED TO BE CREATE THAD
ADDRESS ECONOMIC STABILITY AND
SOCIAL AND COMMUNITY CONTEXT, AS
WELL AS HEALTHCARE ACCESS AND
QUALITY AND ADDRESS ISSUES
AROUND NEIGHBORHOOD AND BUILD
ENVIRONMENT.
CAN WITH THAT I WOULD LIKE TO
THANK THE ORGANIZERS FOR THE
INVITATION AS WELL AS
CONTRIBUTORS FOR THE WORK WE'RE
DOING AND ALL THE PARTNERS WE
HAVE THAT FUND THE WORK WE DO AT
THE FOUNDATION AND WITH THAT I
WILL TAKE ANY QUESTIONS.
[ APPLAUSE ]
>> THANK YOU DR. BOWIE.
NEXT WE HAVE COMING UP ISOTOPA
BELLA OCHOA AND EMILY JONES WHO
ARE PATIENT COORDINATORS AND
THEY WILL BE INTERVIEWING MISS
MARIA ESPINOW.
PLEASE GIVE THEM A WARM WELCOME.
[SPEAKING FOREIGN LANGUAGE ]
>> WE SHARE EVERYTHING, WE SHARE
EVERYTHING IN THE OFFICE, SHE
SHARE THE ROLES, DUTIES, SHARE
THE PATIENTS, SO WHY NOT SHARE
THE MICROPHONE.
[SPEAKING FOREIGN LANGUAGE ]
>> TRANSLATOR: HI MARIA WE WERE
WONDERING IF YOU COULD TELL US
ABOUT YOUR DIAGNOSIS AND HOW
YOU'RE FEELING CURRENTLY.
>> [SPEAKING FOREIGN LANGUAGE ]
>> TRANSLATOR: THANK YOU FOR
INVITING ME AND HAVING ME HERE.
I WAS
>> [SPEAKING FOREIGN LANGUAGE ]
DIAGNOSED IN 2010.
>> [SPEAKING FOREIGN LANGUAGE ]
NCAN IT WAS BECAUSE I HAD
>> [SPEAKING FOREIGN LANGUAGE ]
A
DENTAL EMERGENCY.
>> [SPEAKING FOREIGN LANGUAGE ]
>> I WENT TO A
>> [SPEAKING FOREIGN LANGUAGE ]
CATHOLIC CHARITY
CLINIC.
>> [SPEAKING FOREIGN LANGUAGE ]
>> TRANSLATOR: AND THE DENTIST
NOTICED A MARK
>> [SPEAKING FOREIGN LANGUAGE ]
ON MY FACE.
>> [SPEAKING FOREIGN LANGUAGE ]
>> ASK HE ASKED ASKED ME IF I D
LUPUS AND I TOLD HIM THAT I DID
>> [SPEAKING FOREIGN LANGUAGE ]
NOT KNOW.
>> [SPEAKING FOREIGN LANGUAGE ]
NSO
>> TRANSLATOR: SO HE DIDN'T
WANT TO ATTEND ME AND HE CALLED
THE PRIMARY CARE PHYSICIAN AND
THEY TOOK
>> [SPEAKING FOREIGN LANGUAGE ]
A BLOOD DRAW.
>> [SPEAKING FOREIGN LANGUAGE ]
>> A MONTH LATER A GOT A PHONE
ALL LETTING ME KNOW THAT I
TESTED
>> [SPEAKING FOREIGN LANGUAGE ]
POSITIVE FOR LUPUS.
>> [SPEAKING FOREIGN LANGUAGE ]
>> AND THAT'S HOW I FOUND OUT
THAT I HAD A DISEASE THAT I KNEW
PRACTICALLY KNEW NOTHING
>> [SPEAKING FOREIGN LANGUAGE ]
ABOUT.
>> WOW.
>> [SPEAKING FOREIGN LANGUAGE ]
>> TRANSLATOR: NOT THE BEST
NEWS YOU
>> [SPEAKING FOREIGN LANGUAGE ]
WANT TO RECEIVE.
>> [SPEAKING FOREIGN LANGUAGE ]
>> TRANSLATOR: I WANTED TO ASK
WHAT MOTIVATED YOU TO
PARTICIPATE
>> [SPEAKING FOREIGN LANGUAGE ]
IN CLINICAL STUDIES?
>> [SPEAKING FOREIGN LANGUAGE ]
>> TRANSLATOR: WELL, FIRSTLY I
HAVE TO SAY HOW I GOT TO NIH.
THE CATHOLIC CHARITY CLINIC ARE
THE 1S WHO SPOKE TO
>> [SPEAKING FOREIGN LANGUAGE ]
ME ABOUT
NIH.
>> [SPEAKING FOREIGN LANGUAGE ]
>> TRANSLATOR: AND THANK GOT AT
THAT TIME NIH WAS LOOKING FOR
PATIENTS THAT WERE HISPANIC,
DIAGNOSED IF
>> [SPEAKING FOREIGN LANGUAGE ]
LUPUS AND FEMALE.
>> [SPEAKING FOREIGN LANGUAGE ]
>> TRANSLATOR: SO THEY WERE THE
1 WHO IS SUBMITTED MY CASE TO
NIH AND
>> [SPEAKING FOREIGN LANGUAGE ]
I WAS APPROVED.
>> [SPEAKING FOREIGN LANGUAGE ]
>> TRANSLATOR: AND I'VE BEEN A
PATIENT AT NIH SINCE THEN AND
THAT
>> [SPEAKING FOREIGN LANGUAGE ]
WAS IN 2010.
>> [SPEAKING FOREIGN LANGUAGE ]
>> TRANSLATOR: AT THE BEGINNING
I DIDN'T PARTICIPATE IN ANY
STUDIES BECAUSE MY DISEASE WAS
VERY--IT WASN'T DIAGNOSED FOR A
LONG TIME AND IT WAS REALLY
SEVERE AT
>> [SPEAKING FOREIGN LANGUAGE ]
THE TIME.
>> [SPEAKING FOREIGN LANGUAGE ]
>> TRANSLATOR: AND THE DOCTORS
AT NIH TOLD ME I COULD DIE AT
ANY MOMENT.
SO THEY GAVE ME A VERY
AGGRESSIVE TREATMENT.
WHEN I STARTED TO GET BETTER
THAT'S WHEN I WAS INVITED TO
PARTICIPATE IN
>> [SPEAKING FOREIGN LANGUAGE ]
THE CLINICAL
TRIALS.
>> [SPEAKING FOREIGN LANGUAGE ]
>> TRANSLATOR: DID YOU EVER
FEEL OBLIGATED OR WERE YOU
INFLUENCED TO PARTICIPATE IN ANY
OF THE CLINICAL TRIALS AND THE
RESPONSE WAS NO, I DID IT ON MY
OWN BECAUSE
>> [SPEAKING FOREIGN LANGUAGE ]
I WANTED TO.
>> [SPEAKING FOREIGN LANGUAGE ]
>> TRANSLATOR: THE BENEFIT OF
COMING TO NIH IS THAT YOU GET
EXPOSED TO OTHER MEDICAL TEAMS
THAT CAN HELP YOU WITH OTHER
ISSUES THAT MIGHT BE RELATED TO
LUPUS.
>> NO, NO, NO, WHAT I WANT TO
SAY IS THAT BECAUSE I
PARTICIPATED IN DIFFERENT
TRIALS, I WAS EXPOSED TO OTHER
TEAM MEDICAL SPECIALTIES THAT
IMPROVE MY HEALTH.
ONE EXAMPLE OF THAT, I MEAN,
[SPEAKING FOREIGN LANGUAGE ]
>> TRANSLATOR: AN EXAMPLE OF
THAT IS THAT I WAS HAVING A LOT
OF ISSUES WITH THE DETERIORATION
OF MUSCLE IN MY LEGS SO I WAS
CONNECTED WITH PHYSICAL THERAPY
AND THEY PROVIDED WITH INSOLES
FOR MY FEET AND IT HELPED A LOT
WITH THE PAIN I WAS HAVING IN MY
LEGS.
>> [SPEAKING FOREIGN LANGUAGE ]
>> TRANSLATOR: HOW HAS YOUR
EXPERIENCE BEEN HERE AT NIH BUT
IN ALL HONESTY.
>> OF COURSE!
[SPEAKING FOREIGN LANGUAGE ]
>> TRANSLATOR.
WELL, FIRSTLY THE PATIENT CARE
IS ALWAYS THE FIRST THING SO
IT'S A HUGE BENEFIT
>> [SPEAKING FOREIGN LANGUAGE ]
TO THE
PATIENTS.
>> [SPEAKING FOREIGN LANGUAGE ]
>> TRANSLATOR: BECAUSE WHEN YOU
HEAR THE WORD STUDY, YOU THINK
YOU WILL BE A LAB RAT AND THAT
HAS NOT
>> [SPEAKING FOREIGN LANGUAGE ]
BEEN MY EXPERIENCE.
>> [SPEAKING FOREIGN LANGUAGE ]
>> TRANSLATOR.
I REMEMBER AT 1 POINT I WAS
TAKING A PLACEBO AND A
MEDICATION THAT HAS SOMETHING TO
DO WITH THE VASCULAR SYSTEM AND
I STARTED
>> [SPEAKING FOREIGN LANGUAGE ]
TO GAIN WEIGHT.
>> [SPEAKING FOREIGN LANGUAGE ]
>> TRANSLATOR: AS SOON AS I
INFORMED THE TEAM ABOUT THAT,
MAY IMMEDIATELY STOPPED THE
MEDICATION SO I WOULD STOP WITH
>> [SPEAKING FOREIGN LANGUAGE ]
THE WEIGHT GAIN.
>> [SPEAKING FOREIGN LANGUAGE ]
>> TRANSLATOR: AND THAT SHOWED
ME THAT THEY CARED MORE ABOUT ME
AS A PERSON THAN
>> [SPEAKING FOREIGN LANGUAGE ]
THE STUDY.
>> [SPEAKING FOREIGN LANGUAGE ]
>> TRANSLATOR: IS THERE
ANYTHING ELSE THAT YOU WOULD
LIKE TO SHARE
>> [SPEAKING FOREIGN LANGUAGE ]
WITH EVERYONE?
>> [SPEAKING FOREIGN LANGUAGE ]
>> TRANSLATOR.
SO I HAVE A REALLY FUNNY
ANECDOTE.
NTRANSLATEOR TRANSLATE--
>> TRANSLATOR: I PARTICIPATED
IN A CONFERENCE HERE, AND I WENT
TO THE RESTROOM IN THAT HOTEL
WHERE THE CONFERENCE WAS AND I
TOOK AN UNEXPECTED INVITER OR
PERSON WITH ME WHICH WAS A TICK.
I NOTICED IT AT HOME ONCE I GOT
BACK.
AND SINCE THAT TICK ALSO CAN
CAUSE LYME DISEASE, I GOT REALY
SCARED.
>> SO I CALLED THE TEAM AND I
>> [SPEAKING FOREIGN LANGUAGE ]
SPOKE WITH THEM.
>> [SPEAKING FOREIGN LANGUAGE ]
>> TRANSLATOR.: THIS IS THE LAT
THING I NEED IS LYME DISEASE,
BUT ALSO I WAS DIAGNOSED WITH
SOJGREN'S DISEASE, SO I FORCED
THE TEAM TO SEE ME AND REMOVE
THE TICK.
BUT THE FUNNY THING IS THAT THE
DAY THAT I
>> [SPEAKING FOREIGN LANGUAGE ]
CAME FOR THAT
>> [SPEAKING FOREIGN LANGUAGE ]
>> TRANSLATOR: THE DOCTOR THAT
SPECIALIZES AND STUDIES TICKS
JUST SO HAPPENED TO BE HERE THAT
DAY.
SO 5 DOCTORS IN THEIR ROBES AND
7 NURSES CAME TO SEE ME TO
REMOVE THE TICK.
AND WHEN THEY STUDIED IT, THAT
DOCTOR DID NOT KNOW THAT SPECIES
OF TICK.
SO I CONTRIBUTED WITH SCIENCE.
[
>> [SPEAKING FOREIGN LANGUAGE ]
APPLAUSE ]
[LAUGHTER]
>> [SPEAKING FOREIGN LANGUAGE ]
>> TRANSLATOR: SHE SAID THERE'S
A TICK OUT THERE SOMEWHERE WITH
MY NAME ON IT.
ANY QUESTIONS IN QUESTIONS?
>> OKAY, THANK YOU VERY MUCH:
>> THANK YOU MISS MARIA ESPINALL
FOR SHARING YOUR STORY.
NEXT WE HAVE DR. JAMES
[INDISCERNIBLE] SENIOR
INVESTIGATOR AT NCI AND HE WILL
BE PRESENTING ON CAR T THERAPY
AND REFRACTORY LUPUS NEPHRITIS
WERE AT THE NIH.
PLEASE GIVE HIM A WARM WELCOME.
[ APPLAUSE ]
>> OKAY, THANKS VERY MUCH FOR
THE ORGANIZERS FOR INVITING ME
TO SPEAK TODAY AND IT'S A GREAT
HONOR TO PEEK TO THIS GROUP IN
THIS VERY NEW AREA OF LUPUS
RESEARCH USING CAR T CELLS.
SO FIRST OF ALL BECAUSE
RHEUMATOLOGY AND OTHER
AUTOIMMUNE FIELDS ARE KIND OF
NEW TO THE USE OF CAR T-CELLS, I
PUT IN A SLIDE ABOUT WHAT IS A
CAR, A CAR STANDS FOR CHIMERIC
ANTIGEN RECEPTOR, THESE ARE
FUSION PROTEINS THAT INCLUDE
ANTIIEN RECOGNITION MODEL
CITIZENETY WHICH IS USUALLY
DERIVED FROM A MONOCLONAL
ANTIBODY, HINGE AND
TRANSMEMBRANE DOMAIN AND THEN CO
STIMLATTORY DOMAINS WHICH ARE
USUALLY CD28 OR 41 BB, WHICH ARE
MOLECULES THAT HELP ACTIVATE
T-CELLS, AND THEN A MAIN T-CELL
ACTIVATION DOMAIN WHICH IS A
DOMAIN SUCH AS CD3 ZETA HA IS
USUALLY THE MAIN MECHANISM BY
WHICH ALL OF OUR NORMAL T-CELLS
ARE ACTIVATED.
SO THE IDEA OF THAT THING AT THE
TOP IS TO HAVE THIS STRUCTURE
WHICH IS AN ARTIFICIAL STRUCTURE
TO PUT THAT INTO THE PATIENTS
T-CELLS SO THAT THE T-CELLS CAN
THEN RECOGNIZE SOMETHING THEY
WILL NOT NORMALLY RECOGNIZE.
THIS IS DONE BY GENE THERAPY
USING A VECTOR WHICH IS USUALLY
A VIRUS BUT THERE ARE OTHER
METHODS TO DO THIS WHICH ARE
COMING ONLINE NOW.
AND THIS CAR STRUCTURE IS
ENCODED BY DNA, ENCODED BY THE
VECTOR, THE VECTOR IS USED TO
PUT THE CAR GENE INTO THE
PATIENT'S T-CELLS AND THEN THE
PATIENT'S OWN LIVING T-CELLS
EXPRESS THE CAR WHICH GIVES THE
PATIENTS CELLS THE ABILITY TO
RECOGNIZE SOME TARGET SELECTED
BY THE PEOPLE GENETICALLY
NRNLING THEIR T-CELLS, IN THIS
CASE WE'RE SHOWING A CAR AGAINST
CD19.
SO, I'M GOING TO SHOW SOME
RESULTS, WE ONLY HAVE 10 MINUTES
CROSS LINKINGY THE REASON WHY
PEOPLE ARE SO EXCITED ABOUT CAR
FOR LUPUS IS MAINLY BECAUSE OF
THE WORK OF AT LEAST INITIALLY
THE WORK OF DOCTORS IN GERMANY.
LED BY GEORGE SCHETT, HIS NAME
IS LAST IN THE PAPER ON
MEDICINE.
HE TREATED BY 5 REFRACTORY SLE
PATIENTS.
ALL SUBYEBS HAD CLINICAL
REMISSION WITHIN 3 MONTHS.
ONLY GREAT 1 CYTOKINE RELEASE
SYNDROME WHICH IS VERY, MINOR
TOXICITY OF CAR T-CELLS WITH
PEASHTS JUST HAD FEVERS
OCCURRED.
SO WHY WOULD PEOPLE USE CAR
T-CELLS TO TREAT LUPUS?
IT'S BECAUSE PEOPLE HAVE
DETERMINED THAT B-CELLS ARE VERY
IMPORTANT FOR THE PATHOGENESIS
OF LUPUS.
SO B-CELLS ARE AT LEAST A BIG
PART OF THE CAUSE OF LUPUS.
AND THE ANTICD19 CAR T-CELLS ARE
CAN BASICALLY KILL THE B-CELLS.
SO APPROXIMATE IT WE WIPE OUT
THE PATIENT'S B-CELLS, THE LUPUS
SHOULD GET BETTER AND NOBODY
REALLY KNEW THIS WOULD HAPPEN
UNTIL DR. SCHETT'S WORK AND IT
TURNED OUT IT WORKED VERY WELL,
AT LEAST IT LOOKS GREAT.
SO THEY DEPLETED B-CELLS FROM
THESE PATIENTS AND SO FAR THE
PATIENTS HAVE NOT RELAPSED.
AT THE TIME OF THIS REPORT, OR
THE MOST RECENT REPORT, WE HAD
THE FIRST PATIENT WAS 17 MONTHS
IN REMISSION EMPLOY SO THESE
GRAFS SHOW HAD SOME OTHER DATA
CONCERNING LUPUS, FIRST OF ALL,
THE FIRST GRAFT IS THE CAR
T-CELL LEVEL, THE CAR T-CELLS
LIVE IN THE BLOOD OF THE
PATIENTS, THEY GO UP, THE PEEKS
OF THOSE LINES, THEY COME DOWN,
SO THEY DON'T AT A UP THERE
FOREVER, THEY COME DOWN ASK
EEIVETTUALLY GO ARK WAY.
AND THE OTHER GRAPH WELL IS
B-CELLS SO YOU IS SEE THEY
STARTED OUT WITH THE PEASHTS HAD
B-CELLS IN THE BLOOD AND THEN
THE B-CELLS DISAPPEARED BECAUSE
THE CAR T-CELLS ARE GOOD AT
KILLING BASHING CELLS.
THIS IS THE LUPUS ACTIVITY
GRAPH, YOU CAN SEE THE LUPUS
ACTIVITY BY OBJECTIVE MEASURES
WENT DOWN SUBSTANTIALLY IN ALL
PATIENTS EMPLOY --THESE ARE THE
OTHER MEASURES THAT PEOPLE IN
THE ROOM FOR MORE FAMILIAR WITH
THAN ME, BUT PROTEIN URIA, AND
DOUBLE STRAND ANTIBODYINGS ALL
IMPROVE AFTER THE CAR T-CELL
TREATMENT.
THIS IS A COMPLICATED GRAPH, BUT
IT'S BEEN VERY HELPFUL IN
TREATING SYSTEMIC CLEROSEIS,
IN,A DITION TO LUPUS AND NUMBERS
OF PATIENTS ARE VERY LOW, BUT
THERE IS ACTIVITIES ANTICD19 CAR
T-CELLS AND OTHER TYPES OF
AUTOIMMUNITYS ALSO, AND THE
GRAPHS SHOW VARIOUS MEASURES OF
THESE AUTOIMMUNE DECEASES
IMPROVING AFTER CAR T-CELL
TREATMENT, YOU CAN SEE THINGS
LIKE VARIOUS SCALES OF MEASURING
THESEOT IMMUNE ACTIVITY ARE
IMPROVING IN THESE PATIENTS.
SO THIS COMES NOW FOR THE WORK
THAT IS LED BY DR. HOSNI, AT THE
NE H, I'M BASICALLY JUST AN
ADVISOR ON THIS BUT THE REASON
WHY I'M INVOLVED IS THE KYV101
FULLY HUMAN CD19 CAR WAS
DEVELOPED IN MY LAB AND WE
TREATED 20 PATIENTS WITH
LYMPHOMA WITH THIS, SO WE
ORIGINALLY DEVELOPED THIS TO
TREAT MALIGNANCY AND IT'S VERY
EFFECTIVE FOR TREATING LYMPHOMA,
THE CAR IS ALSO KNOWN AS CD1928
Z, THAT'S WITH WE CALL IT WHEN
WE TREAT IMFOAMA AND THE
AUTOIMMUNE SETTING IT'S CALLED
KYV BY THE COMPANY THAT IS,
WOOING ON DEVELOPING THIS, THIS
CAR HAS AN IMPROVED SAFETY
PROFILE, IT'S STRUCTURES SHOWN
OR THERE TO THE LEFT, IT IS A
HUMAN ANTIBODY DERIVED CAR, THE
RECOGNITION DOMAIN FOR ANTICD19
ANTIBODY THAT'S HUMAN, FULLY
HUMAN, IT HAS A CD8 HINGE, CD8
ALPHA TRANSMEMBRANE DOMAIN, A
CD28 CO STIMLATTORY BRAIN AND A
ZETA T-CELL ACTIVATION DOMAIN SO
THOSE ARE KIND OF A VERY
STANDARD KIND OF CAR DESIGN.
THERE'S NOTHING SUPER EXCITING
ABOUT THAT.
AT THE TIME IT WAS TWEPPED BACK
IN 2011 IT WAS MORE EXCITING BA
UNITED STATES IT WAS FULLY HUMAN
IS EVERYTHING, BUT AT THIS
POINT, THIS IS PRETTY STANDARD.
IT IS NOTABLE BECAUSE IT IS LESS
TOXIC THAN PREVIOUS CARS WE USE
TO TREAT MALIGNANCY, AND IT
PRODUCES LYER LEVELS OF
CYTOKINES AND THE CYTOKINES ARE
THE THINGS THAT CAUSE TOXICITY
IN CAR T-CELL PATIENTS.
I WILL TALK MORE ABOUT THAT
LATER, IT'S ALSO PROBABLY LOWER
IN IMMUNO GENERATEDISSITY
BECAUSE IT'S FULLY HUMAN.
APPROXIMATE TERPIOF THES WELL
WITH THIS, AND IT TREATS WELL
WELL ON LYMPHOMA THAT WERE MORE
TOXIC, SO WE TREATED LIKE I SAID
20 PATIENTS.
SO GENERAL POINTS ON CAR T-CELL
OX TOXICITY SINCE VERY FEW
PEOPLE HAVE TREATED PATES WITH
CAR T-CELLS, THEY RELEASE
CYTOKINE RELEASE SYNDROME,
CYTODIRECTOR OF NATIONAL
INSTITUTE RELEASE SYNDROME,
HYPOTENSION AND TACHYCARDIA
AMONG OTHER SIGNS AND SYMPTOMS,
THEY ALSO CAUSE NEUROLOGIC
TAXISITYS AND THE CAR T-CELL
TOXICITIES ARE TREATED WITH THE
COTTERRIC O STEROIDS AND
TOCILIZUMAB.
WE HAD A 55% COMPLETE REMISSION,
RATE, 40% HAD A PROGRESSION FREE
RADICALS SURVIVAL LASTING MORE
THAN 24 MONTHS MEANING THEY WERE
TREATMENT FREE AND LYMPHOMA FREE
AT MORE THAN 24 MONTHS.
THE MOST STRIKING THING ABOUT
THIS STUDY WAS THE LOW LEVEL
NEUROLOGIC TOXICITY, ONLY 5% OF
THE PATIENTS THAT GOT THIS CAR
HAD HAD GRADE 3 OR 4 NEUROLOGIC
TOXICITY, THAT WAS 1 PATIENT
WITH A GRADE 4 NEUROLOGIC EVENT.
THIS IS COMPARED TO THE OLDER
CAR WHERE WE HAD 50% OF THE
PATIENTS HAD GRADER NEUROLOGIC
TOXICITY SO THIS WAS A STRIKING
REDUCTION IN NEUROLOGIC
TOXICITY, HOWEVER, THIS CAR
STILL COULD CAUSE TOXICITY
IBSITY IN LYMPHOMA PATIENTS, WE
HAD GRADE 2 OR GREATER CRS IN 12
OUT OF 20 PASHTS AND THE
LYMPHOMA PORLD WE DON'T WORRY
ABOUT GRADE 2, CRS TOO MUCH,
IT'S JUST FEVERS AND
TACHYCARDIA, AND IT'S USUALLY
TREATED WITH SUPPORTIVE CARE AND
INJECTION OF TOCILIZUMAB, BUT IT
DOES OCCUR AND IT IS TOXICITY WE
NEED TO BE AWARE OF AND
CONCERNED ABOUT.
WE TREAT THE PATIENTS ON THIS
TRIAL, 1 RECEIVED CORDIC O
STEROIDS FOR TOXICITYIT.
ONE RECEIVED TOCILIZUMAB, AND 1
RECEIVED IT FOR COTTERRIC O
STEROIDS FOR CRS.
SO THE TOXICITY CAN BE TREATED.
NOW THE WHOLE POINT OF THIS
WHICH I MENTIONED A COUPLE TIMES
IS THAT THE CAR T-CELLS ARE
INCREDIBLE LOAMACY I GOOD AT
DEPLETING B-CELLS AND THAT OTHER
PEOPLE HAVE SHOWN THAT B-CELLS
ARE REALLY IMPORTANT FOR LUPUS
SO I WANT TO SHOW WHY THESE ARE,
AND WHY THEY'RE DIFFERENT FROM
OTHER TREATMENT AND WE HAVE
EVIDENCE FROM LYMPHOMA PATIENTS
ABOUT THE POWER OF CAR TISSUING
CELLS DEPLETING THE POWER OF THE
B-CELLS.
SO, I INCLUED ON THESE GRAPHS
PATIENTS WITH HU19-CD828 Z
TREATMENT AND HAD 4 MONTHS MUCH
FOLLOW UP AND NO BLOOD LYMPHOMA
INVOLVEMENT, PEASHTS 5, 6, 13,
YOU CAN SEE THESE PATIENTS HAD
B-CELLS IN THEIR BLOOD
PRETREATMENT WITH THE CAR T-CELL
AND THEN THE B-CELL LEVELS ARE
ALL DOWN NEAR 0 CAN THAT GOES
OUT TO 60 MONTHS, SO AT 5 YEARS,
SOME OF THOSE PATIENTS HAVE
B-CELLS, BASICALLY FONNATES OF
THEM HAVE THESELY AND YOU MIGHT
ASK WHY SOME OF THESE PATIENTS
HAVE LOW B-CELLS AT THE
BEGINNING AND IT'S BECAUSE WE
TREAT THEM WITH CHEMOTHERAPY,
AND REDUCES THEIR B-CELLS TO
BEGIN, THE OTHER GRAPH SHOWS
PATIENT OF LESS THAN 2 BLOOD
CELLS PER B-CELL, AND MICROLITER
AND NOT MILLILITER, AND SOME OF
THE PATIENTS HAD B-CELL RECOVERY
BUT SOME OF THEM HAD B-CELL
BEPLETION LEFTING UP TO 5 YEARS.
--LASTING UP TO 5 YEARS SO CAR
T-CELLS ARE GOOD AT DEPLETING
B-CELLS.
WE ARE PLANNING ON OPENING A
STUDY, AGAIN, DR. HOSNI WILL BE
THE PI FOR THE STUDY FOR
TREATING LUPUS NEPHRITIS AT THE
NIH WITH A KYV 101 CAR I'VE BEEN
TALKING ABOUT, SO ELIGIBLE
PATIENTS WILL HAVE TO HAVE A
FORMAL DIAGNOSIS OF SLE, ACTIVE
BIOPSY PROVEN CLASS 3 OR 4, AND
INADEQUATE RESPONSE TO STANDARD
THERAPIES; AND 9 TO 12 PATIENTS
WILL BE ENROLL IN A DOSE
ESCALATION STUDY TO EVALUATE
SAFETY ANDERABILITY, AND EACH
DOSE LEVEL WILL INCLUDE A
MINIMUM OF 3 SUBRECYCLINGS.
SOME OF THESE ARE
MULTISPONSORRED TRIALS SOME OF
THESE STEPS ARE ALREADY UNDERWAY
AND WE HOPE TO OPEN THE NIH ARM
OF THE STUDY VERY SOON, YOU CAN
SEE THERE THAT THE CAR DOSE
LEVELS ARE PAIRLY MODEST
COMPARED TO WHAT WE TESTED
BEFORE.
WE TESTED HIGHER DOSES OF THIS,
IN PATIENTS WITH LYMPHOMA, BUT
THE DOSES OF 0.5 MILLION, 0.5
TIMES 10 TO THE EIGHTH CAR
T-CELLS ARE 1 TO TIMES TO THE
EIGHTH CAR T-CELLS AND NOTABLY
THE CAR T-CELL THERAPY IS
PRECEDED BY CONDITIONING CHEMO
THERAPY OR AND PSYCHOFOSTER
NURSED FOSTER NURSED MADE
BECAUSE THE CHEMO THERAPY
DROOMATICALLY ENHANCES THE
ACTIVITY OF THE CAR T-CELLS.
SO AGAIN, HOW WILL THE STUDY
GENERALLY BE DONE?
PATIENTS WILL BE KREENED.
IF THEY'RE ELIGIBLE TO UNDERGO
APHORESIS, HERE AT THE NOORK H,
AFTER APHORESIS, THE PATIENT
CELLS ARE SHIPPED TO A CENTRAL
MANUFACTURING FACILITY.
IT'S IF PHILADELPHIA, AND AT
THAT PLACE THE COMPANY HAS
ARRANGED TO HAVE THE CELLS
PRODUCED, THE CELLS WILL BE
SHIPPED BACK TO THE NIH FOR THE
INFEEGZ.
DURING THIS TIME OF
MANUFACTURING THE PATIENTS CAN
RESUME LUPUS NEPHRITIS DISEASE
THERAPY IF NEEDED AND AS I
MEKSED WIDE RECEIVER THE CAR
T-CELL INFUSION THEY WILL GET
DEPLETING CONDITIONING CHEMO
THERAPY.
AFTER THEY GO INTO FOR THE
STUDY, THEY WILL BE MONITORED.
SO THE PRIMARY OBJECTIVES ARE
THE PRIMARY END POINTS ARE ARE
END SEVERITY AND LAB RAARE TOY
ABNORMALITIES.
THE FREQUENCY OF DLTs AT EACH
LEVEL WILL BE A PRIMARY END
POINT AND THERE ARE MANY
SECONDARY OBLIGATIONS YECTIVES
ALL AIMED AT MEASURING LUPUS
RESPONSE AND IN ADDITION BASIC
CAR T-CELL MEASURES SUCH AS THE
CAR TISSUING CELL LEVELS IN THE
BLOOD OF THE PATIENTS.
SO, I ACKNOWLEDGE MY COWORKERS
ON THIS, DOCTOR AS I MENTIONED
HOSNI WILL BE THE PUNISHING I,
THE STUDY, THE PHYSICIAN AND NCI
WORKS CLOSELY WITH ME, MOOMG
THIS PROJECT AMONG MANY OTHERS,
ELAINE PONCIO HAS BEEN WORKING
HARD TO GET THIS STUDY OPEN,
THERE ARE VARIOUS PEOPLE IN MY
LAB, NORRIS LAM AND OTHER WHO IS
WORKED ON MAKING THIS CAR, CAN
IN THE DEPARTMENT OF TRANSFUSION
MEDICINE, VERY IMPORTANT
COLLABORATORS WILL WORK ON THIS
STUDY TO HELP US GET THE CAR
T-CELLS MANUFACTURED AND SHIPPED
AND INFUSED INTO THE PATIENT.
AND THEN WE HAVE COLLEAGUES AND
PATHOLOGY WHO HAVE BEEN WORKING
ON THIS PROJECT DOING VERSE
CLOSE CYTOMETRY AND IMMUNO
CHEMISTRY ASSAYS.
THANK YOU VERY MUCH FOR YOUR
ATTENTION.
[ APPLAUSE ]
DINNER.
>> THANK YOU.
THE NEXT SPEAKER IS MISS JUNE B.
CHU, SHE WILL SPEAK ON QUALITY
INDICATOR SAYS FOR LUPUS
PATIENTS AT THE NIH.
PLEASE GIVE HER A WARM WELCOME.
>> HI, EVERYONE.
SO WE'VE COME A LONG WAY OVER
THE LAUGH THIRD YEARS AND
TODAY'S DC LUPUS CONSORTIUM HAS
HIGHLIGHTED AND VALIDATED SOME
OF OUR FREMEDENDOUS PROGRESS.
MY TOPIC TODAY IS ON
IMPLEMENTING QUALITY INDICATORS
FOR LUPUS PATIENTS AT THE NIH.
YOU WILL SEE A LOT OF
DEFINITIONS ON QUALITY
INDICATORS OR QI BUT IN BROAD
TERPS QI IS TO STANDARDIZE AND
IMPROVE HEALTH OUTCOMES AND
LUPUS IS DESIRABLE BECAUSE IT'S
A MULTIORGAN DISEASE WITH VARIED
MAN TESTACEANS.
PATIENTS CAN ACCUMULATE ORGAN
ISSUES OVER TIME.
SO WHAT TYPE OF QUALITY
INDICATORS HAVE WE IMPLEMENTED
HERE AT NIH.
WE DEVELOPED A STANDARDIZED
VISIT NOTE OF COMPREHENSIVE
LUPUS CLINICAL CARE EMPLOY WE'VE
CREATED A HEALTH NATIVE
AMERICANS IN THE NORTHERNINENCE
SECTION IN EACH OF THE
STANDARDIZED LUPUS NOTES, WE
DEVELOPED A SUNSCREEN BROCHURE
TO PROMOTE SUNSCREEN EDUCATION,
WE DEVELOPED LUPUS DISEASE
ACTIVITY SCORING TOOL IN OUR
ELECTRONIC MEDICAL RECORD
SYSTEM, OR CHRIS, AND
IMPLEMENTED REPRODUCTIVE HELT
INITIATIVE AS WELL AND LASTLY WE
WORK CLOSELY WITH THE COMMUNITY
PROVIDERS.
FOR STANDARDIZED NOTES THAT IS A
CLINICAL NOTE WE USE WITH EACH
VISIT WITH WE SEE OUR LUPUS
COHORT THAT INSURES
INTERNATIONALLY RECOGNIZED
QUALITY INDICATORS ARE MET THIS
HELPS WITHSTAND ARDIZEATION OF
CARE AS WELL AS EFFICIENT AND
DATA EXTRACTION FOR RESEARCH
PURPOSES.
AND WITHIN EACH OF THESE
STANDARDIZED LUPUS NOTES WE HAVE
A HEALTH MAINTENANCE SECTION
WHICH ADDRESSES VACCINATIONS,
RECOMMENDED FOR LUPUS PATIENTS,
BONE HEALTH THAT IMPLEMENTS
CALCIUM VITAMIN D USE, BONE
DENSITY SCREENING AND CHECKING
FOR VITAMIN D LEVELS.
WE ALSO LOOK AT AGE RELATED
HEALTH SCREENINGS SUCH AS
MAMMOGRAMS, COLONOSCOPYS
GYNECOLOGY VISITS AND WE ASSESS
FOR CARDIOVASCULAR RISK AS WELL
EMPLOY PHOTO SENSITIVITY IS A
COMMON LUPUS MANIFESTATION, IT'S
IMPORTANT TO HELP DECREASE LUPUS
FLARE, TO PROMOTE AND EDUCATE
SUNSCREEN USE, WE DEVELOPED A
SUNSCREEN BROCHURE WHICH IS IN
YOUR WONDERFUL PURPLE FOLDERS
TODAY, THEY'RE IN BOTH ENGLISH
AND SPANISH TO DISSEMINATE THE
BEST TYPE OF SUNSCREEN THAT
GIVES THE BEST PROTECTION FOR
LUPUS PATIENTS.
FOR DISEASE ACTIVITY STORES WE
ADD THE LUPUS DISEASE ACTIVITY
INDEX FOR THE DEC DAMAGE ACCRUAL
INDEX, PLIP AND FLOABAL
ASSESSMENT TO EMR TO STABBED
ARDIZE AND INCORPORATE NECESSARY
RESEARCH EXTRACTION, FOR
CLINICAL PURPOSES THIS IS
HELPFUL TO TRACK AND TREND LUPUS
DISEASE AND HELPING WITH LUPUS
MANAGEMENT.
ALL OF THESE SCORES ARE
COMPLOATED AFTER EACH PATIENT
VISIT.
OUR REPRODUCTIVE INITIATIVE IS
SOMETHING I'M INTERESTED IN, WE
HAVE IMPLEMENTED HEALTH
REPRODUCTION, WE,A ASSESSES
HEALTH HISTORY, WE ASK FEMALES
OF CHAILD BEARING AGE IN THERE
INTEREST IN THE FIXTURE 12
MONTHS AND DEPENDING ON THEIR
ANSWERS WE GIVE THEM MEDICATIONS
ARE OR DISCUSS RISK FACTORS
DURING PREGNANCIES AND LASTLY WE
WILL WORK VERY CLOSELY WITH A
PATIENT'S MATERNAL FETAL
MEDICINE AND OBGYN EMPLOY HERE
ARE SOME OF THE PEBLICATIONS AND
PRESCRIBING SEBTATIONS THAT
VILLAIN ACCEPTED AT
INTERNATIONAL CONFERENCES WHERE
WE TALK ABOUT IMPLEMENTING LUPUS
QUALITY IMPLICATORS AND QUALITY
REPRODICTIVE HEALTH MEDICINE.
YOU'VE SEEN THIS PICTURE
THROUGHOUT THE DAY, THIS IS OUR
WONDERFUL TEAM.
KUDOS TO EVERYONE WE'RE AND OUR
COMBINED EFFORTS AND IMPROVING
LUPUS CLINICAL RESEARCH AND
THANK AND YOU PLEASE LET ME KNOW
IF YOU HAVE ANY QUESTIONS.
[ APPLAUSE ]
NTHANK YOU JUN.
NEXT WE HAVE MR. MICHAEL DAVIS
NURSE PRACTITIONER, HE WILL
PROVIDE AN OVERSLEW OF THE
[INDISCERNIBLE] STUDY.
PLEASE GIVE HIM A WARM WELCOME.
>> GOOD MORNING.
I NORMALLY WORK BIGGER VENUES,
BUT THIS IS THE FIRST 1 POST
COVID.
[LAUGHTER]
SO.
YOU KNOW.
YOU'RE WELCOME.
I'M BEING SILLY, I AM ACTUALLY
TOTALLY BLOWN AWAY BY THE NUMBER
OF PEOPLE THAT SHOWED UP AND I
WANT TO SAY THANK YOU TO THE
PATIENTS IN PARTICULAR WHO HAVE
SHOWN UP AS WELL AS TO OUR
REFERRING PHYSICIANS AND OUR
COLLABORATORS OUT THERE BECAUSE
IF YOU WERE NOT THERE, WE WOULD
BE SITTING HERE HANGING OUT
BEING GOVERNMENT EMPLOYEES.
[LAUGHTER]
ALL RIGHT.
SO, THEY GAVE ME 5 MINUTES TO
TALK ABOUT SOMETHING THAT WILL
TAKE 2 MINUTES, SO I'LL GO
IMMEDIATELY OFF TOPIC, IF I
START RAMBLING OFF, IT'S BECAUSE
I HAVE THYROID FOG, I GOT ZAPPED
A COUPLE MONTHS AGO, SHE WAS
LIKE WHAT IS WRONG WITH YOU?
SO I DISCOVERED A NEW TYPE OF
FOG, ANYBODY HEARD OF LUPUS FOG,
NOW I UNDERSTAND YOU QUITE
CLEARLY.
SO THE POINT IS, READ THE
SLIDES, IF I START NOT MAKING
ANY SENSE.
SO, I THINK THE TITLE KIND OF
SUMMARIZES EVERYTHING, IT'S THE
ROLE OF [INDISCERNIBLE] IN
PATIENTS WITH SYSTEMIC LUPUS,
SYSTEMIC LUPUS ERYTHEMATOSUS,
FOR THOSE THAT DON'T KNOW WE
REALLY HAVE GONE DOWN THE HOLE
WITH THE LUPUS CLINICAL RESEARCH
PROGRAM.
I WILL QUICKLY BRIEF WHY, THE
WHO, THE WHAT AND THE WHERE AND
THE WHEN AND WHERE, SO CAN YOU
HAVE AN UNDERSTANDING FELT STUDY
IN PARTICULAR IF YOU WANT TO
REFER SOMEONE, PLEASE DO SO.
ALL RIGHT, SO, THE WHY HAS
ALREADY BEEN ADDRESSED SO I'LL
GLOSS OVER THIS, DR. KAPLAN,
MENTION THE 50% FOLD INCRACY IN
CARDIOVASCULAR RISK THAT
CARDIOVASCULAR EVENTS ACCOUNT
FOR ONE-THIRD OF DEATHS.
WE KNOW THAT TYPE 1 INTERFERONS
ARE IMPORTANT DRIVERS OF THIS
AND VASCULAR DAMAGE.
SO, WE'RE LOOKING TO SEE DO WE
IMPROVE VASCULAR FUNCTION WITH
ANIFROLUMAB, ARE WE DECREASING
VASCULAR INFLAMMATION, WILL
THERE BE A MODULATION BIOMARKERS
OF VASCULAR RISK AND OF COURSE
ALWAYS LOOKING AT SAFETY EMPLOY
NOW ANIFROLUMAB IS ALREADY
APPROVED FOR LUPUS SO WE'RE NOT
EXECKING A MAJOR ISSUE WITH THE
SAFETY.
WHO?
WE ARE LOOKING FOR PATIENTS WHO
18-80, SO ALMOST EVERYBODY.
IT WILL BE MOSTLY WOMEN AND IT'S
GO TO BE LIKE A LOW SCORE OF
ACTIVITY SO SLE OF 6 OR LESS, WE
SRO AN ACCRUAL OF 45 MISHTS WITH
THE PLAN TO COMPLETE 30.
OF COURSE NOT PREGNANT OR
LACTATES AND I SHOULD MENTION
BUT I DIDN'T PUT IN HIRE BECAUSE
WE DON'T KNOW WHO YOU ARE, BUT
APPROXIMATE YOU COME THROUGH US,
WE'RE GOING TO DO A VASCULAR
STUDY ON YOU TO SEE IF YOU'RE
ABNORMAL, IF YOU ARE ABNORMAL
YOU HEAR FROM US BECAUSE YOU'RE
THE PATIENT WE NEED.
THE WHAT IS ANIFROLUMAB, WE WILL
BE LOOKING AT WHAT ANIFROLUMAB
DOES IN A 2-1 RATIO SO WE WILL
HAVE 2 PATIENTS FOR EVERY 1
GETTING PLACEBO.
ANIFROLUMAB YOU MIGHT BE
WONDERING PARTICULARLY IF YOU'RE
A PATIENT, IT'S A MONOCLONAL
ANTIBODY THAT IS BINDING TO
BASICALLY AN INTERFERON RECEPTOR
SO WE CAN BLOCK THAT RECEPTOR.
SO WITH THE PLANS OF BLOCKING
THE INTERFERON SIGNALING AND
BLOCKING OF TYPE 1 INTERFERONS,
IT ALSO REDUCES THE CELL SURFACE
OF THE INTERFERON RECEPTOR WITH
THE HOPES OF DECREASING DOWN
STREAM INFLAMMATION AND
IMMUNOLOGIC PROCESSES RESULTS IN
HOPEFULLY DECREASED DEC ACTIVITY
AND [INDISCERNIBLE] AND WE
ALREADY KNOW AND I JUST
MENTIONED IT'S BEEN WELL
TOLERATED.
ALL RIGHT, THE WHERE, IT SOUNDS
SERIOUS FOR WHAT WE KNOW ABOUT
THIS DRUG BUT IF YOU'RE GOING TO
EXPERIENCE HEADACHE, JOINT PAIN,
VOMITING, I HAVEN'T SEEN ANY OF
THESE YET.
INFECTIONS, LOW GRADE INFECTIONS
IS WHAT WE HAVE SEEN IF THE PAST
AND THAT'S WITH WE EXPECT TO WE
WERE TO BE ANTICIPATORY,
MALIGNANCY ALWAYS REMAINED A
THEORETICAL CONCERN WITH THE
MEDICATIONS WE GIVE, HYPER
TENSATIVITY, IN ANAFLACTIC
REACTIONS OF COURSE ARE ALWAYS A
CONCERN WITH ANY DRUG, BUT
THERE'S A 3.3% INCIDENCE WITH A
DRUG VERSUS 0.8 WITH PLACEBO AND
PRIOR USE.
SO, ANYWAY, THE TAKE HOME POINT
IS MILD-MODERATE TYPE OF ISSUES
HAVE BEEN FOUND EMPLOY WE HAVE A
PROTOCOL PLAN IN PLACE WHERE
WE'RE NOT GOING TO PREMEDICATE
PATIENTS AS A ROUTINE.
IF A PATIENT EXPERIENCES
SOMETHING MILD TO MODERATE, WE
CAN GIVE THINGS LIKE
ANTIHISTAMINES OR SSIT MEN FIN
FOR THE PATIENT'S COMFORT.
IF YOU ARE A PATIENT WHO ENDS UP
HAVING ANAFLACTIC REAC, WE HAVE
A PROTOCOL IN PLACE, BUT THEN
YOU WILL BE WITHDRAWN BECAUSE
WE'RE NOT GOING TO EXPOSE TO YOU
ANY UNNECESSARY RISK.
THIS IS PROBABLY THE MOST
IMPORTANT SLIDE OF WHAT I'M
GETTING MYSELF INTO.
YOU WILL SEE ME, WHICH IS
PROBABLY THE BIGGEST DOWN SIDE
OF THE STUDY.
[LAUGHTER]
WE WILL SCREEN YOU UP IN OP9,
THAT IS OUR CLINIC.
YOU WILL GET VASCULAR STUDIES
AND THE PET CT.
ASSUMING YOU GET THROUGH THAT,
WE'RE GOING TO RANDOMIZE YOU IN
THE 2 TO 1 RATIO I MENTIONED AND
THEN YOU WILL GO ON A 7 MONTH
ODESSY OF GETTING A ONCE A MONTH
INFUSION THAT'S IN SOUTHWEST DAY
HOSPITAL, I KNOW LIKE MARIA
ESPINALL, KNOWS WHERE THAT'S
LOCATED, ANYWAY WE'LL GIVE YOU
AN INFUSION EVERY MONTH AND
WE'LL WRAP UP IN THE CLINIC
AGAIN WITH ANOTHER PET CT,
VASCULAR STUDIES, AND THAT WOULD
CONCLUDE THE STUDY.
OUR CONTACTS, THESE ARE THE--YOU
PROBABLY GOT ALL THESE NAMES
ALREADY, THE MAIN PERSON TO
REACH OUT TO BE WOULD BE LUBNA
HOODA, HERE E-MAIL AND PHONE
NUMBER ARE UP THERE BUT ANY OF
US ARE ACCESS CLINICAL
COMMUNITY.
I WANT TO SAY THANK YOU AND WE
LOVE THIS PICTURE APPARENTLY.
[LAUGHTER]
[ APPLAUSE ]
>> THANK YOU MIKE OUR NEXT
PRESENTERS ARE FROM #LUPUSCHAT,
MISS TIFFANY PETERSON AND FOUND
XER CEO OF AND THE HOST OF LUPUS
CHAT AND AS WELL AS KARLY
HARRISON, CHIEF RESEARCH AND
INNOVATION OFFICER AND CO-HOST
OF LUPUS CHAT AND THEY WILL BE
SPEAKING ON BUILDING RESIL YEBT
HEALTHCARE COMMUNITIES AND
ADDRESSING CHALLENGES AND
EMPOWERING PATES IN THE WAKE OF
THE PANDEMIC AND BEYOND.
PLEASE GIVE THEM A WARM WELCOME.
[ APPLAUSE ]
>> HI, INCH.
SO WE WOULD LIKE TO SHED SOME
LIGHT ON OUR PATIENT'S
COMMUNITIES NEEDS DURING THE PAN
DEMAND HOW WE ADDRESS THOSE
CHALLENGES WHILE EMPOWERING
PATIENTS THROUGHOUT THE DURATION
OF THE PANDEMIC AND BEYOND.
BESIDES MYSELF AND KARLY WE HAVE
2 OTHER LUPUS CHAT EXECUTIVE
TEAM MEMBERS CRYSTAL AND
ELIZABETH.
I KNOW YOU ARE TESTIMONY WITH
TWITTER AND ALTHOUGH IT HAS BEEN
GOING THROUGH SOME QUESTIONABLE
CHANGES, IT'S STULL A VITAL
SPACE FOR THE SHARING OF HEALTH
INFORMATION FOR OUR COMMUNITY.
SO IT'S REALLY IMPORTANT FOR US
TO MEET THEM WHERE THEY ARE.
WE INVITE LUPUS PATIENTS, CARE
PARTNERS, HEALTHCARE
PROFESSIONALS, RESEARCHERS AND
HEALTH INDUSTRY LEADERS TO JOIN
OUR CONVERSATION.
IT'S IMPORTANT FOR US TO DO OUR
PART TO DISPEL MISINFORMATION
AND MAKE SURE THAT WE'RE
PRIORITIZING INCREASING HEALTH
LITERACY.
IT WAS EXTREMELY IMPORTANT FOR
US TO BRING AWARE BUSINESS TO
THE SHORTAGE OF HYDROXY CHLOR O
QUINWHEN WE WERE FACING THAT
DURING THE ONSET OF COVID, IT
REALLY SPARKED A NEW REALITY FOR
MANY PATIENTS WHO BEGAN
RECEIVING NOTICE FROM THEIR
HEALTH PROVIDERS THAT THEY WERE
GOING TO BE LIMITED SUPPLY AND
THAT THEIR MEDICATIONS COULD
POSSIBLY BE STOPPED EMPLOY SO IT
WAS REALLY IMPORTANT FOR US TO
BRING THIS INFORMATION TO OUR
PATIENT COMMUNITY AND WE ASKED
THEM, YOU KNOW WHAT ARE SOME
ADVOCACY EFFORTS YOU'RE AWARE OF
AND THAT IS ADDRESSING THE
HYDROXY COLOR SHORTAGE FOR
PATIENTS AND WE ENCOURAGE THEM
TO GET A 90 DAY SUPPLY SO THAT
WAY THEY WOULDN'T BE ABLE TO GO
WITHOUT MEDICATION, WE FOUND OUT
THAT SOME OF OUR PATIENT
COMMUNITY MEMBERS WERE HALVING
THEIR DOSES ASK JUST HAVING
REALLY EXTREME DELAYS WITH THEIR
PHARMACY IN GETTING ACCESS TO
THESE MEDICATIONS.
THE LUPUS FOUNDATION DID--THE
LUPUS FOUNDATION OF AMERICA DID
LAUNCH A CAMPAIGN CALLED WITHOUT
MY HCQ, AND IT WAS REALLY
PIVOTAL IN GETTING PATIENTS
ACROSS THE GLOBE TO JUST SHARE
THEIR INFORMATION ABOUT WHAT THE
SHORTAGE OF HYDROXY CHLOR O
QUINWAS DOING TO THEM.
I WANT TO MOVE ON TO THE FACT
THAT WE PROVIDE SAFE SPACES FOR
MENTAL AND CERTAINLY--CERTAINLY
MOTIONAL WELLNESS.
WE FOUND IT REALLY CRUCIAL TO
PROVIDE SAFE SPACES AND FOSTER
CONVERSATIONS WITH OUR MEMBERS
WHO ARE FACING FURTHER ISOLATION
DUE TO LOCK DOWN, THEY WERE
EXPERIENCING RACIAL TRAUMS,
UNCERTAINTY, AND THE OVERALL
WEIGHT OF LIVING WITH A CHRONIC
ILLNESS WHILE TRIEFING TO
NAVIGATE LIFE.
WE RECEIVED A LOT OF FEEDBACK
FROM OUR COMMUNITY THAT OUR SAFE
SPACE AND PROVIDED WERE MUCH
APPRECIATED AND YOU CAN SEE IN
SOME OF THESE SPEECH BUBBLES
EXACTLY HOW SOME OF OUR
COMMUNITY MEMBERS FELT ABOUT US
PROVIDING THE SAFE SPACE WITH
THEM DURING SUCH A DIFFICULT
TIME.
SNORKS SO WHILE WE WERE
ATTEMPTING TO NAVIGATE THE
UNKNOWNS OF THE PANDEMIC, AS
WELL AS LIFE WITH CHRONIC
ILLNESS, WE ALSO FACED A RACIAL
UPRISING SPARKED BOO I CONTINUED
STATE SANCTIONED INJUST THE AND
SO WE FELT IT'S FORTUNATE
ADDRESS THAT DURING THAT TIME AS
WE DID SEE MANY PATIENTS WHO
WERE FEELING BURDENED AND
OVERWHELMED BY WHAT WAS GOING ON
AND NONE OF OUR ORGANIZATIONS
WERE ADDRESSING THE ISSUE AND
HOW IT COULD IMPACT US AS A
COMMUNITY AND ALSO IMPACT OUR
DISEASE ACTIVITY, AND SO, WE
FIRST RELEASED A STATEMENT AS
SEEN THERE AND THEN WE THOUGHT
IT IMPORTANT TO DISCUSS HOW
THESE THINGS IMPACTED, YOU KNOW
HEALTH DISPARITIES AND THINGS
LIKE THAT.
THERE WE ARE, AND SO, WE
PRESENTED AT ACR REGARDING
COVID-19 RACISM AND GENDER AND
DISCRIMINATION AND THE FUNCTION
OF STRESS ON WIDENING THE GAP IN
HEALTH DISPARITIES MUCH OF IT
SPEAKING TO DR. BOWIE'S POINT
EARLIER ABOUT HOW STRESS CAN
IMPACT DISEASE ACTIVITY, AS WELL
AS MENTAL HEALTH AND THINGS LIKE
THAT.
AND WORK TOWARD WIDENING THAT
HEALTH DISPARITY.
ANOTHER THING THAT WE DID AS A
COMMUNITY IS WE HOSTED CHAT
CO-HOST BY DR. BLAZER IN THE
AUDIENCE TODAY, WHERE WE DECIDED
TO SPEAK WITH OUR COMMUNITY
MEMBERS AND REALEY GET ENGAGED
AS TO SOME OF THE CHANGES WE CAN
MAKE WITHIN THE HEALTHCARE SPACE
AND SOCIETY AS A WHOLE TO TRY
AND LESSON HEALTH DISPARITIES
AND CLOSE THAT GAP AND YOU ALL
CAN SEE SOME OF THE RESPONSES OF
HOW PATIENTS AND PRACTITIONERS
ALIKE FELT THAT YOU KNOW WE AS A
COLLECTIVE COULD WORK TOWARD
CLOSING THE GAP IN HEALTH
DISPARITIES.
>> ONE OF THE OTHER THINGS THAT
WE FELT IMPORTANT BECAUSE HEALTH
LITERACY AS BEEN AT THE
FOREFRONT OF OUR MISSION SINCE
OUR INCEPTION, OVER THE LAST FEW
YEARS, WE'VE CONTINUED TO WORK
TO PROVIDE PATIENT EDUCATION AND
RESOURCES AROUND RESEARCH,
CLINICAL TRIALS, AND LUPUS AND
IT'S CO-MORBIDITIES.
SO 1 OF THE WAYS DURING THE LAST
FEW YEARS THAT WE DID THIS WAS
GETTING INVOLVED WITH THE
COVID-19 GLOBAL RHEUMATOLOGY
ALLIANCE WHICH MANY OF YOU MAY
BE FAMILIAR WITH WHO WAS A
COALITION BROUGHT TOGETHER
BETWEEN BOTH RHEUMATOLOGISTS AND
PATIENTS JUST OVER A GENERAL
TWITTER CONVERSATION 1 DAY, AS
THE PROJECTION NEURONS OR PIONS
DEMIC FIRST WAS IN THIS EARLY
STAGES AND PATIENTS WANTED TO
KNOW WHAT WAS GOING ON,
RESEARCHERS AND PRACTITIONERS
ALSO WERE IN NEED OF INFORMATION
AND SO WE DECIDED TO COME
TOGETHER AND AND REALLY IT WAS A
NOVEL IDEA OF GETTING THE
PATIENT VOICE FROM THE BOTTOM UP
AND SO, WE RELEASED A PATIENT
EXPERIENCE SURVEY, WE DID 2
ITERATIONS OF THAT THAT REALLY
SPOKE TO THE IMPACTS OF COVID ON
PATIENTS WITH RHEUMATIC DECS AND
FROM THAT AND EVERY OTHER
PUBLICATION WE HAD, WE ALSO
DEVELOPED LAY SUMMARIES ARE IF
PATIENTS SO THAT THEY WOULD BE
ABLE TO YOU KNOW RECEIVE THE
INFORMATION IN THE WAY THAT YOU
KNOW BEST WORKED FOR THEM AND
THEIR LEARNING STYLE SO THEY HAD
THE HIGHLY SCIENTIFIC VERSION
BUT THEN ALSO SOMETHING THAT PUT
IT IN LAY TERMS SO THAT THEY CAN
EASILY DIGEST THE INFORMATION
AND TO UNDERSTAND WHAT WAS GOING
ON AS FAR AS THE PANDEMIC AND
ALSO RESEARCH IN TERMS OF HOW IT
BEING IMPACT THEM AND THEIR
LUPUS AND OTHER RHEUMATIC
DISEASES.
AND SO 1 OF THE OTHER THINGS
THAT WE DID DO, WE HOSTED A CHAT
REGARDING LUPUS RESEARCH
MATTERS, THAT'S 1 OF OUR #S THAT
WE USUALLY USE DURING LUPUS
AWARENESS MONTH WITH
DR. RICHMOND FROM U-MASS MED
CENTER AND AS YOU CAN SEE, WE
COLLECTED SOME PATIENT FEEDBACK
ON WHAT THEY WOULD LIKE TO SEE
GOING FORWARD IN LUPUS RESEARCH
AND WHY IT WOULD BE IMPORTANT.
NON ENCOURAGING SELF-ADVOCACY, I
FEEL LIKE IT'S FORTUNATE
CHAMPION PATIENTS AND WE
ENCOURAGE THE COMMUNITY TO GET
ACT AND I HAVE ADVOCATE FOR
PROPER CARE, TREME, AND
EQUITABLE OPPORTUNITIES AND
HEALTHCARE AND BEYOND.
THIS IS AN EXAMPLE OF A
CONVERSATION THAT WE HAD 1
SUNDAY WITH SOME OF OUR
COMMUNITY MEMBERS AND JUST
ASKING THEM IN WHAT WAYS THAT WE
CAN IMPACT OUR OWN COMMUNITIES
BOTH LOCALLY AND GLOBALLY AND A
LOT OF THEM WERE TALKING ABOUT,
YOU KNOW SPEAKING UP ABOUT
HEALTHCARE DISPARITIES,
ADVOCATING FOR PATIENT
ENGAGEMENT RESEARCH, CONCEPTING
THEIR REPS, DURING DC ADVOCACY
DAYS, AND WE WANTED TO HIGHLIGHT
ELIZABETH SANTA CRUZ WHICH IS AN
EXECUTIVE MEMBER OF THE LUPUS
CHAT AND THE AMAZING WORK SHE
DOES IN NEW JERSEY EVERY YEAR
WITH HER RIBBON CEREMONY FOR
COMMEMORATING MAY'S LUPUS MONTH
IN THE STATE OF NEW JERSEY.
>> ALL RIGHT, SO AS IT SAYS WE
DO WELCOME THE OPPORTUNITY TO
SPEAK WITH EVERYONE REGARDING
COLLABORATIONS SO THAT WE CAN
STRENGTHEN THE TIES BETWEEN THE
PATIENT AND THE PRACTITIONER
COMMUNITY.
WE'RE EXSPECIALLY INTERESTED IN
ADDRESSING FATIGUE, HYDROXY
CHLOR O QUIN AND LUPUS RESEARCH
SO WE ENCOURAGE EVERYONE TO
REACH OUT IF YOU ARE INTERESTED
IN WORKING WITH US AND MAKING
THOSE CONNECTIONS WITH THE
PATIENT COMMUNITY.
[ APPLAUSE ]
>> THANK YOU KARL EXPE TIFFANY.
NEXT WE HAVE OUR LAST PATIENT
INTERVIEW.
MISS [INDISCERNIBLE] WILL BE
INTERVIEWING MISS MALLORY EMPLOY
PLEASE GIVE THEM A WARM WELCOME.
>> [ APPLAUSE ]
NHELLO, EVERYONE, THANK YOU FOR
JOINING US AND PATIENTLY WAITING
FOR US TO BE THE LAST 1.
THIS IS [INDISCERNIBLE].
SHE IS 1 OF OUR NEW PATIENTS
THAT YOINED THE LUPUS PROGRAM.
>> USE YOUR MIC.
>> OH, SORRY.
NTHIS IS [INDISCERNIBLE].
SHE JOINED US THE LAST LIKE 2 OR
3 YEARS AGO TO THE LUPUS PROGRAM
AS A PATIENT.
SO I WILL HAVE SOME QUESTIONS
FOR HER AND TO SHARE HER
EXPERIENCE.
WITH
OKAY, COULD YOU TELL US ABOUT
WHEN YOUR FIRST DIAGNOSED WITH
LUPUS?
>> YES, FIRST OFF THANK YOU FOR
HAVING ME HERE AND SO I WAS
INITIALLY DIAGNOSED IN 2019,
MARCH, I HAD JUST STARTED A NEW
JOB AND A FEW MONTHS LATE ARE I
END UP GETTING SICK, I THOUGHT
IT WAS THE FLU BUT I REALIZED IT
WAS A FLARE AND WAS SICK FOR 2
WEEKS AND HAD A CONSISTENT
HEADACHES AND I WENT TO A
NEUROLOGIST AND THEY HELPED TO
RUN AN A& A TEST WHICH CAME BACK
POSITIVE AND THEN AT A REFERRED
TO ME TO RAY RHEUMATOLOGIST AND
INITIALLY I WAS DIAGNOSED WITH A
TISSUE DISEASE BUT AFTER KIDNEY
BIOPSIES AND A COUPLE SECOND
OPINIONS IT WAS CLASSIFIED LUPUS
OF THE KIDNEYS AND THEN THEY
CHANGED MY DIAGNOSIS IN
JANUARY 2020 TO LUPUS.
>> YOU JOIN US IN 2021, RIGHT?
>> YES.
>> RIGHT.
>> HOW DID YOU HERE ABOUT NIH
AND YOU KNOW WHAT INTERESTED YOU
TO COME TO NIH?
>> SURE, SO ACTUALLY I WAS
TALKING TO A FAMILY FRIEND WHO
ACTUALLY WORKED AT THE NIH, AND
I WAS SHARING MY LUPUS STORY AND
SHE ENCOURAGED ME TO BECOME
INVOLVE WIDE SOME OF THE
CLINICAL TRIALS AND I WASN'T--I
HEARD ABOUT CLINICAL TRIALS BUT
I DIDN'T KNOW SO MUCH ABOUT IT
AND I DECIDED TO LOOK INTO IT,
DO MY OWN RESEARCH AND I DECIDED
TO REACH OUT AND I THINK I
INITIAL CONTACT WAS ELAINE AND
YOU KNOW AFTER OUR CONVERSATION,
I FELT REALLY COMFORTABLE TO GO
FORWARD AND THEN ANOTHER REASON
WHY I DECIDED TO GO FORWARD IS
BECAUSE I WENT HOME TO
PITTSBURGH XI WAS TALKING TO A
COUSIN AND I TOLD HIM I WAS
GOING TO BE INVOLVED IN A
CLINICAL TRIAL AND HIS RESPONSE
WAS, I QUOTE, YOU KNOW THEY'RE
GOING TO USE YOUR DNA FOR AN
UNSOLVED MURDER MYSTERY.
SO MY ANXIETY KICKED IN, I WAS
LIKE WAY, ARE THEY IN BUT THEN,
YOU KNOW IN ALL SERIOUSNESS, YOU
KNOW MAYBE NOT FOR THE EXTREME,
BUT THERE ARE SOME PEOPLE WHO
FILL THAT WAY, AND I THOUGHT
ABOUT HOW MANY PEOPLE OF COLOR
WHO HAVE LUPUS MODEL CITIZEN
MAYBE THINK THAT WAY, MAYBE NOT
TO THAT EXTENT AND AS A PATIENT
AND ADVOCATE MY YOB CORPS IS TO
LEARN, INFORM AND EDUCATE AND
THE BEST WAY IS TO DO THAT
THROUGH SHARED EXPERIENCE AND
SO, IF I CAN CONTRIBUTE TO
CHANGE IN THE NARRATIVE, THAT
WAS 1 OF THE REASONS WHY.
>> YES, KEY TO TELL THAT STORY
SO YOUAR TIS PARTICIPATE IN A
TRIALED IN THE COVID STUDY AND
VASCULAR STUDY HERE WITH US WHAT
INTERESTS YOU EARLY DUE TO
PARTICIPATE IF THIS CLINICAL
RESEARCH STUDY.
>> THAT'S EASY, WHEN I INITIALLY
SIGNED UP I TOLD ELAINE I WAS
INTERESTED AND OPEN TO TRIALS
THAT I WAS A CANDIDATE FOR, SO
WHEN THESE TRIALS CAME UP IT WAS
AN AUTOMATIC YES FOR ME.
>> THANK YOU FOR PARTICIPATING
BECAUSE YOUR PARTICIPATION MAKES
A DIFFERENCE AND AS SENCH
SPEAKING DIVERSITY IS A KEY FOR
BETTER TREATMENT AND BETTER
OUTCOMES, SO CONTRIBUTION IS
VERY IMPORTANT IN THIS RESEARCH
STUDY SPACE WE HAVE HERE AT NIH
AND EVEN OUTSIDE.
SO I KNOW YOU PART OF THE
AUTOIMMUNE ASSOCIATION NONPROFIT
ORGANIZATION, IS THERE ANYTHING
YOU WANT TO SHARE WITH OUR
AUDIENCE, YES, I HAD SEEN THE
PEASHT ADVOCACY WORK WITH THEM
AND THEIR NONPROFIT ORGANIZATION
THAT FOCUSES ON AUTOIMMUNE
AWARENESS RESEARCH EDUCATION AND
EFFICACY AND I'VE BEEN A PART OF
THEIR DEI INITIATIVE AND THEY
HAVE GIVEN ME THE PLATFORM TO
SHARE MY EXPERIENCES WITH
CLINICAL TRIALS WITH THE NIH
BECAUSE WE KNOW AGAIN, LIKE
REPRESENTATION IS IMPORTANT.
AND SO I'VE HAD THE OPPORTUNITY
TO HAVE A SUM AND IT WE HAD A
WHOLE SESSION JUST FOR CLINICAL
TRIALS AND JUST TRYING TO
EDUCATE PEOPLE, MAINLY PEOPLE OF
COLOR AND JUST KIND OF DISPEL
SOME OF THE MYTHS THAT'S
ASSOCIATE WIDE CLINICAL TRIALS.
NYES, YES, YES, YEAH, THAT'S
GOOD.
SO HOW WOULD YOU SUMMARIZE YOUR
OVERALL EXPERIENCE WITH NIH AND
IS THERE ANYTHING YOU WOULD LIKE
PEOPLE TO KNOW?
>> YEAH, I HAVE NOT HAD A
NEGATIVE EXPERIENCE.
LIKE YOU SAID I'M FAIRLY NEW A
FEW YEARS, AND YOU KNOW I LOVE
THAT IT'S FLEXIBLE, I LOVE THAT
1 OF THE THINGS THAT I WASN'T
AWARE OF IS IT'S THOT SOMETHING
THAT YOU HAVE TO CONTINUE, AND
SO WHEN I ENCOURAGE PEOPLE TO
DO, CLINICAL TRIALS, I TELL
THEM, YOU KNOW YOU DON'T HAVE TO
SIGN A CONTRACT, IF YOU'RE IN IT
FOR 3 MONTHS AND DECIDE YOU WANT
TO STOP, LIKE YOU CAN.
AND THEN ALSO, WHEN I TRY TO
TELL PEOPLE, THE BENEFITS OF A
CLINICAL TRIAL, YOU KNOW THERE
ARE SOME PATIENTS WHO MAY NOT
HAVE HEALTHCARE OR ACCESS TO
ADEQUATE HEALTHCARE, OTHER THAN
IT BEING ANOTHER PAIR OF EYES,
OR ANOTHER OPINION REGARDING
YOUR--REGARDING YOUR DISEASE,
YOU KNOW, YOU DON'T HAVE TO HAVE
INSURANCE, I REMEMBER, LAST
YEAR, MY HUSBAND LAST HIS JOB,
HE'S IN THE IT FIELD AND WE WENT
THROUGH MONTHS WITHOUT INSURANCE
BUT I WAS STILL ABLE TO MEET
WITH MY LUPUS DOCTOR HERE, GET
MY LABS DRAWN HERE AND I THINK
THAT'S A HUGE BENEFIT.
SO YEAH, I HAVE NO COMPLAINTS
AND I'M GLAD THAT I TARTED WHEN
I DID, YOU KNOW EARLY IN MY
DIAGNOSIS AND I LOOK FORWARD TO
CONTINUING THE JOURNEY.
>> THANK YOU.
THANK YOU FOR SHARING WITH US
YOUR STORIES AND ANYONE HAVE ANY
QUESTIONS FOR HER?
ALL RIGHT.
THANK YOU VERY MUCH, EVERYONE.
>> THANK YOU.
[ APPLAUSE ]
>> THANK YOU.
OKAY, LAST I WOULD LIKE TO
INVITE DR. HASNI BACK UP FOR
CLOSING REMARKS.
>> ALL RIGHT, SO THANK YOU
EVERYONE FOR STAYING THROUGH AND
I KNOW IT HAS BEEN A BUSY
MORNING AND YOU'VE HEARD A LOT
OF THINGS FROM THE RESEARCHERS
FROM THE PATIENTS, SO THANK YOU
ALL FOR COMING AND STAYING WITH
US AND WE WILL CONTINUE THIS
JOURNEY TOGETHER, COLLABORATE
AND DO MORE AND MORE STRONGER,
MORE STRONGER, MORE BETTER
THINGS.
WE HAVE PATIENT ADVOCACY PEOPLE
HERE, YOU KNOW SO EVERYONE
TOGETHER, WE HAVE TO WORK
TOGETHER AND YOU KNOW DO MORE
BETTER THINGS AND A BRIGHTER
FUTURE.
I WANT TO THANK THE VOLUNTEERS
AGAIN AND I ALSO WANT TO THANK
THE NIAMS LEADERSHIP,
DR. COLBERT HAS BEEN AT AING
THROUGH ALL, I KNOW HE'S SO BUSY
BUT DESPITE HIS BUSY SCHEDULE HE
STAYED HERE AND DR. KAPLAN OF
COURSE, DR. CRISWELL HAS TO
LEAVE A LITTLE EARLY BUT ALL THE
UNWAVERING SUPPORT OF THE
LEADERSHIP HAS HELPED US BUILD
THE PROGRAM TO THIS POINT. 4131 02:37:54,631 ALL PATIENTS HERE