2022-04-11T17:00:01-05:00 2021-12-31 420714325 RSM US LLP 2021 L STREET NW SUITE 400 WASHINGTON DC 20036 990 2021-01-01 953018799 NATIONAL TUBEROUS SCLEROSIS ASSOCIATION NATI 3015629890 8737 COLESVILLE ROAD 400 SILVER SPRING MD 20910 KARI L ROSBECK PRESIDENT & CEO 3015629890 2022-04-08 1 KARI ROSBECK XXX-XX-XXXX ELIZABETH W HELLER P00397829 2022932200 2022-04-08 3 AAL1 IAL1 2021 2022-09-23 18:48:47Z TSC ALLIANCE KARI L ROSBECK 8737 COLESVILLE ROAD 400 SILVER SPRING MD 20910 9061703 0 X WWW.TSCALLIANCE.ORG X 1975 CA FINDING A CURE FOR TUBEROUS SCLEROSIS COMPLEX WHILE IMPROVING THE LIVES OF THOSE AFFECTED. 29 29 22 2234 0 0 4345011 5769053 792685 2234262 11770 5670 -21302 1007002 5128164 9015987 685788 948236 0 0 2382792 2455940 0 0 886523 2353610 3465386 5422190 6869562 -294026 2146425 11657873 14046866 2195312 2437880 9462561 11608986 X THE NATIONAL TUBEROUS SCLEROSIS ASSOCIATION, D/B/A TSC ALLIANCE, IS DEDICATED TO FINDING A CURE FOR TUBEROUS SCLEROSIS COMPLEX WHILE IMPROVING THE LIVES OF THOSE AFFECTED. 0 0 3939485 948236 3190730 RESEARCH PROGRAM STIMULATES AND SUPPORTS BASIC, TRANSLATIONAL, AND CLINICAL RESEARCH ON THE VARIOUS MANIFESTATIONS OF TUBEROUS SCLEROSIS COMPLEX (TSC) TO FURTHER THE DEVELOPMENT OF CLINICAL THERAPIES AND, ULTIMATELY, A CURE FOR TSC. DIRECTED BY STEVEN L. ROBERDS, PHD, CHIEF SCIENTIFIC OFFICER, THE TSC ALLIANCE RESEARCH PROGRAM BUILDS AND FOSTERS COLLABORATIONS BETWEEN BASIC AND CLINICAL RESEARCHERS BY COLLECTING AND DISTRIBUTING TSC NATURAL HISTORY DATA AND BIOSAMPLES, THROUGH COLLABORATIVE PRECLINICAL AND CLINICAL RESEARCH PROGRAMS, AND BY HOSTING BIENNIAL INTERNATIONAL TSC RESEARCH CONFERENCES.SINCE 1984, THE TSC ALLIANCE HAS INVESTED $31 MILLION INTO TSC RESEARCH PROJECTS THROUGH GRANTS AND CONTRACTS: $19.5 MILLION IN RESEARCH GRANTS AND POSTDOCTORAL FELLOWSHIPS, $4.6 MILLION INTO THE NATURAL HISTORY DATABASE AND BIOSAMPLE REPOSITORY, $5.9 MILLION INTO THE PRECLINICAL CONSORTIUM, AND $0.95 MILLION INTO THE CLINICAL RESEARCH CONSORTIUM. GRANT AND FELLOWSHIP APPLICATIONS ARE REVIEWED IN A THREE-STEP PROCESS: (1) ALL APPLICATIONS ARE REVIEWED BY A COMMITTEE COMPRISED OF SCIENTISTS KNOWLEDGEABLE ABOUT THE TOPIC AREA FOR SCIENTIFIC MERIT AND OF CAREGIVERS OR ADULTS AFFECTED BY TSC FOR POTENTIAL IMPACT ON THE LIVES OF THOSE AFFECTED BY TSC; (2) THE SCIENCE AND MEDICAL COMMITTEE OF THE BOARD OF DIRECTORS EVALUATES THE GRANT REVIEW COMMITTEE'S RECOMMENDATIONS AND THE RELEVANCE OF THE APPLICATIONS TO THE TSC ALLIANCE'S FUNDING PRIORITIES; AND (3) THE BOARD OF DIRECTORS THEN REVIEWS THE RECOMMENDATIONS OF THE SCIENCE AND MEDICAL COMMITTEE AND MAKES FINAL APPROVAL FOR FUNDING. FOR A COMPLETE LIST OF CURRENTLY FUNDED PROJECTS AND AN ARCHIVE OF PAST AWARDEES, PLEASE VISIT TSCALLIANCE.ORG/GRANTS.IMPLEMENTED IN 2006, THE TSC NATURAL HISTORY DATABASE (NHD) CAPTURES CLINICAL DATA TO DOCUMENT THE IMPACT OF THE DISEASE ON A PERSON'S HEALTH OVER THEIR LIFETIME. THE DATABASE SERVES AS A RESOURCE OF INFORMATION THAT HELPS TSC RESEARCHERS BETTER UNDERSTAND THE PROGRESSION OF THE DISEASE, DESIGN HYPOTHESIS-DRIVEN QUESTIONS TO HASTEN THE DISCOVERY OF NEW TREATMENTS AND IDENTIFY PERSONS WITH TSC WHO ARE ELIGIBLE TO PARTICIPATE IN RESEARCH STUDIES. AS OF DECEMBER 2021, 2,357 PEOPLE WITH TSC WERE ENROLLED IN THE PROJECT FROM AMONG 18 U.S.-BASED SITES AND THROUGH THE TSC ALLIANCE. THE TSC ALLIANCE PROVIDES FUNDING TO PARTICIPATING CLINICS TO PERFORM DATA ENTRY, MONITORS THE INTEGRITY OF THE DATABASE, AND MAKES DATA AVAILABLE TO INVESTIGATORS TO ANSWER SPECIFIC RESEARCH QUESTIONS AND IDENTIFY POTENTIAL PARTICIPANTS FOR CLINICAL TRIALS AND STUDIES. IN 2020, THE TSC ALLIANCE ADDED A COVID-19 SUB-PROJECT TO ADDRESS DETAILED QUESTIONS ABOUT COVID-19 INFECTION IN INDIVIDUALS WITH TSC. FOR THIS PROJECT, THE TSC ALLIANCE OPENED THIS NHD SUB-PROJECT UP TO PARTICIPANTS WITH SPORADIC LYMPHANGIOLEIOMYOMATOSIS (S-LAM) IN PARTNERSHIP WITH THE LAM FOUNDATION. IN 2021, THE TSC ALLIANCE EXPANDED THE SUB-PROJECT'S VARIABLES TO INCLUDE ENTRIES FOR COVID-19 VACCINATIONS AND ANTIBODY TESTS. BUILDING UPON THE NATURAL HISTORY DATABASE, THE TSC BIOSAMPLE REPOSITORY IS A TSC ALLIANCE-DIRECTED PROJECT INITIATED IN 2014 THAT WILL IMPACT RESEARCH OVER THE NEXT 10 YEARS OR MORE. THE TSC ALLIANCE'S SCIENCE AND MEDICAL COMMITTEE IDENTIFIED THIS AS A GAP THAT CAN ONLY BE FILLED EFFECTIVELY WITH LEADERSHIP OF THE TSC ALLIANCE, GUIDED BY A STEERING COMMITTEE OF CLINICIANS AND RESEARCHERS. HIGH-QUALITY BIOSAMPLES SUCH AS BLOOD, DNA, AND TISSUES LINKED TO DETAILED CLINICAL DATA ARE CRITICAL FOR RESEARCHERS TO UNDERSTAND WHY TSC IS SO DIFFERENT FROM PERSON TO PERSON. SAMPLES IN THE REPOSITORY ARE LINKED TO DETAILED CLINICAL DATA IN OUR EXISTING TSC NATURAL HISTORY DATABASE AND ARE AVAILABLE TO QUALIFIED RESEARCHERS WORLDWIDE. SAMPLES ARE HOUSED AT AND DISTRIBUTED FROM THE VAN ANDEL INSTITUTE IN GRAND RAPIDS, MI, UNDER CONTROL OF THE TSC ALLIANCE. AS OF DECEMBER 31, 2021, THE TSC BIOSAMPLE REPOSITORY HAS ACQUIRED 2,000 BIOSAMPLES: 615 BLOOD SAMPLES FROM INDIVIDUALS WITH TSC ENROLLED IN THE NHD, 367 BUCCAL SAMPLES FROM INDIVIDUALS WITH TSC ENROLLED IN THE NHD, 154 TISSUE SAMPLES FROM INDIVIDUALS WITH TSC ENROLLED IN THE NHD, 465 BLOOD SAMPLES FROM THE TSC CLINICAL RESEARCH CONSORTIUM COLLABORATIVE PROJECTS, AND 389 DNA SAMPLES FROM THE TSC AUTISM CENTER OF EXCELLENCE NETWORK AUTISM BIOMARKER STUDY. THE ORGANIZATION CONTINUES TO GROW THE BIOSAMPLE REPOSITORY BY COLLECTING BLOOD SAMPLES ANNUALLY FROM PARTICIPANTS WHO VOLUNTEER TO DO SO, ENABLING RESEARCHERS TO STUDY CHANGES IN PROTEINS AND OTHER MOLECULES IN THE BLOOD OVER TIME; ADDING ADDITIONAL TSC CLINICS AS PARTICIPATING BIOSAMPLE REPOSITORY COLLECTION SITES; AND COLLECTING BLOOD USING MOBILE PHLEBOTOMY, ENABLING PEOPLE TO DONATE SAMPLES REGARDLESS OF WHERE THEY LIVE IN THE US AND WHETHER THEY ARE SEEN AT A TSC CLINIC. SINCE THE INCEPTION OF OUR MOBILE PHLEBOTOMY INITIATIVE, WE HAVE ACQUIRED 187 BLOOD SAMPLES VIA MOBILE PHLEBOTOMY. UNIQUE ADDITIONS IN 2021 INCLUDED THE ACQUISITION OF 100 TISSUES FROM THE MARYLAND BRAIN AND TISSUE BANK, FIVE CORD BLOOD SAMPLES FROM INFANTS WITHOUT TSC TO USE AS CONTROLS IN FUTURE EXPERIMENTS FOR THE NEWBORN SCREENING INITIATIVE, AND ONE CORD BLOOD SAMPLE FROM AN INDIVIDUAL WITH TSC. TO ENCOURAGE AND ENABLE MORE RESEARCHERS TO UTILIZE BIOSAMPLES, THE TSC ALLIANCE FUNDED THREE SEED GRANTS IN 2021, TOTALING $54,940.80, TO LABS THAT SUBMITTED MERITORIOUS AND INNOVATIVE IDEAS FOR RESEARCH ON TSC BIOSAMPLES.IN 2021, THE TSC ALLIANCE COMPLETED A WHOLE-GENOME SEQUENCING (WGS) PILOT STUDY USING DNA FROM BLOOD SAMPLES IN OUR BIOSAMPLE REPOSITORY TO DEMONSTRATE THE FEASIBILITY OF THIS APPROACH. THE TSC ALLIANCE SUBMITTED 20 DNA SAMPLES WHICH HAD NOT BEEN PREVIOUSLY ANALYZED TO THE TRANSLATIONAL GENOMICS RESEARCH INSTITUTE (TGEN) FOR WGS. RESULTS REVEALED DISEASE-CAUSING VARIANTS IN 19 OF 20 SAMPLES AND DEMONSTRATED FEASIBILITY OF WGS TO DELIVER VALUABLE INFORMATION TO BOTH PATIENTS AND RESEARCHERS. WE INTEND TO SCALE UP WGS IN COMING YEARS AS FUNDS ARE RAISED FOR THIS INITIATIVE.IN DECEMBER 2021, THE TSC ALLIANCE ADDED THE TSC SELF-REPORT PORTAL TO THE TSC NATURAL HISTORY DATABASE. THIS NEW PORTAL WILL PERMIT THE COLLECTION OF PATIENT-REPORTED OUTCOMES ON HOW TSC AFFECTS INDIVIDUALS AND FAMILIES, WHICH WILL COMPLEMENT MEDICAL DATA IN THE NATURAL HISTORY DATABASE. THE PURPOSE OF THIS INITIATIVE IS TO HELP THE TSC ALLIANCE AND TSC RESEARCHERS BETTER UNDERSTAND THE PERSPECTIVE OF THOSE AFFECTED BY TSC TO DEVELOP TOOLS TO MEASURE IMPROVEMENT IN AREAS MOST IMPORTANT TO THE TSC COMMUNITY. EVENTUALLY, THESE MEASUREMENTS CAN BE USED TO IDENTIFY ENDPOINTS FOR CLINICAL TRIALS AND EVIDENCE-BASED GUIDELINES FOR TREATMENT.ONE OF THE MOST IMPACTFUL ASPECTS OF TSC ON THE QUALITY OF LIFE FOR PEOPLE LIVING WITH TSC IS TSC-ASSOCIATED NEUROPSYCHIATRIC DISORDERS (TAND). THEREFORE, THE TSC ALLIANCE'S FIRST USE OF THE PORTAL IS THROUGH COLLABORATION WITH THE TANDEM PROJECT (EMPOWERING FAMILIES THROUGH TECHNOLOGY: A MOBILE-HEALTH PROJECT TO REDUCE THE TAND IDENTIFICATION AND TREATMENT GAP) BY ALLOWING THE COMMUNITY TO COMPLETE THE SELF-QUANTIFIED TAND CHECKLIST (TAND-SQ). THE TSC ALLIANCE IS HELPING THE TANDEM PROJECT TEAM VALIDATE THE UTILITY OF THE TAND-SQ FOR FUTURE USE IN A MOBILE APPLICATION.THE TSC ALLIANCE LAUNCHED THE TSC PRECLINICAL CONSORTIUM IN 2015 TO HELP ADVANCE MORE DRUG CANDIDATES INTO CLINICAL TESTING. TO ACHIEVE THIS END, THE PRECLINICAL CONSORTIUM PROVIDES THE INFRASTRUCTURE TO FOSTER COLLABORATION BETWEEN ACADEMIA AND PHARMACEUTICAL INDUSTRY RESEARCHERS AND FOR ACCESS TO RESOURCES DESIGNED TO HELP FACILITATE DRUG DEVELOPMENT IN TSC. COLLABORATING WITH THE TSC COMMUNITY, THE CONSORTIUM HAS IDENTIFIED AND IMPLEMENTED ROBUST AND REPRODUCIBLE CELL AND ANIMAL MODELS FOR TSC MANIFESTATIONS INCLUDING TUMORS, EPILEPSY, AND TAND. THE TSC ALLIANCE HAS LICENSES TO USE SPECIFIC TSC MOUSE MODELS FOR EXPERIMENTS CARRIED OUT BY THE PRECLINICAL CONSORTIUM, AND ALL MOUSE LICENSE AGREEMENTS INCLUDE THE RIGHTS FOR THE TSC ALLIANCE TO PERFORM EXPERIMENTS UNDER CONTRACT FOR COMMERCIAL ENTITIES. THIS ENSURES DATA GENERATED BY THE PRECLINICAL CONSORTIUM CAN BE USED TO ACCELERATE THE DEVELOPMENT OF NEW TREATMENTS BY COMMERCIAL ENTITIES AS WELL AS ACADEMIC INVESTIGATORS. EXPERIMENTS ARE EXECUTED AT PARTNERING RESEARCH INSTITUTIONS TO ENSURE CONSISTENCY IN TESTING, DATA ACQUISITION AND INTERPRETATION. EPILEPSY STUDIES ARE CONDUCTED AT PSYCHOGENICS (US), AND THE TUMOR GRAFT MODEL AND CELL-BASED ASSAYS ARE CONDUCTED AT PORSOLT (FRANCE). THE VAN ANDEL RESEARCH INSTITUTE, A NON-PROFIT RESEARCH ORGANIZATION, MAINTAINS A COLONY OF TSC2+/- AJ MICE AND BREEDING PAIRS OF L7-CRE-TSC2 MICE ON BEHALF OF THE PRECLINICAL CONSORTIUM. 648010 0 183500 SUPPORT SERVICES DEVELOPS PROGRAMS AND SERVICES THAT PROVIDE INDIVIDUALS WITH TSC DIRECT ACCESS TO INFORMATION, RESOURCES, AND SPECIALISTS EXPERIENCED IN THE DIAGNOSIS, TREATMENT AND MANAGEMENT OF TSC. IN 2021 THE TSC ALLIANCE FACILITATED 8,742 PEER-TO-PEER SUPPORTS FROM ADULT REGIONAL COORDINATORS (12 VOLUNTEERS IN 8 REGIONS NATIONWIDE), CLINIC AMBASSADORS (16 VOLUNTEERS IN 19 TSC CLINICS AND TSC CENTERS OF EXCELLENCE NATIONWIDE), DEPENDENT ADULT TRANSITION RESOURCE COORDINATORS (31 VOLUNTEERS IN 26 STATES AND PUERTO RICO), EDUCATION PARENT MENTORS (29 VOLUNTEERS IN 21 STATES), AND COMMUNITY ALLIANCE LEADERS (47 VOLUNTEERS IN 33 REGIONS ACROSS THE UNITED STATES) WITH ACTIVE FUTURE LEADERS (8 VOLUNTEERS) AND PAST FUTURE LEADERS (6 VOLUNTEERS) WHO HAVE AGREED TO REMAIN AS LEADERS FOR OTHER YOUNG ADULTS WITH TSC.EDUCATION PARENT MENTORS ATTENDED 55 SCHOOL MEETINGS (IEPS, EVALUATION TEAM MEETINGS, 504 PLAN MEETINGS, RESOLUTION MEETINGS, AND MEDIATIONS) IN PERSON, THROUGH SKYPE/ZOOM, AND VIA CONFERENCE CALLS TO SUPPORT FAMILIES IN ATTAINING EDUCATIONAL SERVICES FOR THEIR CHILDREN THROUGHOUT THE COUNTRY. SEVERAL SCHOOL SYSTEMS ALSO REQUESTED "TSC 101" IN AN EFFORT TO HELP THEM UNDERSTAND THE COMPLEXITIES OF TSC AND LEARNING ISSUES FOR CHILDREN WITH TSC. THE COMMUNITY PROGRAMS TEAM SUPPORTS A NETWORK OF 33 VOLUNTEER BRANCHES OF THE ORGANIZATION, CALLED COMMUNITY ALLIANCES, THAT PROVIDE SUPPORT AND COMMUNITY EDUCATION IN ALL 50 STATES. TO ENSURE THE TSC COMMUNITY CONTINUE TO RECEIVE UPDATED INFORMATION ABOUT TSC, TSC-ASSOCIATED NEUROPSYCHIATRIC DISORDERS (TAND),TRANSITION AND RESEARCH, THE TSC ALLIANCE DEVELOPED AN E-WEBINAR SERIES. IN 2021, WE HOSTED 27 E-WEBINARS WITH 1,219 CUMULATIVE LIVE ATTENDEES, 5,567 CUMULATIVE RECORDING VIEWS AND 3,123 CUMULATIVE LANDING PAGES VISITS. ADDITIONALLY, WE HELD THE FOLLOWING WEBINARS: APRIL COVID VACCINE TOWN HALL - 66 LIVE ATTENDEES, 304 VIDEO VIEWS; ABC TO SAP WEBINAR - 50 LIVE ATTENDEES, 458 VIDEO VIEWS; AUGUST COVID UPDATE TOWN HALL - 307 LIVE ATTENDEES, 463 VIDEO VIEWS. ADDING IN LIVE ATTENDEES WITH ADDITIONAL WEBINARS TOTAL LIVE AUDIENCE FOR WEBINARS IS 1,642, TOTAL CUMULATIVE RECORDING VIEWS IS 6,636 AND 3,123 CUMULATIVE LANDING PAGE VISITS.IN 2021, THE TSC ALLIANCE LAUNCHED THE TSC NAVIGATOR, AN EASY-TO-USE, INTERACTIVE ONLINE TOOL TO HELP GUIDE INDIVIDUALS AND FAMILIES THROUGH THE COMPLEXITIES OF TSC ACROSS THE LIFESPAN, PROACTIVELY MANAGE THEIR CARE, AND LIVE THEIR FULLEST LIVES. USERS CAN ACCESS INFORMATION BASED ON THE AGE OF ONE'S DIAGNOSIS, SUCH AS PRENATAL, CHILDHOOD, OR ADULT, TO HELP DETERMINE WHICH STEPS WILL HELP EMPOWER THEM THROUGHOUT THEIR INDIVIDUAL JOURNEYS. REGARDLESS OF AGE, TSC NAVIGATOR ALSO HELPS INDIVIDUALS WITH TSC AND THEIR CAREGIVERS FACE COMPLEX SITUATIONS, OVERCOME ACCESS ISSUES, AND ADDRESS INSURANCE BARRIERS. THE TSC NAVIGATOR LAUNCHED OCTOBER 14, 2019 AND RECEIVED MORE THAN 2,700 VISITS BETWEEN LAUNCH AND YEAR END.587 INDIVIDUALS AND FAMILIES WERE ASSISTED DIRECTLY BY THE DIRECTOR OF MEDICAL AFFAIRS AND TEAM ACTING AS SUPPORT NAVIGATORS. OF THE ASSISTANCE PROVIDED, 78 WERE MEDICATION-RELATED, 60 WERE TAND-RELATED, 88 WERE NEW DIAGNOSIS, AND 252 WERE FOR ASSISTANCE WITH ACCESS TO CARE WITH 46 COVID-19 RELATED. THE REMAINDER SERVED A VARIETY OF OTHER NEEDS. 524283 0 69500 PUBLIC HEALTH EDUCATION HEIGHTENS AWARENESS OF TSC THROUGHOUT THE GENERAL PUBLIC TO BROADEN THE SCOPE OF SUPPORT AND UNDERSTANDING BEYOND TSC INDIVIDUALS AND THEIR FAMILIES. ONE MAJOR PROJECT IN 2021 WAS CHANGING THE ORGANIZATION'S NAME TO TSC ALLIANCE, WHICH WAS FORMALLY ANNOUNCED SUNDAY, MAY 16.DURING 2021, THE TSC ALLIANCE ALSO PRODUCED TWO ISSUES OF ITS NATIONAL MAGAZINE, PERSPECTIVE, WHICH IS MAILED TO APPROXIMATELY 17,900 CONSTITUENTS AS WELL AS POSTED ON THE WEBSITE. THE TSC ALLIANCE'S PODCAST SERIES, CALLED TSC NOW, PRODUCED 12 EPISODES WITH 2,186 DOWNLOADS. SIX ISSUES OF THE ELECTRONIC TSC MATTERS NEWSLETTER WERE DISTRIBUTED TO 1,340 SUBSCRIBERS. THE TSC ALLIANCE'S WEBSITE INCREASES AWARENESS AND PROVIDES EXTENSIVE EDUCATION THROUGH AN AVERAGE OF MORE THAN 24,000 UNIQUE VISITORS EACH MONTH. THE TSC ALLIANCE ALSO RELIES HEAVILY ON SOCIAL MEDIA TO EDUCATE CONSTITUENTS AND PROMOTE NEW RESOURCES AND EVENTS. ITS PRIVATE FACEBOOK TSC DISCUSSION GROUP BOASTS MORE THAN 10,000 MEMBERS, WHILE ITS TWITTER ACCOUNT HAS 2,579 FOLLOWERS AND INSTAGRAM HAS 2,762 FOLLOWERS. TO INCREASE PUBLIC AWARENESS, THE TSC ALLIANCE PARTICIPATED IN THE SEVENTH ANNUAL TSC GLOBAL AWARENESS DAY ON MAY 15 AS WELL AS TSC AWARENESS MONTH THROUGHOUT MAY. THE TSC ALLIANCE ALSO HEAVILY PROMOTED THE INAUGURAL SEIZURE ACTION PLAN AWARENESS WEEK (FEBRUARY 8-14) AND INFANTILE SPASMS AWARENESS WEEK (DECEMBER 1 TO 7). THESE AWARENESS CAMPAIGNS ALONG WITH THE ORGANIZATIONS VARIOUS NEWS RELEASES CULMINATED IN MORE THAN 1 BILLION CUMULATIVE IMPRESSIONS. 353569 34070 GOVERNMENT RELATIONS EFFORTS FOCUS ON INCREASING FEDERAL AND STATE APPROPRIATIONS FOR TSC RESEARCH, RAISING AWARENESS, AND COLLABORATING WITH GOVERNMENT PARTNERS TO DRIVE TSC RESEARCH FORWARD AND IMPROVE CLINICAL CARE AND TREATMENT OPTIONS FOR INDIVIDUALS WITH TSC. ON THE STATE LEVEL, THE TSC ALLIANCE ADVOCATED FOR STATE FUNDING FOR TSC CENTERS IN MISSOURI AND MARYLAND, RESULTING IN AT LEAST $750,000 IN STATE APPROPRIATIONS. THE ANNUAL TSC ALLIANCE MARCH ON CAPITOL HILL TO ADVOCATE FOR FEDERAL FUNDING FOR THE TUBEROUS SCLEROSIS COMPLEX RESEARCH PROGRAM (TSCRP) AT THE DEPARTMENT OF DEFENSE'S (DOD) CONGRESSIONALLY DIRECTED MEDICAL RESEARCH PROGRAM (CDMRP) TOOK PLACE IN EARLY MARCH 2021. MORE THAN 135 PEOPLE PARTICIPATED IN THE VIRTUAL MARCH ON CAPITOL HILL THE WEEK OF MARCH 1-5, 2021. ADVOCATES MADE VIRTUAL APPOINTMENTS WITH 75 SENATE AND 280 HOUSE OFFICES TO ADVOCATE FOR A CONTINUATION OF $8 MILLION IN FUNDING FOR THE TSCRP. THE HOUSE TSCRP DEAR COLLEAGUE LETTER SPONSORED BY REPRESENTATIVES MULLIN (R-OK) AND RASKIN (D-MD) CLOSED WITH 202 SIGNERS. THE SENATE LETTER SPONSORED BY SENATORS CRAMER (R-ND) AND HEINRICH (D-NM) CLOSED WITH 34 SIGNERS. TOTAL FUNDING FOR THE TSCRP HAS BEEN $105 MILLION SINCE 2002. PRESIDENT BIDEN JUST SIGNED THE FY22 DEFENSE BILL INTO LAW ON MARCH 15, 2022, WHICH INCLUDED $8 MILLION FOR THE TSCRP.RESEARCH PERFORMED THROUGH THIS PROGRAM HAS RECENTLY LED TO ADDITIONAL CLINICAL TRIALS INCLUDING DETERMINING IF IMATINIB, A DRUG FDA-APPROVED FOR CANCER, CAN SAFELY IMPROVE LEVELS OF VEGF-D, A BIOMARKER OF LYMPHANGIOLEIOMYOMATOSIS (LAM), A LIFE-THREATENING LUNG MANIFESTATION OF TSC, FUNDED IN FY2013; TESTING A COMBINATION OF TWO DRUGS TO TREAT LAM FUNDED IN FY2012; A MULTI-SITE CLINICAL TRIAL TESTING THE EFFICACY OF AN EXPERIMENTAL TOPICAL RAPAMYCIN CREAM TO TREAT THE DISFIGURING FACIAL TUMORS, CALLED FACIAL ANGIOFIBROMAS, CAUSED BY TSC FUNDED IN FY2010; A CLINICAL RESEARCH NETWORK WAS CREATED TO TEST POTENTIAL NEW THERAPIES, TO VALIDATE BIOMARKERS, AND TO LEARN THE NATURAL HISTORY OF LEADING TO A CLINICAL TRIAL FUNDED IN FY2012. DATA OBTAINED FROM AN FY2010 TSCRP CLINICAL RESEARCH AWARD TO DEFINE EARLY AUTISM PREDICTORS IN TSC AND AN FY2014 TSCRP AWARD FOR A PILOT CLINICAL TRIAL IS BEING TESTED IN A LARGE, NIH-FUNDED CLINICAL TRIAL LOOKING AT THE EFFECTIVENESS OF A BEHAVIORAL INTERVENTION STRATEGY, JASPER, TO IMPROVE OUTCOMES IN CHILDREN WITH AUTISM. THE TSCRP HAS ALSO FUNDED RESEARCH TO DEVELOP ANIMAL MODELS OF TSC THAT HAVE SEIZURES, ENABLING A BETTER UNDERSTANDING OF THE ETIOLOGY OF TSC. BASED ON DATA FROM TSCRP-FUNDED ANIMAL MODELS OF TSC THAT HAVE SEIZURES AND SHARE PATHOLOGY RELATED TO THAT OF TRAUMATIC BRAIN INJURY, AN INDUSTRY-SPONSORED CLINICAL TRIAL DEMONSTRATED THE EFFECTIVENESS THE MTOR INHIBITOR, EVEROLIMUS, AT TREATING EPILEPSY IN MANY INDIVIDUALS WITH TSC. NONE OF THIS PROGRESS WOULD HAVE BEEN POSSIBLE WITHOUT THE CRITICAL SUPPORT PROVIDED THROUGH THE TSCRP.GLOBAL OUTREACH WORKS TO ADDRESS UNMET NEEDS WITHIN THE GLOBAL TSC COMMUNITY. THE PROGRAM PROVIDES THE OPPORTUNITY FOR THE TSC ALLIANCE TO SHARE EXPERIENCES AND ASSIST IN THE START-UP OF SUPPORT OF TSC-RELATED ORGANIZATIONS IN OTHER COUNTRIES. A GLOBAL ALLIANCE IS A STRUCTURED GROUP OF EMPOWERED AND CARING VOLUNTEERS WHO WORK CLOSELY WITH THE TSC ALLIANCE TO FACILITATE LOCAL CONNECTIONS FOR INDIVIDUALS AND FAMILIES AFFECTED BY TSC AND RAISE REVENUE AND AWARENESS WHILE SUPPORTING THE MISSION OF THE ORGANIZATION. THE TSC ALLIANCE HAS SIX GLOBAL PARTNERSHIPS, INCLUDING: TSC ALLIANCE OF ISRAEL, TS CANADA ST, TSC ALLIANCE OF MEXICO, TSC ALLIANCE FOUNDATION (IN THAILAND), HUNGARIAN FOUNDATION FOR TUBEROUS SCLEROSIS, AND TSC ALLIANCE OF INDIA. THE TSC ALLIANCE RECOGNIZES 10 TSC CLINICS IN GLOBAL ALLIANCE COUNTRIES. PROFESSIONAL EDUCATION EXPANDS PROGRAMS TO TARGET RESEARCHERS AND HEALTHCARE PROVIDERS CARING FOR INDIVIDUALS WITH TSC, MEDICAL STUDENTS, GENETIC COUNSELORS AND EDUCATORS TO MINIMIZE THE CONSEQUENCES OF IGNORANCE AND MISINFORMATION. IN AUGUST, TWO NEW PUBLICATIONS WERE ACCEPTED BY PEDIATRIC NEUROLOGY, "UPDATED INTERNATIONAL TSC DIAGNOSTIC CRITERIA AND SURVEILLANCE AND MANAGEMENT RECOMMENDATIONS AND "BEYOND THE GUIDELINES: HOW WE CAN IMPROVE HEALTHCARE FOR PEOPLE WITH TSC AROUND THE WORLD." THE NEW PAPERS PROVIDE THE FIRST SIGNIFICANT UPDATES TO THE INTERNATIONAL GUIDELINES SINCE 2013 BECAUSE OF NEW MEDICATIONS AND ADVANCES IN TREATMENTS. TO ADDRESS THE CHANGES, A WORKING GROUP LED BY DARCY A. KRUEGER, MD, PHD, OF CINCINNATI CHILDREN'S HOSPITAL MEDICAL CENTER, AND HOPE NORTHRUP, MD, MCGOVERN MEDICAL SCHOOL, UNIVERSITY OF TEXAS HEALTH SCIENCE CENTER AT HOUSTON, INCLUDED 80 PARTICIPANTS FROM 16 COUNTRIES.THE TSC ALLIANCE PARTICIPATED IN OR PRESENTED AT 37 PROFESSIONAL MEETINGS IN 2021 INCLUDING CURING THE EPILEPSIES 2020: SETTING RESEARCH PRIORITIES, NIMH 2021 VIRTUAL WORKSHOP: GENE-BASED THERAPEUTICS FOR RARE NEURODEVELOPMENTAL PSYCHIATRIC DISORDERS, TSC ALLIANCE: NEWBORN SCREENING WORKSHOP, ATS BOARD OF DIRECTORS MEETING, RARE DISEASE DAY AT NIH, ISAN MEMBERSHIP MEETING, INAUGURAL O'DONNELL BRAIN INSTITUTE SYMPOSIUM ON AUTISM SPECTRUM DISORDERS, OVERCOMING BARRIERS TO DIVERSIFYING CLINICAL TRIALS (RDCRN), ELHS SPRING LEARNING SESSION, HEALTH RESEARCH ALLIANCE CULTURAL COMPETENCY WORKSHOP WITH BME, RESEARCH!AMERICA ANNUAL MEETING OF THE MEMBERS, MEDICAID AND CHIP PAYMENT AND ACCESS COMMISSION (MACPAC) WEBINAR, ICARE MEETING, TRANS-NIH MEETING, ATS PAR MEET-THE-EXPERTS PATIENT/FAMILY FORUM, ATS SCIENTIFIC SYMPOSIUM, USF DIVERSITY, EQUITY, AND INCLUSION IN THE WORKPLACE SERIES, ISAN MEMBERSHIP MEETING-SUMMER, GLOBAL GENES RARE DRUG DEVELOPMENT SYMPOSIUM: PRECLINICAL MODELING-WHAT ADVOCATES NEED TO KNOW ABOUT ANIMAL & CELL MODELS, INTERNATIONAL TSC RESEARCH CONFERENCE-UK, RARE DISEASE PATIENT-FOCUSED DRUG DEVELOPMENT (PFDD) GUIDANCE COMPENDIUM, GLOBAL GENES ACCESS WORKING GROUP LUNCH BRIEFING: MEDICAID COVERAGE FOR GENOMIC SEQUENCING, NINDS NONPROFIT FORUM ADVISORY BOARD MEETING-INDUSTRY PARTNER, GLOBAL GENES: ACCESS WORKING GROUP, LGS FOUNDATION MEETING OF THE MINDS, EVERYLIFE VIRTUAL NEWBORN SCREENING BOOTCAMP, CNF SYMPOSIUM (WITHIN CNS), RDCRN FALL MEETING, NIAMS, BIO, VIRTUAL INTERNATIONAL TSC & LAM RESEARCH CONFERENCE, ATS PAR ANNUAL PLANNING MEETING, TSCI VIRTUAL WORKSHOP, TSCRP PROGRAMMATIC REVIEW & VISION SETTING, AMERICAN EPILEPSY SOCIETY ANNUAL MEETING, AND INFANTILE SPASMS ACTION NETWORK MEMBERS MEETING. 353569 34070 5465347 1 1 0 1 0 0 0 0 0 1 1 0 0 1 1 0 0 1 0 0 1 1 0 0 1 0 0 1 0 1 0 0 0 0 0 0 0 0 1 0 0 0 0 1 0 0 0 1 31 0 1 22 1 0 0 0 0 0 1 1 0 0 0 0 0 0 X 29 29 0 0 0 0 1 1 0 1 1 0 1 1 1 1 1 1 1 1 1 1 0 AL AR CA FL GA HI IL IN KS KY MD MA MI MN MS NH NJ NM NY NC OR PA RI SC TN UT VA WI WV X X X THE ORGANIZATION 3015629890 8737 COLESVILLE ROAD 400 SILVER SPRING MD 20910 PETER CRINO MD PHD CHAIR 5.00 0.00 X X 0 0 0 WILLIAM JOSEPH VICE CHAIR 5.00 1.00 X X 0 0 0 CHRIS RUSSELL IMMEDIATE PAST CHAIR 5.00 0.00 X X 0 0 0 LAURA MARKS SECRETARY 5.00 0.00 X X 0 0 0 PAUL WAXLAX TREASURER 5.00 1.00 X X 0 0 0 DEREK BAUER BOARD MEMBER 2.00 0.00 X 0 0 0 JULIE BLUM BOARD MEMBER 2.00 0.00 X 0 0 0 MATT BOLGER BOARD MEMBER 2.00 0.00 X 0 0 0 EDWARD BURKHALTER BOARD MEMBER 2.00 0.00 X 0 0 0 MARK CARROLL BOARD MEMBER 2.00 0.00 X 0 0 0 SARAH CHIEFFO BOARD MEMBER 2.00 0.00 X 0 0 0 DAVID COIT BOARD MEMBER 2.00 0.00 X 0 0 0 MARGARET COX BOARD MEMBER 2.00 0.00 X 0 0 0 TIMOTHY DILLS BOARD MEMBER 2.00 0.00 X 0 0 0 BONNIE HOGUE DUFFY BOARD MEMBER 2.00 0.00 X 0 0 0 TANJALA GIPSON BOARD MEMBER 2.00 0.00 X 0 0 0 STEVEN GOLDSTEIN BOARD MEMBER 2.00 0.00 X 0 0 0 ROBERT GRANDIA BOARD MEMBER 2.00 0.00 X 0 0 0 DANA HOLINKA BOARD MEMBER 2.00 0.00 X 0 0 0 JESSICA KREFTING BOARD MEMBER 2.00 0.00 X 0 0 0 DARCY KRUEGER BOARD MEMBER 2.00 0.00 X 0 0 0 HEATHER LENS BOARD MEMBER 2.00 0.00 X 0 0 0 JIM MAGINN BOARD MEMBER 2.00 0.00 X 0 0 0 DIANE MCSWAIN BOARD MEMBER 2.00 0.00 X 0 0 0 MUSTAFA SAHIN BOARD MEMBER 2.00 1.00 X 0 0 0 SEAN SHILLINGER BOARD MEMBER 2.00 0.00 X 0 0 0 MATTHEW SIMONIAN BOARD MEMBER 2.00 0.00 X 0 0 0 VANESSA VAZQUEZ BOARD MEMBER 2.00 0.00 X 0 0 0 TARA ZIMMERMAN BOARD MEMBER 2.00 0.00 X 0 0 0 KARI L ROSBECK PRESIDENT & CEO 54.00 1.00 X 214608 3974 33232 RICHARD GOLLUB CFO 47.00 3.00 X 156734 10004 32878 STEVEN L ROBERDS CHIEF SCIENTIFIC OFFICER 45.00 0.00 X 228854 0 26998 DEAN RAGER-AGUIAR DIRECTOR, PRECLINICAL RESEARCH 45.00 0.00 X 163695 0 32110 JAYE D ISHAM VP, COMMUNICATIONS STRATEGY 45.00 0.00 X 121765 0 4244 LISA M MOSS SR. DIRECTOR, DONOR RELATIONS 45.00 0.00 X 109646 0 26893 APRIL COOPER DIRECTOR, COMMUNITY PROGRAMS 45.00 0.00 X 108589 0 3418 ASHLEY POUNDERS DIRECTOR, MEDICAL AFFAIRS 45.00 0.00 X 101208 0 11671 1205099 13978 171444 8 0 1 0 PSYCHOGENICS INC 20 GRAMERCY PARK SOUTH NEW YORK NY 10003 RESEARCH LAB SERVICES 1564533 VAN ANDEL RESEARCH INSTITUTE 333 BOSTWICK AVE NE GRAND RAPIDS MI 49503 LABORATORY SERVICES 227915 CAVAROCCHI RUSCIO DENNIS AND ASSOCIATES 600 MARYLAND AVE SW WASHINGTON DC 20024 GOVERNMENT RELATIONS 112980 3 50596 4132 955330 226000 4532995 285267 5769053 CONTRACT REVENUE 900099 2120485 2120485 CONFERENCE REVENUE 900099 113777 113777 2234262 3809 3809 1861 0 1861 1861 1861 234482 955330 45716 188766 188766 CHANGE INT-ENDOWMENT 900099 818236 818236 818236 9015987 2234262 0 1012672 833329 833329 114907 114907 797701 489000 223016 85685 1242312 792478 103838 345996 275780 173018 38279 64483 140147 87925 19453 32769 38838 25875 10766 2197 26179 26179 112980 112980 369033 228093 16900 124040 24836 24608 228 256679 140716 8501 107462 172599 90501 20808 61290 128139 80284 17669 30186 13890 8415 1692 3783 76847 76630 55 162 73200 50381 6465 16354 11222 7040 1558 2624 PRECLINICAL CONSORTIUM 1824180 1824180 NHD BIOSAMPLE REPOSIT 266285 266285 DUES AND SUBSCRIPTIONS 48878 29979 12767 6132 MISCELLANEOUS EXPENSES 8527 3443 3848 1236 13074 5280 5898 1896 6869562 5465347 517692 886523 X 33150 16575 0 16575 1097500 1459324 1238876 1242438 1578285 2698495 113491 99173 392003 620148 719159 281990 510367 437169 6727351 7490119 11657873 14046866 435478 472049 422226 676798 1337608 1289033 2195312 2437880 X 5954477 6580605 3508084 5028381 9462561 11608986 11657873 14046866 2146425 0 X 0 1 X 1 0 X 3949953 4105376 5527543 4345011 5769053 23696936 3949953 4105376 5527543 4345011 5769053 23696936 3559289 20137647 10393 13784 27444 11188 3809 66618 642 1243 714 2599 23766153 10327170 0.84730 0.84300 X SCHEDULE A, PART II, LINE 10, EXPLANATION OF OTHER INCOME: INCOME FROM ACTIVITIES NOT REGULARLY CARRIED ON - 2017 AMOUNT: $ 642. 2018 AMOUNT: $ 1,243. 2019 AMOUNT: $ 714. RESTRICTED RESTRICTED RESTRICTED RESTRICTED RESTRICTED RESTRICTED RESTRICTED RESTRICTED 1127 153012 154139 6795167 6949306 497465 124366 0 0 450555 445393 423685 497465 1817098 2725647 149340 165226 123014 154139 591719 112639 111348 105921 124366 454274 681411 823 2491 895 1127 5336 5893521 132476 1053588 333813 34024 6711748 5915805 54645 416431 463721 29639 5893521 5193934 78962 959121 288356 27856 5915805 5655555 82346 -439321 50000 54646 5193934 5293989 92635 739072 403000 67141 5655555 0.84560 0.13100 0.02340 0 1 1 449272 71475 377797 189615 134797 54818 80272 75718 4554 437169 INTEREST IN NET ASSETS OF AFFILIATE 6711748 OPERATING LEASE RIGHT-OF-USE ASSET 778371 7490119 DEFERRED RENT & LEASE INCENTIVE 1285691 DUE TO AFFILIATE 3342 1289033 9050015 34028 34028 9015987 0 9015987 6903590 34028 34028 6869562 0 6869562 PART V, LINE 4: THE ALLIANCE'S ENDOWMENTS CONSIST OF TWO FUNDS ESTABLISHED FOR DIFFERENT PURPOSES. THE ALLIANCE'S ENDOWMENT INCLUDE ONE TRADITIONAL DONOR-RESTRICTED ENDOWMENT FUNDS AND ONE BOARD-DESIGNATED ENDOWMENT FUND. THE BOARD-DESIGNATED ENDOWMENT FUND SOLELY CONSISTS OF THE ENDOWMENT FUND'S UNRESTRICTED NET ASSET BALANCE. 1 EUROPE (INCLUDING ICELAND & GREENLAND) 0 0 GRANTMAKING 19907 MIDDLE EAST AND NORTH AFRICA 0 0 GRANTMAKING 75000 EUROPE (INCLUDING ICELAND & GREENLAND) 0 0 PROGRAM SERVICE ACTIVITIES PRECLINICAL RESEARCH STUDIES 4177 NORTH AMERICA 0 0 GRANTMAKING 20000 0 0 0 0 0 0 119084 0 119084 MIDDLE EAST AND NORTH AFRICA RESEARCH ON TUBEROUS SCLEROSIS COMPLEX 75000 WIRE 0 EUROPE (INCLUDING ICELAND & GREENLAND) PROGRAMMATIC SUPPORT 19907 WIRE 0 NORTH AMERICA GRANTMAKING 20000 CHECK 0 0 3 0 0 0 0 0 0 PART I, LINE 2: GRANTEE ORGANIZATIONS ARE EXPECTED TO FILE ANNUAL PROGRESS REPORTS TO OUTLINED GRANT GOALS AND MILESTONES. THESE REPORTS ARE REVIEWED BY A COMMITTEE OF PEERS. THIS COMMITTEE MAKES DETERMINATIONS BASED ON QUALITY OF WORK TO GOALS AND IF THE GRANTEE WILL CONTINUE TO RECEIVE FUNDING. A FINAL WRITTEN AND FINANCIAL REPORT IS REQUIRED OF ALL GRANTEES. PART III ACCOUNTING METHOD: VIRTUAL WALKS 806174 650809 155365 20365 154 COMEDY FOR A CURE 142870 100282 42588 1093 15891 1400 7 240768 204239 36529 2313 4500 1189812 955330 234482 23771 20545 1400 45716 188766 1 BRIGHAM & WOMEN'S HOSPITAL 75 FRANCIS STREET BOSTON MA 02115 042312909 501(C)(3) 109963 0 RESEARCH GRANTS UNIVERSITY OF TEXAS SOUTHWESTERN MEDICAL CENTER 5323 HARRY HINES BLVD DALLAS TX 75287 756002868 501(C)(3) 18750 0 RESEARCH GRANTS NATIONAL ORGANIZATION FOR RARE DISORDERS 55 KENOSIA AVENUE DANBURY CT 06810 133223946 501(C)(3) 225000 0 TSC TRAVEL AND LODGING ASSISTANCE PROGRAM NORTHWESTERN UNIVERSITY 7TH FLOOR RUBLOFF BUILDING CHICAGO IL 60611 362167818 501(C)(3) 56250 0 RESEARCH GRANTS CHILDREN'S HOSPITAL CORPORATION 300 LONGWOOD AVENUE BOSTON MA 02115 042774441 501(C)(3) 112500 0 RESEARCH GRANTS UNIVERSITY OF CALIFORNIA BERKELEY 1608 FOURTH ST STE 220 BERKELEY CA 94710 946002123 501(C)(3) 28125 0 RESEARCH GRANT VANDERBILT UNIVERSITY 1161 21ST AVE SOUTH NASHVILLE TN 37232 352528741 501(C)(3) 28125 0 RESEARCH GRANT UNIVERSITY OF MARYLAND BALTIMORE PO BOX 41428 BALTIMORE MD 21203 526002033 501(C)(3) 18634 0 RESEARCH GRANT UNIVERSITY OF WASHINGTON 4333 BROOKLYN AVE NE BOX 359472 SEATTLE WA 98195 916001537 501(C)(3) 25000 0 RESEARCH GRANT CHILDREN'S HOSPITAL MEDICAL CENTER 3333 BURNET AVENUE CINCINNATI OH 45229 310833936 501(C)(3) 100000 0 CLINICAL RESEARCH BROWN UNIVERSITY 69 BROWN STREET BOX 1911 PROVIDENCE RI 02912 050258809 501(C)(3) 55000 0 CLINICAL RESEARCH BOSTON CHILDREN'S HOSPITAL PO BOX 414413 BOSTON MA 022414413 042774441 501(C)(3) 57409 0 CLINICAL RESEARCH 12 0 PART I, LINE 2: THE TSC ALLIANCE HAS FUNDED $31 MILLION IN RESEARCH ON TSC SINCE 1984. DIRECTED BY STEVEN L. ROBERDS, PH.D, CHIEF SCIENTIFIC OFFICER, THE TSC ALLIANCE RESEARCH GRANTS PROGRAM FUNDS RESEARCH FOCUSED ON TSC WITH PRIORITIES SET BY THE RESEARCHERS TOGETHER WITH THE TSC ALLIANCE. COLLABORATIONS BETWEEN BASIC AND CLINICAL RESEARCHERS ARE ENCOURAGED AND FOSTERED, AND THE TSC ALLIANCE IS WORKING TO INCREASE FUNDING FOR RESEARCH ON TSC. THROUGH THE TSC ALLIANCE RESEARCH GRANTS PROGRAM, APPLICATIONS CAN BE SUBMITTED FOR POSTDOCTORAL FELLOWSHIPS AND TSC RESEARCH GRANTS. GRANTS ARE REVIEWED IN A THREE-STEP PROCESS: 1. A GRANT REVIEW COMMITTEE COMPOSED OF INDIVIDUALS KNOWLEDGEABLE ABOUT THE CLINICAL AND BASIC COMPONENTS OF TSC AS WELL AS CONSUMERS REVIEW ALL GRANT APPLICATIONS FOR SCIENTIFIC MERIT, RELEVANCY TO THE FUNDING PRIORITIES OF THE ORGANIZATION AND WITH A FOCUS ON UNDERSTANDING THE MECHANISMS OF TSC AND/OR THE DEVELOPMENT OF TREATMENTS AND THERAPIES FOR THE MANIFESTATIONS OF THE DISEASE. 2. THE SCIENCE AND MEDICAL COMMITTEE OF THE BOARD OF DIRECTORS THEN REVIEWS THE GRANT REVIEW COMMITTEE'S CONCLUSIONS AND MAKES FUNDING RECOMMENDATIONS TO THE BOARD OF DIRECTORS. 3. THE BOARD OF DIRECTORS THEN REVIEWS THE RECOMMENDATIONS OF THE SCIENCE AND MEDICAL COMMITTEE AND MAKES FINAL APPROVAL FOR THE FUNDING OF GRANTS. X X X X 0 0 0 1 0 1 0 0 0 STEVEN L ROBERDS CHIEF SCIENTIFIC OFFICER 212421 0 16175 0 258 0 7007 0 24311 0 260172 0 0 0 KARI L ROSBECK PRESIDENT & CEO 196498 3638 17866 331 244 5 6806 0 30738 0 252152 3974 0 0 RICHARD GOLLUB CFO 146390 9344 9972 636 372 24 5207 0 32130 0 194071 10004 0 0 DEAN RAGER-AGUIAR DIRECTOR, PRECLINICAL RESEARCH 157178 0 6379 0 138 0 5180 0 31130 0 200005 0 0 0 PART I, LINE 5 KARI LUTHER ROSBECK, RICHARD GOLLUB, STEVE ROBERDS, DEAN AGUIAR, JAYE ISHAM, LISA MOSS AND APRIL COOPER ALL HAVE INCENTIVE COMPENSATION EQUAL TO A PERCENTAGE OF THEIR SALARIES BASED ON KEY PERFORMANCE OBJECTIVES AS ESTABLISHED BY THE COMPENSATION COMMITTEE. PART I, LINE 6 KARI LUTHER ROSBECK, RICHARD GOLLUB, STEVE ROBERDS, DEAN AGUIAR, JAYE ISHAM, LISA MOSS AND APRIL COOPER ALL HAVE INCENTIVE COMPENSATION EQUAL TO A PERCENTAGE OF THEIR SALARIES BASED ON KEY PERFORMANCE OBJECTIVES AS ESTABLISHED BY THE COMPENSATION COMMITTEE. X 34028 FMV X 724 FMV X 7 250515 FMV 0 0 1 0 PART I, COLUMN (B): REPORTING THE NUMBER OF CONTRIBUTIONS FORM 990, PART III, LINE 4A THE PRECLINICAL CONSORTIUM INVITES COMPOUND NOMINATIONS FROM ACADEMIC AND INDUSTRY RESEARCHERS TO CONTINUE TO REFRESH OUR PIPELINE BASED ON THE LATEST DATA AND NOVEL IDEAS. THE NOMINATIONS ARE PEER-REVIEWED BY CONSORTIUM MEMBERS AND PRIORITIZED BY THE PRECLINICAL CONSORTIUM STEERING COMMITTEE FOR FUNDING BY THE TSC ALLIANCE. IF THE TSC ALLIANCE FUNDS THE STUDY, THE DATA ARE SHARED WITH THE CONSORTIUM FOR TRANSPARENCY AND STIMULATION OF ADDITIONAL HYPOTHESES. AN ANNUAL FACE-TO-FACE MEMBERS MEETING IS TYPICALLY HELD EACH YEAR IN APRIL TO KEEP MEMBERS ENGAGED AND ALIGNED ON THE CONSORTIUM'S STRATEGY AND PRIORITIES. PARTNERSHIP WITH PHARMACEUTICAL COMPANIES IS AN IMPORTANT ASPECT OF THE CONSORTIUM, AS THESE ORGANIZATIONS HAVE THE INFRASTRUCTURE TO EFFICIENTLY MOVE PRECLINICAL RESEARCH FINDINGS TO CLINICAL TESTING AND EVENTUALLY COMMERCIAL DISTRIBUTION. AS OF DECEMBER 2021, 10 COMPANIES WERE ACTIVE MEMBERS OF THE CONSORTIUM. THE CONSORTIUM HAS HAD 17 COMPANY MEMBERS SINCE 2016. THERE IS NATURAL TURNOVER OF INDUSTRY MEMBERSHIP, PRINCIPALLY DRIVEN BY RESEARCH DATA GENERATED BY TESTING THEIR DRUGS. DUE TO INTELLECTUAL PROPERTY CONSIDERATIONS, MOST COMPANIES FULLY FUND THEIR STUDIES THROUGH THE TSC ALLIANCE, WHICH ALLOWS THE COMPANY TO KEEP ITS DATA CONFIDENTIAL. THESE CONFIDENTIAL STUDIES ALSO INCUR A 10% OVERHEAD PAID BY THE COMPANY, WHICH IS A SOURCE OF REVENUE FOR THE TSC ALLIANCE. SINCE INCEPTION, THE CONSORTIUM HAS HELPED ADVANCE THE EVALUATION OF DRUGS FOR TSC BY CONDUCTING 90 STUDIES, EVALUATING 57 CANDIDATE DRUGS. IN 2020, THE TSC PRECLINICAL CONSORTIUM EVALUATED 9 CANDIDATE COMPOUNDS AND CONDUCTED 22 EXPERIMENTS. IN 2012, THE TSC ALLIANCE HELPED CREATE THE TSC CLINICAL RESEARCH CONSORTIUM IN PARTNERSHIP WITH INVESTIGATORS RUNNING CLINICAL STUDIES TO ENSURE CLINICAL RESEARCH IN TSC IS AS EFFICIENT AND EFFECTIVE AS POSSIBLE. SINCE THEN, TSC CLINICAL RESEARCH CONSORTIUM INVESTIGATORS HAVE BEEN AWARDED MORE THAN $39 MILLION BY THE NATIONAL INSTITUTES OF HEALTH (NIH) AND FOOD AND DRUG ADMINISTRATION (FDA) THROUGH COMPETITIVE GRANT PROCESSES. TSC ALLIANCE PERSONNEL SERVE ON THE LEADERSHIP TEAM FOR THE CONSORTIUM, ACTIVELY TRACK ENROLLMENT, AND RAISE COMMUNITY AWARENESS TO HELP IDENTIFY POTENTIAL PARTICIPANTS FOR CLINICAL STUDIES. TSC ALLIANCE ALSO PROVIDES SUPPLEMENTAL FINANCIAL SUPPORT TO ACCELERATE OR EXPAND NIH-FUNDED STUDIES. THE PREVENT TRIAL - PREVENTING EPILEPSY USING VIGABATRIN IN INFANTS WITH TSC - IS THE FIRST PREVENTATIVE TRIAL IN THE UNITED STATES FOR ANY FORM OF EPILEPSY AND HAS COMPLETED ENROLLMENT OF 86 PARTICIPANTS. WE EXPECT THE FIRST RESULTS IN THE FIRST QUARTER OF 2022 AND FINAL RESULTS IN EARLY 2023. THIS TRIAL BUILDS DIRECTLY UPON THE CLINICAL RESEARCH CONSORTIUM'S FIRST CLINICAL STUDY, WHICH IDENTIFIED SPECIFIC EEG CHANGES IN INFANTS WITH TSC PRIOR TO THE ONSET OF SEIZURES. IN THE PREVENT TRIAL, INFANTS WITH TSC RECEIVE REGULAR EEG MONITORING, AND THOSE WHO DEVELOP EEG ABNORMALITIES ARE PLACED ON EITHER VIGABATRIN OR PLACEBO. ANY INFANT WHO SUBSEQUENTLY DEVELOPS CLINICAL SEIZURES IS IMMEDIATELY PLACED ON STANDARD TREATMENT. THIS TRIAL WILL DETERMINE WHETHER TREATMENT WITH VIGABATRIN PRIOR TO THE ONSET OF CLINICAL SEIZURES IN TSC IS BENEFICIAL TO CHILDREN'S DEVELOPMENTAL AND NEUROLOGIC OUTCOMES. MORE DETAILS ABOUT THIS STUDY ARE ONLINE AT WWW.CLINICALTRIALS.GOV/CT2/SHOW/NCT02849457. IMMEDIATELY AFTER THE PREVENT TRIAL ENROLLMENT COMPLETED, A SIMILAR CLINICAL TRIAL BEGAN ENROLLING NEWBORNS WITH TSC BEFORE THE ONSET OF SEIZURES TO TEST THE EFFECTS OF SIROLIMUS ON PREVENTING OR DELAYING SEIZURE ONSET. THE STOPPING TSC ONSET AND PROGRESSION 2 (STOP-2) TRIAL ENROLLED FIVE INFANTS AT CINCINNATI CHILDREN'S HOSPITAL MEDICAL CENTER AS AN INITIAL SAFETY STUDY. THE SECOND STAGE OF THE TRIAL IS NOW CALLED "SIROLIMUS TSC EPILEPSY PREVENTION STUDY (TSC-STEPS) AND THE TSC ALLIANCE HAS CONTRIBUTED $200,000 TO ENABLE THE TRIAL TO BE EXPANDED TO ADDITIONAL SITES ACROSS THE COUNTRY. THE TSC ALLIANCE IS ALSO PROVIDING SUPPLEMENTAL FUNDING AND COLLECTING BLOOD SAMPLES FOR THE DEVELOPMENTAL SYNAPTOPATHIES CONSORTIUM (DSC), AN NIH-FUNDED PROJECT THAT INCLUDES STUDIES OF TSC AND THE RELATED RARE DISORDERS PHELAN-MCDERMID SYNDROME AND PTEN HAMARTOMA SYNDROME. THESE THREE RARE DISEASES SEEM TO AFFECT CERTAIN SHARED PATHWAYS INFLUENCING THE DEVELOPMENT OF BRAIN CONNECTIONS, OR SYNAPSES. RESEARCHERS IN THIS STUDY ARE TRYING TO FIND EARLIER SIGNS OF AUTISM SPECTRUM DISORDER (ASD) AND INTELLECTUAL DISABILITY (ID) TO GAIN A BETTER UNDERSTANDING OF ASD/ID IN INDIVIDUALS WITH TSC AND ENABLE EFFECTIVE TREATMENTS AND INTERVENTIONS FOR ASD/ID TO BE FOUND. THE DSC WAS RENEWED IN 2019 FOR A SECOND 5-YEAR FUNDING PERIOD. THE TSC ALLIANCE FUNDS A PORTION OF CLINICAL RESEARCH COORDINATORS' SALARIES AT 5 SITES. IN 2021, THE TSC ALLIANCE AWARDED A TWO-YEAR CLINICAL RESEARCH TSC-DSC FELLOWSHIP TO FELIX CHAN, PHD, AT BROWN UNIVERSITY. IN ADDITION TO SPECIFIC COLLABORATIVE PROJECTS DETAILED ABOVE, THE TSC ALLIANCE STIMULATES COLLABORATION AND INNOVATION THROUGH CONVENING EXPERTS AND STAKEHOLDERS WITHIN AND OUTSIDE THE TSC FIELD. IN 2020, THE TSC ALLIANCE INTRODUCED THE CONCEPT OF AN AD HOC INNOVATION WORKSHOP FOCUSED ON A SPECIFIC RESEARCH GAP OR OPPORTUNITY. THE FIRST INNOVATION WORKSHOP TOPIC WAS NEWBORN SCREENING, HELD USING ZOOM AND POWERNOODLE IN LATE 2020 AND CULMINATING IN A ZOOM MEETING ON JANUARY 29, 2021. PARTICIPANTS INCLUDED 20 RESEARCHERS WITH TSC EXPERTISE; 19 RESEARCHERS WITH NEWBORN SCREENING EXPERIENCE FROM CDC, NIH, AND ACADEMIC LABORATORIES; AND FIVE REPRESENTATIVES OF NON-TSC ADVOCACY ORGANIZATIONS WITH NEWBORN SCREENING EXPERIENCE. FOLLOWING PEER-REVIEW BY EXPERTS IN TSC AND SCREENING RESEARCH, THE TSC ALLIANCE FULLY FUNDED TWO PROPOSALS TOTALING $164,856 FOR HYPOTHESIS-DRIVEN RESEARCH IN ASSAY DEVELOPMENT FOR EARLY DETECTION OF TSC. THE 2021 VIRTUAL INTERNATIONAL TSC & LAM RESEARCH CONFERENCE: DRIVING DISCOVERIES BEYOND BOUNDARIES HELD OCTOBER 28-30, 2021, WELCOMED 179 PEOPLE FROM 18 COUNTRIES. CO-SPONSORED BY THE TSC ALLIANCE AND THE LAM FOUNDATION, THE CONFERENCE FEATURED THREE PLENARY SESSIONS WITH 17 ORAL PRESENTATIONS AND TWO POSTER SESSIONS WITH 14 POSTERS. ADDITIONALLY, TWO HALF-DAY SESSIONS OF "TOPIC-BASED DISCUSSION" BROUGHT TOGETHER CLINICAL AND BASIC SCIENCE RESEARCHERS TO DISCUSS CROSS-CUTTING TOPICS, INCLUDING BIG DATA AND CLINICAL TRANSLATION. FORM 990, PART VI THE FORM 990 IS REVIEWED, IN DETAIL, BY THE BOARD OF DIRECTORS' AUDIT COMMITTEE. RECOMMENDATIONS ARE MADE BY THE COMMITTEE MEMBERS FOR ANY CHANGES/EDITS/ADDITIONS. AFTER THOSE HAVE BEEN INCORPORATED, THE AUDIT COMMITTEE VOTES WHETHER TO APPROVE AND THEN FORWARD THE 990 TO THE FINANCE AND EXECUTIVE COMMITTEES. THE FINANCE AND EXECUTIVE COMMITTEES PERFORM THE FINAL REVIEW AND THEN VOTE WHETHER TO APPROVE ON BEHALF OF THE BOARD OF DIRECTORS. A COPY OF THE APPROVED 990 IS SHARED WITH THE ENTIRE BOARD PRIOR TO ITS FILING WITH THE IRS. FORM 990, PART VI, SECTION A, LINE 6 MEMBERSHIP IS AVAILABLE TO ANY PERSON WHO SUBSCRIBES TO THE PURPOSES AND OBJECTIVES OF THE CORPORATION, WITHOUT REGARD TO RACE, RELIGION, GENDER, SEXUAL ORIENTATION, AGE, COLOR, NATIONAL ORIGIN OR MENTAL OR PHYSICAL HANDICAP OR DISABILITY. THERE SHALL BE NO LIMIT TO THE NUMBER OF MEMBERS IN THE CORPORATION. 1) THERE MAY BE ONE OR MORE CLASSES OF MEMBERSHIP AS DETERMINED BY THE BOARD. 2) MEMBERSHIP IS NOT TRANSFERABLE OR ASSIGNABLE. FORM 990, PART VI, SECTION A, LINE 7A THE TSC ALLIANCE IS A MEMBERSHIP-BASED ORGANIZATION, WHICH MEANS MEMBERS HELP ELECT THE BOARD OF DIRECTORS. THE TSC ALLIANCE MEMBERSHIP PROGRAM ALLOWS INDIVIDUALS TO STATE THEIR INTENT TO BE A MEMBER FOR THE PURPOSE OF GOVERNANCE. THERE WERE NO LEVELS TO MEMBERSHIP IN 2021. ANYONE CAN BE A MEMBER AT NO COST. FORM 990, PART VI, SECTION B, LINE 11B THE FORM 990 IS REVIEWED, IN DETAIL, BY THE BOARD OF DIRECTORS' AUDIT COMMITTEE. RECOMMENDATIONS ARE MADE BY THE COMMITTEE MEMBERS FOR ANY CHANGES/EDITS/ADDITIONS. AFTER THOSE HAVE BEEN INCORPORATED, THE AUDIT COMMITTEE VOTES WHETHER TO APPROVE AND THEN FORWARD THE 990 TO THE FINANCE AND EXECUTIVE COMMITTEES. THE FINANCE AND EXECUTIVE COMMITTEES PERFORM THE FINAL REVIEW AND THEN VOTE WHETHER TO APPROVE ON BEHALF OF THE BOARD OF DIRECTORS. A COPY OF THE APPROVED 990 IS SHARED WITH THE ENTIRE BOARD PRIOR TO ITS FILING WITH THE IRS. FORM 990, PART VI, SECTION B, LINE 12C ANNUALLY EACH MEMBER OF THE BOARD OF DIRECTORS WILL RECEIVE NOTICE OF THE ORGANIZATION'S CONFLICT OF INTEREST STATEMENT. EACH MEMBER WILL BE PROVIDED WITH A STATEMENT TO MAKE DISCLOSURE OF ANY POTENTIAL CONFLICT OF INTEREST. IF DURING THE COURSE OF THE YEAR A POTENTIAL CONFLICT OF INTEREST ARISES THAT HAS NOT PREVIOUSLY BEEN DISCLOSED, THE BOARD MEMBER WILL MAKE WRITTEN NOTICE OF A POTENTIAL CONFLICT OF INTEREST AND RECUSE HIMSELF OR HERSELF FROM ANY DISCUSSIONS AND VOTES IN CONNECTION WITH THE ISSUE IDENTIFIED. ANY TIME A MEMBER IS RECUSED FROM DISCUSSION ON AN ISSUE, THE MINUTES OF COMMITTEE MEETING AND BOARD MEETING WILL DULY RECORD SUCH ACTIONS. THE FOLLOWING POTENTIAL CONFLICTS OF INTEREST WERE DISCLOSED FOR 2021: BOARD MEMBER MUSTAFA SAHIN, PH.D., M.D., IS EMPLOYED AT BOSTON CHILDREN'S HOSPITAL, WHICH RECEIVED $113,659 IN GRANTS AND $9,015 IN FEES FOR SERVICES FOR PARTICIPATION IN THE TSC NATURAL HISTORY DATABASE. BOARD MEMBER DARCY KRUEGER, MD, MPA, IS EMPLOYED AT THE CINCINNATI CHILDREN'S HOSPITAL MEDICAL CENTER, WHICH RECEIVED A $100,000 GRANT PAYMENT FOR HIS WORK ON STOP-2 AND $8,190 IN FEES FOR SERVICES FOR PARTICIPATION IN THE TSC NATURAL HISTORY DATABASE. BOARD MEMBER JESSICA KREFTING, RN, IS EMPLOYED AT UNIVERSITY OF ALABAMA AT BIRMINGHAM, WHICH RECEIVED $7,715 IN FEES FOR SERVICES FOR PARTICIPATION IN THE TSC NATURAL HISTORY DATABASE. BOARD MEMBER AND CHAIR PETER CRINO, MD, PHD, IS CHAIR OF THE DEPARTMENT OF NEUROLOGY AT THE UNIVERSITY OF MD, WHICH RECIEVED A $18,634 GRANT PAYMENT. FORM 990, PART VI, SECTION B, LINE 15 THE COMPENSATION COMMITTEE REVIEWS AND APPROVES THE SALARIES OF THE PRESIDENT/CEO, CHIEF SCIENTIFIC OFFICER, CFO, AND ANY EMPLOYEE APPEARING ON THE FORM 990, IN ACCORDANCE WITH THE TSC ALLIANCE BYLAWS. SUCH REVIEW AND APPROVAL OCCURS INITIALLY UPON HIRING, UPON ANNUAL REVIEWS, AND WHENEVER MODIFIED. THE ORGANIZATION'S EXECUTIVE REMUNERATION HAS BEEN STRUCTURED TO ENSURE THAT IT: IS REASONABLE; PROVIDES A COMPETITIVE COMPENSATION PROGRAM TO RETAIN, ATTRACT AND REWARD KEY EMPLOYEES AND ACHIEVES CLEAR ALIGNMENT BETWEEN TOTAL REMUNERATION AND DELIVERED BUSINESS AND PERSONAL PERFORMANCE OVER THE SHORT AND LONG-TERMS. THE COMPENSATION IS REVIEWED BY THE COMPENSATION COMMITTEE TO ENSURE: - COMPARABILITY, - PROPER REVIEW, AND - SUBSTANTIATION IN SETTING THE COMPENSATION. FORM 990, PART VI, SECTION C, LINE 19 ALL GOVERNING DOCUMENTS, CONFLICT OF INTEREST POLICY AND FINANCIAL STATEMENTS ARE AVAILABLE UPON WRITTEN REQUEST. TSC ALLIANCE ENDOWMENT FUND INC 8737 COLESVILLE ROAD NO 400 SILVER SPRING MD 20910 521926919 SUPPORT THE MISSION OF THE TSC ALLIANCE MD 501(C)(3) LINE 12B, II N/A 0 0 0 1 0 0 0 0 0 0 0 0 0 0 1 1 0 0 0 0 TSC ALLIANCE ENDOWMENT FUND INC C 226000 CASH