Programs

  • Program 1 [2018]

    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 TS ALLIANCE RESEARCH PROGRAM FUNDS RESEARCH FOCUSED ON TSC PROPOSED BY RESEARCHERS AND ALIGNED WITH THE ORGANIZATION'S MISSION. THE TS ALLIANCE 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.THE TS ALLIANCE HAS FUNDED MORE THAN $20.7 MILLION IN RESEARCH GRANTS ON TSC SINCE 1984. THROUGH THE TS ALLIANCE RESEARCH GRANTS PROGRAM, APPLICATIONS CAN BE SUBMITTED FOR POSTDOCTORAL FELLOWSHIPS AND RESEARCH GRANTS. GRANTS ARE REVIEWED IN A THREE-STEP PROCESS: (1) ALL GRANT APPLICATIONS ARE REVIEWED BY A COMMITTEE COMPRISED OF SCIENTISTS KNOWLEDGEABLE ABOUT THE TOPIC AREA FOR SCIENTIFIC MERIT AND OF ADULTS OR PARENTS 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 TS 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. A TOTAL OF 12 RESEARCH AWARDS WERE FUNDED DURING 2018. THE BOARD OF DIRECTORS APPROVED FUNDING TO BEGIN IN 2018 FOR FOUR MAJOR RESEARCH PROJECTS: (1) DR. YOU FENG (BRIGHAM AND WOMEN'S HOSPITAL) TO STUDY DYSREGULATED PHOSPHATIDYLCHOLINE METABOLISM IN TSC AND LAM PATHOGENESIS AND THERAPY; (2) DR. CHRISTINE OCHOA ESCAMILLA (UNIVERSITY OF TEXAS SOUTHWESTERN MEDICAL SCHOOL) FOR A POSTDOCTORAL FELLOWSHIP TO STUDY NEURAL CIRCUITS UNDERLYING AUTISM-RELATED BEHAVIORS IN TSC; (3) DR. ANGELIQUE BORDEY (YALE UNIVERSITY) TO STUDY NON-CELL AUTONOMOUS EFFECTS IN TSC BRAIN MALFORMATIONS; AND (4) DR. ALAN DOMBKOWSKI (WAYNE STATE UNIVERSITY) TO STUDY THE ROLE OF EXOSOMES IN EPILEPSY OF TSC. THE TS ALLIANCE ALSO FUNDED FOUR SEED GRANTS IN RESPONSE TO A CALL FOR PROPOSALS TO UTILIZE BIOSAMPLES AND DATA IN THE TSC BIOSAMPLE REPSOITORY AND NATURAL HISTORY DATABASE: (1) DR. LAURA FARACH (UNIVERSITY OF TEXAS HEALTH SCIENCE CENTER AT HOUSTON) FOR DEVELOPING A GENETIC RISK PREDICTION MODEL FOR EPILEPSY IN PATIENTS WITH TSC; (2) DR. HILAIRE LAM (BRIGHAM AND WOMEN'S HOSPITAL) TO STUDY MITOCHONDRIAL DNA HETEROPLASMY IN THE PATHOGENESIS AND THERAPY OF TSC; (3) DR. JOANA MARQUES (UNIVERSITY OF PORTO FACULTY OF MEDICINE) TO STUDY EPIGENETIC AND TRANSCRIPTIONAL ANALYSIS OF TSC2 IN LEUKOCYTE DNA FROM TSC PATIENTS; AND (4) DR. JANE YU (UNIVERSITY OF CINCINNATI) FOR QUANTIFICATION OF PLASMA LEVELS OF SPHINGOLIPIDS AND CERAMIDES IN PATIENTS WITH TSC. ALSO IN 2018, WE CONTINUED TO SUPPORT FOUR RESEARCH GRANTS AWARDED IN PREVIOUS YEARS: (1) DR. HENG-JIA LIU (BRIGHAM AND WOMEN'S HOSPITAL) FOR A POSTDOCTORAL FELLOWSHIP TO STUDY T-CELL DYSFUNCTION IN TSC; (2) DR. PETER TSAI (UNIVERSITY OF TEXAS SOUTHWESTERN MEDICAL CENTER) TO STUDY THE IMPACT OF CEREBELLAR-MEDIAL PREFRONTAL CORTICAL CIRCUITS IN TSC; (3) DR. BRENDAN MANNING (HARVARD SCHOOL OF PUBLIC HEALTH) TO STUDY THE REPURPOSING OF INHIBITORS OF PURINE SYNTHESIS FOR TSC TREATMENT; AND (4) DRS. MARK THOMAS AND ROSEMARY EKONG (UNIVERSITY COLLEGE LONDON) TO CURATE ADDITIONAL MUTATIONS IN TSC1 AND TSC2 GENES AND MAKE THEM OPENLY AVAILABLE TO THE PUBLIC IN A DATABASE THAT IS HIGHLY UTILIZED BY GENETIC RESEARCHERS IN TSC.IMPLEMENTED IN 2006, THE TSC NATURAL HISTORY DATABASE CAPTURES CLINICAL DATA TO DOCUMENT THE IMPACT OF THE DISEASE ON A PERSON'S HEALTH OVER THEIR LIFETIME. AS OF DECEMBER 2018, 2,179 PEOPLE WITH TSC WERE ENROLLED IN THE PROJECT FROM AMONG 18 U.S.-BASED SITES. THE TS 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 2018, THE TS ALLIANCE INVESTED $170,893 IN THE TSC NATURAL HISTORY DATABASE, INCLUDING SUB-PROJECTS ON TAND, EPILEPSY, RENAL ANGIOMYOLIPOMAS, AND SUBEPYNDYMAL GIANT CELL ASTROCYTOMAS TO ADDRESS DETAILED QUESTIONS ABOUT THESE LIFE-THREATENING MANIFESTATIONS OF TSC. A CONTRACT WITH NOVARTIS EXECUTED IN NOVEMBER 2012 PROVIDED TS ALLIANCE WITH FUNDING TO ENHANCE AND GROW THE DEPTH OF DATA IN THE TSC NATURAL HISTORY DATABASE THROUGH 2018.THE TSC BIOSAMPLE REPOSITORY IS A TS ALLIANCE-DIRECTED PROJECT INITIATED IN 2014 THAT WILL IMPACT RESEARCH OVER THE NEXT TEN YEARS OR MORE. HIGH-QUALITY BIOSAMPLES SUCH AS BLOOD, DNA, AND TISSUES LINKED TO DETAILED CLINICAL DATA ARE REQUIRED FOR RESEARCHERS TO UNDERSTAND WHY TSC IS SO DIFFERENT FROM PERSON TO PERSON.THE TS ALLIANCE'S SCIENCE AND MEDICAL COMMITTEE IDENTIFIED THIS AS A GAP THAT CAN ONLY BE FILLED EFFECTIVELY WITH LEADERSHIP OF THE TS ALLIANCE, GUIDED BY A STEERING COMMITTEE OF CLINICIANS AND RESEARCHERS. THE TSC BIOSAMPLE REPOSITORY ENDED 2018 WITH 756 BIOSAMPLES: 524 BLOOD, BUCCAL CELL, OR TISSUE SAMPLES FROM INDIVIDUALS WITH TSC ENROLLED IN THE TSC NATURAL HISTORY DATABASE, AND AN ADDITIONAL 232 SAMPLES FROM THE RARE DISEASE CLINICAL RESEARCH NETWORK'S DEVELOPMENTAL SYNAPTOPATHIES CONSORTIUM AND THE PREVENTING EPILEPSY USING VIGABATRIN IN INFANTS WITH TUBEROUS SCLEROSIS COMPLEX (PREVENT) CLINICAL TRIAL. 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, MICHIGAN, UNDER CONTROL OF THE TS ALLIANCE. THE TS ALLIANCE LAUNCHED THE TSC PRECLINICAL CONSORTIUM IN 2015, WHICH ENABLES PRIORITIZATION OF CANDIDATE TREATMENTS BASED ON COMPARING HEAD-TO-HEAD DATA USING CONSISTENT ANIMAL MODELS AND RIGOROUS TESTING PROCEDURES. THE TS ALLIANCE IS IDEALLY POSITIONED TO DRIVE THIS COLLABORATION - WITH INPUT FROM ACADEMIC, REGULATORY, AND INDUSTRY STAKEHOLDERS - BY MAINTAINING CONSTANT FOCUS ON THE NEEDS OF PEOPLE WITH TSC. TO DRIVE THIS PROJECT FURTHER IN 2019 AND BEYOND, THE TS ALLIANCE IN NOVEMBER 2018 HIRED DEAN J. AGUIAR, PHD, AS THE DIRECTOR OF PRECLINICAL RESEARCH. DEAN HAS MORE THAN 17 YEARS OF RESEARCH AND DEVELOPMENT (R&D) LEADERSHIP IN BIOPHARMACEUTICAL AND MEDICAL DEVICE INDUSTRIES, LEADING TEAMS AND TECHNOLOGIES FROM DISCOVERY TO INVESTIGATIONAL NEW DRUG (IND) AND INVESTIGATIONAL DEVICE EXEMPTION (IDE), A PRE-REQUISITE FOR CLINICAL TRIAL EVALUATION. THE TS 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 TS 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. THE CONSORTIUM BEGAN RUNNING EPILEPSY EXPERIMENTS IN AUGUST 2016 AT PSYCHOGENICS, A CONTRACT RESEARCH ORGANIZATION. TUMOR EXPERIMENTS BEGAN IN AUGUST 2017 AT PORSOLT, ANOTHER CONTRACT RESEARCH ORGANIZATION. THREE PHARMACEUTICAL COMPANIES JOINED THE PRECLINICAL CONSORTIUM IN 2018, BRINGING THE TOTAL SINCE INCEPTION TO NINE INDUSTRY PARTNERS, SEVEN OF WHOM REMAIN ACTIVE MEMBERS. THE TSC PRECLINICAL RESEARCH CONSORTIUM RAN 12 STUDIES TO TEST 10 COMPOUNDS IN 2018, COMPARED TO 10 STUDIES AND 8 COMPOUNDS IN 2017. SOME OF THE CONSORTIUM'S RESULTS WERE SHARED BY PIQUR AT THE EPILEPSY PIPELINE CONFERENCE AND BY DR. ANGELIQUE BORDEY AT THE AMERICAN EPILEPSY SOCIETY ANNUAL MEETING. THIS DESCRIPTION IS CONTINUED ON LINE 4D.
    Geographies
    Not indicated
    Dates
    Jan 1, 2018 – Dec 31, 2018
    Source
    990
    No causes provided
    No populations provided
    –
    $2.5M

  • Program 2 [2018]

    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 2018 THE TS ALLIANCE FACILITATED 24,852 PEER-TO-PEER SUPPORTS FROM ADULT REGIONAL COORDINATORS, CLINIC AMBASSADORS, DEPENDENT ADULT TRANSITION RESOURCE COORDINATORS, EDUCATION PARENT MENTORS, AND COMMUNITY ALLIANCES THROUGH THE TSC CONNECT PROGRAM.THE SUPPORT SERVICES DEPARTMENT ALSO PROVIDED DIRECT SUPPORT AND RESOURCES TO 4,083 INDIVIDUALS AND FAMILIES DEALING WITH TSC THROUGHOUT 2018. THE VICE PRESIDENT, SUPPORT SERVICES AND EDUCATION PARENT MENTORS ATTENDED 83 SCHOOL MEETINGS (IEPS, EVALUATION TEAM MEETINGS, 504 PLAN MEETINGS, RESOLUTION MEETINGS, AND MEDIATIONS) IN PERSON, THROUGH SKYPE/ FACETIME, AND VIA CONFERENCE CALLS TO SUPPORT FAMILIES IN ATTAINING EDUCATIONAL SERVICES FOR THEIR CHILDREN THROUGHOUT THE COUNTRY. THROUGH COLLABORATIONS WITH THE PARENT TRAINING AND INFORMATION CENTERS IN 2018, 4,473 FREE PARENT TRAININGS/WEBINARS ON EDUCATIONAL ADVOCACY WERE OFFERED TO FAMILIES IN 36 STATES DEALING WITH EDUCATIONAL ISSUES FOR THEIR CHILDREN. IN ADDITION, 110 SCHOOL SYSTEMS WERE OFFERED THE SUPPORT OF THE EDUCATOR MENTOR PROGRAM IN 2018. THERE WERE 12 SCHOOL SYSTEMS PROVIDED TRAINING ON "TSC 101" FOR SCHOOL DISTRICTS IN AN EFFORT TO HELP THEM UNDERSTAND THE COMPLEXITIES OF TSC AND LEARNING. THROUGH A NETWORK OF 37 VOLUNTEER BRANCHES OF THE ORGANIZATION, CALLED COMMUNITY ALLIANCES, LOCAL EDUCATION AND SUPPORT GROUP MEETINGS WERE HELD THROUGHOUT THE COUNTRY EDUCATING AND SUPPORTING 1,500 FAMILIES/INDIVIDUALS LIVING WITH TSC. THE COMMUNITY PROGRAMS TEAM WAS DEVELOPED IN 2018 WHEN THE COMMUNITY ALLIANCES WERE DIVIDED INTO THREE REGIONS, WITH A COMMUNITY PROGRAMS MANAGER ASSIGNED TO EACH REGION TO MANAGE THE DAY-TO-DAY ACTIVITIES WITHIN THEIR COMMUNITIES. IN 2018, 16 OF 75 LEADERSHIP POSITIONS (37 COMMUNITY ALLIANCE CHAIRS AND 38 WALK CHAIRS) WERE FILLED, AND THREE REMAINED OPEN AS OF DECEMBER 31, 2018. THE 2018 WORLD TSC CONFERENCE WAS CO-HOSTED BY THE TS ALLIANCE AND TUBEROUS SCLEROSIS COMPLEX INTERNATIONAL IN DALLAS, TEXAS FROM JULY 26-29, 2018. THIS EVENT DREW 933 TOTAL ATTENDEES FROM 26 COUNTRIES. NINETY-NINE SCHOLARSHIPS WERE AWARDED TO ASSIST FAMILIES/INDVIDUALS WITH THE COST. THE SUCCESS OF THIS EVENT IS DO TO BOTH THE IN-PERSON AND ONLINE PARTICIPATION. THE CONFERENCE CONSISTED OF FOUR EDUCATIONAL TRACKS: PEDIATRIC, TRANSITION, ADULT, AND SPECIALTY. IN ADDITION, THERE WERE CONCURRENTLY RUN GROUP SESSIONS (4) AND MEET-THE-EXPERT SESSIONS (2). GROUP SESSIONS PROVIDED A CONVERSATIONAL FORUM TO DISCUSS TOPICS RELEVANT TO A SMALLER POPULATION OF THOSE AFFECTED BY TSC (E.G.: WOMEN'S HEALTH ISSUES). MEET-THE-EXPERT SESSIONS PROVIDED A QUESTION-AND-ANSWER FORMAT WITH SIX EXPERTS PER SESSION PARTICIPATING FROM VARIOUS SPECIALTIES. GENERAL SESSIONS TOPICS WERE SELECTED BASED ON HOT TOPICS THAT ARE APPLICABLE TO THE MAJORITY OF TSC INDIVIDUALS AND FAMILIES AND INTERNATIONAL RELEVANCE. GLOBAL OUTREACH WORKS TO ADDRESS UNMET NEEDS WITHIN THE GLOBAL TSC COMMUNITY. THE PROGRAM PROVIDES THE OPPORTUNITY FOR THE TS 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 TS ALLIANCE TO FACILITATE LOCAL CONNECTIONS FOR INDIVIDUALS AND FAMILIES AFFECTED BY TSC, AND RAISE REVENUE AND AWARENESS WHILE SUPPORTING THE MISSION OF THE ORGANIZATION. IN 2018, THE TS ALLIANCE SIGNED A GLOBAL AGREEMENT WITH THE TS ALLIANCE OF INDIA. THE TS ALLIANCE NOW HAS SIX GLOBAL PARTNERSHIPS, INCLUDING: TS ALLIANCE OF ISRAEL, TS CANADA ST, TS ALLIANCE OF MEXICO, TS ALLIANCE FOUNDATION (IN THAILAND), HUNGARIAN FOUNDATION FOR TUBEROUS SCLEROSIS, AND TS ALLIANCE OF INDIA.
    Geographies
    Not indicated
    Dates
    Jan 1, 2018 – Dec 31, 2018
    Source
    990
    No causes provided
    No populations provided
    –
    $1.5M

  • Program 3 [2018]

    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. DURING 2018, THE TS ALLIANCE PRODUCED TWO ISSUES OF ITS NATIONAL MAGAZINE, PERSPECTIVE, WHICH IS MAILED TO APPROXIMATELY 14,000 CONSTITUENTS AS WELL AS POSTED ON THE WEBSITE. THE TS ALLIANCE'S WEBSITE INCREASES AWARENESS AND PROVIDES EXTENSIVE EDUCATION THROUGH AN AVERAGE OF MORE THAN 35,000 UNIQUE VISITORS EACH MONTH. THE TS ALLIANCE ALSO RELIES HEAVILY ON SOCIAL MEDIA TO EDUCATE CONSTITUENTS AND PROMOTE NEW RESOURCES AND EVENTS. ITS FACEBOOK GROUP BOASTS MORE THAN 9,200 MEMBERS, WHILE ITS TWITTER AND INSTAGRAM ACCOUNTS HAVE 2,000-PLUS AND 1,300-PLUS FOLLOWERS RESPECTIVELY. TO INCREASE PUBLIC AWARENESS, THE TS ALLIANCE ONCE AGAIN PARTICIPATED IN TSC GLOBAL AWARENESS DAY ON MAY 15 AS WELL AS TSC AWARENESS MONTH THOUGHOUT MAY; A TSC GLOBAL AWARENESS DAY NEWS RELEASE GARNERED 15.7 MILLION IMPRESSIONS. THE TS ALLIANCE AGAIN HEAVILY PROMOTED INFANTILE SPASMS AWARENESS WEEK, DECEMBER 1 TO 7. THIS AWARENESS CAMPAIGN INCLUDED TARGETED SOCIAL MEDIA OUTREACH AND A SATELLITE RADIO TOUR, WHICH GAINED COVERAGE REACHING A POTENTIAL OF 1.8 MILLION. ADDITIONAL NEWS RELEASES REACHED ANOTHER 76.1 MILLION. EDUCATIONAL SESSION VIDEOS FROM THE ORGANIZATION'S 2018 WORLD TSC CONFERENCE WERE VIEWED 4,510 TIMES.
    Geographies
    Not indicated
    Dates
    Jan 1, 2018 – Dec 31, 2018
    Source
    990
    No causes provided
    No populations provided
    –
    $323K