SOLVING KIDS' CANCER INC

SKC AND OUR ADVISORS SURVEY, ACTIVELY DISCUSS, AND PRIORITIZE THE AREAS OF GREATEST UNMET NEEDS WITHIN THE PEDIATRIC CANCER RESEARCH LANDSCAPE AND DEVELOP SPECIFIC PROGRAMS TO ADDRESS THEM. (1) THE PH I TITAN CLINICAL TRIAL IS PROVIDING AN EFFECTIVE TARGETED DRUG FOR CHILDREN WITH ALK MUTATIONS IN FRONTLINE TREATMENT WHEN NEWLY DIAGNOSED AND IS GENERATING CRITICAL DATA TOWARDS AN APPROVAL FOR USE AS A STANDARD OF CARE. WORKING TOWARDS HAVING THIS BE ONE OF THE FIRST TARGETED AGENTS BECOMING APPROVED FOR HR NB AND UTILIZED AS A COMBINATION THERAPY STRATEGY, MAKING A SIGNIFICANT IMPACT ON OVERALL SURVIVAL. (2) OUR COLLABORATION WITH THE NATIONAL CANCER INSTITUTE TO VALIDATE AND DEVELOP AN IMMUNOTHERAPY TREATMENT FOR CHILDREN WITH ETMR AND MEDULLOBLASTOMA BRAIN TUMORS USING A T CELL CAR TO TARGET GPC2 IS THE FIRST EVER IMMUNOTHERAPY RESEARCH PROGRAM SPECIFIC FOR ETMR BRAIN TUMORS. IF THE RESULTS ARE POSITIVE THIS WORK WILL BE RAPIDLY TRANSLATED INTO A CLINICAL TRIAL TO INCLUDE CHILDREN WITH ETMR AS WELL AS MEDULLOBLASTOMA USING THIS CUTTING-EDGE IMMUNOTHERAPY. (3) THE PH I CLINICAL TRIAL USING A TRIPLET COMBINATION HAS BEEN A LIFELINE FOR CHILDREN WITH NEUROBLASTOMA WHO RELAPSE AND THE STUDY HAS BEEN SO SUCCESSFUL THAT IT WILL STAY OPEN TO ACCRUE EVEN MORE PATIENTS THAN ORIGINALLY PLANNED. WHILE THE CLINICAL TRIAL IS PROVIDING TREATMENT BENEFIT TO PATIENTS, THE TRIAL IS GENERATING VERY IMPORTANT DATA THAT CAN BE USED TO FURTHER ADVANCE THIS TRIPLE TREATMENT COMBINATION INTO MORE POINTS IN THE TREATMENT CONTINUUM FOR EVEN GREATER PATIENT BENEFIT. (4) SKC TARGETS ONLY THE MOST CHALLENGING AND POOREST OUTCOME CHILDHOOD CANCERS THAT REQUIRE SIGNIFICANT HELP AND HAVE THE GREATEST UNMET NEED INCLUDING HIGH-RISK BRAIN TUMORS AND SOLID TUMORS. (5) TAKES A GLOBAL APPROACH TO SUPPORTING PROGRAMS TO LEVERAGE THE COLLECTIVE POWER OF COLLABORATION ACROSS SEVERAL CENTERS-OF-EXCELLENCE TO BREAK DOWN BARRIERS, SPEED PROGRESS AND HAVE MORE CHILDREN ACCESS PROMISING TREATMENTS. (6) PRIORITIZES ADVANCING TREATMENT OPTIONS THAT HAVE THE HIGHEST POTENTIAL FOR CURATIVE RESPONSES RATHER THAN PARTIAL TUMOR SHRINKAGE OR TEMPORARY LIFE EXTENSION. (7) TAKES LEADERSHIP ROLES WITHIN INFLUENTIAL NATIONAL AND INTERNATIONAL RESEARCH GROUPS' ADVISORY BOARDS AND CO-AUTHOR INFLUENTIAL PAPERS IN PROFESSIONAL JOURNALS TO DRIVE NEW DIRECTION TO POSITIVELY IMPACT PATIENTS. (8) BUILD COALITIONS AND PARTNERSHIPS WITH OTHER NONPROFITS IN COLLABORATIVELY DRIVING AND FUNDING RESEARCH PROJECTS FOR GREATER IMPACT AND SCALE BECAUSE WE ARE MUCH MORE EFFECTIVE TOGETHER THAN WORKING ALONE. SKC PRIORITIZES THE DELIVERY OF TRANSFORMATIVE, NEXT-GENERATION CANCER THERAPIES TO THIS POPULATION THAT INCLUDES IMMUNOTHERAPY, VACCINES, CELLULAR THERAPY, ONCOLYTIC VIROTHERAPY, THERAPEUTIC ANTIBODIES, CHECKPOINT INHIBITORS, SYNERGISTIC COMBINATION TREATMENTS, NEW GENOMICALLY TARGETED DRUGS, INTERVENTIONAL RADIOLOGY TECHNIQUES AND NOVEL DELIVERY MECHANISMS. OUR CRITERIA FOR IDENTIFYING AND SUPPORTING THESE NOVEL APPROACHES AIMS TO PROVIDE MORE EFFECTIVE AND LESS TOXIC TREATMENT OPTIONS THAN THE CURRENT STANDARD-OF-CARE. (9) WE ARE PLANNING A NEW INITIATIVE FOR MEDULLOBLASTOMA TO IDENTIFY PROMISING COMBINATION THERAPIES THAT CROSS THE BLOOD-BRAIN BARRIER AS WELL AS NEW DELIVERY MECHANISMS.