Organization: PROGERIA RESEARCH FOUNDATION INC
Mission: DISCOVER TREATMENTS & CURE FOR PROGERIA & ITS AGING-RELATED DISORDERS, INCLUDING HEART DISEASE.
Activities: RESEARCH GRANTS: PRF HAS AWARDED 76 RESEARCH GRANTS TOTALING $8.37 MILLION THROUGH PEER REVIEW BY OUR VOLUNTEER MEDICAL RESEARCH COMMITTEE. AWARDS OF UP TO $100,000 PER YEAR, FOR UP TO THREE YEARS, HAVE ALLOWED INNOVATIVE NEW RESEARCH IN PROGERIA TO THRIVE.
PROGERIA DRUG TRIAL: THE PROGERIA RESEARCH FOUNDATION AND CHILDREN'S HOSPITAL BOSTON CONTINUE THEIR PARTNERSHIP FOR CONDUCTING CLINICAL DRUG TRIALS FOR CHILDREN WITH PROGERIA.THE CLINICAL TRIAL TEAM: A TEAM OF PROFESSIONALS AT CHILDREN'S HOSPITAL BOSTON AND BRIGHAM & WOMEN'S HOSPITAL IS WORKING WITH CHILDREN WITH PROGERIA FROM AROUND THE GLOBE. MEMBERS OF THE TEAM HAVE EXPERTISE NOT ONLY IN PROGERIA, BUT ALSO IN THE DRUGS ADMINISTERED IN THE TRIALS.(SEE SCHEDULE O FOR CONTINUATION) THE PROGERIA TRIPLE DRUG TRIALIN 2007, RESEARCHERS IDENTIFIED TWO DRUGS CALLED PRAVASTATIN AND ZOLEDRONATE THAT, WHEN USED IN COMBINATION WITH THE FTI DRUG THAT WAS TESTED IN THE FIRST-EVER PROGERIA CLINICAL DRUG TRIAL, MIGHT PROVIDE AN EVEN MORE EFFECTIVE TREATMENT FOR CHILDREN WITH PROGERIA THAN FTI'S ALONE.IN 2014, THE TRIPLE DRUG TRIAL DESIGN WAS AMENDED BASED ON THE CONTINUAL RE-EVALUATION OF TRIAL PROTOCOL AND THE RESULTS OF THE FIRST TRIAL SHOWING THE EFFECTIVENESS OF ONE OF THE THREE DRUGS TESTED. THE TRIAL TEAM DETERMINED THAT THE BEST COURSE OF ACTION WAS TO CONTINUE STUDYING THE EFFECTS OF LONAFARNIB ALONE FOR A PERIOD OF 2 YEARS, AND EXPAND ENROLLMENT TO UP TO 80 PARTICIPANTS. THE APPROPRIATE APPROVALS WERE SECURED, AND CHANGES IMPLEMENTED AND THE PROGRESS IN 2015-2017 IS DETAILED BELOW. IN 2015, 15 ADDITIONAL TRIAL SUBJECTS ENROLLED FROM 13 COUNTRIES SPEAKING 10 DIFFERENT LANGUAGES. IN 2016, 3 ADDITIONAL TRIAL SUBJECTS ENROLLED FROM 3 COUNTRIES. DURING 2017, PRF AND THE TRIAL TEAM CONTINUED TO WORK WITH OTHER FAMILIES TOWARD TRIAL PARTICIPATION, INCLUDING SEVERAL NEWLY DIAGNOSED CHILDREN. THE PROGERIA 2-DRUG, PHASE I/II TRIALIN 2015, PRF OBTAINED ALL NECESSARY APPROVALS FOR ITS THIRD CLINICAL TRIAL WHICH WILL ASSESS A TWO-DRUG COMBINATION OF LONAFARNIB AND EVEROLIMUS. EVEROLIMUS IS A FORM OF THE DRUG RAPAMYCIN, BUT EVEROLIMUS CAN BE MORE EASILY GIVEN TO CHILDREN WITH PROGERIA. EVEROLIMUS TARGETS A DIFFERENT PATHWAY THAN LONAFARNIB AND THE TRIAL IS DESIGNED TO DETERMINE IF THE COMBINATION PROVIDES A BETTER TREATMENT THAN LONAFARNIB ON ITS OWN. THE PROGRESS OF THIS TRIAL IN 2016 IS DETAILED BELOW.IN 2016, 15 TRIAL PARTICIPANTS ENROLLED IN PHASE I OF THIS TRIAL FROM 6 COUNTRIES SPEAKING 6 DIFFERENT LANGUAGES. THE PURPOSE OF A PHASE I TRIAL IS TO DETERMINE THE SAFEST MAXIMUM DOSAGE OF A DRUG.IN 2017, THE 17 CHILDREN THAT PARTICIPATED IN PHASE I OF THE DRUG TRIAL HAVE BEED MOVED ONTO PHASE 2. PHASE II OF THE TRIAL TESTS THE EFFECTIVENESS OF THE 2-DRUG COMBINATION. THE ENROLLMENT OF ADDITIONAL CHILDREN TOOK PLACE IN JULY OF 2017. 39 CHILDREN FROM 18 DIFFERENT COUNTRIES TRAVELED TO BOSTON TO PARTICIPATE IN THE DRUG TRIAL.
FUTURE TRIAL EFFORTS: PRF ENGAGES MEMBERS IN THE SCIENTIFIC COMMUNITY WITH EXPERTISE IN VARIOUS DRUGS AND/OR GENETIC THERAPIES FOR THE TREATMENT AND POTENTIAL CURE FOR PROGERIA. THE INVESTIGATION AND COLLABORATIVE EVALUATION OF DRUG THERAPIES IS PURSUED TO IDENTIFY DRUG CANDIDATES/GENETIC THERAPIES THAT CAN LEAD TO FUTURE CLINICAL TRIALS.
NTEE category: Medical Research
Secondary category: Voluntary Health Associations & Medical Disciplines