IFOPA Research Blueprint 2018-2021
20% Basic Research
Answering Priority Research Questions
The IFOPA will support research that addresses key unanswered questions which will have a near term impact for drug discovery, biomarker identification, and/or for identifying key disease processes in FOP that will lead to a curative treatment.
30% Drug Discovery
Advancing Drug Candidates Towards Clinical Trials
The IFOPA will support discovery research that expands the development and identification of treatments that alone, or in combination, will prevent or reverse heterotopic ossification and FOP flares. The IFOPA will build and maintain research infrastructure that reduces the barriers and accelerates drug development.
35% Clinical Trials
Accelerating Drug Development
The IFOPA will invest in the development and validation of tools, as well as the analysis of data, that support conducting rigorous clinical trials, requiring the fewest FOP participants and the shortest duration to achieve definitive results.
15% Clinical Care
Supporting Early Diagnosis and Achieving Optimal Clinical Care
The IFOPA will advance research efforts to minimize time to diagnosis and to ensure optimal clinical care for those living with FOP.
IFOPA Research Strategy 2018-2021
Basic Research
Research Objective: Answering Priority Research Questions
- Establish and maintain a global FOP Biorepository to expand biospecimen access to researchers, enabling the study of FOP biology and testing new treatments
- Fund research that delineates the immunologic mechanisms in FOP
- Support research that identifies disease mechanisms that will lead to drugable targets for FOP and/or are markers of disease progression
- Fund research that further elucidates the mechanisms for muscle biology and regeneration as well as tendon and ligament repair, post bone resection
- Support in research that explores progenitor fate determination into bone (vs muscle)
- Support research that uncovers the genetic and epigenetic modifiers that predict variable phenotypic mosaicism within classic and variant FOP
- (Long-term) Establish a genetic database of FOP patients, which is linked to the FOP Registry, with the goal of looking for genetic modifiers
Drug Discovery
Research Objective: Advancing New Drugs Towards Clinical Trials
- Explore combination treatments that may provide better efficacy, allow for more persistent treatment, or to allow treatment for pediatrics
- Fund research that evaluates repurposed drugs that already have a known safety profile and could be a complementary treatment option
- Invest in preclinical research that investigates drugs’ effectiveness following post-resection of heterotopic bone
- Invest in new therapies that either provide a novel approach to treating FOP or have convincing animal data to warrant further research
- Maintain and ensure unencumbered distribution of FOP mice to test new therapeutic compounds
- Promote the replication of promising preclinical; support research that explores the limitations of existing FOP models or the refinement /development of new models
- (Long-term) invest invalidated research that has the potential to be curative for FOP
Clinical Trials
Research Objective: Accelerating Drug Development
- Support the IFOPA FOP Registry and the resulting data analysis to further our clinical understanding of FOP; ensure that all data covered by sharing agreements is transferred into the registry and available for future researchers
- Support efforts to identify biomarkers capable of measuring and predicting early disease progression and/or treatment response. Encourage research that validates surrogate endpoints that have utility in clinical trials
- Fund research that assesses novel imaging techniques sensitive enough to detect early disease progression
- Promote responsible trial design to maximize benefit for the FOP population (e.g. minimizes patient time per benefit gained in each trial)
- Develop educational resources on clinical trials to better educate and enable trial enrollment decisions
- (Long-term) Encourage bio-pharmaceutical companies to identify genetic or other characteristics that lead to treatment "Responders" from "Non responders“
Clinical Care
Research Objective: Answering Priority Research Questions
- Explore non-genetic contributions to phenotypic heterogeneity in FOP, including the role of vaccination, injury, exercise, dietary influences, etc.
- Support efforts to educate the international and local medical community on FOP to ensure earlier diagnosis and appropriate management; support efforts to advance pediatric screening for FOP
- Fund translational/clinical research that evaluates optimal pain management strategies for FOP
