Much research is underway worldwide to find a treatment for rare diseases as FOP. Most research centers are located in the US, UK, Japan, Italy, France, and the Netherlands. Investigators have frequent contact with each other and with FOP patients and their associations.
Compounds have been discovered in recent years that may lead to a medicine and possibly a treatment for FOP.
Research Amsterdam Free University
The FOP Expertise Centrum VUmc in Amsterdam has been researching FOP extensively (see also http://bit.ly/2o1vJRO). In the years 2017/2018 research included:
- Designing and carrying out clinical trials with new medication for people with FOP before and during flare-ups.
- Designing future surgery protocols in FOP persons under the protection of new medication.
- Research of the nature and course of FOP using the 18F sodium fluoride PET/CT scan which can accurately image the level of FOP activity.
- Impact of FOP on other organs such as the heart and lungs.
- Study of cell cultures from skin biopsies in FOP persons showing that FOP increased bone formation, possibly during flare-ups especially. In cell models, old and new medicines are tested for their potential to inhibit bone formation and their clinical applicability.
- Designing animal experiments in mice that have the human FOP gene and the same FOP disease to test promising compounds from cell culture research. Cell biological research using material after removal of teeth and molars in FOP persons, increasing our understanding of the FOP processes in addition to more fundamental general understanding of bone remodeling.
- 2 clinical trials will be initiated with new medications in 2018 (see also https://clinicaltrialsregister.eu/ and particularly https://clinicaltrials.gov/ and enter medicine)
- Palovarotene (MOVE study) see http://bit.ly/2nJOn5i
- REGN2477 (Lumina -1 study) see http://bit.ly/2kObMll
Outline of clinical drug trials in FOP:
Explanation: the following diagram outlines current and upcoming clinical drug trials using the following compounds:
Regn2477 (Anti-Activine A antibodies), Anti-Alk2 antibodies, Saracatinib, Imantinib, Ranamycin, Palovarotene