Since the discovery of the causative gene (ACVR1) in myositis ossificans or fibrodysplasia ossificans progressiva (FOP), several therapeutic avenues have been explored, including the potent ALK2/ACVR1 kinase inhibitor saracatinib:
- this drug has the advantage of having been developed for the treatment of ovarian cancers with a good safety profile;
- the current trial, codenamed STOPFOP, is a double-blind, randomized phase IIa study and is being conducted in Europe (but not in France);
- the investigators hope to decrease the volume of heterotopic bone formations as evaluated by a bone scan;
- twenty patients with FOP are being enrolled in the study, which will last six months.
