Two of the therapeutic avenues explored in fibrodysplasia ossificans progressiva (FOP) have been the subject of recent publications:
- a report of four cases of administration of interleukin-1 inhibitors (anakinra and canakinumab) for up to six years in patients aged between 23 months and 15 years, with a significant reduction in disease flare-ups from 1.51 to 0.36 per month, on average (i.e. a 61% to 89% reduction depending on the patient), with no major adverse effects;
- the preclinical results of fidrisertib, a small molecule inhibitor of the ACVR1 (or ALK2) gene which, when administered orally on a daily basis in a mouse model of FOP, prevents the formation of heterotopic ossifications and pinch-induced edema.
Fidrisertib is currently being evaluated in the Falkon clinical trial, which is taking place in France.
