Children with fibrodysplasia ossificans progressiva (FOP) experience painful episodes of bone formation at extra-skeletal sites, such as tendons and ligaments, eventually resulting in life-threatening complications. Unfortunately, to date there is no cure for FOP. With the support of AFM-Telethon, Duffhues and his collaborators have established cultures of circulating endothelial cells from control and FOP donors during routine blood tests. They found strong evidence pointing at an aberrant behavior of endothelial cells in FOP patients, in favor of bone formation at extra-skeletal sites. The Trampoline grant will allow them to combine a unique patient-derived endothelial cell-model with state-of-the-art organ-on-a-chip platforms and to perform transcriptomic and proteomic assays to identify novel biomarkers and therapies for FOP.
On February 18th, Gonzalo Sánchez-Duffhues was awarded a €50.000 Trampoline grant for his project on fibrodysplasia ossificans progressiva by the French Muscular Dystrophy Association (AFM-Telethon).