Dr Antonella Forlino of the Department of Molecular Medicine, Biochemistry Unit, University of Pavia, Italy – Development of a novel combined anabolic and anti-catabolic bone specific drug to treat OI.
No definitive cure is available for OI and novel therapies are an urgent patients’ need. In the last decade, it became clear that the skeletal deformity and fragility associated to reduce bone mineral density, typical features of the disease, are not only due to the abnormal type 1 collagen in the bone matrix. The compromised function of bone forming cells, caused by intracellular retention of the mutant protein, is also contributing to OI severity. Thus, targeting simultaneously the cellular compartments and the bone matrix seems promising. We aim to develop and in vivo test a new drug that will combine the stimulation of osteoblasts protein secretion to the bone resorption reduction by conjugating the chaperone 4-phenylbutyrate to the alendronate.
