Researchers from the University of Barcelona and IDIBELL describe a new treatment to cope with two bone diseases
Researchers of the Faculty of Medicine and Health Sciences of the UB and the Bellvitge Biomedical Research Institute (IDIBELL) described the ability of an inhibitor of PI3Kα (BYL719) to block the ectopic bone formation in mice models. The study was led by the expert Francesc Ventura, professor from the Department of Physiological Sciences of the Faculty of Medicine and Health Sciences of the UB and head of the Research Group on Cell Signalling and Bone Biology at IDIBELL.
Researchers of the Faculty of Medicine and Health Sciences of the UB and the Bellvitge Biomedical Research Institute (IDIBELL) described the ability of an inhibitor of PI3Kα (BYL719) to block the ectopic bone formation in mice models. The study was led by the expert Francesc Ventura, professor from the Department of Physiological Sciences of the Faculty of Medicine and Health Sciences of the UB and head of the Research Group on Cell Signalling and Bone Biology at IDIBELL.
The results of the new study, published in the journal EMBO Molecular Medicine, could contribute to improve the treatment of patients with heterotopic ossification of fibrodysplasia ossificans progressiva.
The heterotopic ossification (OH) is a pathology which shows the apparition of ossifications in inappropriate places (ectopic formation), such as tendons, muscles and connective tissue. Some risk factors associated with this pathology are prolonged immobility, spinal injuries, burns, hip operations and muscle trauma. “Despite having this knowledge, we do not have a complete understanding on the causes nor the proper treatment for this disease”, notes Professor Francesc Ventura.
Moreover, fibrodysplasia ossificans progressiva (FOP) is a rare disease generated by mutations in the ACVR1 gene, which encodes bone growth factor receptors (BMPs). In this pathology, which has no treatment at the moment, the progressive formation of bones in muscles, tendons and ligaments reduces the mobility and life expectancy of the affected patients.
“What we saw in the study is that, in culture stem cells as well as preclinical models of this disease, the inhibitor BYL719 prevents the ectopic bone formation induced by the mutations of the ACVR1 gene”, comments Francesc Ventura. “In this study, we showed this inhibitor was approved by the U.S Food and Drug Administration (FDA) in May to treat metastatic breast cancer and it can play an important role in the ossification and treatment of these bone diseases”.
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