Fibrodysplasia ossificans progressiva (FOP) is a rare disease in which the muscles and connective tissues (e.g. tendons and ligaments) slowly turn into bone. There is no treatment; as the disease progresses, the build-up of bone material around the joints gradually limits patients’ mobility, and can also result in difficulties eating, speaking and even breathing.
The aim of STOPFOP is to see if a drug called AZD0530 could be used to treat FOP. FOP is caused by a mutation in a gene that codes for a protein called ALK2 kinase. Studies in the lab have shown that AZD0530 blocks the action of ALK2 kinase, and in mice, the drug successfully stopped the formation of bone material in the soft tissues and kept the mice’s limbs moving.
AZD0530, also known as saracatinib, has already been tested for safety in humans in healthy volunteers and as a treatment for certain cancers. Now, the STOPFOP team plans to run a clinical trial of AZD0530 in 16 adults with FOP to see if it reduces the formation of new bone. They will work with patients, FOP experts and regulatory authorities throughout the project.