Hope that a cancer drug can stop FOP, one of the rarest diseases in the world
STOPFOP are trialling the cancer drug saracatinib, which has shown promise in stopping muscle and tissue from turning to bone in FOP patients
The ACVR1 gene. Image by By Pleiotrope (public domain via Wikipedia )
Fibrodyplasia ossificans progressiva (FOP) is a rare and devastating disease in which muscles, tendons and ligaments slowly turn to bone. People with FOP gradually get ‘locked in’ their bodies, making it difficult to move and breathe. It’s caused by a mutation in a single gene, called ACVR1, which encodes for the protein kinase ALK2. The mutation causes ALK2 to become overactive, causing muscles and connective tissues to turn into bone. There is no drug to treat this disease.