- Fibrodysplasia ossificans progressiva (FOP) is a rare, genetic disease in which damaged muscle and soft tissue turn into bone
- It affects just 800 people worldwide and there is currently no known cure
- Three drugs have entered clinical trials as potential treatments for the condition
- They target the genes and proteins responsible for the hyperactive bone growth to prevent – and, in some cases, reverse – it
- One of the drugs is being submitted for FDA approval and could receive it by next year
People with a rare, genetic disorder that turns tissue into bone may finally have a treatment.
Three experimental drugs are currently being tested in human trials as potential therapies for fibrodysplasia ossificans progressiva (FOP).
Developed by research universities and pharmaceutical companies, the drugs target genes and proteins responsible for the hyperactive bone growth to prevent – and, in some cases, reverse – it.
With any luck, one of them could be approved by the Food and Drug Administration (FDA) by next year, reported STAT News.
FOP is a rare, genetic disorder in which damaged muscles and soft tissues – such as ligaments and tendons – regrow as bone.
These new bones eventually form a second skeleton and severely limit sufferers’ ability to move.
It was only in 2006 that researchers discovered FOP is caused by a mutation in the ACVR1 gene, which controls the body’s growth and development of bones and muscles.
Naturally, cartilage is replaced by bone from birth to adolescence. But scientists say that the mutation may leave gene receptors constantly ‘on’, which causes bones to overgrow and joints to fuse together.
Those with the disorder usually experience their first ‘flare-up’ by age 10. Children with FOP start growing extra bones in their neck and shoulders, proceeding through the body down to the feet.
According to the National Center for Advancing Transnational Sciences, the disorder occurs in roughly one in 1.6 million newborns.
There are only about 800 people in the world known to have the condtion.