Children with rare bone disease set to benefit from arthritis charity grant
YOUNGSTERS suffering from a very rare form of the bone-thinning disease, osteoporosis, are set to benefit from a major grant of £238,787 from leading medical research charity the Arthritis Research Campaign.
A three-year Senior Research Fellowship has been awarded to the new Professor of Paediatric Bone Disease at the University of Sheffield, Professor Nick Bishop.
Professor Bishop, aged 39, is to set up two clinical trials at the Children's Hospital involving about 100 of the 200 children in the UK who are severely affected by osteogenesis imperfecta, an inherited form of osteoporosis. Children with OI from all over the country are treated at the Children's.
Children born with the condition suffer from recurrent fractures which often result in severe deformities and growth stunting. Around half are in wheelchairs by their teens.
The clinical trials will test a new group of drugs only recently used for children with osteoporosis, the bisphosphonates, which have been used successfully with adult patients for about 20 years. Doctors will test the effectiveness of the drugs in tablet and injection form.
"These drugs are a major advance in the treatment of youngsters with osteoporosis, they slow down the rate at which bone is eaten away, and they may actually help to re-build bone," explained Professor Bishop, who recently arrived in Sheffield from Addenbrookes Hospital in Cambridge.
"Preliminary results are very encouraging, with reductions in fracture rates and increased density in many of the children studied," he said. Many more youngsters are at risk of fractures and disability because of their having to take steroids over long periods of time because of arthritis and other inflammatory diseases. "We hope that these studies will be extended to children with steroid-induced osteoporosis in due course."
Although osteoporotic conditions are the main focus of Prof Bishop's work, inherited forms of rickets and conditions which affect the growth of bone and cartilage leading to very short stature will also be targeted.
In the longer term, genetic therapy aimed at correcting the fundamental defects, which result in a wide variety of bone diseases, is planned . "However, much work will be needed to turn that dream into a reality," added Prof Bishop.
The ARC currently funds nearly £2m worth of research in Sheffield.
