Children with rare bone disease given new hope by Sheffield trial
CHILDREN with a rare form of bone disease are to take part in a major new clinical trial of a promising new drug therapy to be co-ordinated in Sheffield.
Nick Bishop, Professor of Paediatric Bone Disease at Sheffield Children's Hospital, is to run a clinical trial of a drug called risedronate on 60 children aged between four and 17 who have osteogenesis imperfecta, a form of osteoporosis which causes multiple fractures, deformity and stunted growth.
He will be funded by a five-year grant of £345,676 from the Chesterfield-based medical research charity the Arthritis Research Campaign.
Professor Bishop is already leading a clinical trial treating other children with osteogenesis imperfecta using a similar drug called pamidronate, which has been shown to have beneficial effects in strengthening the youngsters' bones.
"The biggest drawback to the pamidronate drug treatment used at the moment is the need for it to be given through a drip over a period of three days every three to four months," explained Professor Bishop, an ARC Senior Research Fellow.
"Many of the children are frightened of needles, perhaps because of bad experiences associated with many surgical procedures that they have undergone, such as limb-straightening.
"Risedronate is taken in tablet form, and as we don't know from any studies so far published what the best dose of this type of treatment is, we will be testing three different dosages in order to identify the best response to the drug with the least amount of side effects."
Over the past ten years, bisphosphonate drugs used to treat adult osteoporosis - such as pamidronate and risodronate - have been used in children with osteogenesis imperfecta with astonishing results.
Professor Bishop has found that the children taking the drugs experience an increase in their bone density of 40 per cent a year - compared to normal children whose bone density growth is just ten per cent. In the most severe cases the youngsters' bone density actually doubled. It also reduced their chronic pain and the number of fractures.
