Studies confirm new treatments for Paget's disease
Sep 5, 2005, 23:56, Reviewed by: Dr.
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"The new treatment quickly gives sufferers a much improved quality of life, and has a much lower rate of relapse than the conventional treatment."
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By The University of Auckland,
Two studies led by researchers at The University of Auckland's Faculty of Medical and Health Sciences have pioneered new, more effective treatment for two forms of Paget's disease - a chronic and often painful bone disease.
The results of the studies, one led by Professor Ian Reid, who heads the University's Bone Research Group, and the other by his colleague, Professor Tim Cundy have been published in separate articles in the latest edition of the prestigious New England Journal of Medicine.
Professor Reid led an international study which confirmed a new treatment for Paget's disease, giving a more rapid, effective and longer-lasting result than existing medication.
Professor Cundy's research focused on Juvenile Paget's disease, a rare genetic bone disease with some similarities to adult Paget's. Having earlier been involved in the identification of the genetic cause of the disease, he used an experimental new treatment that substantially relieved bone pain, improved bone density and reversed the metabolic abnormalities of the disease in two patients.
Paget's disease is the second most prevalent bone disease after osteoporosis and affects up to five percent of the older population in New Zealand. It is caused by a malfunction of the body's regular bone building process and results in bone pain, skeletal deformity, bone fractures, deafness and neurological complications. Generally onset is in people over the age of 40.
Juvenile Paget's disease, although very rare is very debilitating. It usually presents in infancy or childhood and results in progressive deformity, growth retardation and deafness.
"Paget's is very painful, affects mobility and can greatly affect the quality of life for sufferers, which is why new treatments are so worthwhile," Professor Reid says.
Professor Reid's international study pioneered the use of zoledronic acid, and showed that a single injection gave a rapid and long-lasting improvement in bone health.
"The new treatment quickly gives sufferers a much improved quality of life, and has a much lower rate of relapse than the conventional treatment."
A further benefit is that it is much easier on the patients - the conventional treatment with tablets requires patients to take daily oral medication in a strict daily regimen for up to six months.
The study involved nearly 360 patients from 10 countries around the world, including 15 from New Zealand.
Based on the results of the study, the new drug under the brand name, Aclasta, was licensed for treatment of Paget's disease in the European Union in April this year, Canada in June and is currently under regulatory review in the United States.
It has not yet been made available in New Zealand for Paget's disease.
Professor Cundy's patients were a brother and sister, who had been wheel-chair bound with Juvenile Paget's since they were 15. He used an experimental new treatment giving them recombinant osteoprotergerin, the protein that is lacking in sufferers and deficiency of which causes the condition.
"While the disease in these patients was too advanced for any treatment to correct deformity, it made enough of an improvement in their bones, and reduction in bone pain for us to believe it could be very useful in halting progress of the disease in infants and children." 
- New England Journal of Medicine
The University of Auckland
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