Campaign launched to find a cure for rare disease linked to osteoarthritis
Written by Oliver, published 4 months ago.
Liverpool, UK, 18 November 2011 - A campaigning patient group has today launched a five million-pound appeal to fund a ground-breaking clinical trial to test the drug nitisinone as a potential treatment for alkaptonuria (AKU), commonly known as black bone disease.
Although a rare disease, medical researchers at the AKU Society believe an effective treatment for the condition could also open new lines of inquiry into treating the millions of patients in the UK who suffer from osteoarthritis.
“Anything we can do to understand AKU, which is a severe form of arthritis, will undoubtedly help us to understand other forms of arthritis such as osteoarthritis” said Dr Lakshminarayan Ranganath, Medical Director for the AKU Society. “Osteoarthritis is a highly prevalent disease affecting at least eight million people in the UK."(i)
“Normally, clinical trials are designed and developed by pharmaceutical companies as it’s a complicated and expensive business,” said Nick Sireau, AKU Society Chairman, whose two young sons suffer from the rare genetic condition. “No pharmaceutical company as yet has committed to take on the Phase III trial. We hope they will, but if necessary we will take on both the development and the fundraising - and pay for it ourselves.”
The AKU Society will launch the campaign at the opening of its annual conference in Liverpool today, attended by more than 80 medical researchers from around the world. The society believes it could soon lead to the first treatment for AKU, with the potential to improve patients’ quality of life dramatically.
Nick’s hard work could mean his two young sons, diagnosed as babies with AKU, will not have to suffer the debilitating consequences of the disease, which typically leads to severe joint pain, early-onset osteoarthritis, an inability to work and the need for joint replacement surgery in middle age.
Written by Oliver, published 4 months ago.
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