Promise of gene therapy for arthritis

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Promise of gene therapy for arthritis

Reported by Aldridge, PhD, medical journalist

Gene therapy is both safe and feasible for arthritis, according to a

trial in a group of patients with advanced disease.

Delivering genes into the body has the potential for curing or at least

alleviating many different diseases. A team at the University of

Pittsburgh now reports on the first trial of gene therapy for

rheumatoid arthritis, using a gene that can target the underlying

inflammatory process.

Nine women with advanced rheumatoid arthritis had genetically modified

cells introduced into their knuckles - the first time a gene had ever

been introduced into a human joint. The women were followed up for five

years to check for any safety issues linked to the therapy. The gene

used blocks the action of interleukin-1, a molecule that helps trigger

the inflammatory process in rheumatoid arthritis.

Tests showed the gene to be active in the patients' joints. In fact,

these patients had been scheduled for joint replacement surgery so the

treated knuckles were actually removed during this process and studied

in the laboratory. Five years on, the patients have not suffered any

adverse effects. The next step is to conduct some trials where the gene

is injected and the patients followed up to see if their arthritis

improves. So far, the gene therapy approach looks promising.

Source

Proceedings of the National Academy of Sciences online 6th June 2005