Promise of gene therapy for arthritis

Reported by Susan 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

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