Gene Therapy for Rheumatoid Arthritis

Filed in archive Gene Therapy on January 27, 2009

Photo courtesy of iStockphoto, Image# 852788

A study published in the journal Human Gene Therapy reports of the first clinical evidence that gene therapy reduces symptoms in patients with rheumatoid arthritis, based on data from two patients. The experiment build on a previous study published by the researchers in 2005 demonstrating that the IL-1Ra gene could be safely transferred to human joints in patients with RA.

By implanting a gene in the affected joint, he was able to stimulate production of a human interleukin-1 receptor antagonist protein, which serves to block actions of the interleukin-1 protein.

"The idea is that by remaining in place, the new gene can continuously block the action of the interleukin-1 within the joints," says Evans. "In essence, the gene becomes its own little factory, continuously working to alleviate pain and swelling."

The study, although involving only 2 patients, shows that the therapy was not only safe, but that it was of therapeutic benefit.