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Gene therapy demonstrates benefit in patients with rheumatoid arthritis
Date:1/26/2009

But, he adds, once he had this answer, another question was not far behind: How could he effectively reach the joints to block the actions of this protein?

Gene therapy provided the answer.

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."

In 2005, in a study published in the Proceedings of the National Academy of Sciences (PNAS), Evans and colleagues demonstrated that the IL-1Ra gene could be safely transferred to human joints in patients with RA. In this new paper, the authors aimed to prove that the therapy was not only safe, but that it was of therapeutic benefit.

Two study subjects were recruited. (The number reduced from six study subjects following severe adverse events in an unrelated gene therapy trial taking place elsewhere, according to Evans.) Both subjects were postmenopausal females under the age of 75 with a diagnosis of advanced rheumatoid arthritis. After tissue was removed from the subjects' knuckle joints, a harmless retrovirus was inserted into the tissue cells, in order to serve as a "vector" to transport the gene into the patients' joints. After being placed in culture to grow and replicate, the cells were injected back into the afflicted joints.

After four weeks, patients reported reduced pain and swelling, according to Evans. "In one of the two subjects, these effects were dramatic, and the gene-treated joints remained pain-free even though other joints experience flares." Subsequent laboratory tests showed that tissues removed from the subject's joint tissue synthesized lower amounts o
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Contact: Bonnie Prescott
bprescot@bidmc.harvard.edu
617-667-7306
Beth Israel Deaconess Medical Center
Source:Eurekalert

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