Gene Therapy Test Shows Arthritis Relief in Two Women

The first clinical test to find that gene therapy helps people with rheumatoid arthritis showed two women had less pain and reduced swelling in their hands after receiving the treatment, Harvard University researchers said.

The patients’ hands were injected with tissue containing genes, carried by a retrovirus capable of invading host cells. The therapy was designed to integrate the genetic material with the cells in knuckles, according to the report in the February issue of Human Gene Therapy.

While the gene therapy was used in only two people over four weeks, the results suggest a new strategy for treating rheumatoid arthritis. Earlier research found that patients treated with drugs for relief often continue to experience pain and swelling after taking the medicines, including Abbott Laboratories’ Humira, Johnson & Johnson’s Remicade and Amgen Inc.’s Enbrel.

“There are finally some green shoots coming up in gene therapy,” said Christopher Evans, author of the study and a professor of orthopedic surgery at Harvard Medical School in Boston in a telephone interview today. “The idea is to treat arthritic joints by genetically modifying them so they make their own treatments.”

The study was funded by the National Institutes of Health and closely held Orthogen LLC. Rheumatoid arthritis is a disabling, painful disease of the joints that affects 1.3 million Americans, according to the U.S. Centers for Disease Control and Prevention.

Rheumatoid arthritis causes swelling and stiffness in the joints, as the body’s immune system begins to attack the joint tissue. Doctors don’t know what causes the ailment, according to the National Institutes of Health.

Blocking Inflammation

In the study, the genes were implanted in tissue taken from the knuckles of the two patients, and injected back into the joints. The genetic material helped create a protein that blocked cytokine interleukin-1, a molecule that causes inflammation. That worked to ward off swelling and pain, the study authors said.

“In one of the two subjects, the effects were dramatic, and the gene-treated joints remained pain-free even though other joints experienced flares,” Evans said in a statement. “In essence, the gene becomes its own little factory, continuously working to alleviate pain and swelling.”

The patients in the report were scheduled for surgery to remove some of their joint tissue after four weeks of gene therapy, the authors said. For this reason, it isn’t clear how long the effects of the treatment may last. No side effects were reported in the study.

The scientists tested the tissue removed from the patients’ hands and found that after gene therapy, there were smaller amounts of disease-related proteins. Future work will focus on gene therapy as a possibility for osteoarthritis as well as rheumatoid arthritis.