Gene Therapy: A Breakthrough for Rheumatoid Arthritis Pain Relief
Gene Therapy Reduces Rheumatoid Arthritis Pain
A team of scientists used gene therapy to significantly reduce joint pain in two patients with rheumatoid arthritis (RA). This data is “the first documented clinical evidence of local gene therapy to relieve symptoms of RA in humans,” according to Dr. Christopher H. Evans in the journal Human Gene Therapy.
Understanding Rheumatoid Arthritis
RA occurs when, for unknown reasons, the immune system attacks the body and causes inflammation in the joints. If left unchecked, the tissues of the joint begin to collapse.
RA has no cure and is estimated to affect more than 2 million people in the U.S. alone.
“RA is an extremely painful disease that affects multiple joints in the body. Arthritis is a good target (for gene therapy) because the joints are a confined area that can be injected genes,” said Evans, Harvard Medical School in Boston.
Previous studies have shown that the interleukin-1 molecule plays a key role in breaking down cartilage in patients with arthritis.
The Gene Therapy Approach
In the new study, researchers removed tissue from the knuckle joints of two patients with severe RA and injected a harmless virus into the cells of the tissue. This virus acted as a “vector” to carry a gene that inhibits the action of the protein interleukin-1 into the joint.
After a phase of cell growth and division, the researchers injected the modified cells back into the damaged joints.
One patient who received gene therapy in two joints felt 85 percent less pain in one joint during the day. Both joint pain disappeared after the first week.
“It was amazing,” the researchers said, noting that the therapy protected the joints from disease outbreaks during the study.
The second patient also responded to gene therapy, experiencing a 70 percent decrease in pain between the second and third week.
“The treatments for RA are expensive and must be administered regularly,” said Evans, adding that not all patients respond well, and there can be adverse effects.
“This study provides the first evidence that painful symptoms can be controlled by gene therapy,” he concluded.