Gene Therapy for Arthritis

We don't hear as much about RA, short for Rheumatoid arthritis, because new drugs are pretty effective. But their side effects can be serious, so a new gene therapy that only targets problem joints is ideal.

RA is the most common form of arthritis that happens when the body's own immune system attacks the synovium, a membrane that lines our joints. This membrane contains blood vessels which provide nutrients for the cartilage and lubrication in the joint. When the body attacks the synovium, the joint swells and over time, the bones and cartilage there erode. Then the tendons and ligaments that bind the joint weaken and stretch causing deformity. RA's triggers are unknown but it may be hereditary and it's more common in women between the ages of forty and sixty.

One treatment for RA is the drug Humira but since it affects the immune system, some patients have developed life threatening diseases such as tuberculosis. The new therapy, however, only treats the joints. ART-one-oh-two is injected into the affected joints and uses an engineered version of a virus into which researchers placed a gene. This gene encodes human interferon beta.

The gene is turned on or off by an additional piece of DNA that only turns on the gene when there's inflammation in the joint. Once on, interferon Beta is produced which reduces inflammation and progression of RA in that joint.

ART-one-oh-two should wrap up clinical trials by the end of two thousand eighteen. If it's safe and effective, the next hurdle is the price. With nearly a million and a half Americans and five million worldwide suffering from rheumatoid arthritis, hopefully the drug will be affordable enough and insurance companies will pay for it.