(Ivanhoe Newswire) -- New research shows gene therapy is
possible and safe to treat patients with rheumatoid arthritis.
Researchers from the University of Pittsburgh conducted
the world’s first human study of gene therapy on patients with rheumatoid
arthritis. The study included nine women who received genetically modified
cells that were injected into their knuckles, where arthritis was rampant.
It was the first time a gene was injected into human joint. The study was
conducted between 1996 and 1999.
Results show gene therapy can target joint inflammation,
and it opens the door for new gene-based therapies for millions of arthritis
sufferers. Researchers from the University of Pittsburgh say they used the
same vector as a recent French study for X-linked severe combined
immunodeficiency. In the French study, three children later developed
leukemia. However, researchers from Pitt say the vector -- a virus that
carries the gene into cells -- is the only similarity between the two
trials. The studies had different targets. In the arthritis trial, the cells
were removed after one week during a routine joint replacement surgery. Pitt
researchers were concerned with the safety but say no clinical side effects
have been reported up to five years later.
Researchers found joints treated with gene therapy did
well. Cells that contained large amounts of the gene were found near a
specific type of tissue and produced significantly less of the
inflammation-provoking substances than cells found in joints that did not
receive the gene.
Authors of the study write, “It is possible to transfer a
potentially therapeutic gene safely to human rheumatoid joints and to obtain
intraarticular, transgene expression. This conclusion justifies additional
efficacy studies and encourages further development of genetic approaches
tot the treatment of arthritis and related disorders.”