After nearly three decades of research, Dr. Christopher Evans, a Mayo Clinic researcher, has made significant progress in developing a new gene therapy targeting osteoarthritis, one of the most common types of arthritis that affects more than 32.5 million adults in the United States alone.
Dr. Evans, director of the Musculoskeletal Gene Therapy Research Laboratory at Mayo Clinic, and a team of 18 researchers and physicians published the results of the first phase 1 human clinical trial of this treatment in the journal Science Translational Medicine.
The results showed that the treatment is safe, results in sustained gene expression within the joint, and has shown preliminary signs of clinical improvement in patients.
"This could revolutionize the treatment of osteoarthritis," said Dr. Evans, explaining that the disease causes the cartilage lining the ends of bones to wear away, and can also affect the bone itself, making it a major cause of disability and difficult to treat.
He noted that drugs injected into the joint are often rapidly removed from the body, limiting their effectiveness. He added, "Gene therapy may be the only way to overcome this significant drug barrier.
The new treatment approach relies on genetically modifying cells in the joint to secrete anti-inflammatory molecules from within the body itself, making the joints more resistant to disease progression.
Laboratory research has identified a key role for a molecule known as interleukin-1 (IL-1) in promoting inflammation, pain, and cartilage loss. Fortunately, this molecule has a natural inhibitor known as interleukin-1 receptor antagonist (IL-1Ra), which has become the basis for proposed gene therapy.
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