A promising gene therapy treatment approach to osteoarthritis

What is it about?

The present study evaluated a novel treatment for osteoarthritis. A gene previously implicated in cartilage regeneration (FGF18) was delivered to joints of healthy rodents using a viral vector. The treatment appeared safe and durable over a broad range of doses. The delivered therapy appeared localized to the joint and upregulated a number of cartilage repair genes, while downregulating genes associated with osteoarthritis and cartilage degeneration. The treatment had a modest increase on the thickness of healthy cartilage, which was mainly apparent after the cartilage thickness was normalized by the weight of the animal. This finding suggests that the FGF18 gene therapy approach may be safe, localized, and durable when delivered directly to the joints. In parallel, cartilage thickness may be auto-regulated to prevent excessive overgrowth of tissues.

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Photo by Eagle Media Pro on Unsplash

Photo by Eagle Media Pro on Unsplash

Why is it important?

Osteoarthritis affects 1 in 7 adults and is estimated to be the most prevalent unmet need in rheumatology. With nearly 500 million people affected worldwide, there is currently no disease modifying therapy available. Many patients will eventually progress to terminal joint replacement surgery, which is not only expensive, but is itself associated with secondary side-effects. The current approach offers a potential single injection treatment for osteoarthritis, or at least a means to meaningfully delay terminal joint replacements. Further studies will be required to assess the efficacy of the treatment in diseased joints and in clinical trials.

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