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Will arthritis gene therapy become a clinical reality?

Nature Clinical Practice Rheumatology volume 2, pages 344–345 (2006)Cite this article

Gene therapy suffers the negative perception of being unsafe, expensive and ultimately inapplicable in clinical practice. After more than a decade of prime billing and over 1,020 clinical trials, the first gene-therapy product Gendicine™ (SiBiono GeneTech, Shenzhen, China) is finally being marketed for the treatment of cancer in China. What, then, are the chances of a gene therapy for arthritis in the foreseeable future? In the authors' view, they are actually quite good. In order for this to happen, there are, of course, certain requirements that have to be satisfied: there has to be a clinical need for such a therapy; the therapy will have to be effective, safe and affordable; and there will be substantial funding requirements.

With the advent of new biological and small-molecule therapies, it is reasonable to question the need for yet more drugs for rheumatoid arthritis (RA). The success of these new therapies should not, however, obscure the fact that they are only treatments, and not cures; moreover, only about a quarter of patients treated with biological agents achieve an American College of Rheumatology 70% improvement score.1 Biological treatments are also very expensive, and, because they require frequent intravenous or subcutaneous administration, there are still concerns about systemic side effects that have not been resolved. We suggest that gene therapies for RA will be more effective, cheaper, safer, and will produce a more persistent effect than currently available drugs.

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Authors and Affiliations

  1. Robert Lovett Professor of Orthopedic Surgery and Director of the Center for Molecular Orthopedics, Harvard Medical School, Boston, MA, USA

    Christopher H Evans

  2. Associate Professor in the Department of Orthopedics and Rehabilitation, University of Florida College of Medicine, Gainesville, FL, USA

    Steven C Ghivizzani

  3. Professor in the Department of Molecular Genetics & Biochemistry and the Director of the Viral Vector Core Facility, University of Pittsburgh School of Medicine, Pittsburgh, PA, USA

    Paul D Robbins

Authors
  1. Christopher H Evans
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  2. Steven C Ghivizzani
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  3. Paul D Robbins
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Correspondence to Christopher H Evans.

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Competing interests

The authors are inventors on several patents and patent applications relating to arthritis gene therapy.

CH Evans and PD Robbins are on the scientific advisory board of TissueGene Inc. and Orthogen AG.

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Evans, C., Ghivizzani, S. & Robbins, P. Will arthritis gene therapy become a clinical reality?. Nat Rev Rheumatol 2, 344–345 (2006). https://doi.org/10.1038/ncprheum0215

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