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Getting arthritis gene therapy into the clinic

Nature Reviews Rheumatology volume 7, pages 244–249 (2011)Cite this article

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Abstract

Gene transfer technologies enable the controlled, targeted and sustained expression of gene products at precise anatomical locations, such as the joint. In this way, they offer the potential for more-effective, less-expensive treatments of joint diseases with fewer extra-articular adverse effects. A large body of preclinical data confirms the utility of intra-articular gene therapy in animal models of rheumatoid arthritis and osteoarthritis. However, relatively few clinical trials have been conducted, only one of which has completed phase II. This article summarizes the status in 2010 of the clinical development of gene therapy for arthritis, identifies certain constraints to progress and suggests possible solutions.

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Figure 1: Basic concept behind local, intra-articular gene therapy for arthritis.
Figure 2: Local gene therapy for arthritis in clinical trials.

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Acknowledgements

The authors' work in this area has been funded by NIH grants R01 AR43623, R21 AR049606, R01 AR048566, R01 AR057422 and R01 AR051085, and by Orthogen.

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Authors and Affiliations

  1. Center for Advanced Orthopedic Studies, Beth Israel Deaconess Medical Center, Harvard Medical School, 330 Brookline Avenue, RN-115, Boston, 02215, MA, USA

    Christopher H. Evans

  2. Department of Orthopedics and Rehabilitation, Gene Therapy Laboratory, University of Florida College of Medicine, PO Box 100137, Gainesville, 32610–0137, FL, USA

    Steven C. Ghivizzani

  3. Department of Microbiology and Molecular Genetics, University of Pittsburgh School of Medicine, 523 Bridgeside Point II, 450 Technology Drive, Pittsburgh, 15219, PA, USA

    Paul D. Robbins

Authors
  1. Christopher H. Evans
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All authors contributed equally to researching data for the article, providing a substantial contribution to discussion of content, writing the article and review and/or editing of the manuscript before submission.

Corresponding author

Correspondence to Christopher H. Evans.

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Competing interests

C. H. Evans and P. D. Robbins are consultants for, and have received honoraria from, TissueGene. C. H. Evans is on the supervisory board of and owns stock in Orthogen. P. D. Robbins and S. C. Ghivizzani are founders of Molecular Orthopaedics.

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Evans, C., Ghivizzani, S. & Robbins, P. Getting arthritis gene therapy into the clinic. Nat Rev Rheumatol 7, 244–249 (2011). https://doi.org/10.1038/nrrheum.2010.193

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