Orthopedic gene therapy-lost in translation?

Christopher H Evans, S. C. Ghivizzani, P. D. Robbins

Research output: Contribution to journalArticle

25 Citations (Scopus)

Abstract

Orthopedic gene therapy has been the topic of considerable research for two decades. The preclinical data are impressive and many orthopedic conditions are well suited to genetic therapies. But there have been few clinical trials and no FDA-approved product exists. This paper examines why this is so. The reasons are multifactorial. Clinical translation is expensive and difficult to fund by traditional academic routes. Because gene therapy is viewed as unsafe and risky, it does not attract major funding from the pharmaceutical industry. Start-up companies are burdened by the complex intellectual property environment and difficulties in dealing with the technology transfer offices of major universities. Successful translation requires close interactions between scientists, clinicians and experts in regulatory and compliance issues. It is difficult to create such a favorable translational environment. Other promising fields of biological therapy have contemplated similar frustrations approximately 20 years after their founding, so there seem to be more general constraints on translation that are difficult to define. Gene therapy has noted some major clinical successes in recent years, and a sense of optimism is returning to the field. We hope that orthopedic applications will benefit collaterally from this upswing and move expeditiously into advanced clinical trials.

Original languageEnglish (US)
Pages (from-to)416-420
Number of pages5
JournalJournal of Cellular Physiology
Volume227
Issue number2
DOIs
StatePublished - Jan 2012
Externally publishedYes

Fingerprint

Gene therapy
Orthopedics
Genetic Therapy
Technology transfer
Intellectual property
Clinical Trials
Intellectual Property
Technology Transfer
Biological Therapy
Frustration
Industry
Drug Industry
Financial Management
Compliance
Pharmaceutical Preparations
Research

ASJC Scopus subject areas

  • Clinical Biochemistry
  • Cell Biology
  • Physiology

Cite this

Orthopedic gene therapy-lost in translation? / Evans, Christopher H; Ghivizzani, S. C.; Robbins, P. D.

In: Journal of Cellular Physiology, Vol. 227, No. 2, 01.2012, p. 416-420.

Research output: Contribution to journalArticle

Evans, Christopher H ; Ghivizzani, S. C. ; Robbins, P. D. / Orthopedic gene therapy-lost in translation?. In: Journal of Cellular Physiology. 2012 ; Vol. 227, No. 2. pp. 416-420.
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