The Safety and Efficacy of a Novel Cell-Based Gene Therapy for Knee Osteoarthritis

A promising new gene technology has been developed for the treatment of osteoarthritis, utilizing transduced human cells expressing transforming growth factor-β1. The safety and efficacy of this treatment modality has been demonstrated in laboratory studies, as well as Phase I, II, and current Phase III human clinical trials. Due to a misidentification error, there have been concerns that this cell-based gene therapy is based on a different cell than the one that was initially approved. However, its safety profile has been demonstrated by over 10 years of data revealing no evidence of tumorigenicity or other long-term safety concerns. In all studies to date, there have been no treatment-related serious adverse events. Although the nomenclature of one component of the drug product has changed, the product itself has not. In this review, we will present the technology, history of use (animal and clinical studies), development, efficacy, and, despite the recent misidentification error, the overall safety of this treatment modality.