title: A placebo-controlled randomised trial to assess the effect of TGF-ß1-expressing chondrocytes in patients with arthritis of the knee.
abstract: The aim of this study was to assess the effect of injecting genetically engineered chondrocytes expressing transforming growth factor beta 1 (TGF-β1) into the knees of patients with osteoarthritis. We assessed the resultant function, pain and quality of life. A total of 54 patients (20 men, 34 women) who had a mean age of 58 years (50 to 66) were blinded and randomised (1:1) to receive a single injection of the active treatment or a placebo. We assessed post-treatment function, pain severity, physical function, quality of life and the incidence of treatment-associated adverse events. Patients were followed at four, 12 and 24 weeks after injection. At final follow-up the treatment group had a significantly greater improvement in the mean International Knee Documentation Committee score than the placebo group (16 points; -18 to 49, vs 8 points; -4 to 37, respectively; p = 0.03). The treatment group also had a significantly improved mean visual analogue score at final follow-up (-25; -85 to 34, vs -11 points; -51 to 25, respectively; p = 0.032). Both cohorts showed an improvement in Western Ontario and McMaster Osteoarthritis Index and Knee Injury and Osteoarthritis Outcome Scores, but these differences were not statistically significant. One patient had an anaphylactic reaction to the preservation medium, but recovered within 24 hours. All other adverse events were localised and resolved without further action. This technique may result in improved clinical outcomes, with the aim of slowing the degenerative process, leading to improvements in pain and function. However, imaging and direct observational studies are needed to verify cartilage regeneration. Nevertheless, this study provided a sufficient basis to proceed to further clinical testing.
authors.affiliation: Department of Orthopedic Surgery, Seoul National University Hospital, Seoul National University College of Medicine, Korea.
authors.forename: M-C
authors.initials: MC
authors.lastname: Lee
authors.affiliation: Samsung Medical Center, 81 Irwon-Ro Gangnam-gu, Seoul, Republic of Korea.
authors.forename: C-W
authors.initials: CW
authors.lastname: Ha
authors.affiliation: Sinai Hospital of Baltimore, Baltimore, Maryland 21215, USA.
authors.forename: R K
authors.initials: RK
authors.lastname: Elmallah
authors.affiliation: Sinai Hospital of Baltimore, Baltimore, Maryland 21215, USA.
authors.forename: J J
authors.initials: JJ
authors.lastname: Cherian
authors.affiliation: Kolon Life Science Inc, 1-22 Byeoryang-dong Gwacheon-si, Gyeonggi-do, 427-709, Seoul, Republic of Korea.
authors.forename: J J
authors.initials: JJ
authors.lastname: Cho
authors.affiliation: Kolon Life Science Inc, 1-22 Byeoryang-dong Gwacheon-si, Gyeonggi-do, 427-709, Seoul, Republic of Korea.
authors.forename: T W
authors.initials: TW
authors.lastname: Kim
authors.affiliation: Asan Medical Center, 88, Olympic-Ro 43-Gil, Songpa-Gu, Seoul 138-736, Republic of Korea.
authors.forename: S I
authors.initials: SI
authors.lastname: Bin
authors.affiliation: Sinai Hospital of Baltimore, Baltimore, Maryland 21215, USA.
authors.forename: M A
authors.initials: MA
authors.lastname: Mont
mesh_terms: D000203:Activities of Daily Living; D000368:Aged; D019902:Chondrocytes / Q000378:metabolism* / Q000637:transplantation*; D005260:Female; D006801:Humans; D007270:Injections, Intra-Articular; D008297:Male; D008875:Middle Aged; D020370:Osteoarthritis, Knee / Q000601:surgery*; D011788:Quality of Life; D016037:Single-Blind Method; D053773:Transforming Growth Factor beta1 / Q000096:biosynthesis*
chemical_list: D053773:Transforming Growth Factor beta1
keywords: TGF-beta; genetically engineered chondrocytes; knee; osteoarthritis