RT Journal Article
SR Electronic
T1 Allogeneic Disc Progenitor Cells Safely Increase Disc Volume and Improve Pain, Disability, and Quality of Life in Patients With Lumbar Disc Degeneration—Results of an FDA-Approved Biologic Therapy Randomized Clinical Trial
JF International Journal of Spine Surgery
JO Int J Spine Surg
FD International Society for the Advancement of Spine Surgery
SP 8609
DO 10.14444/8609
A1 Gornet, Matthew F.
A1 Beall, Douglas P.
A1 Davis, Timothy T.
A1 Coric, Domagoj
A1 LaBagnara, Michael
A1 Krull, Angela
A1 DePalma, Michael J.
A1 Hsieh, Patrick C.
A1 Mallempati, Srinivas
A1 Schranck, Francine W.
A1 Kelly, Colleen
A1 Foley, Kevin T.
YR 2024
UL https://www.ijssurgery.com/content/early/2024/06/25/8609.abstract
AB Background Progenitor cells derived from intervertebral disc tissue demonstrated immunomodulatory and regenerative properties in preclinical studies. We report the safety and efficacy results of a US Food and Drug Administration–approved clinical trial of these cells for the treatment of symptomatic degenerative disc disease.Methods Sixty patients with symptomatic single-level lumbar degenerative disc disease (mean age 37.9 years, 60% men) were enrolled in a randomized, double-blinded, placebo-controlled Phase I/Phase II study at 13 clinical sites. They were randomized to receive single intradiscal injections of either low-dose cells (N = 20), high-dose cells (N = 20), vehicle alone (N = 10), or placebo (N = 10). The primary endpoint was mean visual analog scale (VAS) pain improvement >30% at 52 weeks. Disc volume was radiologically assessed. Adverse events (AEs), regardless of whether they were related to treatment, were reported. Patients were assessed at baseline and at 4, 12, 26, 52, 78, and 104 weeks posttreatment.Results At week 52, the high-dose group had a mean VAS percentage decrease from baseline (−62.8%, P = 0.0005), achieving the endpoint of back pain improvement >30%; the mean change was also significantly greater than the minimal clinically important difference of a 20-point decrease (−42.8, P = 0.001). This clinical improvement was maintained at week 104. The vehicle group had a smaller significant decrease in VAS (–52.8%, P = 0.044), while the low-dose and placebo groups showed nonsignificant improvements. Only the high-dose group had a significant change in disc volume, with mean increases of 249.0 mm3 (P = 0.028) at 52 weeks and 402.1 mm3 (P = 0.028) at 104 weeks. A minority of patients (18.3%) reported AEs that were severe. Overall, 6.7% of patients experienced serious AEs, all in the vehicle (n = 1) or placebo (n = 3) groups, none treatment related.Conclusions High-dose allogeneic disc progenitor cells produced statistically significant, clinically meaningful improvements in back pain and disc volume at 1 year following a single intradiscal injection and were safe and well tolerated. These improvements were maintained at 2 years post-injection.Level of Evidence 1.Clinical Trial Registration NCT03347708—Study to Evaluate the Safety and Preliminary Efficacy of Injectable Disc Cell Therapy, a Treatment for Symptomatic Lumbar Intervertebral Disc Degeneration.