(F1238) STEM CELL THERAPY FOR CHRONIC LUMBAR DISC DISEASE: PHASE 2 CLINICAL SAFETY AND FEASIBILITY DATA OF INTRADISCAL INJECTIONS OF HYPOXIC CULTURED MESENCHYMAL STEM CELLS
VP of Research and Development BioRestorative Therapies Melville, NY, United States
Abstract: Lumbar disc disease (LDD) is a leading cause of disability with no effective therapy. Orthobiologics have emerged as a strategy for regenerative therapy. Hypoxic culturing of MSCs results in increased therapeutic activity of MSCs post-transplant into the nutrient-poor, low oxygen microenvironment of the disc. The use of this biologic to treat LDD is a promising strategy, due to their hypoxic engineering and known, immuno-modulatory/anti-inflammatory properties. We report early safety/feasibility data in subjects (n=10) dosed in an ongoing Phase 2 trial.
The study is double-blind randomized, controlled, and multicenter to determine the safety/efficacy of a single injection of hypoxic cultured MSCs combined with platelet lysate to treat LDD with 12 month safety/efficacy endpoints. 99 subjects will be randomized 2:1 to treatment or control. Data collected at baseline, week 2, week 12, week 26, and week 52. Pain and function scales used; Visual Analog Scale (VAS), Oswestry Disability Index (ODI). Primary endpoint is safety and secondary a clinical response (30% decrease in pain and a 30% increase in function at Week 52).
10 subjects were dosed with 40 x 106 hMSCs or control. At 52 weeks there were no AEs/SAEs related to cell dose. At 12 weeks average baseline ODI was 44 vs 12 week ODI of 43 (2.27% change), at 26 week average baseline ODI was 48 vs 26 week ODI of 24 (50% change), at 52 week average baseline ODI was 43 vs 52 week ODI score of 12 (75% change). For VAS, subjects at 12 weeks had an average baseline VAS score of 63.7 vs 12 week VAS score of 36.4 (42.8% change), at 26 week average baseline VAS score was 69.6 vs 26 week VAS score of 35.5 (48.9% change), at 52 week average baseline VAS score was 71.5 vs 52 week VAS score of 17 (76.2% change).
Studies suggest that the harsh microenvironment of the disc could impact cell viability resulting in non-efficacy or worsening symptoms. At 52 weeks in our trial AEs/SAEs reported were not related to cell dose and although blinded, early clinical data at 12 week, 26 week and 52 weeks demonstrated a positive trend with no worsening of pain and function.
Results demonstrate that hMSCs may be used for treating LDD. Early data from the present study suggest that a cell dose of 40x106 of hMSCs are suitable for intra-discal injection, not limited by dose limiting toxicity and may be an effective therapy for treating LDD.
