Abstract
The lack of clinically predictive potency assays for cell products significantly impedes translation of these therapies. Here, we describe a microfluidic on-chip 3D system for rapid evaluation of a subset of patient-derived bone marrow aspirate concentrate (BMAC) samples used in a phase 3 multicenter trial (NCT03818737) evaluating autologous cells for relieving knee osteoarthritis pain. BMAC clinical samples cultured in the on-chip 3D system exhibit elevated levels of immunomodulatory and trophic proteins compared to 2D culture. Using analyte information from in vitro assays and patient-matched clinical data, we build linear regression prediction models for clinical outcomes. We demonstrate improved clinical prediction by cross-validation accuracy for the on-chip 3D platform compared to 2D culture. Additionally, on-chip 3D assay metrics display higher correlative power with patient pain scores compared to the 2D assay. This study establishes a potency assay with improved prediction power to accelerate translation of cell therapies.