Abstract
Background aims. Shipment of therapeutic somatic cells between a current good manufacturing practice (cGMP) facility and a clinic or between different cGMP facilities requires validated standard operating procedures (SOP). Under National Heart Lung & Blood Institute (NHLBI) sponsorship, the Production Assistance for Cellular Therapies (PACT) group conducted a validation study for the shipping SOP it has created, including shipments of cryopreserved somatic cells, fresh peripheral blood specimens and apheresis products. Methods. Comparisons of pre- and post-shipped cells and cell products at the three participating facilities included measurements of viability, phenotypic profiles and cellular functions. The data were analyzed at the University of Pittsburgh Biostatistics Facility. Results. No consistent shipping effects on cell viability, phenotype or functions were detected for cryopreserved and shipped peripheral blood mononuclear cells (PBMC), monocytes, immature dendritic cells (iDC), NK-92 or cytotoxic T cells (CTL). Cryopreserved mesenchymal stromal cells (MSC) had a significantly decreased viability after shipment, but this effect was in part because of inter-laboratory variability in the viable cell counts. Shipments of fresh peripheral blood and apheresis products for the generation of CTL and dendritic cells (DC), respectively, had no significant effects on cell product quality. MSC were successfully generated from fresh bone marrow samples shipped overnight. Conclusions. This validation study provides a useful set of data for guiding shipments of therapeutic somatic cells in multi-institutional clinical trials.
Acknowledgments
This work was supported in part by PACT (NHLBI contract numbers N01-HB-37163, N01-HB-37164, N01-HB-37165 and N01-HB-37166). The authors would like to thank the staff at Baylor College of Medicine, University of Minnesota and the University of Pittsburgh for sample collection, shipping and testing. In addition, we would like to thank the EMMES Corporation for their assistance and dedication in bringing this manuscript to fruition.
Declaration of interest: The authors report no conflicts of interest. The authors alone are responsible for the content and writing of the paper.