Sterility Testing for Hematopoietic Stem Cells.

Over the last two decades, rapid technological advances have led to the wide adoption of cell and gene therapy products for the treatment of a variety of disease states. In this study, we reviewed the literature between 2003 and 2021 to provide a summary of overarching trends associated with microbial contamination in hematopoietic stem cells (HSCs) derived from peripheral blood, bone marrow, and cord blood. We provide a brief background on the regulatory context for human cells, tissues, and cellular and tissue-based products (HCT/Ps) as regulated by the US Food and Drug Administration (FDA), sterility testing expectations for autologous (Section 361) and allogeneic (Section 351) HSC products, and discuss clinical risks associated with the infusion of a contaminated HSC product. Finally, we discuss the expectations for current good tissue practices (cGTP) and current good manufacturing practices (cGMP) for the manufacturing and testing of HSC based on Section 361 and Section 351 categorization, respectively. We provide commentary on what is practiced in the field and discuss the critical need for updates to professional standards that keep pace with advancing technologies with an aim to clarify expectations for manufacturing and testing facilities to improve standardization across institutions.

Current and Future Cell Therapy Standards and Guidelines.

Cell biology researchers, cellular engineers, and clinicians are teaming together to create powerful drugs. The use of cell-derived products as biologics is rapidly advancing. These human cell-based products have great potential for treating serious conditions but may have unidentified risks. Manipulations, expansions, and gene modifications increase the risks of unexpected outcomes. Implementation of the 21st Century Cures Act is opening avenues for accelerated approvals of these drugs for use in clinical trials and licensure. Although overwhelming, collaboration between regulators, industry, and research and medical communities enables the field to safely meet the needs of critically ill patients.