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As part of the International Society of Cell Therapy (ISCT) 2018 Annual Meeting, the Mesenchymal Stem/Stromal Cell (MSC) committee organized a pre-conference, which covered methods of improving MSC engraftment and potency in vivo and clinical efficacy using MSC potency assays. The speakers examined methods to improve clinical efficacy using MSC potency assays and methods to improve MSC engraftment/homing/potency in vivo. Discussion of patient "responders" versus "non-responders" in clinical trials and working toward ways to identify them were also included.

The International Society for Cell & Gene Therapy (ISCT®) Mesenchymal Stromal Cell (ISCT MSC) committee offers a position statement to clarify the nomenclature of mesenchymal stromal cells (MSCs). The ISCT MSC committee continues to support the use of the acronym "MSCs" but recommends this be (i) supplemented by tissue-source origin of the cells, which would highlight tissue-specific properties; (ii) intended as MSCs unless rigorous evidence for stemness exists that can be supported by both in vitro and in vivo data; and (iii) associated with robust matrix of functional assays to demonstrate MSC properties, which are not generically defined but informed by the intended therapeutic mode of actions.

The Cellular Therapy Coding and Labeling Advisory Group of the International Council for Commonality in Blood Banking Automation and the International Society for Cell & Gene Therapy mesenchymal stromal cell (MSC) committee are providing specific recommendations on abbreviating tissue sources of culture-adapted MSCs. These recommendations include using abbreviations based on the ISBT 128 terminology model that specifies standard class names to distinguish cell types and tissue sources for culture-adapted MSCs. Thus, MSCs from bone marrow are MSC(M), MSCs from cord blood are MSC(CB), MSCs from adipose tissue are MSC(AT) and MSCs from Wharton's jelly are MSC(WJ). Additional recommendations include using these abbreviations through the full spectrum of pre-clinical, translational and clinical research for the development of culture-adapted MSC products. This does not apply to basic research focused on investigating the developmental origins, identity or functionalities of endogenous progenitor cells in different tissues. These recommendations will serve to harmonize nomenclature in describing research and development surrounding culture-adapted MSCs, many of which are destined for clinical and/or commercial translation. These recommendations will also serve to align research and development efforts on culture-adapted MSCs with other cell therapy products.

The serious consequences of the global coronavirus disease 2019 (COVID-19) pandemic have prompted a rapid global response to develop effective therapies that can lessen disease severity in infected patients. Cell-based approaches, primarily using mesenchymal stromal cells (MSCs), have demonstrated a strong safety profile and possible efficacy in patients with acute respiratory distress syndrome (ARDS), but whether these therapies are effective for treating respiratory virus-induced ARDS is unknown. According to the World Health Organization International Clinical Trials Registry Platform and the National Institutes of Health ClinicalTrials.gov databases, 27 clinical investigations of MSC-based cell therapy approaches have begun in China since the onset of the COVID-19 outbreak, with a growing number of academic and industry trials elsewhere as well. Several recent published reports have suggested potential efficacy; however, the available data presented are either anecdotal or from incomplete, poorly controlled investigations. Therefore, although there may be a potential role for MSCs and other cell-based therapies in treatment of COVID-19, these need to be investigated in a rationally designed, controlled approach if safety and efficacy are to be demonstrated accurately. The authors urge that the field proceed by finding a balance between swift experimentation and communication of results and scientifically coherent generation and analysis of clinical data.

Background & Aim Jordan's Syndrome (JS), also known as PPP2R5D-related intellectual disability (ID), is a neurodevelopmental disorder that is caused primarily by de novo missense mutations in the PPP2R5D gene. PPP2R5D encodes a 602-amino acid protein PPP2R5D (also known as the B56δ subunit), which is a regulatory subunit of the heterotrimeric enzyme serine/threonine-protein phosphatase 2A (PP2A). Mutations in phosphatases are known to cause human overgrowth due to their involvement in cellular regulation. With the recent identification of Jordan's Syndrome using Whole Exome Sequencing, PPP2R5D mutations specifically have been associated with various patient intellectual disabilities (IDs), malformations, autism spectrum disorders, seizures, and behavioral challenges. The wide range of the severity of patient IDs, along with the association of PPP2R5D to Alzheimer's, autism, Parkinson's-like symptoms, and cancer, has generated an impetus for further research to elucidate the specific cellular pathways that are affected by PPP2R5D. In order to study JS in vitro, various biological tools need to be generated including neural cellular models. Methods, Results & Conclusion Induced pluripotent stem cells (iPSC) are highly proliferative cells that can differentiate into all three germ layers in vitro. Reprogramming patient somatic cells in to iPSC offers a means of generating difficult to source neural stem cells and neurons to study PPP2R5D mutations. Our team has generated five chromosomally stable iPSC clones from JS patient-derived skin fibroblasts representing four of the most common PPP2R5D disease-causing mutations. We have also generated chromosomally stable isogenic clones of these iPSC lines using precision CRISPR editing to provide genetically matched unmutated cells as controls. Neural stem cell (NSC) lines and neurons have been derived from each line to help study disease biology and novel therapeutic agents in a disease relevant cell type, as well. iPSC and NSC provide a theoretically unlimited supply of disease-specific cells that can be used to study JS. They are important and necessary tools for advancing the field of JS research and developing therapies for this debilitating disorder.