Abstract

Preclinical Stem Cells Studies: Safety and Regulatory Considerations related to Human-derived Cell-based Therapeutics

Itschak Lamensdorf, CEO, Pharmaseed Ltd.

Stem cells (SCs) therapy has been attempted for many clinical indications and is thus tested in various animal models. The different indications and their implications on efficacy and safety issues will be discussed. The various sources of stem cells, their multiple applications and the novel mechanisms of action of stem cell-based therapies require well-designed efficacy and safety pre-clinical studies. Important parameters, unique to cell therapy, guide the design of these studies. The regulation of stem-cells pre-clinical studies is still evolving. It is now clear that a major objective of efficacy studies is to evaluate the safety and bio-distribution of cells’ administration. Since there is no single cells’ manufacturing procedure accepted, comparative screening models should be adopted. These models aim at comparing different cell batches and different sources or manufacturing procedures in relatively quick schedule. Following definition of the proper cell batch, Proof of Concept studies are performed. The translation of the pre-clinical model to the human disease is another key issue that should be addressed. The mechanism and mode of action of stem cells are also studied in controlled ex-vivo systems, also fit for screening tests. The presentation will include several examples.