A Manually Curated Database on Clinical Studies Involving Cell Products Derived from Human Pluripotent Stem Cells

Abstract

The last 5 years have witnessed a significant increase in the number of clinical studies based on human pluripotent stem cells (hPSCs). In parallel, concern is increasing about the proliferation of unregulated stem cell treatments worldwide. Regulated clinical testing is a de facto standard to establish the safety and efficacy of new cell therapies, yet reliable information on clinical studies involving hPSCs is scattered. Our analysis of a multitude of resources found 54 clinical studies involving several types of hPSCs, which are performed in ten countries. While the majority of those studies is based on human embryonic stem cells (hESCs), clinical studies involving human induced pluripotent stem cells increased more strongly in the past 2 years than the number of hESC-based studies. A publicly accessible database was created using the human pluripotent stem cell registry (https://hpscreg.eu) platform, providing a steadily updated comprehensive overview on hPSC-based clinical studies performed worldwide.

The Future of Regenerative Medicine: Cell Therapy Using Pluripotent Stem Cells and Acellular Therapies Based on Extracellular Vesicles

“…hPSCs are compatible with large-scale industrial productions in good manufacturing practices (GMP) facilities and quality controlled as any other more conventional pharmaceutical products. Whereas the majority of hPSC-based clinical trials have used hESCs up to now, the field is moving toward hiPSCs as they do not require the destruction of embryos and consequently can be used worldwide without restriction [ 1 ].…”

“…Since then, a number of indications were evaluated, including eye diseases, diabetes and ischemic hearts. A recent manually curated database identified 54 planned or initiated clinical trials based on hPSCs worldwide [ 1 ]. Starting from 2018, a switch was observed regarding the source material with the number of hiPSC-based clinical trials beginning to be more important compared to that of hESC-based trials.…”

“…Most of these clinical trials focus on hPSC-derived RPE (24 clinical trials) to target macular degeneration, some forms of RP and Stargardt’s disease [ 1 , 3 ]. This could be explained by the easy access and available live imaging technologies, as well as low number of required cells and immune privilege of the eye.…”

“…In the background of many diseases, the degeneration and/or dysfunction of a particular cell type affects tissues and organs, which will deteriorate over time and lose their functions. In this context, regenerative medicine offers new perspectives for pathologies that otherwise remain untreated [ 1 , 2 ]. Hundreds of clinical trials have been initiated in the last few years for a large panel of indications.…”

Cell Therapy With Human ESC-Derived Cardiac Cells: Clinical Perspectives

“…Future clinical studies will tell whether such an expectation can be really met. In the meantime, the observation that by the end of 2019, there were at least 54 registered trials testing PSC derivatives (Kobold et al, 2020) provides compelling evidence that regardless of the cell source (ESC or iPSC), the clinical indication and the delivery strategy, PSC-derived lineage-specific cells have now integrated the armamentarium of therapies against a wide variety of diseases.…”