Clinical trials using human pluripotent stem cells is published in Cell Stem Cell

A comprehensive overview of clinical trials using human pluripotent stem cells is published in Cell Stem Cell by Associate Professor Agnete Kirkeby and colleagues.

Human pluripotent stem cells can self-renew indefinitely in culture while maintaining the ability to become any cell type in the human body. Over the last decade, a growing number of clinical trials using human pluripotent stem cells to develop novel cellular treatments for diseases, such as Parkinson’s Disease, blindness, diabetes and cancer, have been initiated in various parts of the world.

Now, a review paper that systematically categorizes and summarizes all completed and ongoing clinical trials has been published in the journal Cell Stem Cell by co-authors Associate Professor Agnete Kirkeby, Dr Heather Main and Dr Melissa Carpenter.

“Dr Main is a consultant from Australia and Dr Carpenter is a consultant based on the West Coast of the US, while I myself is based in Copenhagen. Collectively, we’ve therefore been in a good position to cover activities around the world. Our contact with trial sponsors also allowed us to, for the first time, actually count how many patients have been transplanted with human pluripotent stem cell products in total, ever since these products first came into clinical trial, ” said Associate Professor Agnete Kirkeby.

The review uncovers that more than 1200 patients have now received treatment with pluripotent stem cell products as part of 116 different clinical trials, and more than 100 billion cells have been administered in total to patients across the globe. The trials are targeting 34 different indications, the majority of which are eye diseases, brain diseases and cancer.

Much of the initial underlying information in the review came from the US based website clinicaltrials.gov, which hosts information on clinical trials worldwide. In addition to this information, manual curation of trials through information from other national databases, publications, press releases and personal communication with trial representatives was necessary to validate the information going into the review.

The review is the first of its kind to provide compiled information on immunosuppression used in the different pluripotent cell therapy clinical trials. Immunosuppression inhibits the body’s immune system and prevents rejection of the transplanted cells and it is therefore an important part of designing and developing safe stem cell trials.

Despite concerns about tumor formation, no cases of this have been reported from the more than 1200 patients receiving pluripotent stem cell treatments as part of controlled clinical trials so far. Overall, the review thereby concludes that there does not appear to be any generalizable safety concerns for these kinds of novel cellular therapies.

It is anticipated that this review on the landscape of human pluripotent stem cells in clinical use will be an invaluable resource to stem cell scientists all over the world. The authors have plans to update the review on a regular basis, as the field is rapidly progressing with new therapies and new clinical trials each year.

Read the review: https://doi.org/10.1016/j.stem.2024.12.005