STEM CELL TRIALS
Q. Who are stem-gene clinical trial sponsors? A. Although currently most of the presentations at outsourcing clinical trials conferences are focused on the supply needs of pharmaceutical and biopharmaceutical sponsors, there is an emerging industry for the supply of stem-gene clinical trials. Stem-gene clinical trials fall into two categories. The first category evaluates treatments with human tissue stem cells, like hematopoietic stem cells (HSCs) and mesenchymal stem cells (MSCs). The vast majority of stem-gene clinical trials are for stem-cell treatments of this type. Examples of sponsors developing stem-cell clinical trials are Athersys, Pluristem, Celularity, Sorrento Therapeutics, and Talaris Therapeutics. More recent and more highly publicised stem-gene clinical trials are gene therapy and gene-editing clinical trials. Well-known sponsors are Sangamo Therapeutics, Bluebird Bio, CRISPR Therapeutics, Beam Therapeutics, Editas Medicine, and Intellia Therapeutics. All gene therapy clinical trials are “stem-gene” clinical trials because the therapies target tissue stem cells (usually HSCs) to ensure that the effects of transduced genes or gene edits are maintained for long periods. Only tissue stem cells provide this essential treatment quality. Other cells in the body mature, die, and are lost rapidly.
Q. What do stem-gene cell clinical trial sponsors need? A. Sponsors of stem-gene clinical trials need all the usual trial execution resources supplied by vendors for pharmaceutical and biopharmaceutical clinical trials. But they also have supply needs that are unique to their special treatments. These include the tissue stem cells and the gene vectors that constitute their treatments.
As in other new clinical trial disciplines, companies in this industry have either created or in-licensed the technologies needed for producing their treatments. In most cases, their treatment basis is a key proprietary element of the sponsors’ business models. This feature makes it challenging for vendors to successfully supply such services in this early period. However, like all current mature areas of clinical trials supply, as the stem-gene clinical trials industry matures, sponsors will extend their clinical programs to outsourcing even the
production of their treatments to gain the advantages of more economical and efficient expert outsourcing.
Q. Why don’t stem-gene clinical trial sponsors count their stem cells? A. This article is titled with the above question and is full of related questions. Many reading this article are already puzzled by the title. Of course, companies conducting stem-gene clinical trials know the number of stem cells in their treatments, don’t they? Another question. They must know the dosage of stem cells in their treatments, right? If not, how can they design clinical trials whose outcomes they can soundly interpret? How do they certify the quality of their treatments if they don’t know how many stem cells – natural, expanded, genetically-engineered, or gene-edited – are in them? Those well-versed in the challenges of tissue stem-cell expansion know that expanded cell products often have far fewer stem cells than their starting preparation. In addition, patients who are compared in stem-gene clinical trials usually get independent stem cell treatments, not treatments sampled from the same pool or bank. So, how do companies know that that they are comparing treatment outcomes for the same stem cell dosage? They don’t.
Some who are willing to acknowledge this wide-spread stem cell counting problem in stem-gene clinical trials may suggest that it is not a problem. After all, the same problem exists for the one approved stem cell medical treatment, HSC transplantation (HSCT), and it seems to be doing fine. Though convenient, this assessment is naïve. During the development of bone marrow HSCT therapy, and subsequent related mobilised peripheral blood HSCT
“Why should stem-gene clinical trial sponsors count their stem cells? The simple answer to this question is, if they do, they can reduce their costs, design and evaluate clinical trials better, and improve the effectiveness of their treatments.”
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