The recent regulatory approval of the first hematopoietic stem and progenitor cell gene therapy (HSPC-GT) signals the start of a new era for gene therapy and highlights the potential contribution by high-throughput cell culture technologies in propelling HSPC-GT from curing rare diseases to curing more common diseases.
Hematopoietic stem cells (HSCs) are the “fountain” for all blood cells that circulate in our bodies throughout life. Arguably, no other cell type has more profound and far-reaching influence on our well-being than HSCs. They reside in our bone marrow and continuously produce a variety of cells with vital tasks, for example, oxygenation via red blood cells, termination of bleeding via platelets, and immunity via leukocytes, which also provide immune defense to the central nervous system.
There is, however, a flipside to the pre-eminence of HSCs. When faulty HSCs emerge, devastating outcomes can ensue, such as autoimmune diseases like multiple sclerosis and blood cancers like leukemia. Thankfully, a solution to these life-threatening indications is well at hand because HSCs can be removed and replaced with healthy HSCs using HSC transplantation (HSCT)—a highly effective procedure pioneered by Nobelist E. Donnall Thomas over five decades ago.