Our Mission
The mission of CSCT is to translate basic stem cell science into clinical application. The goal is to develop innovative stem cell therapies with a focus on blood, lung, and intestine and implement pluripotent stem cell-based therapies developed elsewhere.
The term ‘stem cell’ covers pluripotent stem cells (PSCs), which either arise in the inner cell mass of preimplantation embryos or can be generated by reprogramming of somatic cells and give rise to all lineages of the embryo, and postnatal stem cells that endow specific organs and tissues in which they reside with regenerative capacity.
There is a deep biological and clinical underpinning to this effort. Treating diseases using approaches that try to counter evolutionary mechanisms is difficult. Immune suppression for alloreactivity, such as in transplantation, is a challenge. Cancer is a recalcitrant disease because it highjacks evolutionary selection mechanisms.
On the other hand, truly game-changing therapies often harness evolved physiological mechanisms. Hormone replacement and antagonism therapies replace or complement mechanisms that provide organismal homeostasis. Antibiotics take advantage of evolved fungal defense mechanism. Vaccination and immunotherapy harness evolved, physiological host defense mechanisms. The most established application of stem cell therapy, hematopoietic (blood) stem cell transplantation for malignant and genetic blood diseases, is based on the fact that blood stem cells migrate in and out of the bone marrow where they generate billions of blood cell every day, even in steady-state. Surgery harnesses the process of wound and injury repair, and owes its success to endogenous, mostly stem cell-based regenerative mechanisms. The generation of mature cells from pluripotent stem cells is based on developmental biology, a process that is the result of millions of years of evolution. The promise of stem cell-based approaches to revolutionize medical care is therefore very high.
While our basic understanding of how stem cells function in their native environment and how they contribute to tissue regeneration has increased exponentially, translation of this knowledge into clinical applications has been challenging. Targeting stem cells to specific niches is a major hurdle. Transplanted blood stem cells home to bone marrow as they do during normal physiology. However, such mechanism are not at play in the homeostasis most other organs. The adult heart and brain, furthermore, only possess very limited regenerative capacity, and here transplantation of appropriate mature cells is envisaged. Pluripotent stem cell-derived tissues are mostly still at a fetal stage of development and are rarely sufficiently homogenous, which presents a significant obstacle for clinical application. Another barrier is the fact that ‘off-the-shelf’ pluripotent stem cells that are not rejected by the host are required for widespread clinical application. Finally, generating appropriate cells in sufficient numbers in good manufacturing practice (GMP) conditions is needed to bring stem cell therapies to the clinic.
Overcoming these obstacle requires a major, concerted effort. This is the challenge that CSCT is trying to meet, by becoming a multidisciplinary hub that actively reaches out to basic scientists, translational scientists, engineers and bioengineers, bioinformaticians and clinicians, as well as regulators, product R&D and intellectual property specialists.