Some of the most important work taking place in the stem cell research community is not in fact directly focused on producing treatments. Instead it consists of infrastructure improvements: creating ways to obtain more cells of a specific type, more reliably, more rapidly, and at a lower cost. This is important because falling costs accelerate further research and development, such as by broadening the number of laboratories that can budget projects in the field, and by expanding what can be accomplished within the budget of any given research group. Ultimately this will also make the resulting treatments cheaper and better, but at this point that is somewhat less important given where the field stands today. Cell therapies deployed to date have proved beneficial, but are just a first pass at the problem space, a fragment of what is possible. That initial success over the past decade has served to draw in enough money and interest for the next cycle to expand considerably. It will produce a panoply of far better, far more diverse approaches to the control of cells in medicine. It will be an explosion of variety and utility for patients, and the cheaper the tools the greater the result and the sooner it will arrive.
This is why it doesn't hurt to keep a weather eye on progress in enabling technologies and infrastructure in stem cell research. For those of us likely to need or benefit from regenerative treatments a decade or two from now, the pace of progress in tools today provides some insight as to the likely future landscape of therapies. Take these few items as illustrated of current work on the creation of reliable and low-cost sources of stem cells, for example:
Using the new technique in mice, the researchers increased the number of stem cells obtained from adult skin cells by more than 20-fold compared with the standard method. They say their technique is efficient and reliable, and thus should generally accelerate research aimed at using stem cells to generate virtually any tissue.
The standard method for reprogramming skin, blood, or other tissue-specific cell types into "induced pluripotent stem cells" (iPSCs) involves the artificial expression of four key genes dubbed OKSM (for Oct4, Klf4, Sox2 and myc) whose collective activity slowly prods cells into an immature state much like that of an early embryonic cell. Converting most cell types into stable iPSCs occurs at rates of 1 percent or less, and the process can take weeks. Researchers throughout the world have been searching for ways to boost this efficiency, and in some cases have reported significant gains. These procedures, however, often alter vital cellular genes, which may cause problems for potential therapies.
Adding to fibroblasts engineered to express OKSM either vitamin C, a compound to activate Wnt signaling, or a compound to inhibit TGF-β signaling increased iPSC-induction efficiency weakly to about 1% after a week of cell culture. Combining any two worked a bit better. But combining all three brought the efficiency to about 80 percent in the same period of time.
Investigators have just published the announcement of the discovery of a new molecule, the first of its kind, which allows for the multiplication of stem cells in a unit of cord blood. Umbilical cord stem cells are used for transplants aimed at curing a number of blood-related diseases, including leukemia, myeloma and lymphoma. For many patients this therapy comprises a treatment of last resort. The research has the potential to multiply by 10 the number of cord blood units available for a transplant in humans. In addition, it will considerably reduce the complications associated with stem cell transplantation.
The small number of hematopoietic stem and progenitor cells in cord blood units limits their widespread use in human transplant protocols. We identified a family of chemically related small molecules that stimulates the expansion ex vivo of human cord blood cells capable of reconstituting human hematopoiesis for at least 6 months in immunocompromised mice. The potent activity of these newly identified compounds, UM171 being the prototype, is independent of suppression of the aryl hydrocarbon receptor, which targets cells with more-limited regenerative potential. The properties of UM171 make it a potential candidate for hematopoietic stem cell transplantation and gene therapy.