Principles of Responsible Translational Stem Cell Research
December 15, 2008 by Kevin Graham
The recent surge of interest in stem cell research has fuelled the hope for a future where today’s discoveries have been translated into patient treatments or cures. A tricky question is how do we know when that future has arrived? When has enough basic research been done to justify clinical trials in human patients and how do we know when enough trials have been done, and the benefits and risks are clear enough, that stem cell therapies can pass clinical trials and be made available to everyone?
Currently there are many unscrupulous people and businesses who have hijacked the hope that stem cell research holds, and have used it to prey upon people battling terminal or debilitating disease. This topic has been explored in Ian Clark’s post, and in a recent article written by Tim Caulfield in the highly respected medical journal Cell Stem Cell. Dr. Caulfield is member of the Department of Public Health Sciences at the University of Alberta (Canada). His research team examined the claims made by websites engaging in direct to patient marketing of their stem cell therapies. After evaluating the claims of 19 websites, who together have claimed to have treated thousands of patients with “stem cell therapies”, Caulfield’s group concluded that the treatments being offered were overly optimistic and unsupported by published clinical evidence. In short, patients are paying large sums of money based on claims of health benefits that are completely unsubstantiated, and are possibly putting their health at further risk by doing so.
Of course it is possible that these treatments offer some form of benefit to the patient, the problem here is that the science to back up the claims has not been done in a rigorous and transparent fashion, and in such a way that invites critical evaluation and review by world experts. Bearing this in mind the ISSRC (2The International Society for Stem Cell Research) has released a set of guidelines that states the required steps and actions to design and implement stem cell research protocols. There are 40 recommendations in total covering everything from the initial basic science to human clinical trials.
One overriding theme that is repeated throughout the 40 recommendations is that every decision and result, positive or negative, must be documented and made available for independent critical review and oversight by other experts in the field. This means that international experts must be able to evaluate every step in the process, from the basic science to the design of preclinical animal trials to the eventual clinical trials in humans. Companies that engage in direct to patient marketing of stem cell therapies generally bypass this step, which is why their claims are held to such scepticism. Having your results reviewed by others allows for widespread acceptance of your findings, leading to a bigger (and a more profitable) customer base. So if peer review is the accepted way to evaluate research findings, and this leads to a potentially larger market share, why don’t all companies subscribe to this process? One must assume it is because they know that their practices and claims will not hold up under the scrutiny of impartial review.
Thus, for your own protection questions on therapy options should be referred to a regulated health professional who can provide concrete peer-reviewed evidence of the benefits and risks to any suggested therapy. After you have all the available information you may do you own review process and take the course of action that you are comfortable with.
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Rett Syndrome and induced Pluripotent Stem cells goes “Green”
June 17, 2009 by Aaron Cheung
Stem cell researchers at The Hospital for Sick Children have now devised a method, reported in the scientific journal Nature Methods, that will facilitate the generation of induced Pluripotent Stem (iPS) cells. I have previously blogged about the promise of iPS cells (see related articles). iPS cells are cells which have been reprogrammed from a differentiated somatic cell (such as your skin cells) to a pluripotent stem cell (like an embryonic stem cell, only that you don’t need to use embryos!). The generation of iPS cells is inherently very rare which occurs at a frequency of about 0.01% meaning you can generate about one iPS cell line out of hundred thousand cells. Out of which, many lines fail to satisfy the different rigorous tests necessary to be given the prestigious name of “iPS cell”.
A group led by Dr. James Ellis, and lead author Dr. Akitsu Hotta, at The Hospital for Sick Children has generated a reporter, named EOS, that will facilitate the generation of iPS cells for both mouse and human. The reporter has DNA elements (called promoters) that will drive the expression of a fluorescent protein (GFP) and an antibiotic resistance gene (puromycin) that will only turn on in pluripotent stem cells, but remain off in differentiated cells. Therefore, scientists can now generate iPS cells from different sources and the iPS cells will literally “light up with a green colour” once reprogramming has been achieved. Furthermore, by adding puromycin to your culture, it will keep the iPS cells in a pluripotent state as iPS cells will normally spontaneously differentiate.
To demonstrate the applicability of EOS, Ellis also derived iPS cells from patients who have a neurodevelopmental disorder called Rett Syndrome. Rett Syndrome is an Autism Spectrum Disorder which affects almost exclusively females at an incidence of 1 in 10,000 and is one of the leading causes of mental retardation in girls. These Rett Syndrome iPS cells were green when placed under a fluorescent microscope and Ellis demonstrated that neurons can be generated from these Rett Syndrome iPS cells, the cell type affected in Rett Syndrome.
In conclusion, the EOS reporter will facilitate the generation of iPS and will be particularly useful for new labs going into this rapidly developing field. Furthermore, Rett Syndrome iPS cells that were also generated in this study will be invaluable for future drug screens that may be beneficial to those with Rett Syndrome.
Article:
Hotta, A. et al. Isolation of human iPS cells using EOS lentiviral vectors to select for pluripotency. Nature Methods 6, 370–376 (2009).
Related articles:
Breakthrough of 2008 – Reprogramming
A New Era for Disease Modeling and Drug Screening
Some of our experts
Stem Cell Origins
Cancer stem cells
Stem Cells and the Nervous System
[...] unknown wrote an interesting post today onPrinciples of Responsible Translational Stem Cell Research …Here’s a quick excerptHis research team examined the claims made by websites engaging in direct to patient marketing of their stem cell therapies. After evaluating the claims of 19 websites, who together have claimed to have treated thousands of patients … [...]
iI have had Parkinson DISEASE for 15 years.-I ALSO HAVE A WIFE AND A FAMILY -I WANT STEM CELL RESEARCH