Each month CIRM President Alan Trounson gives his perspective on recently published papers he thinks will be valuable in moving the field of stem cell research forward. This month’s report, along with an archive of past reports, is available on the CIRM website.
This month’s review has to start by noting a key milestone for the field: the first published data from a clinical trial using cells derived from embryonic stem cells (ESCs). This Lancet paper from UCLA and Advanced Cell Technologies (ACT) describes results from a trial testing a potential therapy for two causes of blindness. The fact that two patients in the trial showed some preliminary improvements in vision created a historical timeline marker for the field. The date 1-23-12 can now go up alongside 3-09-09, the date President Obama lifted federal restrictions on ESC funding.
However, you must remember that this latest milestone is based on very limited, preliminary data. It involved only two patients and its suggestions of safety and potential benefit are based on following those patients for only four months. Many more patients and more follow-up data are needed, as my colleague blogged about here.
Another paper this month on eye disease provides a great example supporting CIRM’s philosophy of funding all types of stem cells because you never know which ones will work most effectively for which patients. Where the UCLA/ACT team was using embryonic stem cells to derive the retinal pigment epithelial (RPE) cells that are degraded in macular degeneration, a New York team isolated cells from the RPE of cadaver eyes that were able to revert to a stem cell state when placed in culture. The team was then were able to redirect those cells to create an RPE monolayer like the one that naturally occurs in the eye. Although they used donor tissue, these cells exist in everyone and could be harvested by needle biopsy from a patient. This theoretically could give patients replacement tissue that matches their own and would not raise issues of immune rejection.
Even though I have discussed disease-in-a-dish models from reprogrammed iPS cells several times in this blog, I can’t resist mentioning one more. A recent Nature paper cites three CIRM comprehensive grants and one training grant, and several of the authors are now housed in the Sanford Consortium for Regenerative Medicine building, constructed in part through CIRM funding. In that paper, the researchers reprogrammed skin cells from people with Alzheimer’s disease then matured those stem cell into nerves. (We blogged about that work here.) It makes major strides in understanding the cellular basis for Alzheimer’s disease and points to opportunities for earlier diagnosis and to potential avenues to therapy.