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 literature produced a number of studies that can be glibly summarized as: Hey they work; they really work. The publications show we are starting to get a real handle on how to get stem cells to integrate and function like the desired tissue, and even to form complex structures.
The same Japanese team that was able to create a complex optic cup in a dish earlier this year, now reports that they have used embryonic stem cells to create a pituitary gland. That gland secretes the appropriate hormones and is able to correct hormone deficiency when transplanted into a mouse that lacks the pituitary gland, making the formerly lethargic animals active again.
Two teams turned embryonic stem cells into neurons that were able to integrate into the brain and show evidence that they function like the specific type of neuron that each team sought. A New York team produced dopamine-producing neurons that were able to correct some movement disorders in Parkinson’s disease models in mice, rats, and monkeys. (We blogged about that work here.) Meanwhile a Wisconsin team chose to mature the stem cells into neurons designed for memory, those in the hippocampus, and showed that they were able to integrate and fire neural signals like neighboring cells.
A Carolina team used a type of stem cell found in bone marrow and fat, mesenchymal cells, taken from the father of sheep with hemophilia, and correct the hemophilia in their sons. They did it by harvesting the stem cells and then genetically altering them so they produced Factor VIII, the blood-clotting factor that hemophiliacs don’t produce. When given to the sons, their bleeds stopped including the bleeds into their joints that had made them limp badly. The animals were able to walk normally again after the injections.
I start this month’s report with a pair of papers that seem to sort out some of the conflicting data that has been reported on stem cells for heart repair. One showed that stem cells from the bone marrow given in a couple of weeks after injury do not do improve heart function. Another showed that a different cell type—cardiac stem cells harvested from the patient’s heart—given even months after evidence of heart failure, were able to improve heart function, and in some patients for at least a year. (We blogged about that work here.) This is a critical area to gain some clarity in what cells to use and when; the burden of heart failure is a huge drain on our nation and the world.
My full report of this month’s highlights is here. I hope you will find it interesting.
A.T.
The Stem Cellar 
It looks like people are getting tired of waiting…will this trend accelerate?
Citizen Scientists
Ordinary people are taking control of their health data, making their DNA public and running their own experiments. Their big question: Why should science be limited to professionals?
Members of this loose collective of amateurs, who call themselves “health hackers” and “citizen scientists,” also perform their own analyses and use the Internet to create and run experiments and clinical trials. They all believe that too much science happens behind closed doors.
There are now a number of companies dedicated to helping citizen scientists devise experiments, and medical journals are publishing the results of their trials.
Ms. Terry's run-in with research scientists led her to start a patient-run database to collect the medical data of people with her children's disease—PXE, which can cause vision loss and other serious problems. After posting information about the data bank on the Web, she put together kits with blood vials and FedEx envelopes and distributed them to participants. They, in turn, took the packages to their doctors, had blood drawn, and then sent the samples to a central lab, where they are available to researchers willing to disclose their findings.
She is now helping to run another effort, launched earlier this year, that is even more far-reaching. Called “That's My Data!'' it aims to facilitate the flow of patients' detailed genetic data to researchers in exchange for open access to the results for those who contributed samples.
The questions that most people have about their DNA—such as what health risks they face and how to prevent them—aren't always in sync with the approach taken by pharmaceutical and academic researchers, who don't usually share any potentially life-saving findings with the patients.
http://online.wsj.com/article/SB10001424052970204621904577014330551132036.html
On November 14, Geron, a pioneer in the field of human embryonic stem cell research, announced that it would discontinue its stem cell programs. This abrupt decision, which shocked the science and business communities, raises important ethical questions about clinical trials conducted by for-profit corporations.
Geron has said that it will “continue to follow all enrolled patients, accruing data and updating the Food and Drug Administration and the medical community on their progress.” Moreover, Stephen Kelsey, Geron’s chief medical officer, is reported to have said that the results “will be a fair reflection of what would have happened if we had completed the study.” This statement is deeply problematic, however.
No clinical trial should involve too few or too many participants. It is important that the trial not be underpowered and thus unable to generate scientific knowledge. It is equally important than no more research participants than necessary be exposed to potential research risks. If only five participants were needed to generate the scientific knowledge, then why would Geron and the F.D.A. have agreed to expose additional persons to the potential harms of trial participation?
It is one thing to close a trial to further enrollment for scientific reasons, such as a problem with trial design, or for ethical reasons, such as an unanticipated serious risk of harm to participants. It is quite another matter to close a trial for business reasons, such as to improve profit margins.
Read more: http://www.thehastingscenter.org/Bioethicsforum/Post.aspx?id=5640&blogid=140#ixzz1fVbS4zSO