Stem cell stories that caught our eye: diabetes, Down Syndrome and the long road to gene therapy

Here are some stem cell stories that caught our eye this past week. Some are groundbreaking science, others are of personal interest to us, and still others are just fun.

Role of blood vessels in diabetes. I have been a skeptic of the few reports that have come out suggesting mesenchymal stem cells, the second type of stem cell found in bone marrow, can be used to treat Type 1 diabetes. But a new study in the journal Diabetes has erased a bit of that skepticism. It provides a plausible rational for why those cells might provide some benefit. A team from the University of Missouri found that when a patient’s immune system attacks their insulin-producing Beta cells it also attacks the blood vessels that nourish those key cells. They report that transplanted bone marrow cells were able to induce new blood vessel growth and that in turn fostered the Beta cells to replenish themselves. They do not claim the stem cells became Beta cells. Because mesenchymal stem cells are proven to foster blood vessel growth and not the growth of endocrine tissue that would be needed for Beta cells, they offer a reasonable scenario. This online news article is a little over the top using words like “cure,” but it does give the basics of what the team has done and found.

Down syndrome defects at the cell level. We often write about the power in creating disease-in-a-dish models of genetic diseases by reprogramming skin cells from a patient to become the stem cells known as iPS cells. But this research into Down Syndrome is a particularly fine example of what those cells can tell us. They found that the nerves grown from cells with the genetic defect produced 40 percent fewer synapses—the connections nerves use to communicate with each other. Those cells also had an unusual number of active genes that are usually only turned on when cells are under a type of chemical stress called oxidative stress. The lower number of synapses could explain part of the cognitive deficit seen in many of these individuals, and the chemical stress could explain the premature aging most experience as this article notes. Understanding these underlying causes often points to potential therapies to ameliorate the disease. Here’s a story about that work.

BMT—the original stem cell transplant. Discussions about stem cell therapy often overlook the fact that stem cell therapies have been in the clinic for more than 30 years in the form of bone marrow transplants (BMT) for patients with blood cancers. In the early years, BMT was a quite dangerous procedure reserved for patients with no other option. Researchers and clinicians have constantly refined it over the years and the outcomes have improved dramatically to the point that it is now presented to many patients as one of multiple options. The Journal of Clinical Oncology just published a good review of the progress, which is discussed in this press release from the National Marrow Donor Program.

The long road to gene therapy. Observers often compare the novelty and hurdles to successful gene therapy to those being experienced by the stem cell field as it moves beyond bone marrow transplants. I actually think these comparisons are usually over done. I really don’t think it will take as long to get an approved second generation stem cell therapy as it did for the first gene therapy approved (in Europe) just in the past year. This press release from the publisher Mary Ann Liebert highlights two articles in its journals this month from pioneers in gene therapy detailing the many ups and downs of that field. Even if stem cell therapies only have a fraction of these issues, the article provides a good reminder of what could be. (Unlike many articles from this publisher, these pieces are not behind a pay wall.)

History of the ties between IVF and stem cells science. ABC TV, in this case the Australian Broadcasting Corp, has aired and posted a half hour interview with Alan Trounson, who is not only our president, but also one of the pioneers of in vitro fertilization and one of the earliest folks from that field to make the connection and move to stem cell research. The interview provides a nice history of the two fields and Alan’s rich and divergent career.

D.G.

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