Friday, November 15, 2013

Stem cell stories that caught our eye: LouGehrig’s disease, diabetes and enlisting stem cells to protect our military

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. 

iPS cells like these were involved in three of this weeks top stories (Image from the lab of Kathrin Plath at UCLA)
Bases loaded for Lou Gehrig’s disease. The international journal BioTechniques did a nice review of how reprogrammed iPS type stem cells can provide tremendously useful insights about disease, in this case ALS (Lou Gehrig's disease), the disease that struck down one of baseball’s greatest hitters and eventually came to bear his name. It discusses the great difficulty researchers had prior to iPS technology because they could not test living tissues that had the genes that make a person susceptible to ALS; it’s not feasible to get sample of spinal cord tissue from patients. But you can take skin samples, convert them to iPS cells and then encourage those cells to become nerves in a lab dish. One such model led researchers to the most common genetic mutation for the disease. The article provides one of the most readable descriptions of both the potential and the limitations of using these models, with a narrative about the progress being made in Lou Gehrig’s disease, from the “first pitch” to a “home run.”

You can read about the research CIRM funds in the area on our stem cells for ALS fact sheet.

Diabetes in a dish. John Ferrell who frequently writes about stem cells for Forbes, has picked up and expanded upon a press release from the New York Stem Cell Foundation on a study published in Diabetes that points to a reason why insulin-producing beta cells stop working in the disease. They created iPS type stem cells from patients with a rare form of diabetes called Wolfram syndrome and turned those into beta cells. They found that those cells reacted abnormally to stress. The New York team is now looking for existing drugs that act on that stress pathway to see if one could be tested on patients.

You can read about the research CIRM funds for the disease on our stem cells for diabetes fact sheet.

The protein that keeps us organized. Researchers at the University of Southern California have found a protein that keeps our bodies organized. In the developing embryo it controls how immature cartilage matures to become various parts of our skeleton. Abnormal versions of this protein have been implicated previously in several diseases. Understanding this protein’s role in the normal progression from cartilage-forming stem cells to mature skeleton should provide targets for therapy. There's a good story about this work in HealthCanal.

The work was done in the lab of Andrew McMahon who holds a Research Leadership grant from CIRM.

Stem cells enlisted by the Air Force. During the week in which we commemorate the service of our armed forces veterans it was nice to read that the Air Force is funding a major research project using stem cells to make the work of our military safer. The Air Force gave the Sanford-Burnham Medical Research Institutes a three-year grant to use iPS type stem cells to screen thousands of chemicals for toxic effects. Our military personnel come in contact with a soup of chemicals and although many have been tested in animals, those tests cannot provide as accurate an assessment as human cells growing in a lab dish. The web site ClinicaSpace, picked up the press release from Sanford-Burnham.

Reducing the side effects of bone marrow transplant. The Australian company Mesoblast published data in the journal Biology of Blood and Marrow Transplantation that showed a significant reduction in deaths in children from the most lethal side effect of donor bone marrow transplants. In nearly half of those patients the immune system cells created by the donor stem cells start to attack the patient’s own tissues, a condition called Graft Versus Host Disease (GVHD). In the most acute form of GVHD only about 30 percent of patients survive 100 days. Survival bumped up to 45 percent in the published data. But you should note the company used a statistic in its release that can be misleading to a lay reader. They state 76 percent were alive at 100 days, however that figure is based on the subset of patients who responded to the therapy at all.

While GVHD is a result of one type of stem cell in the marrow, the blood forming cells, Mesoblast has a product that is from of the second type of stem cell found in marrow, mesenchymal stem cells. Those cells have a natural anti-inflammatory property and the company claims it has a way to enhance those cells’ natural ability to tame the immune system.

CIRM funds a major disease team effort aimed at using an antibody protein to reduce the chances of GVHD in stem cell transplants.

Don Gibbons

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