Recent progress in stem cell research

Stem cells are undifferentiated biological cells that can become specialized cells in various parts of the body and divide to produce more stem cells. The ability of these cells to regenerate tissues and organs of the body have made them a topic of great interest and research. Treatment with stem cells has the promise to treat a wide variety of diseases and injuries; thus, laboratories around the world are working overtime, making great strides in basic research that are beginning to translate into successes in the clinic.

Stem cell treatment for stroke patients

Researchers at Stanford University, interested in studying the safety of injecting stem cells directly into the brain of stroke victims, performed the relatively straightforward surgery on a group of 18 individuals.

Colony of undifferentiated stem cells
The individuals in the study had each suffered a stroke between six months and three years before the treatment, and were not expected to experience additional improvement. Dr. Gary Steinberg, Professor and Chair of Neurosurgery, led the study using modified bone marrow-derived mesenchymal stem cells. He and his colleagues were “stunned” by the results, which were recently published in the journal Stroke. Seven of the 18 patients had significant improvements after the injection of the cells. Patients who had trouble walking and one that was even wheelchair-bound were able to walk post-injection. While the researchers caution that this trial only included a small sample size of subjects, it is certainly very promising to the millions of stroke patients living with complications.

Adult stem cells can help repair injured muscle

Muscle weakening or wasting is a characteristic of more than thirty debilitating genetic diseases, termed myopathies, including Duchenne muscular dystrophy. Recently Dr. Robert Schneider and colleagues at New York University’s Langone Medical Center – along with collaborators at the University of Colorado at Boulder – identified a single protein that determines if stem cells retain their ability to regenerate. The study, published in Cell Reports, found that AUF1, a protein previously thought to participate in mundane cellular activities, is a critical controller of cell fate and determines whether stem cells can regenerate muscle after injury or during the aging process in mice.

The researchers found that AUF1 blocks a matrix metalloprotease called MMP9 that degrades the skeletal muscle matrix, preventing stem-cell regulated regeneration. They found that mice with stem cells lacking the auf1 gene had muscle cells that deteriorated and were not repaired after injury. The hope is that additional work on AUF1 and MMP9 will lead to treatments for certain muscular disorders and injured muscle tissue.

Using stem cells to assess safety of drug cardiotoxicity

Another group at Stanford University used induced pluripotent stem cells (iPSCs) to create heart muscle that can act as a model to test the cardiotoxicity of pharmaceuticals. Authors Dr. Joseph Wu and Dr. Elena Mata published the study recently in Cell Stem Cell. According to Dr. Wu, “Thirty percent of drugs in clinical trials are eventually withdrawn due to safety concerns, which often involve adverse cardiac effects.”

The team used iPSCs from seven different individuals who were considered cardiovascularly healthy and induced them to grow muscle cells. They sequenced the RNA and analyzed gene expression patterns among individuals, finding that the patterns differed significantly, especially for genes involved in metabolism and stress response. They then treated the heart muscle cells with rosiglitazone, a drug that has caused concern because of its negative effect on cardiac function. Of the seven tested, cells from one of the subjects, which had an abnormal expression pattern of genes in a particular metabolic pathway, responded poorly to the rosiglitazone. This study suggests that a person’s own stem cells may be used in a personalized medicine manner to predict how they will react to a therapeutic before administration of the drug to the individual.

Do you work in the area of stem cells or genetic diseases? In the Kerafast catalog you will find Prader-Willi and Angelman Syndrome iPSCs, as well as a cystic fibrosis human bronchial epithelial cell line and Drosophila adult muscle progenitor-like cells.