Precision medicine seems to be the new hot topic in the research world. President Obama spoke about precision medicine in his State of the Union speech on January 20, his budget released today requests $215M for precision medicine, and NIH just announced plans for a study of a million or more volunteers to explore precision medicine. What precisely is it? The White House website has a useful definition: getting the right treatment at the right time to the right person. The President, in an event devoted to precision medicine in the East Room last Friday [January 30, see video, below], told the story of ivacaftor, a drug that effectively treats the underlying causenot the symptomsof cystic fibrosis, but works in only 4% of patients who have a specific mutation in the gene causing this disease. Most of the conversation about precision medicine focuses on cancer. Because cancer is a disease of genetic mutations leading to unregulated cell division, defining the precise mutations in the affected tissue have already led to breakthrough treatments for both blood and solid tissue cancers. In fact, the same mutation can occur in different parts of the body, so cancer is increasingly diagnosed in terms of … Continue reading →

Adult Stem Cells (ASCs), by definition, are unspecialized or undifferentiated cells that not only retain their ability to divide mitotically while still maintaining their undifferentiated state but also, given the right conditions, have the ability to differentiate into different types of cells including cells of different germ-origin an ability referred to as transdifferentiation or plasticity.1,2 In vitro, the conditions under which transdifferentiation occurs can be brought about by modifying the culture medium in which the cells are cultured. In vivo, the same changes are seen when the ASCs are transplanted into a tissue environment different to their own tissue-of origin. Though the exact mechanism of this transdifferentiation of ASCs is still under debate, this ability of ASCs along with their ability to self-renew is of great interest in the field of Regenerative Medicine as a therapeutic tool in being able to regenerate and replace dying, damaged or diseased tissue. Clinically, however, there are a few criteria that ASCs need to fulfill before they can be viewed as a viable option in Regenerative Medicine. These are as follows:3 Adds Millions of Stem Cells Back into Circulation. Adipose Tissue Yields an Abundance of ASCs Compared to any other source, the high concentrations … Continue reading →

Among patients with severe aplastic anemia who received stem cell transplant from an unrelated donor, longer leukocyte (white blood cells) telomere length (a structure at the end of a chromosome) was associated with increased overall survival at 5 years, according to a study in the February 10 issue of JAMA. Telomeres protect chromosome ends and are essential for maintaining chromosomal stability. Telomere length is a biological marker for cellular aging and the capacity to replicate. Aplastic anemia is a blood disorder where the bone marrow fails to make new blood cells, with one of the causes potentially being defects in telomere biology. Allogeneic (genetically different) hematopoietic (blood marrow) cell transplantation (HCT) is recommended as initial therapy for young patients with acquired severe aplastic anemia when a matched sibling donor is available, according to information in the article. Shahinaz M. Gadalla, M.D., Ph.D., of the National Cancer Institute, National Institutes of Health, Rockville, M.D. and colleagues evaluated the association between recipient and donor pretransplant leukocyte telomere length with outcomes after unrelated donor allogeneic HCT for 330 patients with severe aplastic anemia. The patients and their unrelated donors had pre-HCT blood samples and other clinical results available at the Center for International … Continue reading →

On the weekend, a whos who of hockey legends gathered to pay tribute to Gordie Howe in his hometown of Saskatoon. In addition to sharing memories about Mr. Hockey, a constant theme of the festivities was his miracle recovery from stroke. Mr. Howe, 86, suffered two strokes last year and, according to his family, was near death before he travelled to Clinica Santa Clarita in Tijuana, Mexico, in December for experimental stem-cell treatment. Afterward, Mr. Howe was able to walk again. He regained a lot of weight and he began to resemble his old self. (Most of this is second-hand; Mr. Howe also suffers from dementia and has not or cannot speak of his symptoms or treatment first-hand.) After his stem-cell treatment, the doctor told us it was kind of an awakening of the body, his son, Marty Howe, told The Canadian Press. They call it the miracle of stem cells and it was nothing less than a miracle. Mr. Howes Lazarus-like recovery makes for a great tug-at-the-heartstrings narrative for a man whose career has been the embodiment of perseverance and longevity. But if you step back a moment and examine the science, all sorts of alarm bells should go … Continue reading →