Stem Cells in the News - September 2017
Wednesday, September 06, 2017 - 13:49
We have captured this month's most interesting, innovative, and maybe some of the strangest examples of stem cells in the news from around the world.
A significant number of blood cancers have something in common, a mutation in the TET2 gene. This gene encodes an enzyme that drives stem cells to develop into fully mature blood cells that will eventually die. A TET2 mutation can cause blood cancer stem cells to have a prolonged life, perpetuating cancer progression. Studies by researchers at NYU Langone Health suggest that aberrant TET2 activity can be restored via high doses of Vitamin C. This mechanism holds promise in blood cancers lacking the mutation, as Vitamin C upregulates TET2 activity, encouraging cell maturation with or without the mutated TET2 gene.
A landmark study was recently published in Nature that utilized the power of CRISPR-Cas9 to correct pathogenic mutations in human embryos. Specifically, this study generated induced pluripotent stem cells (iPSCs) from skin biopsy tissues obtained from a male with Hypertrophic cardiomyopathy (HCM). The research teams used CRISPR-Cas9 on the iPSCs to target the mutated gene causing HCM, effectively splicing out the “bad” gene. When testing the gene-edited cells in a fertilization experiment, the team saw that a high percentage of embryos contained the repaired gene. They also noted no off-target mutations or genomic instability. While future testing is needed, this is a huge advancement for the utility of gene-editing in stem cell research.
A team of researchers at the Max Planck Institute for Infection Biology in Berlin have found a link between Helicobacter pylori and stomach epithelium stem cells that may lead to a breakthrough in the diagnosis and treatment of gastric carcinoma. The study identified two distinct subpopulations of stem cells in the gut, one of which, when exposed to H. pylori infection increases its rate of self-renewal and the production of a thick mucosa that is commonly used to identify gastric carcinoma. This study provides some of the first hard evidence of chronic bacterial infections promoting cancer development.
In light (no pun intended) of the total solar eclipse that traveled across North America this summer, we would like to highlight a review recently published in Stem Cells that discusses the use of stem cells in corneal wound healing. Specifically, this review is the first to include data and therapies addressing damage to the major corneal cell types, all of which have unique responses to wound healing processes and cell therapy treatment strategies.
In a collaborative effort with scientists at the University of California - San Diego (UCSD), Neuralstem has been awarded a Phase 2 Small Business Innovation Research (SBIR) grant by the NIH to the tune of $997,068 over the next two years. Their grant will fund more in-depth research into their novel small molecule compound, NSI-189, for the protection and improvement of nerve cell function in patients with Type 1 and Type 2 Diabetes. They have already shown promising results in the protection of motor and sensory nerve function in both Type 1 and Type 2 Diabetes in a mouse model.
Over the last 20 years, genetic engineering and utilization of hematopoietic stem cells has dramatically changed the treatment strategies for diseases of the blood and immune system. This review, published recently in Current Opinions of Hematology, provides an overview of the latest technology surrounding the gene editing of hematopoietic stem cells, including research findings during the discovery stage as well as from current preclinical and clinical trials for the treatment of hematopoiesis-related genetic diseases.
A team at the University of California - Los Angeles has discovered that lactate production in hair follicle stem cells is key to the progression of balding. Using genetic engineering in mouse hair follicle stem cells, this research team found that blocking lactate production from glucose halted stem cell proliferation. Conversely, over-production of lactate activated hair follicle stem cells. These studies have been conducted on mice, but hold promise in humans suffering from alopecia, hormone imbalances, and even old age.
Scientists at the University of North Carolina School of Medicine and North Carolina State University have investigated a new stem cell therapy option for those suffering from fibrotic diseases of the lung, including Chronic Obstructive Pulmonary Disease (COPD) and Cystic Fibrosis. The study utilized a minimally invasive technique to isolate and expand cells from rat lung tissue. These cells were then used to replace damaged fibrotic tissues. The transplantation did not trigger a harmful immune response, even when the cells were not autologous. These results outperformed other studies using mesenchymal stem cells, which are also a new treatment option for fibrosis.
BlueRock Therapeutics is expanding its presence in Toronto with a new site in the MaRS Discovery District. The facility will focus on research, development, and manufacturing of their therapies for degenerative diseases. This expansion comes alongside their announced collaboration with the McEwen Centre for Regenerative Medicine, which will grow their expertise through a new network of experts in the field. They also announced a partnership with the Centre for Commercialization of Regenerative Medicine (CCRM), to help accelerate their manufacturing capabilities.
Researchers at the Francis Crick Institute in London have found a solution to male infertility caused chromosomal diseases, like Klinefelter Syndrome or Double Y Syndrome. Men suffering from either of these conditions have difficulty producing fertile sperm, but with advances in induced pluripotent stem cell (iPSC) technology, the team at Francis Crick created viable sperm in a dish. The catch? When the iPSC-derived pre-spermatogenic cells are injected into the testes for final development, the treatment resulted in cancer 29-50% of the time. At this point, in vitro sperm development and maturation is the only option to ensure normal sperm are used for fertilization and to avoid carcinogenesis.
With recent stories of unapproved stem cell therapies resulting in blindness and death, an unassuming scientist from University of California - Davis, Dr. Paul Knoepfler, has emerged as an unofficial watchdog for patients, patient advocates, and many researchers. Since 2010, Knoepfler has used his blog, The Niche, as a platform to discuss findings in stem cell research, progress in clinical trials, and advise potential patients on the risks and validity of stem cell treatments at clinics all over North America. Read more about why he does it in the linked article.
Last month, Viacyte Inc., a medical device company outside San Diego, CA, implanted a revolutionary stem cell treatment into two people with Type 1 Diabetes. Their patented technology is a sub-dermal implant that houses progenitor cells derived from embryonic stem (ES) cells. The credit card-sized implant contains pores that allow vessels to penetrate inside to nourish the progenitor cells, which differentiate into functional islet cells approximately 3 months after transplantation. If successful, this implant could change the way we treat Type 1 Diabetes. However, generating a limitless supply of these progenitor cells will be key to the success of this revolutionary treatment strategy.
It has been widely believed that stem cells lose their circadian rhythm over time, resulting in the acceleration of aging in these cells. However, researchers at the Institute for Research in Biomedicine in Barcelona, Pompeu Fabra Univerisity in Spain, and the University of California - Berkeley, have found this not to be the case. Their findings, recently published in Cell, show that aged stem cells actually conserve their circadian rhythms. They attribute the acceleration of aging in these cells to their shift in focus from tissue regeneration and maintenance to protection from cell stress. Their findings, using a mouse model system, suggest that a low-calorie diet can preserve the tissue maintenance function in stem cells.
A new therapy to treat Osteoarthritis (OA) is emerging from The Emory Orthopedics and Spine Research Center at Emory University. The treatment is, simply: extraction, concentration, and reinjection of mesenchymal stem cells directly from patients. Emory University started using stem cell-based therapies about 5 years ago, but this study focused on specifically growing back cartilage. Their process is unique in that the entire treatment takes only minutes - from extraction to concentration and reinjection. While more work and approvals need to be addressed, these promising first results have many excited and hopeful for a new treatment option for those affected.