Researchers at Hospital De San Jose in Colombia have utilized autologous (the patients’ own) stem cells to regenerate bone in children with cleft palates, greatly improving their quality of life by replacing an often arduous, surgically invasive procedure with a stem cell graft.The children partaking in the study were born with cleft palates, which typically require surgery and extensive grafting with bone from elsewhere in the body to create enough bone matter to support future teeth. When the children were born, their parents made the wise decision to bank their children’s powerful cord blood stem cells, which became vital to the success of this later treatment. This groundbreaking study used the patients’ own stem cells and a biological scaffold to allow the stem cells to grow into bone and fill the cleft. The ability to use autologous stem cells posed no risk of rejection to the patients, and in 5 and 10-year follow ups, the patients showed healthy bone development and experienced no adverse effects.
Researchers at the University of Texas Medical Branch are using autologous (the patient’s own) stem cells to successfully transplant entire lungs without the risk of rejection. In animal models, researchers obtained a lung from a donor and removed all blood and cells, leaving a lung scaffold. Then, they obtained autologous lung stem cells from the subject and seeded the lung scaffold so that the lung would be repopulated. This created a brand new lung for transplantation, comprised of cells that would not be rejected because they are the patient’s own. When implanted back into the body, the engineered lungs were able to grow and vascularize with no additional treatments or infusions. This protocol could potentially be expanded to provide life-saving organs for hundreds of thousands of patients waiting for organ transplants, which, besides the obvious shortage, still pose a risk of immune rejection.
Organs-on-Chips are set to be studied in zero gravity at the International Space Station. Astronauts who go into space have been known to experience changes in their health and immune response, but until recently, the reasons for these changes remained largely unknown. Previously, animals were sent as a way to determine the long-term health effects of being in space. However, since every organism functions differently, this approach, while useful, had obvious drawbacks. Organs-on-Chips [OOCs] are an innovation created by a collaborative effort of the Wyss Institute of Harvard University and the Massachusetts Institute of Technology, among others. OOCs are small vessels that utilize stem cells to create various tissue types to simulate the conditions inside human organs. If the tests prove successful, these tiny chips will be the closest researchers get to estimating the effects of space travel on human organ function - aside from sending out actual astronauts.
A mesenchymal stem cell treatment for patients with cardiac muscle degeneration and ventricular failure is being conducted at the MedStar Heart and Vascular Institute. The patients currently being recruited for the study are those who require left ventricular assist devices (LVADs) in order to pump their heart, and these patients are in severe stages of cardiac failure. In pre-clinical models, intravenous mesenchymal stem cell injections have greatly improved left ventricular function, which is responsible for pumping and pressurizing the blood to the rest of the body. Additionally, there was a significant decrease in the inflammatory response that is indicative of damaged cardiac muscles. By reducing inflammation researchers hope to not only provide immediate relief for the strained cardiac muscle, but also slow or stop the progression of heart failure.
A major obstacle to successful bone marrow transplants (BMT) is rejection due to the age discrepancy of the donor and recipient, with older donors presenting problems due to the donor stem cells’ loss of efficacy with age. The older stem cells’ compromised ability to actively regenerate (given that older stem cells are less active than younger stem cells) increases the risk of age-related rejection significantly. In a groundbreaking study, researchers have discovered that the in-vitro (outside the body) introduction of young mesenchymal stem cells (MSCs) to aged donor hematopoietic stem cells (HSCs) used for transplants resulted in the rejuvenation of the donor cells likely improving the efficacy of the transplant.
Families choosing to bank their stem cells – usually in the form of cord blood and/or dental pulp stem cells, typically view their decision as “biological insurance.” A Phase II clinical trial is investigating the safety and efficacy of autologous [the patient’s own] cord blood stem cells to treat children with behavioral and social difficulties associated with Autism Spectrum Disorder (ASD). In a clear demonstration of the value of banking your own stem cells, only families that chose to bank their children’s cord blood were qualified to participate in the study.
Phase III clinical trials were announced for an autologous [the patient’s own] stem cell treatment to restore blood vessels and reestablish blood flow following critical limb ischemia (CLI). Ischemia is a lack of blood to an area in the body, typically due to a blood clot, and is common in diabetics and other patients whose conditions result in damage or clotting in the blood vessels. In extreme cases, ischemia can lead to painful ulcers, gangrene and even amputation, given that cells in the area of decreased blood flow begin to die rapidly. The treatment seeks to facilitate re-vascularization through the administration of both blood stem cells and endothelial progenitor cells, which form the walls of blood vessels.
Researchers at Cornell University are working on a stem cell-infused implant that could cure insulin deficiency for diabetics. Type I diabetes results from inadequate or malfunctioning insulin-producing beta cells in the islets of the pancreas, as well as an autoimmune response that attacks the body's insulin-producing cells. This treatment utilizes stem cells and directs them to differentiate into these cells. As opposed to daily insulin injections, the treatment is designed to provide a long-term solution that eliminates the need to constantly monitor blood sugar. It utilizes a naturally derived hydrogel to create a thread packed with stem cells induced to become pancreatic islets which is then implanted into the abdomen. Additionally, the treatment addresses what no other current treatment addresses: the body’s immune system attacking the insulin-producing cells. Encasing the cells protects them from the autoimmune response, increasing their efficacy and lifespan.
UCLA researchers are using stem cells and gene therapy to reverse the effects of HIV. The treatment utilizes stem cells to carry the chimeric antigen receptor (CAR) genes that have been successfully used to treat leukemia and are being explored for other cancers. The modified stem cells can trigger the immune system to specifically target and destroy HIV infected cells without harming nearby healthy cells. The stem cells carrying the gene are able to directly interrupt the mechanism between the virus and body cell surface receptors that allow the virus to infect the cells by binding to the virus and destroying it.
UCLA researchers have developed a potential stem cell based treatment for Duchenne Muscular Dystrophy (DMD). This degenerative muscular disorder - caused by a genetic mutation in a protein essential for skeletal muscle function - primarily affects young men and boys, degenerating skeletal muscle to the point where it eventually wastes away. This severe degeneration of skeletal muscle results in almost a complete lack of voluntary movement. The UCLA team used the gene editing technology, CRISPR, to repair the mutated gene for the protein in stem cells obtained from the subjects and reintroduced the re-programmed muscle cells back into the subjects. In an animal model, the re-programmed cells successfully produced the dystrophin protein, reversing the degeneration of the skeletal muscle. The team has also conducted a second set of biological markers to ensure complete differentiation into adult muscle cells.