In a clinical study, researchers at Queen Mary University of London will utilize autologous stem cells to reboot the immune system of Crohn’s disease patients, with the aim of greatly alleviating the inflammation of the bowel thus significantly improving the patients’ quality of life. Crohn’s disease is an autoimmune disorder in which portions of the bowel are attacked by the immune system leading to severe inflammation, malnutrition and debilitating abdominal pain. Though there is currently no effective cure, this stem cell treatment has shown promise in treating the erroneous attacks of the immune system on the bowel tissues. The treatment involves a stimulation of the bone marrow to release stem cells, which are then harvested, followed by an irradiation of the body to eliminate the malfunctioning immune system. The recovered healthy immune stem cells are then reinfused into the body to reboot the immune system and eliminate the inflammation of the bowels.
A study cited in the Journal of Medical Cases has demonstrated successful results utilizing autologous (the patient’s own) stem cells to repair a torn meniscus without surgical intervention. Meniscal injuries are the most frequent knee injuries, and they are particularly common in athletes, but can also occur due to aging. Typically, meniscal tears are treated with surgical intervention, which requires “stitches” meant to hold the tear together until it heals, or cutting out the torn part entirely. The surgical intervention method, though safe, requires extensive physical therapy to strengthen supporting muscles, as well as weeks of recovery time on crutches. Imaging of a tear following a stem cell therapy showed that 9 months after a stem cell injection, the tear healed to the point where it was virtually unnoticeable.
A phase II clinical study investigating the efficacy of mesenchymal stem cells [MSCs] to treat moderate to severe lupus symptoms has been launched by the Lupus Foundation of America, in collaboration with the National Institute of Allergy and Infectious Diseases (part of the NIH). Lupus is a chronic autoimmune disorder in which the immune system can affect virtually any tissue in the body, including skin, joints and organs. MSCs represent a promising treatment option in that, in addition to the inherent plasticity of MSCs, they also possess immune modulation properties. The NIH is providing resources and oversight for the study, which will investigate how mesenchymal stem cells can effectively regulate and limit the autoimmune response of Lupus sufferers. Currently there are no effective options for their lupus symptoms other than steroid treatments, which have significant side effects, as they are detrimental to vital organ function.
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 collaborative effort between researchers at Stanford University, the Joint Institute of Metrology and Biology, and the National Institute of Standards and Technology has developed a modified and more targeted version of CRISPR, which is more efficient at editing single nucleotide mutations. The new system is called MAGESTIC (multiplexed, accurate genome-editing through short, trackable, integrated cellular barcodes), and it has been shown to successfully modify genes by accurately targeting the location of defective genes. MAGESTIC ameliorates and addresses the current shortcomings of gene-editing technology by enhancing the ability of CRISPR to target single genes [out of millions] with the purpose of correcting specific mutations.
A genetically modified stem cell therapy for Diffuse Large B-Cell Lymphoma (DLBCL) has been approved by the FDA. Researchers at the Abramson Cancer Center, in collaboration with Novartis, have successfully administered a CAR-T Cell therapy, called Kymriah, for the most common type of non-Hodgkin Lymphoma. DLBCL is a fast growing cancer that affects B lymphocytes, which are responsible for producing antibodies that help fight infections in the body. This groundbreaking treatment involves obtaining autologous (the patient’s own) T cells, which are a more specialized type of stem cell, and genetically engineering the cells to track down and destroy cancerous cells.
A Phase I clinical trial to test the efficacy of genetically modified autologous (the patient’s own) stem cells to treat beta-thalassemia has been initiated. This condition is an inherited disease that affects the production of hemoglobin, which is responsible for carrying oxygen in the body and delivering it to tissues and vital organs. With thousands of new cases every year, this condition often results in fatigue, bone fragility and extreme anemia (a deficiency of iron in the blood). This trial aims to create a groundbreaking protocol that would obtain autologous stem cells from the patients, genetically alter them to produce the missing protein responsible for the condition and, reintroduce the stem cells back into the body through a transfusion.
A $225m investment from pharmaceutical giant Bayer catapults BlueRock Therapeutics onto the stem cell therapy stage. BlueRock will initially focus on cardiovascular and neurological treatments. A team led by Dr. Michael Laflamme and Dr. Gordon Keller will concentrate on regenerating heart muscle for patients who have suffered a heart attack, whilst a team led by Dr. Lorenz Studer and Dr. Viviane Tabar will concentrate on restoring dopamine-producing cells in patients with Parkinson's disease .
Scientists from the RIKEN Center for Developmental Biology in Japan have recently grown skin tissue from transformed stem cells. Their work demonstrated an advancement from previous efforts to grow skin in that the transplanted stem cells developed as integumentary tissue – the tissue between the outer and inner skin, which holds the functional properties of the skin, including sweat glands and hair follicles.
Scientists from the University of Nottingham and Harvard University have developed a treatment that takes advantage of the unique regenerative characteristics of stem cells to enable teeth to heal themselves. The treatment represents an advancement over the current methods to treat severe cavities by eliminating the need for root canals. By stimulating the dental stem cells [mesenchymal stem cells] within the tooth, the growth of dentin, the bony material that makes up the majority of the tooth, is encouraged. This allows patients to regrow damaged teeth effectively.