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.
In a phase 1 clinical trial, a novel mesenchymal stem cell [MSCs] therapy was successfully applied to treat often fatal steroid resistant graft versus host disease [GVHD]. A significant concern with life-saving organ and bone marrow transplants is the risk of immune rejection. GVHD occurs when the transplanted immune cells attack the patient’s body, with patients typically suffering from painful ulcers all over the body, and in extreme cases, death. The therapy, created by Cynata Therapeutics, is called CYP-001, and utilizes mesenchymal stem cells [the same type of cells found in teeth] to treat steroid resistant GVHD. This treatment gives hope to thousands of patients around the world receiving bone marrow transplants and risking the extremely fatal immune response.
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.
Dr. Patricia Braga and her team at the University of Sao Paolo, in collaboration with Alysson Muotri, professor of pediatrics and cellular and molecular medicine at UC San Diego, are using dental stem cells from donated baby teeth to grow neurons and examine the role of astrocytes in the expression of Autistic traits such as language impairment, repetitive behaviors and sleeping difficulties. Dr. Braga has used dental pulp stem cells from two groups of patients - children with Autism and a non-autistic control group, and directed their stem cells to differentiate into brain cells in vitro. When allowed to grow, the stem cells developed into clusters that contained the star-shaped brain cells called astrocytes, as well as fully grown neurons. Upon closer inspection, the astrocytes and neurons from children with Autism showed significant functional differences compared to the control group cells. Autistic astrocytes release excessive amounts of an inflammatory molecule called interleukin-6 (or IL6), which, in concentrated amounts, can harm nearby neurons and hinder their functionality. Additionally, the neurons from Autistic children were found to fire less frequently, form fewer synapses (connections with other neurons) and release less glutamate, which is used to excite surrounding neurons and transmit signals.
Phase III clinical trials for a stem cell based ALS treatment has been initiated. ALS, or amyotrophic lateral sclerosis, is a disorder in which motor neurons in the body rapidly degenerate, and the treatment aims to prolong the survival rate of afflicted individuals by using autologous [the patient’s own] mesenchymal stem cells (the same stem cells found in teeth), which can be differentiated into fully-functioning neurons. The trials, to be conducted by BrainStorm Therapeutics, exploits the company’s proprietary technology [NurOwn], which utilizes mesenchymal stem cells. BrainStorm obtains these cells from the patient, expanding and differentiating the stem cells prior to application. The stem cells begin producing neurotrophic factors that facilitate neuronal growth and regeneration.
Artist and researcher Amy Karle is collaborating with researchers at the California Academy of Science and Autodesk to develop a method to 3D print a human arm using stem cells. Karle created a 3D printed scaffold and a culture medium that will direct the stem cells to grow into the structure of the bones in the arm. Her methods could be essential for future limb transplants, which could be grown in a lab rather than obtained from a donor.
Researchers at Cincinnati Children’s Hospital Medical Center Heart Institute have discovered a possible method of directing stem cells to fuse together and form functional skeletal muscle. Utilizing gene therapy, their research has provided insight into correcting disorders like muscular dystrophy, and congenital and metabolic myopathy. By identifying the genes Gm 7325 and Tmem8c, as well as their corresponding proteins, researchers have determined their paired role in directing muscle stem cells (myoblasts) in fusing and creating functional striated muscle tissue, which is responsible for movement.
Researchers at the Mayo Clinic are researching stem cell treatments for amyotrophic lateral sclerosis (ALS), the onset of which remains unknown and the cure for which has yet to be discovered. For patients with ALS, motor neurons in the brain, which are in charge of basic muscle movement, are destroyed, causing paralysis or severe lack of muscle control. The phase I clinical study is investigating the use of mesenchymal stem cells (MSCs), known for their ability to support neural growth and survival, to prevent the premature neuron degradation normally associated with ALS, and/or their ability to slow the progression of the disease.
Wan-Ju Li and Tsung-Lin Tsai, researchers at the University of Wisconsin-Madison, have developed a more efficient method to regrow large masses of bone. Using the proteins lipocalin-2 and prolactin, the researchers were able to slow and neutralize senescence, a naturally occurring process that negatively impacts the ability of stem cells to divide and grow, thereby preserving the regenerative capabilities of the mesenchymal stem cells and facilitating bone growth. The combination of these two proteins in culture provides sustenance for the stem cells to remain in prime condition until they are ready to be implanted into the patient.