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.
In a clinical trial conducted at Augusta University Medical College of Georgia, doctors have found that autologous [the patients’ own] stem cell injections can restore estrogen levels in the ovaries, the lack of which contributes to Premature Ovarian Failure (POF). Patients who received the injections saw an increase in estrogen levels in 3 months following the injections and, in 6 months, resumed regular menstruation cycles, which had ceased due to premature menopause. POF primarily affects women under 40 and causes the ovaries to cease their normal functions, leading to premature menopause, and rendering the women incapable of conceiving a child. The procedure involves the collection of the patients’ own stem cells, which are then isolated and re-introduced into the body to rejuvenate the prematurely aged ovaries. The study uses a minimally invasive treatment to inject the stem cells into one ovary, leaving the other untreated as a control. Through ultrasound imaging, the treated ovary has increased in size compared to the control, signifying a restoration in normal ovarian function.
Researchers at UC Davis have created lab-grown brain organoids that are complex and vascularized, dramatically furthering research for brain disorders. Given that the human brain is one of the most complex anatomical structures and researchers are still discovering new functions and neuronal pathways, having brain organoids in vitro greatly expedites this research. When several small brain organoids joined together, researchers observed nerve impulses among the structures, signifying cellular communication that resembles that of fully-grown human brains. In a recent development, these organoids have vascularized and have brought researchers one step closer to both understanding neurological disorders, as well as helping patients replace damaged neurons from conditions like strokes, Alzheimer’s etc.
Researchers at the University of Chile have found that mesenchymal stem cell (MSC) injections could treat a genetic predisposition for excessive alcohol consumption. In an animal model, rats that were bred to consume high volumes of alcohol received intracranial MSC injections, which are meant to treat the neuroinflammation exhibited in individuals who chronically abuse alcohol [and drugs]. The study revealed that following the injections, voluntary alcohol intake decreased dramatically. These are promising results for the many people suffering from a predisposition to alcoholism. Additionally, the study addressed the significant hurdle of devising an effective method of delivery for the stem cells. While intracranial injections ensure the most concentrated delivery of stem cells, it is not ideal. Intravenous delivery also proves difficult given the highly selective blood-brain barrier. The researchers addressed both issues by effectively reducing cell size by growing MSCs in a spheroid aggregate and then administering them intravenously. This enabled the MSCs to be delivered to the brain effectively and efficiently, reducing the alcohol intake by 90% in as little as 48 hours.
Researchers at Tufts University School of Dental Medicine are creating bioengineered teeth from dental stem cells, with the ultimate goal of replacing dental implants. The innovative approach utilizes a hydrogel that has been developed to encapsulate dental stem cells enabling them to differentiate and grow into buds that can be implanted to grow into healthy teeth. In a pre-clinical model, the procedure involves using the hydrogel to provide a scaffold for the stem cells to grow in vitro, followed by the implantation of the scaffold in vivo, where the stem cells developed into whole tooth structures.
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.
In a major breakthrough, researchers are one step closer to growing functional kidneys from human stem cells with the potential of eliminating the need for donated kidney transplants. The collaborative effort by the researchers at Murdoch Children’s Research Institute, University of Melbourne and Leiden University Medical Center has made progress in vascularizing a lab grown kidney organoid. Kidney tissue has been successfully grown in a lab - with all requisite cell types. However, vascularizing the tissue (allowing for blood flow) has proved difficult. This breakthrough research effort has overcome this obstacle to kidney replacement. In an animal model, researchers implanted the human stem cell-derived organoid into healthy kidney tissue, with the organoid maturing and vascularizing into fully fledged kidney tissue in vivo in 4 weeks.
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.
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.