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.
The Hospital for Special Surgery has been awarded an $800,000 grant to conduct a Phase II clinical trial utilizing autologous (the patient’s own) stem cells to help mend rotator cuff tears. This sizable grant will be used to treat one of the most common musculoskeletal conditions, particularly in athletes. Surgical repair for rotator cuff tears leave patients with resilient discomfort and pain, and many patients re-tear the muscle and are unable to return to full capacity. Stem cells can ameliorate the treatment process by facilitating recovery. The trial will investigate the efficacy of stem cells in skeletal muscle and tendon tissue regeneration, as well as the reduction of inflammation.
Researchers at the Technion-Israel Institute have directed stem cells to differentiate into neurons with the potential to repair spinal damage that causes paralysis in the legs, known as paraplegia. In an animal model, subjects suffering injury to their spinal cords, causing them to lose all mobility and feeling in their hind limbs, were treated with human stem cells cultured to differentiate into support factors that promote neural growth and survival. Three weeks after administering the stem cell treatment, 42% of the subjects began either walking or showing significant improvements in bearing weight on their hind legs. Furthermore, over 75% of the subjects responded to stimulation in their hind legs. When compared to the placebo group that received no stem cells, the results were impressive and demonstrate the immense potential of utilizing stem cells to restore the neural connections in the spine following a traumatic injury.
A collaborative effort from German and Italian researchers allowed a child dying from severe epidermolysis bullosa (EB) to lead a healthy, normal life. EB is a genetic disorder which causes the top layer of the skin (epidermis) to become extremely fragile and easily blister-prone. Patients with EB typically do not live past the age of 30, given the exorbitant risk of infections and other complications of having “paper thin” skin, and there is currently no cure. However, a recent experimental skin graft, made from the patient’s own stem cells, allowed a young boy to return to normalcy. The graft’s success comes from a technique of genetic engineering to correct the defective gene that causes EB in immature stem cells, and then develops those stem cells into layers of epithelial tissue and applies them to the patient’s affected areas. Though the risk of such procedures is high, using the patient’s own cells minimizes the risk of rejection and provides a safer alternative to merely enduring this disease.
A clinical trial has shown that autologous [the patient’s own] stem cell infusions can accelerate improvements in motor function of children with cerebral palsy. Cerebral palsy occurs when the brain is damaged either before or during birth and has varying levels of severity, but in all cases, affects movement and speech. CP children typically receive physical and occupational therapy and will make subtle improvements with age, as their bodies develop. In the double blind clinical trial in which some children were given a placebo and others were given varying amounts of stem cells, those who received approximately 25 million cells per kilogram of body weight showed substantial improvement in motor skills when tested a year following the treatment. The improvement was significant when compared to the expected normal yearly improvement CP children typically make, and was also greater than that of the children who received the smaller dosage. In the next phase of the clinical trial, researchers seek to determine whether continuous stem cell infusions could improve motor function even more significantly.