A Phase II clinical trial is currently underway to evaluate the efficacy of retinal progenitor cells in treating a retinitis pigmentosa that causes visual impairment and, in severe cases, blindness. In an earlier clinical trial, the stem cells were used to create new photo receptors, with patients reporting improvements in vision. In follow-ups conducted between 18 days and 2 months following treatment, patients were able to read three additional lines in a standard eye test chart.
Researchers at University of California San Francisco (UCSF) have created insulin producing cells in vitro that successfully produced insulin in vivo for Type I Diabetes patients. Type I diabetics experience an autoimmune disorder which attacks and destroys the body’s insulin-producing beta cells. These patients have to take continuous insulin injections and closely and constantly monitor their blood sugar levels, since extremely high or low blood sugar levels cause diabetic ketoacidosis or hyperglycemic shock, leading to coma and death. Though diabetes is currently manageable, patients must be constantly vigilant since their bodies’ inability to regulate blood sugar often leads to other systemic diseases such as blood vessel damage, neuropathy and nephropathy, just to name a few. The study from USCF involved directing human pancreatic stem cells to become insulin-producing islets cells in the lab. In an animal model, the cells were then implanted back into the body and were shown to produce insulin in response to blood sugar spikes. Additionally, the islets produced other essential hormones for blood sugar regulation, fully resembling normal pancreatic islets.
The U.S. Department of Defense [DOD] has approved a grant of $2 million to the University of Arizona [UA] to advance the development of their technology combining 3D printing and stem cell grafting to create a better alternative to conventional bone replacement. Current standard of care for shattered bones involves using cadaver bones and support rods to replace bones entirely. However, these treatments are often ephemeral since the cadaver bone is dead and becomes increasingly fragile over time. The technique being developed by UA utilizes advanced 3D printing to create a scaffold that mimics the structure of bone and then seeds it with the patient’s own stem cells, along with calcium, to grow a bone that will be sturdier. Since the technique will use the patient’s own stem cells, it virtually eliminates the possibility of rejection.
Researchers at the University of Pennsylvania have developed bio-engineered replacement spinal discs. Intervertebral discs are located between the bones of the spine to absorb shock, prevent the bones from painfully rubbing together and protect the nerves of the spinal cord. Degraded discs cause intense chronic pain, which is often debilitating and diminishes a person’s quality of life. The current standard of care involves replacing a damaged disc with a synthetic replacement, which does alleviate some pain, but does not compare to real cartilage. In an animal model, autologous (the patient’s own) mesenchymal stem cells (MSCs) were seeded into a biological scaffold where they differentiated into cartilage tissue. When the disc was fully-formed, it was surgically inserted back into the spine, and in a 20 week follow-up the disc maintained its structure and performed as normal.
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.
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.
In a recently published study at Cedars-Sinai Heart Institute, stem cells obtained from younger subjects and injected into aging subjects resulted in improved heart function, and an overall increase in stamina and activity levels. As we age, our heart muscles begin to stiffen, causing fluid to build up in the heart and preventing the muscles from relaxing properly. This is similar to hearts of patients who have experienced heart failure with ejection fraction. Therefore, this research is pivotal in treating both heart failure and age-related deterioration. In an animal model, mice that received the progenitor cells (a more specified type of stem cell) obtained from younger mice showed multifaceted beneficial results. Not only did the older mice display improved heart function, but their activity levels increased, and their telomeres, which shorten as cells age, were regenerated. The implications of this research show that though the stem cells were injected into the heart, beneficial effects were seen all over the body, in addition to showing that younger stem cells are in fact far more proliferative than older cells.
Researchers at the University of California Irvine have created a method of engineering mesenchymal stem cells (MSCs) to specifically target and help destroy cancer metastasis, which is an indicator of cancer spreading and the cause of approximately 90% of cancer deaths. The researchers are utilizing MSCs that have been engineered to detect stiffened tissues, a typical indicator of breast cancer metastases. These stem cells then release an enzyme upon detection of the cancer cells that triggers the activation of a localized chemotherapy. This is a revolutionary method of treating cancer given that one of the biggest concerns with chemotherapy is its ability to not only harm cancer cells, but also harm healthy cells as well.
A phase III clinical trial utilizing autologous [the patient’s own] mesenchymal stem cells (MSCs) has begun, and could offer relief to the millions suffering from ALS. The study is being conducted by Brainstorm Cell Therapeutics with a grant of $16 million from the California Institute for Regenerative Medicine [CIRM].Brainstorm has developed a proprietary method [called NurOwn] for inducing MSCs to secrete neurological growth factors, which exhibits the ability to perpetuate the life of neurons experiencing rapid degradation in ALS patients. In previous clinical trials the treatment demonstrated the ability to slow the progression of ALS immediately following the treatment. The new trial seeks to prolong these beneficial effects.