A new study has demonstrated the ability of an experimental stem cell therapy to repair brain damage and improve memory function that can be caused by strokes and dementia.
The UCLA study has found that a one-time injection of an experimental stem cell therapy can promote recovery by repairing brain damage and improving memory function in mice with conditions that replicate human strokes and dementia. Dementia is characterised by an array of symptoms including problems with memory, attention, communication, and physical coordination with the two most common causes of dementia bring Alzheimer’s disease and white matter strokes.
The study has been published in Science Translational Medicine.
In the study, the researchers used a specialised type of glial cells which surround and support neurons in the central nervous system. Dr S. Thomas Carmichael, senior author of the study and interim director of the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA and his team evaluated the effects of the glial cell therapy by injecting it into the brains of mice with brain damage similar to that seen in humans in the early to middle stages of dementia.
The therapy was developed in collaboration with Bill Lowry, a UCLA professor of molecular, cell and developmental biology. The team used a method, previously discovered by Lowry, for quickly producing large numbers of glial cells by treating human induced pluripotent stem cells with a drug called deferoxamine.
“Upon injection, our cell therapy travelled to damaged areas of the brain and secreted chemicals called growth factors that stimulated the brain’s stem cells to launch a repair response,” said Dr Irene Llorente, the paper’s first author and an assistant research professor of neurology at the David Geffen School of Medicine at UCLA.
This repair response limited the progression of damage, enhanced the formation of new neural connections, and increased the production of myelin, a fatty substance that covers and protects the connections.
Francesca Bosetti, a programme director at the National Institutes of Health’s National Institute of Neurological Disorders and Stroke, which supported the study, said: “Understanding the role that glia play in repairing white matter damage is a critically important area of research that needs to be explored. These preliminary results suggest that glial cell-based therapies may one day help combat the white matter damage that many stroke and vascular dementia patients suffer every year.”
In the future, if the therapy is shown to be safe and effective through clinical trials in humans, the researchers envision it becoming an “off-the-shelf” product. This would mean that the cells would be mass manufactured, frozen, and shipped to hospitals, where they could be used as a one-time therapy for people with early signs of white matter stroke – setting the treatment apart from patient-specific cell therapies, which are created using each individual patient’s own cells.
“The damage from white matter strokes is progressive, so you do not have months to spend producing a treatment for each patient,” said Carmichael, who is also chair of neurology at the medical school. “If you can have a treatment that is already in the freezer ready to go during the window of time when it could be most effective, that is a much better option.”
He added that: “Because the cell therapy is not directly repairing the brain, you do not need to rely on the transplanted cells to persist in order for the treatment to be successful.”
The team is now conducting the additional studies necessary to apply to the Food and Drug Administration for permission to test the therapy in a clinical trial in humans.