A study has found that bone marrow transplants can restore nerve cell damage in mice with Friedreich’s ataxia (FA), forming new nerve cells and improving movement.
FA is a genetic disorder caused by an FXN gene mutation for frataxin. Frataxin is a protein that allows the mitochondria in cells to function properly. Without Frataxin, energy production in cells is impaired, and the unbound iron in the body, as a result of Frataxin not performing properly, causes cell damage.
This often leads to neurological disability in patients, leading to loss of coordination in limbs, vision impairment, hearing loss, and many other common symptoms. But despite an increasing understanding of FA and its causes, there is still no treatment available to repair damaged nerve tissue.
However, research is finding that bone marrow stem cells might be able to migrate and integrate into the nervous system and create new nerve and glial cells. According to researchers, transplants from healthy donors could be a remarkable improvement for treatment and slowing progression in diseases like FA.
Using a mouse model of FA, scientists recently looked into bone marrow transplants as a way to repair cells carrying a normal FXN gene. They tagged the cells with a fluorescent light, and monitored the progress of the mice’s mobility over the course of six months. The mice were then euthanized to evaluate the proteins and changes in their nervous systems.
Movement in the mice over the six months increased dramatically, and led to higher levels of frataxin. The disease manifested at a much lower rate, and the stem cells were able to integrate into areas of injury and create new mature nerve cells.
SCF and G-CSF, both proteins that can aid in stem cell therapy, were found to increase the number of bone marrow-derived cells in the blood, as well as lead to additional improvements in movement and less inflammation in the brain and spinal cord. However, researchers admit that further work must be done to determine the mechanism by which G-CSF and SCF generate such benefits.
Though not a cure, this study offers hope in showing opportunity for neuro-regenerative treatment in patients with FA and similar neurodegenerative diseases.