A new study from Northwestern Medicine and the Mayo Clinic has described how stem cells can be used to treat a rare neurological disease. The condition, called neuromyelitis optica, can cause sufferers to become blind and lose the ability to walk within a few years of diagnosis.
Most of the study’s participants remained healthy for five years after the transplant. They also didn’t require the expensive drug treatments normally associated with the condition.
Neuromyelitis optica was formerly considered a rare subtype of multiple sclerosis (MS) but is now considered a unique disease. Unlike other auto immune conditions, neuromyelitis optica has a biological marker (called AQP4) which is directly correlated with the progression of the illness.
The patients in the study who received a stem cell transplant did not have discernible levels of AQP4 in their blood. This was a major breakthrough as lead author Dr. Richard Burt explained: “No prior therapy has caused AQP4 to consistently disappear or allowed patients to become treatment free.”
He continued “There is marked difference between a transplant and the drug. The transplant improved patients’ neurological disability and quality of life. They got better, and the disease maker disappeared for up to five years after transplant.”
The Mayo Clinic holds a patent on the AQP4 marker and performed the analysis in collaboration with researchers from Northwestern.
The trial involved a total of 12 patients who had just been diagnosed with neuromyelitis optica. After five years, only 2 of the patients had relapsed, requiring the use of drug therapy.
The scientists used hematopoietic stem cell transplantation (HSCT) to “reboot” the immune systems of each patient. HSCTs are blood-forming stem cells which can produce both white and red blood cells.
The process begins with patients receiving chemotherapy to destroy their immune system, before receiving a stem cell transplant to re-establish a “rebooted” immune system. The rebooted immune system does not have the abnormalities which previously caused it to attack healthy tissue — which is the root cause of autoimmune diseases.
Source: Stem cell transplant reverses disabling MS-like disease
{{cta(‘d62560c2-336a-4f88-9683-44a37a5bd7fd’)}}