Can gene therapy be used to treat autoimmune encephalitis?
Back in July, we briefly discussed how a Clinical Research Unit at the Universitätsmedizin Berlin was funded with 6.2 million euros to study autoantibodies in neurological diseases. Their goal was to identify the prevalence, targets, and functions of autoantibodies in neurological diseases while also developing diagnostic tools and innovative therapies.
Today, they are testing a new treatment for autoimmune encephalitis. Using gene therapy, this treatment targets and eliminates disease-causing cells through reprogrammed white blood cells.
In this case, scientists focused on anti-NMDA receptor encephalitis, which occurs when the immune system attacks the brain's NMDA receptors that are central to the formation of memory and learning. Patients with this type of encephalitis may experience impaired consciousness, epileptic seizures, psychosis, and memory impairment. Currently, treatment involves immunotherapy such as corticosteroids or intravenous immunoglobulin (IVIG).
With the new treatment, researchers have genetically reprogrammed T-cells—a type of white blood cells—from the patient's blood into chimeric autoantibody receptor T cells, or CAAR-T cells. Once these newly reprogrammed T-cells are reintroduced into the body, they then attack specific cells that produce misdirected antibodies. This treatment has shown success in a laboratory setting and is expected to be tested on patients and healthy controls in the next few years.
What's remarkable about this type of treatment is that it only targets the cells that produce the misdirected antibodies as opposed to systemically shutting down a patient's immune system. Moreover, current evidence shows that once these aberrant cells are destroyed, they do not usually reproduce, leading to the possibility of a short-term treatment that can potentially cure the autoimmune disease.
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