Extracellular vesicle-based therapy for amyotrophic lateral sclerosis

Amyotrophic lateral sclerosis (ALS) is a disease that causes the fast breakdown of motor neurons in the brain and spinal cord. These motor neurons are essential for controlling muscle movement, and their loss leads to disability. There are few treatments for ALS, so new methods to prevent further damage to motor neurons are urgently needed. One way to protect motor neurons could be by fixing the barrier between the blood and the central nervous system (CNS). This barrier helps to prevent harmful substances in the blood from entering the CNS and causing damage. In ALS, this barrier is not working properly, which may lead to the motor neuron damage. Researchers have found that certain cells from human bone marrow, called endothelial progenitor cells (hBM-EPCs), may help repair this barrier.

These cells can release factors that help heal damaged cells in the barrier. Additionally, they produce small particles called extracellular vesicles (EVs) that carry helpful substances, which can further assist in fixing the damaged cells. In a laboratory study, these EVs were taken up by mouse brain cells, and the damage to these cells was significantly reduced. However, when a molecule called β1 integrin was blocked, the EVs could not be taken up by the cells. This research suggests that hBM-EPCs, and specifically the EVs they produce, may have potential as a new treatment for ALS by repairing the damaged blood-CNS-barrier.