ALS is a severe neurological disease-causing neuron death in the brain and spinal cord. Primarily affecting people over 55, it's more common in military veterans and those with prior brain injuries or infections. Early symptoms include muscle weakness or stiffness, leading to the loss of voluntary muscle movement for basic functions like chewing, walking, talking, and breathing. Unfortunately, ALS patients usually die from respiratory failure within five years of diagnosis, and no effective treatments exist to halt its progression. Recent findings reveal that the barrier between circulating blood and the nervous system breaks down early in the disease, even before neuron death. This barrier is made up of semipermeable endothelial cells, which prevent solutes from entering the nervous system where neurons are located. Our research shows that EVs from various cells can improve the function of this barrier. In our project, we are testing the therapeutic potential of EVs in animal ALS models to assess their ability to reduce neuron death and slow the disease's progression by repairing the damaged blood-nervous system barrier. If successful, this study could pave the way for future clinical trials, aiming to enhance the quality of life for ALS patients and potentially extend their survival.
Funding Support: The US Department of Defense (Award # HT94252310138)
