Specific inhibition in a type of nervous system cell, called astrocytes, of a mutated protein associated with ALS helps protect motor neurons and muscle function in mice.
The death of motor neurons in ALS results in muscle paralysis and wasting, dramatically impacting quality of life for patients as paralysis progresses. This is because as the motor neurons die they no longer connect to muscles and are unable to convey signals from our brains and spinal cord to the muscles. As patients can no longer move their muscles they start to waste away. To find a treatment that helps preserve motor neurons and keep them connected to muscles would be very beneficial to ALS patients. Astrocytes exist in close proximity to motor neurons. In healthy conditions, astrocytes support the motor neurons in many ways. This support can be reduced in disease, and in the case of ALS, astrocytes containing mutant SOD1 can become actively toxic to motor neurons, contributing to their death.