Preventing Synuclein Accumulation as a Strategy for Improving Neuronal Survival and Regeneration after Spinal Cord Injury

Protein aggregation is associated with a number of diseases (Alzheimer’s, Parkinson’s, and amyloidosis), and is believed to be responsible for tissue damage after traumatic injury to the spinal cord or brain. It is hypothesized that removing such aggregates can alleviate symptoms and minimize tissue damage.

This invention describes a new application of a known drug, called CLR01. CLR01 is a “molecular tweezer” that can disaggregate amyloidogenic proteins, such as amyloid-β. According to new findings, CLR01 can be applied to the injury site of the spinal cord, and improve neuronal survival by inhibiting the post-injury aggregation of α-synuclein.

There are currently no therapeutics that specifically target toxic protein aggregates following spinal cord and traumatic brain injury.

The molecular tweezer has been tested in a lamprey model of spinal cord injury. Not only was significantly less synuclein aggregation observed, neuronal survival was also greatly increased.