Tackling toxic alpha-synuclein

Three major lines of clinical investigation are currently underway to determine if stopping the clumping together of the protein alpha-synuclein in Parkinson’s can help to slow down the progression of the disease. Cure Parkinson’s is proud to be supporting two of these efforts, and very much looks forward to future developments in this important target area.

Alpha-synuclein is one of the most abundant proteins found inside neurons in the brain. It is believed to have many different biological functions, particularly in terms of signalling from one neuron to the next.

In people with Parkinson’s, alpha-synuclein clumps together, forming dense clusters called Lewy bodies. These structures are believed to be toxic, and may in turn be involved in the death of neurons. For this reason researchers are pursuing this important target area, to identify existing drugs and new compounds that can help clear the toxic clumps of alpha-synuclein from affected cells. These efforts have led to a number of clinical trials which are testing them.

One example of these clinical trials is the ADepT-PD study, a Cure Parkinson’s supported trial, assessing a drug called nortriptyline. Nortriptyline is an anti-depressant drug that is being re-purposed to assess its potential to treat Parkinson’s. In pre-clinical studies nortriptyline has shown to reduce alpha-synuclein clumping in models of Parkinson’s.