New molecules targeting alpha-synuclein have potential for slowing Parkinson’s
Researchers in Japan have developed molecules that could be key to slowing the progression of Parkinson’s.
The results, published in Scientific Reports, showed the molecules reduced the amount of the protein alpha-synuclein made in nerve cells and reduced the symptoms of Parkinson’s in a mouse model of the condition.
A problem protein
The protein alpha-synuclein is the main component of Lewy bodies that form in cells affected by Parkinson's.
The normal function of this troublesome protein isn't fully understood, but we know it has one or more toxic forms that are related to Parkinson’s. Recent research also suggests that the protein is responsible for the spread of Parkinson's from cell to cell. As such, there’s a lot of interest in targeting alpha-synuclein to slow or stop the progression of Parkinson’s.
But while most therapies to date have concentrated on targeting the protein once it's already been made. In this study, the researchers have developed molecules that can reduce the amount of alpha-synuclein protein being made. They suggest it could be used alongside other therapies targeting alpha-synuclein.
A fresh approach
The type of molecule being developed, called antisense oligonucleotides (ASO), have already been approved for treating neuromuscular diseases, including spinal muscular atrophy (SMA) and Duchenne muscular dystrophy.
By developing ASO molecules that bind to alpha-synuclein RNA (the intermediate step between DNA and protein) the researchers were able to target the RNA for destruction, reducing the amount of alpha-synuclein protein made.
In the mouse model of Parkinson's, one of the molecules in particular, ASOA19, was seen to reduce some of the symptoms associated with Parkinson's.
Dr Beckie Port, Research Manager at Parkinson’s UK, said:
"Targeting the alpha-synuclein protein holds promise for one-day slowing or stopping the progression of Parkinson's, something no current treatment can do.
"These results look very promising. However, it is early days for Parkinson's - more research is needed to understand the long term safety and effectiveness of targeting alpha-synuclein in this way, and there are also challenges to overcome to ensure the ASO molecules would reach the affected brain cells."