A new study published in the journal Nature Communications reveals a new drug designed to break down the accumulation of toxic proteins associated with Parkinson’s disease, effectively slowing disease in mouse models, and researchers are now looking to push the new technology into human trials. Molecular tweezers are new compounds with the ability to unwrever abnormal protein lumps.
The main symptom of Parkinson’s disease: neurodegeneration is thought to be caused by a protein aggregation called alpha-synuclein. These toxic protein blocks eventually kill the dopamine-producing neurons, leading to the disease’s common motor degradation.
CLR01 is a molecular tweezer previously discovered that prevents alpha-synuclein from gathering in the first place and potentially disassembles the formed clumps. The newly published study builds on a growing body of evidence from different animals that CLR01 is safe and effective in slowing and, in some cases, reversing the pathological symptoms of Parkinson’s disease.
Perhaps the most important finding in this new study is that the observation of CLR01 may only be effective if applied at a specific point in the progress of the disease. The researchers treated mice genetically modified to simulate Parkinson’s neurodegeneration, but CLR01 was only partially valid when the animals were given at 6 months of age and only partially when given at 18 months of age. The study points out that the most effective treatment window is when the animal is 12 months old. This suggests that CLR01 may only be effective if applied as a preventive therapy in the early stages of the disease.
“This is a very exciting work that shows that drug treatments can be developed to unrap toxic protein clusters to save neurons in the Parkinson’s model,” said Richard Wade-Martins, lead author of the study. “Our focus is on developing new ways to save neurons before they start to lose function early.”
Beckie Port, from Parkinson’s in the UK, said the new molecular tweezers were exciting and hoped it would be tested in humans as soon as possible.
“We urgently need treatments that protect brain cells from Parkinson’s disease, and these results suggest that this innovative ‘molecular tweezer’ approach has exciting potential in the laboratory. We now need to move this treatment to clinical trials so that we know if it will play the same role in people with Parkinson’s disease. “
The new study was published in the journal Nature Communications.