New answers for future Parkinson’s therapy
13 June 2023
New molecules could hold promising future for the treatment of Parkinson’s disease by preventing the build-up of toxins in the brain, finds new research from Cardiff University.
The dysfunction of mitochondria in brain cells can lead to the accumulation of toxins in the brain, leading to the death of brain cells and the development of Parkinson’s disease, a form of dementia that affects 1 in 37 people in the UK.
New research from Cardiff University’s School of Pharmacy and Pharmaceutical Sciences has designed first-of-its-kind molecules that have the potential of becoming a new therapy for Parkinson’s disease in the future, by potentially slowing the progress of the disease by targeting the function of mitochondria.
“The build-up of toxins in brain, leading to the death of the brain cells, is among the main contributing mechanisms to the development of Parkinson’s disease. This happens when the cell’s mitochondria malfunction. To help treat Parkinson’s disease, we need to find a treatment that targets the malfunctioning mitochondria.
“In Parkinson’s disease, we see the dysfunction of an enzyme called PINK1 which leads to mitochondria in brain cells not performing how they should.
“We aimed to design and create chemical compounds that target the PINK1 enzyme, which triggers the repair or recycling of the damaged mitochondria, as well as preventing the damaging toxin accumulation in brain cells,” said Dr Youcef Mehellou, Cardiff University’s School of Pharmacy and Pharmaceutical Sciences.
“Our research identified a new class of molecules that have the potential to treat Parkinson’s disease. Critically, our research shows that it is possible to have molecules that clear damaged mitochondria and also prevent the accumulation of toxins in the brain of Parkinson’s disease patients. This is an exciting discovery, as to date, there have been no reports of molecules that can do both.
“This research marks a step forward in finding targeted treatments for this form of dementia. We have more research to do before this becomes accessible to patients, but our research brings us a closer to a new targeted therapy for Parkinson’s disease,” added Dr Rhys Thomas, Cardiff University’s School of Pharmacy and Pharmaceutical Sciences.
The research, PINK1-Dependent Mitophagy Inhibits Elevated Ubiquitin Phosphorylation Caused by Mitochondrial Damage, was published in the Journal of Medicinal Chemistry on May 29th, 2023.