Potential new target in the fight against early onset Parkinson’s
13 February 2017 - An international team led by Patrik Verstreken has identified one of the mechanisms underlying early onset Parkinson’s disease. By blocking a specific protein, the researchers were able to change the composition of the mitochondria or 'energy factories' of the nerve cells. This reduced the symptoms of Parkinson's disease.
An estimated 10 million people worldwide are currently suffering from Parkinson’s disease. A small percentage of them is confronted with the disease before the age of 40. While the causes of the disorder are not yet known, scientists believe a combination of genetic and environmental factors is to blame. In genetic Parkinson’s disease, a mutation in the PINK1 gene causes changes in the mitochondria of the nerve cells. As a result, these nerve cells start degenerating.
Professor Verstreken (KU Leuven - VIB) and his international team, including researchers from Belgium, Germany, and Portugal, observed that a protein responsible for lipid creation in cells, FASN, bypasses the genetic defect in mitochondria. “Several drugs that block FASN already exist," says Professor Verstreken. "After all, this protein is also important in cancer research and treatments. Many of these drugs have already been used in clinical trials. Thanks to this research we can now test the drugs in the context of Parkinson’s disease.”
The researchers used cells from fruit flies and mice as well as patient-derived cells. They found that FASN has a significant impact on the mitochondria.
"The genetic defect reduces the amount of lipids in the mitochondria," says Professor Verstreken. "We were surprised to find that blocking FASN actually sidesteps the mitochondrial effects of the PINK1 mutation. Blocking FASN increases the level of these lipids in the mitochondria, reducing the degradation of nerve cells.”
This study is an important new step, but further research is necessary. “Before we can develop new therapies, we will have to get a clearer picture of the link between these lipids and early onset Parkinson's disease," Professor Verstreken concludes. "Furthermore, we have already shown the positive impact of these lipids on the mitochondria in flies, mouse models, and in human cells, but we still need to explore its effects in actual patients.”