Parkinson’s disease cutting-edge research by Scherzer

Clemens Scherzer: What keeps me going in the morning and the day and night is that we are looking for a new medication that can halt Parkinson’s disease.

Clemens Scherzer is a neurologist at Harvard Medical School. In fall, 2010, he published a study on Parkinson’s disease, an illness that disrupts movement and slowly destroys the brain. Today, the disease has no cure. But Scherzer said his team had made a potential breakthrough in understanding Parkinson’s, one that could help with treatment.

Clemens Scherzer: We found the genes that the mitochondria need to function are working sluggishly in patients with Parkinson’s.

Mitochondria are in every human cell. They’re like tiny power plants. They convert sugar into a substance our cells use as fuel. Scherzer discovered that, in patients with Parkinson’s, the genes that control these mini power plants are working too slowly. He told us there’s a way to speed up these sluggish genes.

Clemens Scherzer: Our study identifies PGC1alpha as a new pathway in treatment in Parkinson’s disease, particularly in the early stages.

He said PGC1alpha is a “master gene” that controls the activity of the genes in charge of mitochondria – the power plants inside cells. Scherzer said a medication that targets PGC1alpha and revs up mitochondria could potentially slow down Parkinson’s disease. Scherzer suggested such a medication could be available within five to ten years.

Clemens Scherzer: It’s actually an excellent target for new medicines because drug companies have been working on this target for many years for other diseases, particularly for diabetes. There are many compound of chemicals out there and already medications out there that work on this energy pathway. It gives us a jump start. So the medications that work on the PGC1alpha pathway work everywhere in the body. We have to refine this medicine so they get to a greater degree into the brain, so they can do their job primarily in the brain.

Scherzer also added that his research on genes linked to Parkinson’s – he found ten sets of genes that were linked – might help experts develop a blood test for Parkinson’s.

Clemens Scherzer: We didn’t look at one gene at a time, we looked at gene sets, and a gene set is really a group or a pathway that encodes a certain process.

He said one of the issues with Parkinson’s is that, by the time it’s detected through a clinical diagnosis – that is, patient history, observation – it’s too late for a good medical intervention:

Clemens Scherzer: Currently the diagnosis and monitoring a disease is entirely based on a doctor’s exam, a neurologist like myself who examines the patient. That does not work very well because once a patient has tremors, and slow movement and a diagnosis of Parkinson’s disease, 70% of the dopamine neurons are already dead. So what we need to cure this disease are tests that allow us to catch patients at the very early disease stages.

Scherzer talked more about finding a blood test that might catch genetic markers for Parkinson’s early.

Clemens Scherzer: We are looking into this. So far, in this study we chiefly focused on the brain, we looked at 300 brain samples, we also looked at 88 blood samples. The most dramatic changes were in the brain samples. So we don’t know whether this can be developed into a blood test or not. That’s something we’re working on, and it’s something that’s very important.

He explained why Parkinson’s has been so difficult to cure.

Clemens Scherzer: It’s a prototype of a common complex disease. And what do these fancy words mean? It means it’s a disease caused by multiple factors. There is a little bit of genetic susceptibility, there is a large portion of exposure to bad environment, pesticides, head trauma, welding, professional exposures. And then there is a significant contribution of the aging process. Therefore it’s so difficult to tease out the combination of these. I strongly believe it’s not one factor.

Dr. Scherzer and his international team published their research in October 2010 in the journal Science Translation Medicine.