It’s long been understood that the motor symptoms of Parkinson’s disease (PD) stem from a loss of dopamine-producing cells in the brain.
But the past few years have added weight to an emerging hypothesis in the field: that the central nervous system (CNS) disorder may in fact have its origins in a place that is far from the brain, both in distance and prestige — the intestine.
With Michael J. Fox Foundation (MJFF) support, Berwyn, Pennsylvania pharmaceutical company QR Pharma is running with this idea, and building on their own work suggesting a gastrointestinal connection to Parkinson's. They hope to develop their compound Posiphen, and its ability to target and break up clumps of alpha-synuclein in nerve cells in both the brain and intestine.
So far, the lion’s share of studies in alpha-synuclein research has targeted only clumps in the brain. But going after clumps in the intestine is particularly interesting because, if it’s true that they occur in the gut first (still a relatively big if), and if researchers can find a way to break up these clumps before they reach the brain, it may become possible to treat Parkinson's before major neurological damage occurs.
More reason to be intrigued about Posiphen: The drug has already proven to be safe in early clinical trials for Alzheimer’s disease (AD). Since it has already navigated the rigors of early clinical testing, if QR Pharma can build the case that their drug is effective in PD pre-clinical models (which is what they’re doing with MJFF funding) they may be able to speed up the often long and arduous journey to bringing their drug to pharmacy shelves.