Parkinson’s UK Award Targets New Therapies

Researchers at the Sheffield Institute for Translational Neuroscience (SITraN) have been awarded £1.2 million ($1.67 million) from Parkinson’s UK to develop new treatments that slow progression of the disease.

“This new funding will greatly accelerate the project and shows our commitment to working with ground-breaking academic researchers in the UK and taking their discoveries from the lab towards clinical trials,” Arthur Roach, PhD, director of research at Parkinson’s UK, said in a press release.

Parkinson’s is characterized by the loss of neurons that produce the neurotransmitter dopamine — a chemical messenger that is essential, among other functions, for muscle control. These brain cells are continuously active and rely on mitochondria (cells’ powerhouses) to work properly.

SITraN’s team of researchers, led by Heather Mortiboys, PhD, has been investigating the function of mitochondria in Parkinson’s disease.

The team now will work with drug discovery experts from Parkinson’s UK’s Virtual Biotech initiative to develop novel candidate molecules that can restore mitochondrial function in the brain and halt disease progression.

These novel candidate therapies, if successful in cellular models of the disease, then can move forward to studies in animal models and eventually clinical trials in humans.

“We’re progressing both a novel mitochondrial therapeutic target and novel molecules which act upon this target. The aim is to have a lead molecule which is active at the mitochondrial target, can restore mitochondrial function in dopaminergic neurons derived from people with Parkinson’s,” said Mortiboys, PhD, senior lecturer at SITraN.

Previous research by Mortiboys, funded by a Senior Fellowship grant from Parkinson’s UK, identified two molecules with these restorative properties.

“The team has identified not only molecules which can restore mitochondrial function in dopaminergic neurons from people with Parkinson’s, but also a novel mechanism by which they do this,” Mortiboys said.

Researchers modified these newly identified molecules to maximize their ability to restore mitochondrial activity without causing side effects. The team will continue to use brain cells from patients with Parkinson’s disease to test the molecules.

The research project will further develop these therapeutic candidates by analyzing how they work and with which proteins they interact inside brain cells.

“[I]t’s incredible to see how this project had its origin in a small academic grant from Parkinson’s UK to screen for molecules that rescue defective mitochondria inside neurons. Since then, the charity and University have continued to work closely together to improve those initial compounds,” said Roach.

“There is a desperate need for new and better treatments and we hope this project will one day deliver a life-changing drug for people living with the condition,” he added.