New drug molecule could help treat Parkinson's disease in young patients

More than 100,000 Canadians are currently living with Parkinson's disease. A novel drug molecule being studied by researchers at McGill University could reactivate housekeeping functions in the brain cells of young Parkinson's patients, paving the way for possible future treatments for this incurable, degenerative disease.

“We are excited about this drug because it offers the possibility of a cure for Parkinson's disease for a subgroup of patients,” said Kalle Gehring, professor of biochemistry at McGill University.

The new compound, developed by biotechnology company Biogen, has shown promising results in activating Parkin, a key protein in the brain responsible for “cleaning” and recycling damaged mitochondria. When Parkin doesn't work properly, these damaged mitochondria accumulate, eventually leading to Parkinson's disease.

In studies conducted in 2013 and 2018, Gehring was able to clarify the functions of Parkin using data collected at the Canadian Light Source (CLS) at the University of Saskatchewan (USask).

In this new follow-up study, Gehring used the CMCF beamline at CLS to determine how the Biogen compound activates Parkin. They found that it glues together Parkin and a natural activator present in the cell. This molecular-level information is critical for future drug development.

“The drug molecule activates Parkin through a secondary pathway that is effective in certain mutations of Parkin that occur in younger patients,” he said.

After turning proteins into tiny crystals, Gehring and his team used a technique called protein crystallography to identify their 3D structures and figure out where the new drug binds and how it affects the proteins. The results were published in the journal. Nature communication.

“We need high-quality data to decipher protein structures and see their 3D images. Only with a facility like the CLS can Canadian research be raised to an international level,” said Gehring.

While the symptoms of Parkinson's disease – slowed movements, tremors and balance problems – often only appear in people over 60 years of age, an earlier onset of the disease is not uncommon: five to ten percent of people diagnosed are affected before they reach the age of 40.

“We hope that one day we will find drugs that can be used to treat Parkinson's disease in general,” said Gehring. “As Canada's population ages and better treatments for other diseases become available, neurodegenerative diseases like Parkinson's will become a major health problem.”

While Gehring will continue to investigate the pathways of Parkin activation as part of his ongoing research to understand the causes of Parkinson's disease, it will be up to Biogen to apply the results of this new study to future drug development, he said.