Brock-industry partnership focused on treating Duchenne muscular dystrophy

The Brock-Niagara Validation, Prototyping and Manufacturing Institute (VPMI) connects Brock University’s advanced scientific and applied research expertise, state-of-the-art equipment assets, and testing and training capabilities with industry in the bioproducts, bioscience, bioagriculture and chemical manufacturing sectors. Examples of these successful VPMI-industry partnerships are highlighted in this series. For more information, contact:

It’s a genetic condition that takes people young. Duchenne muscular dystrophy (DMD), characterized by progressive muscle wasting and weakness, is a disease Val Fajardo is determined to treat.

“Without a cure, affected individuals will live a shortened lifespan,” says the Brock Assistant Professor of Kinesiology. “In addition to muscle weakness, many patients will experience cognitive impairment, which will further dampen their quality of life.”

Fajardo, who is Canada Research Chair in Tissue Remodelling and Plasticity throughout the Lifespan, studies an enzyme called GSK3.

While GSK3 is involved in a cell’s metabolism, immunity and differentiation, it can also lead to muscle wasting and declines in heart and brain function if overactivated.

Fajardo’s ongoing research on how and why this happens, and how to suppress GSK3, led him to contact AMO Pharma Inc., a biopharmaceutical company developing drugs to treat neuromuscular and central nervous system (CNS) symptoms of rare diseases.

The U.K.-based company produces a drug called tideglusib, which has been proven to inhibit GSK3.

“I connected with AMO Pharma a few years ago because of our shared interest in tideglusib being a GSK3 inhibitor, and I wanted to test its efficacy in treating the muscle damage and weakness that occurs with Duchenne muscular dystrophy,” Fajardo recalls.

Although their discussions were positive and exciting, the timing for a research partnership wasn’t right, he says.

His research team stuck with their GSK3 investigations not only within the context of muscular dystrophy but also for other conditions such as obesity and spaceflight.

The American space agency NASA awarded the team two separate batches of muscle samples – one in 2020 and another last year – from subjects originally aboard the International Space Station so that the researchers could see what happens to GSK3 and other muscle signalling pathways during spaceflight.

Also last year, the Canadian Space Agency granted Fajardo and his team $150,000 to carry out similar research investigating whether stopping the enzyme GSK3 can prevent space travellers from experiencing muscle loss and weakness, bone fragility and cognitive decline, which occur when spending time in space.

“If you can figure out ways to stop or slow down muscle loss in space, why not apply that here on Earth for aging or other diseases?” says Fajardo.

Duchenne muscular dystrophy was at the top of his mind.

The team got the break they needed when Fajardo was named Canada Research Chair in 2020.

“With the support of this award, we were able to collaborate with other talented researchers at Brock University on stopping GSK3 for Duchenne muscular dystrophy,” he says. “We have accumulated promising data with the drug tideglusib.”

When the Brock-Niagara Validation, Prototyping and Manufacturing Institute (VPMI) put out a call for university-industry research partnerships, Fajardo remembered his animated discussions with AMO Pharma and reached out once more — finding it was now a fitting time to connect.

From the days of their initial discussions, “Dr. Fajardo has progressed his studies in Duchenne muscular dystrophy, which were compelling in the first instance and have attracted worldwide interest when presented recently in their most current iteration,” says AMO Pharma Chief Science Officer Mike Snape.

“In discussion it became clear to us that Dr. Fajardo has a much better understanding of DMD than any other researchers we have spoken with in the past,” he says.

In the VPMI-AMO Pharma partnership, Fajardo and his team will test the effectiveness of the company’s drug, tideglusib, in a more severe model of Duchenne muscular dystrophy. The researchers will focus their attention on cardiac and skeletal muscle health and function.

“The development of a new medicine that was universally applicable to all DMD patients and able to treat all organ systems would be a game changer,” says Snape.

Fajardo has also joined forces with Associate Professor of Health Sciences Rebecca MacPherson to co-supervise PhD student Emily Copeland, who will determine whether tideglusib treatment and GSK3 inhibition in the brain can reduce the cognitive impairments that are often found in patients living with Duchenne muscular dystrophy.

“This work has implications for research into tissue degeneration in many other muscle-wasting conditions and potentially aging more generally, so we anticipate the potential to build a larger collaboration,” says Snape.

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