At the Ottawa Hospital Research Institute, Michael Rudnicki is helping to chart the future of regenerative medicine. Dr. Rudnicki works with stem cells, a key to treatments and cures for a vast range of serious diseases.
Dr. Rudnicki is head of the Institute’s regenerative medicine program, and directs its Sprott Centre for Stem Cell Research. Launched in 2006 after Bay Street money manager Eric Sprott and his wife Vizma donated $7-million to establish an endowment fund, this facility houses a range of staff, from researchers and clinicians to computer scientists and specialists in bioinformatics.
Scientists derive stem cells from adult, fetal and embryonic tissue. These so-called master cells can replicate themselves indefinitely. They develop into blood, brain and other specialized cells, and repair damage to the body.
In one facet of their research, Dr. Rudnicki and his team study muscle stem cells. They recently discovered that a protein called Wnt7a stimulates the growth and repair of muscle tissue – a finding that could lead to new treatments for afflictions such as muscular dystrophy.
Wnt7a is part of normal muscle growth and repair, but adding more of it accelerates the process. Young children’s bodies can repair severe trauma, Dr. Rudnicki notes. “What we’re doing is harnessing the stem cells that remain in our body to behave as if they’re in a young individual,” he says. “We want to stimulate the intrinsic repair processes that are already there, but have become quite dormant as we age.”
So far, Dr. Rudnicki has only tested the protein on mice, but he’s exploring its clinical potential with Fate Therapeutics Inc., a San Diego-based developer of stem cell medicines; Dr. Rudnicki is one of the company’s founding scientists.
“We’ve found that we can inject protein and have the muscle respond very robustly,” he explains.
Dr. Rudnicki is also Canada Research Chair in Molecular Genetics and scientific director of the Stem Cell Network, a Canadian not-for-profit organization that promotes excellence in stem cell research.
Already in clinical use through therapies such as bone-marrow transplants and cartilage repair, regenerative medicine has only started to realize its potential. Although scientists may eventually use stem cells and biomaterials to cure everything from heart disease to neurodegenerative illnesses, developing and commercializing these technologies will take time.
“What we all hope is that there will be new curative treatments for debilitating diseases,” says Peter Zandstra, a professor at the University of Toronto’s Institute of Biomaterials and Biomedical Engineering and a Canada Research Chair in Stem Cell Bioengineering. “It’s not clear right now if our approaches will be successful, but I think there’s a lot of hope and promise.”
Cell delivery is one of two main approaches to regenerative medicine, Dr. Rudnicki says. Surgeons do this in bone-marrow transplants. Then there are drugs based on the scientific knowledge of how stem cells function in adult tissues. “That kind of approach – stimulating the repair with a specific targeted bullet that only acts on that stem cell of interest – I think is the future,” Dr. Rudnicki says. “[It] has very exciting potential for ushering in the age of regenerative medicine.”
In the years ahead, Dr. Rudnicki says, protein biologics could perform tasks like regenerating cardiac tissue damaged by a heart attack. “A few weeks later, you’d come out of the hospital and you’d be walking, [instead of] having to go out in a wheelchair,” he explains.
Among other regenerative medicine projects, U of T’s Dr. Zandstra is utilizing new technologies, such as tissue engineering. “We generate cells such as cardiac cells [and] we’re interested in formulating these cells into micro-tissues that mimic adult heart tissue,” he says. “Then we can use those for drug screening.”
Dr. Zandstra, who works closely with Toronto’s McEwen Centre for Regenerative Medicine, stresses the importance of collaboration with fellow researchers here and abroad. He also wants to see innovative technology reach the marketplace. In addition to his research duties, Dr. Zandstra is chief scientific officer of the new Centre for the Commercialization of Regenerative Medicine (CCRM).
The Toronto-based centre takes early-stage technologies from Ontario institutions and adds value to help commercialize them, he explains. “One of the problems that we face in regenerative medicine – and there are many – is that numerous technologies that come out are too early for real commercialization, and we need this extra step of turning them into products.”
At the Stanford University School of Medicine, neurosurgery professor Gary Steinberg knows that commercializing therapies to regenerate the nervous system won’t happen quickly. In a clinical study a decade ago, Dr. Steinberg transplanted progenitor cells, which are similar to stem cells, into 14 patients with chronic stroke.
“These were patients who were partly paralyzed in their arms and hands,” he says. “Some of them did show improved recovery in terms of ability to do things with their hands, such as turning doorknobs and manipulating objects.”
Dr. Steinberg recently performed the first North American injection of stem cells into the brain of a stroke victim.
“We have great hope that over the next two decades, we’re going to witness some quantum leap changes in terms of recovering function and regenerating the nervous system, in ways we could never imagine even five years ago.”
