Ottawa scientists have transformed one of the body’s natural proteins into what could become the basis for a powerful drug—one with the potential to treat a range of muscle-wasting diseases, according to Dr. Michael Rudnicki of the Ottawa Hospital Research Institute, who led the research. It’s the latest milestone in the booming field of regenerative medicine, which aims to use the body’s own building blocks to fix what ails us. “A new wave of therapeutics will be entering the clinic,” Rudnicki says, “drugs and proteins that target stem cells, and stimulate repair.” It’s happening already.
Rudnicki’s team targeted a protein called Wnt7a, part of a family of proteins that stimulates stem cells and promotes tissue regeneration. “This class of protein is quite difficult to work with,” says Rudnicki, who’s also a professor of medicine at the University of Ottawa, and has been tackling this for several years. “It’s difficult to manufacture,” a necessary step before it can be used to treat patients, “and difficult to disperse through the body.” Rudnicki and colleagues discovered that one small portion of this protein—just 130 amino acids long—packs just as much regenerative power as the entire thing, while doing away with longstanding challenges. “This raises the possibility it can be used in the clinic.”
Those suffering from Duchenne muscular dystrophy, an inherited disorder that affects about 1 in 3,500 boys, could be among the first to benefit. “We think that by stimulating repair in its early stages, we can delay the downward spiral where muscle tissue is lost,” he says. Such a drug could provide a better quality of life for patients. “It won’t cure the disease,” he says, “but conceivably, it could make it chronic, rather than fatal.” Other muscle-wasting diseases could be targeted, too, from other genetic conditions to the muscle loss that occurs in some forms of cancer, maybe even age-related loss of muscle mass and strength, although that has yet to be tested in mice (let alone humans).
Clinical trials in humans should begin within a few years, led by Fate Therapeutics, a public company where Rudnicki is co-founding scientist. The field of regenerative medicine is “approaching a tilting point,” he says. “It will transform the practice of medicine, within our lifetimes.”