Dr. Michael Tymianski
Krembil Brain Institute researcher and Sprott Department of Surgery neurosurgeon, Dr. Michael Tymianski, has worked on developing nerinetide (NA-1) for two decades (Photo: The Globe and Mail)

A new study published in the journal The Lancet and presented at the International Stroke Conference in Los Angeles, shows strong promise for efficacy of a drug called nerinetide (NA-1), the first neuroprotective therapeutic agent in the world to demonstrate improvement in the functional and mortality outcomes of patients with acute stroke, which occurs when there is a blockage in an artery that supplies blood to the brain.

Nerinetide acts by enhancing the resilience of brain cells to withstand the lack of blood supply during a stroke. It was developed by Dr. Michael Tymianski, a neurosurgeon at the Sprott Department of Surgery and Senior Scientist with the Krembil Brain Institute at Toronto Western Hospital, University Health Network (UHN), and Professor, Departments of Surgery and Physiology, University of Toronto.

"This is a terrific example of the importance of investments in fundamental research and how they fuel the development of new therapies that can impact the patients we serve," says Dr. Brad Wouters, Executive Vice President, Science and Research at UHN. "Dr. Tymianski and his team are motivated by the unmet needs of our patients and they are creating change through discoveries in basic research."

"Therapeutics in stroke is an area that has been a wasteland for decades and I think today's announcement represents a step in the right direction," adds Dr. Donald Weaver, co-director of the Krembil Brain Institute. "It demonstrates that drugs that can meaningfully treat people with stroke are possible, and this research shows us the route to how to get there."

This work is the result of more than two decades of scientific research by Dr. Michael Tymianski and his research and development team, which first began at UHN in Toronto and continued under NoNO Inc., a biotechnology company founded by Dr. Tymianski.

There have been many milestones along the way, including the discovery of a protein called PSD95, which can impact the severity of stroke damage and the identification of a molecule (the first iteration of nerinetide), which is able to block the protein in preclinical models.

This study is the result of a Phase III clinical trial called ESCAPE-NA-1, involving more than 1100 stroke patients worldwide. Nerinetide was administered to patients selected for endovascular thrombectomy, a procedure in which a tube is inserted through an artery in a patient's groin, then, using imaging, is guided to the brain, where a retrievable stent removes the clot.

The study found that implementation of nerinetide, without prior administration of alteplase (also known as tPA), improved functional outcome, reduced mortality and decreased infarct volume (a measure of the extent of brain injury) among acute ischemic stroke patients.

The trial was led by Dr. Michael Hill, a neurology professor and director of the Calgary Stroke Program, and Dr. Mayank Goyal, a professor of radiology at the University of Calgary. It is the largest trial involving endovascular thrombectomy in history and the largest ongoing acute stroke trial globally.

"It takes many years and the talents of countless individuals to take an idea, a concept, and to translate that to a drug," says Dr. Weaver. "Dr. Tymianski has been working on this approach to stroke for two decades.

"I think that a lot of people would have given up. He did not. Kudos to him for having the persistence and the diligence to continue with this idea and kudos to UHN for fostering an environment where research like this is possible."

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