Shaping Tomorrow's Treatments

Clinical Trials Have Changed Medicine

Examples of Canadian Contributions

Canadian researchers have made significant contributions to the practice of medicine through findings proven in clinical trials. Below are some examples that you might know of and be familiar with, but did you know that they are home-grown?



A drug called 3TC has been responsible for helping many people live with AIDS and for significantly decreasing the transmission rates of HIV/AIDS from mothers to their children. Children may acquire HIV/AIDS from their mother during pregnancy, labour, or breast-feeding, however 3TC greatly lowers that risk by affecting the amount of virus in the mother. This drug was developed by BioChem Pharma in Montreal and has impacted millions of children’s lives. (Reference 1)


Colorectal Cancer

Canadian clinical trials have been key to helping establish the characteristics of patients with colorectal cancer that will benefit significantly from treatment with cetuximab (also called Erbitux). It has been found that only patients who carry a molecular marker called wild-type KRAS respond best to cetuximab through studies on their survival, quality of life, and economics. Because of these studies, oncologists know that patients with wild-type KRAS will respond to this treatment, and these patients have thus been treated with personalized therapy for their colorectal cancer. (References 2, 3, 4)


Congenital hypothyroidism

Drs. Jean Dussault and Paul Walfish developed a neonatal screen for children with congenital hypothyroidism. Congenital hypothyroidism is when a person has very low metabolism because they cannot make enough thyroid hormone – a condition that results in mental retardation. The ability to screen for this condition has allowed babies with congenital hypothyroidism to be identified and treated immediately, sparing thousands of children from mental retardation. (Reference 5)



Before insulin was discovered, Type 1 Diabetes led to premature death. Important work about diabetes itself, the isolation and purification of insulin from the pancreas, and clinical research using insulin to treat Type 1 Diabetes were all undertaken at the University of Toronto. This work was contributed to by Drs. Frederick Banting, Charles Best, John MacLeod and J.B. Collip, and is now credited with allowing people with Type 1 Diabetes to live successfully with this chronic illness. (Reference 6)


Further research and innovation in Type 1 Diabetes has also lead to the development of the Edmonton Protocol. The Edmonton Protocol is the transplantation of Islet cells to a person with Type 1 Diabetes. The Islet cells are the part of a person’s pancreas that makes insulin. People with Type 1 Diabetes do not make insulin. While others have experimented with Islet transplantation, the Edmonton Protocol is a much more robust operation and has been done with much greater success. (Reference 7)


Heart Attack

Dr. Salim Yusuf and his Canadian colleagues lead a 52-country wide study in nearly 30,000 participants called INTERHEART. Their findings showed that irrespective of where people live, their racial/ethnic background, and their sex, they have 9 major risk factors for heart attack. These factors are highly modifiable (meaning they can be controlled or affected by a person) and are: smoking, levels of fat in the blood, high blood pressure, diabetes, obesity, psychosocial factors, daily fruit and vegetable intake, alcohol use, and regular physical activity. Knowing these factors allows for better prevention to be undertaken. (Reference 8)



Dr. Henry Barnett of the Robarts Research Institute led the NASCET (North America Symptomatic Carotid Endarterectomy Trial) that had over 50 sites in Canada and the United States with close to 1,500 participants. NASCET’s findings are critical to stroke prevention and defined the use of Aspirin to prevent heart attacks and stroke. (Reference 9)




2. Karapetis CS, et al., N Engl J Med, 359:1757-65, 2008

3. Mittmann N, et al., J Natl Cancer Inst, 101:1182-92, 2009

4. Jonker DJ, et al., Br J Cancer, 110: 648–65, 2014

5. Dussault, JH, et al., J Pediatr, 86 (5); 670-74, 1975


7. Shapiro, AM James, et al., N Engl J Med, 343:230-38, 2000

8. Yusuf, S, et al., Lancet, 364 (9438): 937-52, 2004

9. Barnett, JM, et al., N Engl J Med, 325:445-53, 1991


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