For most of Fred Van Goor's bachelor of science degree, he never wavered from his mission of following in his father's footsteps and attending medical school at the University of Alberta.
"I wanted to be a medical doctor. And that's what brought me to the University of Alberta in the first place," remembers Van Goor.
However, a summer job at the tail end of that degree in former U of A professor John Chang's "hardcore" zoology lab — complete with crowded shelves of specimen jars — studying growth and reproduction in goldfish, took the budding scientist's medical dreams in a different direction.
"I'm quite proud of that job because that gave me my love of really trying to understand how cells that nobody else knows about work, answering questions that are unknown, and using science to be able to do that," he says.
"I credit the University of Alberta for really showing me what I really wanted to do in life, which was to do research."
That fateful decision decades ago would eventually lead to the development of a therapy that has enhanced and will ultimately save the lives of thousands of people with cystic fibrosis, a life-limiting inherited disease caused by a gene mutation that leads to untold damage to the lungs, pancreas and other organs. It also led to Van Goor being named a recipient of the 2024 Breakthrough Prize in Life Sciences.
Originally from Calgary, Van Goor started at Vertex Pharmaceuticals in 2001, just five years after earning his PhD in biological sciences at the U of A and on the heels of a postdoctoral stint with the National Institutes of Health in Bethesda, Maryland.
"What was really neat for me is I took my love of science and the love of figuring out the unknown, and turned that into using science to transform the lives of people with a serious unmet medical disease. And I just think that's the coolest job in the world."
Now the vice-president and head of cystic fibrosis research for the biotech pharmaceutical company based in San Diego, Van Goor has spent more than two decades developing life-transforming drug combinations that repair the multiple defects in the protein found in people with cystic fibrosis. When this protein is not working correctly, chloride — a component of salt — becomes trapped in cells, such as those found on the surface of the lungs. The trapped chloride prevents proper hydration of cellular surfaces, which leads to the mucus buildup and the resulting damage to the lungs found in those with cystic fibrosis.
The genius of the current standard of care, Vertex's Trikafta, is that it combines three drugs that work through different but complementary mechanisms of action to combat the multiple defects in the protein caused by mutations in the gene that causes cystic fibrosis.
Under the previous best standard of care, someone living with cystic fibrosis had a life expectancy of 38 years. Today, the same person, given they start early enough with Trikafta, is projected to live into their 70s.
The benefits also include a decrease in lung transplantation and a decrease in pulmonary exacerbations, which can result in permanent loss of lung function — all leading to a reduction in hospitalizations.
"The fact that it all started in a zoology lab under Dr. Chang and later as a PhD student with (biological sciences professor) Jeff Goldberg is probably not your traditional path to where I am today."
As for what's next, Van Goor is leading Phase 3 clinical trials testing another drug combination that will be an upgrade on Trikafta. Vertex is also collaborating with Moderna on a new therapy for the roughly 5,000 of the estimated 88,000 people worldwide with cystic fibrosis who don't respond to current therapies.
"Since winning the award, people have been asking me, 'Where did it all start? Where did you come from?'" he said. "I've really been thinking about that a lot lately, and that experience at the University of Alberta and being from Canada is pretty neat. I am thankful."
At $3 million for each prize category, the Breakthrough Prizes are the richest awards in recognition of scientific advances. The annual prizes are given in mathematics, fundamental physics and life sciences, and are sponsored by a host of tech entrepreneurs including Mark Zuckerberg and Priscilla Chan of Facebook and Sergey Brin, co-founder of Google.
Van Goor was joined by fellow U of A graduate Michel Sadelain, who was also awarded a 2024 Breakthrough Prize in Life Sciences in recognition of his discovery of cancer-fighting immunotherapy based on the genetic engineering of a patient's own T cells.
—with files from Caitlin Crawshaw
