In a time of despair for U.S. scientists, Gairdner Award winners shine brighter than usual
Since they were established in 1957, the Canada Gairdner International Awards have celebrated achievements in biomedical research, from the discovery of stem cells to the development of mRNA vaccines.Founded by James A. Gairdner, a Toronto financier and philanthropist, with added supported from the Canadian government starting in 2008, the awards, which are now valued at $250,000 each, are among the most prestigious that can be won by any scientist in the world in the fields of medicine and human biology.Each year, five names are added to the growing roster of laureates. A separate $100,000 prize for those who have improved global health and two for mid-career researchers who have done exceptional work within Canada completes the slate of eight recipients.The latest set of five Canada Gairdner International Award winners, announced on Friday, are all based in the United States.The sweep is a testament to the resources that the U.S. invests in biomedical research, accounting for nearly half of all the money spent in the field worldwide. Much of this is underwritten by the U.S. National Institutes of Health, including more than 100 projects currently led by Canadian investigators.It is no small irony that this year’s American Gairdner winners are being honoured just as medical researchers across the United States are in a state of collective despair.Funding cuts to the NIH and research universities ordered by the Trump administration have thrown the medical research world into turmoil by sidelining studies, upending clinical trials and threatening to pull the plug on an entire generation of scientists in training.“A catastrophe,” is how one of this year’s American Gairdner winners bluntly described the situation.Janet Rossant, the Gairdner Foundation’s president and scientific director, said that at a time when trust and support in science and scientists are under threat, the awards not only celebrate excellence in research but also its importance to the health and well-being of people everywhere.“We stand up for diverse voices at the table,” Dr. Rossant said. “We stand up to support the next generation of Canadian scientists who are going to make Canada a leader and trusted partner in research and innovation worldwide.”The duo that cracked the code on cystic fibrosis drugsMichael Welsh and Paul Negulescu, Canada Gairdner International AwardOpen this photo in gallery:Open this photo in gallery:Michael Welsh can still summon the multi-sensory memory of his first cystic fibrosis patient.He can hear her loud, violent coughs. He can see her neck muscles straining with every laboured breath. And he can smell the fruity odour of Pseudomonas aeruginosa, a bacterium that commonly infects the lungs of CF sufferers. She was only 7 or 8, but – as with so many CF patients at the time – she was unlikely to survive her teen years.“As a physician, there are certain patients that are burned into your memory,” says Dr. Welsh, a professor of internal medicine with the University of Iowa. “My inability to do something that would truly impact the underlying problem was a terrible feeling.”This was 1973, when Dr. Welsh was still a medical student in Iowa and physicians were powerless to stop the progression of cystic fibrosis, a rare and lethal inherited disease. But today, if Dr. Welsh were to encounter that same patient – and determined her to have the most common type of CF – he could prescribe a drug that not only treats the root cause of her disease but unfurls the possibility of an entire future: High school. Adulthood. Parenthood. Maybe even old age.These drugs, known as CFTR modulators, have transformed cystic fibrosis from being a life-shortening diagnosis to a manageable chronic disease for patients with access to these therapies. And they exist because of groundbreaking research conducted by Dr. Welsh, which paved the way for pioneering drug development led by Paul Negulescu, a senior vice-president with Boston’s Vertex Pharmaceuticals.Cystic fibrosis, which today affects an estimated 160,000 people worldwide, occurs when there are mutations in a gene that encodes a protein called CFTR. This protein helps move chloride – a component of salt – across our cell membranes, a process that maintains the fluidity of mucus in our lungs and other organs. When CFTR malfunctions, it results in the tar-like mucus that builds up in CF patients’ airways, increasing their risk of infections, causing progressively worsening lung damage and eventually leading to an early death.Following the CFTR gene’s discovery in 1989, Dr. Welsh and his team set out to unlock its mysteries. What does CFTR do? How does it work? How do mutations corrupt its function? And, critically, can a faulty CFTR protein be fixed? Dr. Welsh’s studies suggested that the answer to that question was yes, thus providing a road map for developing targeted CF therapies.In the late 1990s, the Cystic Fibrosis Foundation was searching for a company willing to tackle this difficult challenge and heard about a San Diego startup called Aurora Biosciences, co-founded by Roger Tsien, Dr. Negulescu’s undergraduate physiology professor.Aurora’s expertise was screening molecules at high speeds in search of potential drugs. “We knew the protein could be fixed,” said Dr. Negulescu, who was Aurora’s fifth hire in 1996. “And even though the odds were long, we were willing to try.”In 2001, Aurora was acquired by Vertex, which has screened millions of molecules to develop five CFTR modulators in the span of two decades. Trikafta, a triple-combination drug that got FDA approval in 2019, has been the biggest breakthrough, effective in 90 per cent of CF patients who have the most common gene mutation. Trikafta is now taken by more than 68,000 patients worldwide, representing more than two-thirds of eligible patients, according to a Vertex spokesperson. More work remains, including developing drugs for patients with the rarest mutations and making CFTR modulators accessible to all. (Its list price is more than $300,000, making it one of the world’s more expensive drugs).Today, the drug’s impact can be seen at the University of Iowa hospital where Dr. Welsh once treated patients. Its CF centre once had maybe a dozen CF patients at any given time, he said; “Now we have maybe one, sometimes none.”– Jennifer YangFrom a fruit fly’s wings, they learned how bodies are builtSpyros Artavanis-Tsakonas, Iva Greenwald and Gary Struhl, Canada Gairdner International AwardOpen this photo in gallery:Open this photo in gallery:Open this photo in gallery:The wingtips of a fruit fly are as round as a butter knife. But when a fruit fly carries a certain mutation, its wings form with irregular notches.That mutation, linked to a gene on the fly’s X-chromosome, was first noticed by geneticists more than a century ago. What no one realized then was this was a clue to one of the key mysteries of life: How do animals, including humans, develop from a single fertilized egg?The answer involves a molecular messaging system called Notch signalling, after the fly wings, which cells use to co-ordinate the building of a body.“If you have one cell that decides to become something and, as a consequence of that, the cell next door decides to become something else, these cells have to be talking to each other,” said Spyros Artavanis-Tsakonas, a professor emeritus of cell biology at Harvard Medical School in Boston, whose work was key to discovering how the system works.Born in Greece, Dr. Artavanis-Tsakonas studied chemistry as an undergraduate. But when the opportunity arose in 1972 to do a PhD in molecular biology at Cambridge University in England, he was hooked.Cambridge was then at the focal point of a scientific renaissance as biologists deployed new molecular tools to understand cell development and function. Surrounded by past and future Nobel Prize winners, Dr. Artavanis-Tsakonas said the experience was life-changing. “Even if you were a complete idiot, by osmosis you’d get something,” he said.His training provided the impetus to take on the Notch gene during post-doctoral stints in Europe and the United States. By the 1980s when he was an assistant professor at Yale University, he and his colleagues had successfully cloned and then sequenced the gene, setting the stage for its further investigation.A key insight from this work was that the gene carried instructions for making a protein that resides on the cell membrane. This was a sign that the protein was involved in exchanging information with other cells, a possibility that had also been discovered by Iva Greenwald.A native of Brooklyn, Dr. Greenwald earned her PhD at MIT where she, too, became interested in how genes orchestrate development. But rather than working with fruit flies she was drawn to a simpler organism – the roundworm – as a basis for studying cellular processes.This led her to a gene called lin-12 that performs the same function in roundworms as Notch does in fruit flies. While doing post-doctoral work at Cambridge she further discovered that some of its components were similar to those found in human cells. Here was an exciting hint that something more universal was at work.“Just knowing if I kept working I might discover something else new and unexpected was thrilling,” Dr. Greenwald said.Through the 1980s, Dr. Greenwald continued working on the idea that the lin-12 protein operated like a switch that could steer a cell’s fate based on the signal it received from a neighbouring cell. In this way, cells destined for different functions would know which path to take.By 1991, she was at Princeton University and had teamed up with Gary Struhl, a developmental biologist and fellow New Yorker whose academic journey had similarly included time at MIT and Cambridge before he became a professor at Columbia University.He was also her husband – Dr. Greenwald and Dr. Struhl had married only months before, but now they discovered they had a shared professional interest in establishing how the Notch/lin-12 system worked.Aided by methods that Dr. Struhl had previously developed to study other genetic pathways in fruit flies, their collaboration led to the discovery of an elegant molecular pathway that would prove to be common to animal cells.The pathway begins with the receptor that resides on the cell’s membrane. During communication, it can be latched onto and pulled by a counterpart structure on a neighbouring cell. This allows the receptor to be cut, triggering the release of an interior component that makes its way to the cell’s nucleus. There, it interacts with the cell’s DNA to promote particular genes, such as those that can determine the cell’s destiny.Variants in the human version of Notch genes have been linked to forms of cancer and neurodegenerative disease. Researchers have also imitated the mechanism to create a synthetic version of Notch signalling for new therapies and for engineering new tissues.Dr. Struhl said the applications opened up by the discovery underscore the values inherent in basic research, particularly involving organisms such as roundworms and fruit flies, which allow ideas about gene function to be tested.“For me, the award represents the recognition and justification of these values,” he said.– Ivan SemeniukFighting malnutrition and saving millions of lives with a revolutionary peanut pasteAndré Briend, John Dirks Canada Gairdner Global Health AwardOpen this photo in gallery:In the late 1990s, about 20 million children worldwide were suffering from severe malnutrition annually and more than half of them died. Standard treatments included administering IV fluids in hospital and feeding malnourished children powdered milk formulas mixed with water.But in developing countries where children were starving to death, health facilities and clean water were in short supply.Dr. André Briend, a pediatric nutritionist, was frustrated by the inability to get high nutrition food to children in a form that didn’t require clinical, sanitary conditions.Working with nutritional engineer Michel Lescanne, Dr. Briend tried making cookies, bars and other products, but they all failed.Then, one day, at his breakfast table, he mulled over a jar of Nutella – a product that requires no cooking and no liquid.The pair went on to invent Plumpy’Nut, a high-energy peanut paste containing sugar, vegetable oil and skimmed milk powder, and enriched with vitamins and minerals. Packaged in foil, it can easily be transported anywhere. Three sachets a day for six to eight weeks is all it takes to save a child’s life – and, in the past 25 years, the product has saved millions of lives.But it was a tough sell at first. “Most people refused to use the product. They were skeptical,” Dr. Briend said.But, studies were done in Chad and Senegal, followed by a high-profile study published in The Lancet.The ready-to-use therapeutic food invention is used to treat more than five million children a year in 50 countries.“It’s gratifying to see a child come back to life,” said Dr. Briend, a long-time researcher at the Institut de Recherche pour le Développement in Marseilles.– André PicardQueen of pain: The nurse who helps children rest easierJennifer Stinson, Peter Gilgan Canada Gairdner Momentum AwardOpen this photo in gallery:As a nurse and clinician-scientist who “fell in love with pain” early in her career, Jennifer Stinson has devoted her life to better understanding pediatric pain and innovating digital interventions that are kid-friendly, evidence-based and scalable.This work has already “revolutionized pediatric pain management,” thanks to Dr. Stinson’s unique ability to conduct research that is as humane as it is scientifically rigorous, according to Bonnie Bassler, a member of the advisory committee for the Peter Gilgan Canada Gairdner Momentum Award. As a 2025 recipient of this award, Dr. Stinson becomes the first nurse to ever win a Gairdner prize.When she started nursing at Toronto’s Hospital for Sick Children, Dr. Stinson saw first-hand the profound impact that pain can have on young patients, robbing them of their personalities and childhood. Painful procedures in early life can also set children up for a lifelong trajectory of being more sensitive to pain, which chronically afflicts one in five Canadian children.Yet, pediatric pain remains largely invisible. Its sufferers are often unable to articulate or communicate their pain, and pediatric pain has been historically overlooked by both researchers and health care providers.Dr. Stinson aims to change that. She involves patients and families at every step, from setting the research priorities to guiding study design. She uses technology from kids’ everyday lives – everything from mobile apps to robots and virtual reality – to create validated tools for helping kids manage pain. Just one example: an app shown to reduce pain and improve mood for sickle cell patients, which Dr. Stinson is now working to roll out in clinics across Canada.Dr. Stinson recognizes that lasting impact comes from training the next generation. At SickKids, where she is co-director of the Pain Centre, a program she oversees has now trained 400 pediatric pain researchers, making Canada a leader in the field. “No one discipline owns pain. Pain should be the responsibility of every single health care professional.”– Jennifer YangHis findings on ‘jumping genes’ were a leap forwardDaniel De Carvalho, Peter Gilgan Canada Gairdner Momentum AwardOpen this photo in gallery:DNA is often portrayed as a kind of central library where cells can access instructions on how to perform a host of biological functions.In reality, DNA is far more dynamic, full of “jumping genes” that move about in pursuit of their own selfish ends, like a library where the books can rearrange themselves and battle each other for shelf space.Under normal circumstances, cells work to keep a lid on such shenanigans, but that can change when a cell becomes cancerous and loses control of its own genome. At such times jumping genes are far less constrained, to the point that a cell can look like it is under attack by a virus trying to insert its own foreign DNA into the cell’s genetic mix.It is this situation that most intrigues Daniel De Carvalho, a senior scientist at the Princess Margaret Cancer Centre and professor at the University of Toronto.When too many jumping genes are on the loose, he said, “you can activate a very strong antiviral response against the cancer cell, which we call ‘viral mimicry.‘”His research involves harnessing viral mimicry both as a means of treatment and as a form of early cancer detection.A native of Brazil, Dr. De Carvalho grew up in the capital city, Brasilia, where he attended high school and dreamed of being a professional soccer player. But he was also interested in science and was particularly fascinated when he read about Dolly the Sheep, the first mammal cloned from an adult cell in 1996.His interest in biology led him to do a PhD in Sao Paulo followed by post-doctoral research at the University of Southern California. A job opening brought him to Toronto in 2012 and it proved a perfect fit.Since then, he has worked on clinical trials involving thousands of patients where viral mimicry has been used to make cancer cells more visible to the body’s immune system, and as a screening tool for genetic factors that may reveal where in the body a cancer cell is active well before any symptoms are apparent.“At first, the reward was the science itself. I was driven by my deep need to understand how things work,” he said. “But more recently, as these things move to the clinic and you start to see the impact on patients – that’s become the biggest motivation.”– Ivan Semeniuk