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Peter Goadsby, MD , PhD, Wins the Largest Brain Research Prize for Migraine Research

Article In Brief

Neurologist Peter Goadsby, MD, PhD, and three of his colleagues received the 2021 Brain Prize, created by the Lundbeck Foundation in Denmark, for their work in determining the cause of migraine, and the breakthrough therapies that followed on the heels of their discoveries. Here, Dr. Goadsby discusses his early years and the mentors who inspired his research and career as a clinician-scientist.

Peter Goadsby, MD, PhD, ended up in medicine quite by accident, and his journey to understanding migraine began with a challenge from his one life-long mentor, neurologist James W. Lance, MD. And so when he was awarded the Brain award in March, for his pivotal research on migraine, the president of the American Headache Society was circumspect about his journey from a schoolboy in Australia to his receiving one of the largest research prizes in brain research.

His aspirations to go large started early in life, he told Neurology Today. At 17, he wanted to be the treasurer of Australia—he thought he'd do a better job than the guy at the helm. At the time, in the 1970s, the way you got into a university was to complete a final state exam and submit a sheet that included things you wanted to do in the world. A computer would match a student based on his/her scores on the state exam and the person's interests. Economics, for sure. Law, yes, that too.

But one night, he and his mother had a blazing argument. Those were the years he fashioned his hair long, and sported a leather jacket that matched quite nicely with his motorcycle—and her concept of work was that you give 100 percent. She wasn't quite sure that her son had it in him to do that. The night before he sent in the state form, he expanded the list of interests to include medicine. The computer would decide his future. And so it did.


“If we could figure out why these changes are happening, and where it is stemming from in the brain, maybe we can stop the onset of the migraine itself. Why wouldnt we want to do that?”—DR. PETER GOADSBY

The results of the university matches were published in the Sydney Morning Herald. His mother read down the list and didn't see his name on economics or law. She folded up a paper and hit him in the head.

“You didn't get into law,” she admonished. It didn't matter that virtually no one in his rough-and-tumble neighborhood in Blacktown, Australia, would even go off to a university. He continued down the list and found that he matched to medical school.

Heading off to the University of South Wales was a culture shock, he said. He wasn't even sure he could do it. But then, by chance, he sat in on a lecture on non-dominant parietal lobe syndrome. The speaker was Dr. Lance. He gave a subsequent lecture on migraine.

Peter Goadsby walked up to his professor after the lecture and said: “I think the experiment that you talked about is not logical.” And Dr. Lance, a master educator, simply said: “Well, if you think it is so silly why don't you come and do research to help sort it out?”

He did. He completed an experiment or two and they worked, and he was hooked. He identified a novel finding about the trigeminal autonomic reflex, and presented his discovery during a lecture. A neurologist in the audience got up and challenged his results, saying that no one had ever reported such a finding. Dr. Lance stood up and said: “Well, now it has.”

It was that sense of discovery—“that for a few seconds or hours I knew something that no one else know,” he explained decades later—that led him to be a neurologist and scientist.

Dr. Goadsby went on to figure out the mechanisms of a number of forms of headache, and has spent his career so far listening to his patients describe what often takes place outside of his office. His seminal findings with his collaborators led to some of the most powerful medicines for migraine and cluster headaches the world has known. Dr. Lance also taught him about doctoring headache patients. Of course, history is everything in diagnosing and understanding migraine and other headache disorders. But it is also developing a strong relationship with patients, and a lot of listening. While his mentor was a “proper professor,” he recalls a time when he was treating an 8-year-old boy with a gait problem.

“Oh, why don't you ride the horse up those stairs,” the neurologist said, and leaned down to lift the boy onto his back and bounded up the stairs.

Dr. Goadsby has made similar connections with his patients over his career. One of his young patients with cluster headaches was a candidate for oxygen therapy—a technique that he helped put on the treatment map— and his challenge was to get a 6-year-old to sit long enough for the process. He made up a story about a dragon and a slayer who blew the beast away with oxygen. The child recounted the story to his teachers and friends, and said: “A wizard told me this story.”

Dr. Goadsby would also be asked to give a eulogy at a patient's funeral. He sat at the bedside of his patient, who was dying of cancer, and talked. “Just tell them the truth about me,” Mike, his patient, said. He had cluster headaches and was one of many patients who sat down to listen to Dr. Goadsby explain the importance of placebo in treatment trials.

“Of course it's important and I wouldn't expect you to get answers otherwise,” said Mike, who was enrolled in the landmark oxygen study where patients with cluster headaches were randomized to receive air or 100 percent oxygen. The study was published in JAMA in 2009. “It works like gangbusters,” Dr. Goadsby said. The treatment is used worldwide.

“It is such a privilege to be a doctor,” he added. “I have the most interesting conversations with my patients.”

Dr. Goadsby is also amused by somehow being a part of the first cluster headache support group. He was running exceptionally late in clinic and a group of his patients were sitting in the waiting room, doing just that. After an hour of talking about their conditions, they decided it would be good to get together and continue the conversations. OUCH-UK was the result. The first official cluster headache support group.

Collaborative Research

But the Brain award is about his collaborative research that led to the discovery that calcitonin gene-related peptide, or CGRP, is key to many migraine attacks. [See “More About the Brain Prize.”] This finding led to the preventive migraine treatments—a game-changer for the field.

Dr. Goadsby worked with Lars Edvinsson, MD, PhD, of Lund University in Sweden and showed that the trigeminal nerve releases CGRP during a migraine attack. They went on to help develop the first CGRP antagonists and tested them as acute treatments to block the start of a migraine.

The US Food and Drug Administration approved the first anti-CGRP medication in 2018, other approvals have followed, and there are even more medications in development. Knowing the path to migraine led to the development of CGRP-monoclonal antibodies that are now given subcutaneously or intravenously once a month to every third month. This has been shown to reduce future attacks by about 50 percent.

The Importance of Mentorship

Dr. Goadsby has gone on to train many headache neurologists. His key to a successful training, he said, is simple: Start with a good mentor—and Dr. Lance was the best he could have hoped for—and find good collaborators (which he did) and be willing to try, and keep on trying. Dr. Goadsby and Dr. Lance remained close friends throughout their lives, and he was honored to have had the opportunity to write his mentor's obituary in 2019 in the journal Headache, which in many ways was a love letter to an amazing teacher, clinician, scientist, and friend.

He has no plans to stop asking questions of migraine. He is now working to understand the premonitory changes that arrive on the scene in the hours before the actual migraine. Patients have reported mood changes, exhaustion, yawning, and passing urine. “If we could figure out why these changes are happening, and where it is stemming from in the brain, maybe we can stop the onset of the migraine itself,” he said. “Why wouldn't we want to do that?”

Dr. Goadsby has also been studying visual snow, where migraine sufferers say that they see small dots moving around their visual field. It is independent of aura, and it is something that few have been studying. In talking about his pursuit to understand visual snow, he recalls something that his mentor once said when they started working together: “If you take something on when no one is studying it, it doesn't take much to advance things. Well, Peter, there is a lot of room to move in migraine.”

More About The Brain Prize

Peter Goadsby, MD, PhD, president of the American Headache Society, and professor of neurology at the David Geffen School of Medicine at UCLA, and three of his colleagues— Lars Edvinsson, MD, PhD, of Lund University Hospital, and Jes Olesen, MD, of the University of Copenhagen, and Michael Moskowitz, MD, of Harvard University—received the 2021 Brain Prize, created by the Lundbeck Foundation in Denmark, for their work in determining the cause of migraine, and the breakthrough treatments that followed on the heels of their discoveries.

The Brain Prize, the world's largest brain research prize, was launched in 2011 by the Lundbeck Foundation in Denmark. Each year, the foundation awards a $1.3 million prize to one or more neuroscientists who have dramatically changed the landscape of their fields.

This year, the four winners have been at the lead in migraine research. So far, 34 scientists from around the world have received the prestigious prize. There will be an award ceremony in Copenhagen that will be presided over by His Royal Highness, The Crown Prince of Denmark.

The Lundbeck Foundation created the prize as a way to educate the public in the importance of brain research.

Brain Prize winners are nominated and a selection committee–which includes nine top neuroscientists from five countries—determines whether the scientist's body of work stands out in advancing what is known in their field of study.

The Lundbeck Foundation was established in 1954 by Grete Lundbeck, a businesswoman and widow of the founder of H. Lundbeck A/S, Hans Lundbeck. The foundation awards more than $80 million dollars to Danish-based biomedical research, primarily in neuroscience. The foundation says that their goal is to “create powerful ripple effects that bring discoveries to lives through investing actively in business and science at the frontiers of their fields.”

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• Goadsby PJ. James W. Lance MD Headache 2019;59:825–827.