Presenters for the 2023 Breakthrough Prize
Getty Images for Breakthrough Prize
Researchers at Vertex Pharmaceuticals were honored for their innovative work in the treatment of a genetic disorder, alongside other researchers in biology, physics, and mathematics.
In 1989, scientists first discovered one of the genetic factors responsible for cystic fibrosis, a disorder that causes a progressive lung disease in children. Currently, around 40,000 Americans are affected by the disease, and at the time of the genetic discovery, the average life expectancy for patients was less than 30 years.
In 2019, the FDA approved Trikafta, a drug developed by researchers at Vertex Pharmaceuticals. This drug is remarkable because it is effective for over 90% of the patient population. For many patients, this was the first approved treatment. Trikafta is a combination of three drugs developed by Vertex that target the disease by repairing the proteins responsible for its onset.
Dr. Fredrick van Goor in his lab.
Vertex Pharmaceuticals
Fred van Goor, one of the researchers involved in developing Trikafta, explains that their approach was to develop multiple medicines that could work together to fight the disease, which was an unconventional approach at the time but is now more common. “We knew from the beginning that this would require a combination of medicines,” he stated in an interview with Forbes.
It took more than 20 years for van Goor and his colleagues to reach this point, and they developed treatments along the way that helped smaller segments of the cystic fibrosis population. Despite the progress made, van Goor and his colleagues remain committed to further advancing the fight against the disease.
“There’s still more we need to do,” he emphasized. “We’re working on more cystic fibrosis drugs that could potentially be even better than Trikafta. We want to ensure that we complete the task and provide therapy for all cystic fibrosis patients.”
On Thursday, van Goor and two other Vertex researchers, Sabine Hadida and Paul Negulescu, received one of the five $3 million Breakthrough Prizes for 2024. The award was shared with eight other scientists working in Fundamental Physics, Life Sciences, and Mathematics. Additionally, 15 early-career researchers were awarded $100,000 prizes for their contributions to physics and mathematics.
The Breakthrough Prize Foundation provides the funding for these awards. The organization was established by Yuri and Julia Milner, with additional co-funding from Facebook CEO Mark Zuckerberg and his wife, Priscilla Chan; Google co-founder Sergey Brin; and 23andMe co-founder & CEO Anne Wojcicki. Over the past 12 years, the prizes have awarded a total of $308 million to researchers in various fields.
Breakthrough Prizes in Life Sciences
In addition to the prize awarded to the Vertex team, two other Life Sciences prizes were awarded. The first went to Carl June and Michel Sadelain, who advanced the use of CAR-T immunotherapy in leukemia. This therapy involves genetically engineering a patient’s immune cells to target and destroy cancer cells. CAR-T cells have revolutionized cancer treatment and are now being explored for use against various cancers and infectious diseases.
The other Life Sciences prize was awarded to Thomas Gasser, Ellen Sidransky, and Andrew Singleton for their work in uncovering the risk factors in the human genome that can lead to the development of Parkinson’s disease. Their research has also deepened our understanding of the disease, revealing that it is not caused by a single factor but rather is a collection of diseases with different causes, similar to cancer.
Breakthrough Prize in Mathematics
Columbia University mathematician Simon Brendle received a $3 million prize for his discoveries in differential geometry. His work, particularly in relation to the concept of Ricci flow, has practical applications in various fields, including computer vision and medical imaging.
Breakthrough Prize in Fundamental Physics
Physicists John Cardy and Alexander Zamolodchikov were awarded $3 million for their discoveries in fundamental physics, particularly in the field of quantum field theory. Their work has had practical applications in materials science, with Cardy’s contribution being instrumental in predicting when certain materials can achieve superconductivity near absolute zero temperatures.
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