Presenters for the 2023 Breakthrough Prize
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Researchers at Vertex Pharmaceuticals were honored for their revolutionary work in the treatment of the genetic disorder, along with other researchers in biology, physics and mathematics.
In 1989, scientists first identified one of the genetic factors that can cause cystic fibrosis, a disorder that causes a progressive lung disease in children. Around 40,000 Americans have the disease right now, and at the time the genetic factors were discovered, the average life expectancy for those with the disease was less than 30 years.
In 2019, the FDA approved the drug Trikafta, which was developed by researchers at Vertex Pharmaceuticals. What’s remarkable about the drug is that it’s effective for over 90% of patients, who receive a treatment that combines three Vertex drugs to tackle the disease by repairing the proteins that cause it.
Dr. Fredrick van Goor in his lab.
Vertex Pharmaceuticals
According to Fred van Goor, one of the researchers who helped develop Trikafta, part of the approach he and his colleagues had in mind was to develop multiple medicines that could work in tandem with each other to fight the disease — an unusual approach at the time that is becoming more common today. “We knew from the beginning that this would require a combination of medicines,” he told Forbes.
It took over 20 years for van Goor and other researchers at Vertex to reach that point, along the way developing approved treatments that helped smaller parts of the cystic fibrosis population. But while he’s been gratified by the work he’s done on cystic fibrosis and the patients he’s been able to help so far, he says he and his colleagues aren’t done tackling the disease.
“There’s still more we need to do,” he says. “We’re working on more cystic fibrosis drugs that could be even better than Trikafta. We want to make sure we finish the job and ensure that all cystic fibrosis patients will have a therapy.”
On Thursday, van Goor and two other Vertex researchers, Sabine Hadida and Paul Negulescu, won one of five $3 million Breakthrough Prizes for 2024, sharing the awards with 8 other scientists working in Fundamental Physics, Life Sciences and Mathematics. In addition, 15 early-career researchers were awarded $100,000 prizes for their contributions to physics and mathematics.
The money for the awards is provided by the Breakthrough Prize Foundation. The organization was founded by Yuri and Julia Milner, and the prizes were additionally cofounded by Facebook CEO Mark Zuckerberg and his wife, Priscilla Chan; Google cofounder Sergey Brin and 23andMe cofounder & CEO Anne Wojcicki. Over the past 12 years, the prizes have awarded $308 million to researchers in a variety of fields.
Breakthrough Prizes In Life Sciences
In addition to the prize awarded to the Vertex team, two other prizes in Life Sciences have been awarded. First was a $3 million prize awarded to Carl June and Michel Sadelain. These two scientists advanced the first use of CAR-T immunotherapy in leukemia. This type of therapy involves taking a patient’s own immune cells and genetically engineering them to target and kill cancer cells. This technology has helped revolutionize cancer therapies. CAR-T cells are now being applied to a number of different cancers and being explored for use against infectious disease.
The other $3 million Life Sciences prize was awarded to Thomas Gasser, Ellen Sidransky and Andrew Singleton. The work of these researchers helped uncover the risk factors in the human genome that can lead to the development of Parkinson’s disease. Additionally, their work helped inform our current understanding of the disease, which is that it doesn’t have a single cause but rather, like cancer, is better thought of as a family of diseases that can be caused in different ways.
Breakthrough Prize In Mathematics
Columbia University mathematician Simon Brendle was awarded $3 million for his discoveries in differential geometry. In particular, Brendle has done significant work on mathematical problems involved in the concept of Ricci flow. The math discovered with respect to this concept has practical applications in a number of fields, especially 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. These two scientists are focused on quantum field theory, and their work has had practical applications in materials science. Cardy, for example, invented the math that helps predict when certain materials can become superconductors as their temperatures approach absolute zero.