Presenters for the 2023 Breakthrough Prize
Getty Images for Breakthrough Prize
Researchers at Vertex Pharmaceuticals were recognized for their groundbreaking work in the treatment of cystic fibrosis, along with other researchers in the fields of biology, physics, and mathematics.
In 1989, scientists discovered one of the genetic factors that can lead to cystic fibrosis, a disorder that causes a progressive lung disease in children. Currently, approximately 40,000 Americans are affected by the disease, and at the time of the genetic discovery, the average life expectancy for individuals with the condition was less than 30 years.
In 2019, the drug Trikafta, developed by Vertex Pharmaceuticals, was approved by the FDA. What makes this drug remarkable is its effectiveness in over 90% of patients, as it combines three Vertex drugs to address the disease by repairing the proteins that cause it.
Dr. Fredrick van Goor in his lab.
Vertex Pharmaceuticals
Fred van Goor, one of the researchers involved in the development of Trikafta, highlighted the approach they had in mind which involved developing multiple medicines that work together to combat the disease. Although this approach was uncommon at the time, it is now becoming more prevalent. “We knew from the beginning that this would require a combination of medicines,” he told Forbes.
It took van Goor and other researchers at Vertex over 20 years to reach this point, developing approved treatments along the way that benefited a portion of the cystic fibrosis population. While van Goor is satisfied with the progress made thus far, he emphasizes that there is still more work to be done in addressing cystic fibrosis. “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 have access to a therapy.”
On Thursday, van Goor and two other Vertex researchers, Sabine Hadida and Paul Negulescu, were awarded one of the five $3 million Breakthrough Prizes for 2024. They shared the awards with eight other scientists working in the fields of Fundamental Physics, Life Sciences, and Mathematics. Additionally, 15 early-career researchers received $100,000 prizes for their contributions to physics and mathematics.
The funding for these prizes is provided by the Breakthrough Prize Foundation, founded by Yuri and Julia Milner. The prizes were further co-founded by Facebook CEO Mark Zuckerberg and his wife, Priscilla Chan, as well as Google co-founder Sergey Brin and 23andMe co-founder & CEO Anne Wojcicki. Over the past 12 years, the prizes have awarded $308 million to researchers across 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 were awarded. The first was a $3 million prize awarded to Carl June and Michel Sadelain for their advancements in the use of CAR-T immunotherapy in leukemia. This therapy involves genetically engineering a patient’s own immune cells to target and eliminate cancer cells, revolutionizing cancer treatments. CAR-T cells are now being explored as potential treatments for various types of cancer and infectious diseases.
The second $3 million Life Sciences prize was awarded to Thomas Gasser, Ellen Sidransky, and Andrew Singleton. These researchers contributed to the understanding of the risk factors in the human genome that can lead to the development of Parkinson’s disease. Their work also shed light on the complex nature of the disease, which is now recognized as a family of diseases with multiple causes, similar to cancer.
Breakthrough Prize in Mathematics
Columbia University mathematician Simon Brendle received a $3 million award for his groundbreaking discoveries in differential geometry. His work in this area has practical applications in fields like 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, with applications in materials science. Cardy’s contributions include developing mathematical models that predict when certain materials can become superconductors as their temperature approaches absolute zero.
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