“It is a breakthrough to be able to make progress in an area like this. I think that is something that is really worth celebrating.”
Frank Bennett of Ionis Pharmaceuticals ©2015 Ken West Photography, Inc.
C. Frank Bennett, the head of research at Ionis Pharmaceuticals, and Adrian R. Krainer, the St. Giles Foundation professor at Cold Spring Harbor Laboratory, will share a $3 million Breakthrough Prize for their role in developing Spinraza, a drug against spinal muscular atrophy that is marketed by Ionis and Biogen.
The Breakthrough Prizes, an “Oscars of Science” sponsored by Sergey Brin, Priscilla Chan and Mark Zuckerberg, Ma Huateng, Yuri and Julia Milner and Anne Wojcicki, pay out larger amounts than any other scientific award. This year, a total of $22 million will be awarded in a televised awards ceremony on November 4.
Breakthrough awards will also be given to Angelika Amon, of the Massachusetts Institute of Technology and Howard Hughes Medical Institute, for research into how having abnormal numbers of chromosomes disrupts cell function; to Xiaowei Zhuang, of Harvard University and Howard Hughes Medical Institute, for using super-resolution imaging to study cells; and Zhijian “James” Chen University of Texas Southwestern Medical Center and Howard Hughes Medical Institute, for understanding how immune responses are triggered in cells.
Cold Spring Harbor Laboratory’s Double Helix Medals 2016 Victor Hugo
The sore thumb in the group, obviously, is Bennett. Four of the five biologists winning a Breakthrough Prize this year are academics. But from their inception, the Breakthrough Prizes have been more likely than other awards, most notably the Nobel Prizes, to recognize the role of the scientists in industry who are frequently the inventors and almost always the developers of new medicines. One of the first Breakthrough Prizes was given to Napoleone Ferrera, then at Genentech, for his role in developing two Roche drugs: Avastin, for multiple types of cancer, and Lucentis, for macular degeneration, a leading cause of blindness.
Bennett joined Ionis, originally called Isis Pharmaceuticals, three decades ago, when Isis’ founder, Stan Crooke, was leaving big pharma to start a smaller biotechnology firm to focus on a technology called antisense. Antisense works by using a strand of nucleotides, the type of chemical in DNA, to keep particular genes from functioning. In 2003, Bennet noticed a paper that Krainer had written about using a less elegant type of antisense to treat spinal muscular atrophy, the disease that Spinraza treats, in cells. The results of their collaboration were stunning. In the clinical trials that led to Spinraza’s approval, half of infants treated with the drug reached a major motor milestone, compared with none on placebo. In later-onset forms of the disease, kids on Spinraza saw their motor function improve, while those on placebo saw it worsen.
Spinraza is expensive: It costs $750,000 for the first year, when a higher dose is given, and $350,000 for each year thereafter.
Cori Bargmann, a previous winner of the Breakthrough Prize and a member of the committee that picks the winners (she’s also a top scientist at Rockefeller University and, in her extra time, the leader of the Chan-Zuckerberg Inititative’s science work), says she can’t reveal anything about the committee’s deliberations but that it doesn’t consider drug prices. “Everyone who thinks seriously about medicine thinks the U. S. system needs work,” she says.
But she does say she thinks the Breakthrough Prize is open to innovation whether it happens in industry or academia. She also notes that there’s a commitment that one of the prizes will focus on neurodegenerative disease, which has proved especially hard to target with drugs. But she also says that part of the goal of the prizes is to be like an Oscars for science—to tell people who don’t pay attention to science what they should be paying attention to, because it is the best, most wondrous, and most impactful work.
“It is a breakthrough to be able to make progress in an area like this,” she says. “I think that is something that is really worth celebrating.”
I believe this is biology’s century. I’ve covered science and medicine for Forbes from the Human Genome Project through Vioxx to the blossoming DNA technology changing the world today.
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