SMA Drug Developers Share $3 Million Breakthrough Prize

Rare Daily Staff

Ionis Pharmaceuticals pharmacologist Frank Bennett and Cold Spring Harbor Laboratory Biochemist Adrian Krainer will share a $3 million Breakthrough Prize for their work in the development of Spinraza, an antisense therapy for children with spinal muscular atrophy, a rare and deadly neurodegenerative disease.

The Breakthrough Prize Foundation awards the prize, which it bills as the “Oscars of Science.” The prize is sponsored by notable figures in Silicon Valley including Sergey Brin, Priscilla Chan and Mark Zuckerberg, Ma Huateng, Yuri and Julia Milner and Anne Wojcicki. A total of $22 million in 2019 prizes to nine researchers were announced for achievements in the life sciences, fundamental physics and mathematics. The prize announcements included six New Horizon prizes for $600,000 each.

The 2019 Breakthrough Prize and New Horizon Prize recipients will be recognized at the seventh annual Breakthrough Prize ceremony Sunday, November 4, at NASA Ames Research Center in Mountain View, California and broadcast live on National Geographic. The Breakthrough Prize is believed to be the science prize that carries the greatest dollar award.

SMA is the leading genetic cause of infant death. Many children with SMA die before their second birthday. The foundation says thank to the work of Bennett and Krainer, it is no longer a death sentence.

The two built upon their discoveries about antisense technology and the natural process of RNA splicing to produce the first drug to treat SMA, which today is marketed by Biogen as Spinraza. The U.S. Food and Drug Administration approved the drug in 2016.

The work has also paved the way for the possibility of new therapies using gene silencing modalities for Huntington’s, ALS, spinocerebellar ataxias, Parkinson’s and Alzheimer’s. The two scientists, previously aware of each other’s work, joined forces in 2004 to research SMA and have collaborated ever since.

“I strongly believe in the importance of collaboration between academic research and industry,” Krainer said. “Frank and I, and our respective teams, have different areas of expertise–on RNA splicing and antisense pharmacology. It was by combining our unique strengths that we managed to accomplish something so important, from which thousands of SMA patients are now benefitting.”

October 17, 2018
Photo: Adrian Krainer, a biochemist at Cold Spring Harbor Laboratory