SMA researchers Frank Bennett and Adrian Krainer win the prestigious Breakthrough Prize

Excerpted from Breakthrough Prize press release:

 

A Total of $22 Million Awarded for Breakthroughs in Treatment for the Leading Genetic Cause of Infant Death, Super-Resolution Imaging, Discovery of a New Kind of Electronic Material, and Other Major Achievements.

 2019 Breakthrough Prize in Life Sciences Awarded to C. Frank Bennett and Adrian R. Krainer.

 All Laureates – including Jocelyn Bell Burnell, Recipient of 2018 Special Breakthrough Prize in Fundamental Physics – to be Honored at Live Televised Ceremony on Sunday, November 4.

October 17, 2018 – (San Francisco) – The Breakthrough Prize Foundation and its sponsors – Sergey Brin, Priscilla Chan and Mark Zuckerberg, Ma Huateng, Yuri and Julia Milner and Anne Wojcicki – announced today the recipients of the 2019 Breakthrough Prize, awarding a collective total of $22 million to nine researchers for important achievements in the Life Sciences, Fundamental Physics and Mathematics.

The 2019 Breakthrough Prize and New Horizon Prize recipients will be recognized at the seventh annual Breakthrough Prize ceremony, known as the “Oscars of Science,” hosted by acclaimed actor, producer and philanthropist Pierce Brosnan, on Sunday, November 4, at NASA Ames Research Center in Mountain View, California, and broadcast live on National Geographic.

Considered the world’s most generous science prize, each Breakthrough Prize is for $3 million. Now in its seventh year, the Prize recognizes achievements in the Life Sciences, Fundamental Physics and Mathematics, the disciplines that ask the biggest questions and find the deepest explanations.

In addition to the seven main-stage prizes, a total of six New Horizons Prizes, worth $100,000 each, were awarded to seven physicists and five mathematicians for early-career achievements in their respective fields.

The 2019 Breakthrough Prize and New Horizon Prize recipients will be recognized at the Breakthrough Prize ceremony, or the “Oscars of Science,” on Sunday, November 4. The gala-event, hosted by acclaimed actor, producer and philanthropist Pierce Brosnan, will bring together luminaries of the science and tech communities with celebrities, athletes and musicians, all attending with the common goal of celebrating science and scientific achievement. This year’s ceremony will be broadcast live on Nat Geo, YouTube and Facebook Live from the NASA Ames Research Center in Mountain View, California.

Breakthrough Prize In Life Sciences

  1. Frank Bennettand Adrian R. Krainer– Ionis Pharmaceuticals and Cold Spring Harbor Laboratory, respectively

Citation: For the development of an effective antisense oligonucleotide therapy for children with the neurodegenerative disease spinal muscular atrophy.

Description: Spinal Muscular Atrophy (SMA) is a rare but devastating disease – the leading genetic cause of infant death. Many children with SMA die before their second birthday. Now it is no longer a death sentence. Frank Bennett, a pharmacologist, and Adrian Krainer, a biochemist, built upon their discoveries about antisense technology and the natural process of RNA splicing to produce the first drug to treat SMA – Nusinersen (marketed by Biogen as Spinraza). It was approved by the FDA in 2016 and is one of the first of a promising new breed of antisense therapies now in development for familial dysautonomia (FD), glioblastoma and liver cancer. 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. Bennett grew up in his family’s hotel, in Aztec, New Mexico. With a nudge from guidance counselors, he pursued pharmacology instead, and developed a passion for finding cures for really awful diseases. Krainer grew up in Montevideo, Uruguay, of Eastern European descent. He admired Gregor Mendel’s work, and as a high school student developed an interest in genetics. Bennett and Krainer, previously aware of each other’s work, joined forces in 2004 to research SMA and have collaborated ever since.

 

About the Breakthrough Prizes

For the seventh year, the Breakthrough Prizes will recognize the world’s top scientists. Each prize is $3 million and presented in the fields of Life Sciences (up to four per year), Fundamental Physics (one per year) and Mathematics (one per year). In addition, up to three New Horizons in Physics and up to three New Horizons in Mathematics Prizes are given out to junior researchers each year. Laureates attend a live televised award ceremony designed to celebrate their achievements and inspire the next generation of scientists. As part of the ceremony schedule, they also engage in a program of lectures and discussions.

The Breakthrough Prizes are sponsored by Sergey Brin, Priscilla Chan and Mark Zuckerberg, Ma Huateng, Yuri and Julia Milner, and Anne Wojcicki. Selection Committees composed of previous Breakthrough Prize laureates in each field choose the winners.

Information on the Breakthrough Prizes is available at breakthroughprize.org.