C. Frank Bennett
2019 Breakthrough Prize in Life Sciences
For the development of an effective antisense oligonucleotide therapy for children with the neurodegenerative disease spinal muscular atrophy.
I am grateful and honored to receive the Breakthrough Prize. I would like to thank my co-recipient Adrian Krainer for his scientific brilliance and collaborative spirit and the commitment of his fellow, Yimin Hua. The patients and their families who participated in the clinical trials are the trailblazers and my source of inspiration. Loren Eng and Karen Chen of the Spinal Muscular Atrophy Foundation helped me navigate the SMA field. Drs. Richard Finkel and Darryl De Vivo and the many other physicians, physical therapists, and staff who take care of SMA patients worked tirelessly to demonstrate the benefit of nusinersen. Kathy Bishop, Frank Rigo, and my other colleagues at Ionis Pharmaceuticals and Biogen helped transform an idea into reality. I thank Don Cleveland and Richard Smith for prodding me down the path of developing therapies for neurodegenerative diseases. Stanley T. Crooke, my mentor and colleague, taught me that it is possible to do creative and impactful science in industry and importantly, the power of saying yes. I thank my parents, who encouraged me to pursue my scientific interests, my wife, Paula, who has been my rock and has supported my endeavors, and my sons, Chris, Nick, and Alex, who keep me grounded.