“For elucidating the molecular basis of neurodegenerative and cardiac transthyretin diseases, and for developing tafamidis, a drug that slows their progression.”
Pioneering biochemist Jeffery Kelly, PhD, has been awarded a $3 million Breakthrough Prize for transformative advances in the understanding of neurological disease.
The 2022 Breakthrough Prizes in Fundamental Physics, Life Sciences and Mathematics are recognized as one of the world’s most prestigious science prizes. Kelly’s award, one of three life science prizes announced Sept. 9, recognizes his research into a broad set of disorders where incorrectly folded proteins clump and lead to progressive damage to heart and/or nervous system tissue. Kelly invented a first-in-class medication, called tafamidis, to treat the condition. His work has implications for many other conditions, including Alzheimer’s and Parkinson’s, noted the Breakthrough Prize Foundation.
“It is a tremendous honor to receive the Breakthrough Prize and to be included in the terrific company of this year’s other recipients and those of years past,” says Kelly, the Lita Annenberg Hazen Professor of Chemistry at Scripps Research, a professor of molecular medicine, and a Scripps Research trustee.
Kelly has spent much of his career studying the clumping of a normal protein into amyloid plaques. These toxic substances can wreak havoc beyond the brain. Around 40 diseases have been named after the amyloid structure, including a rare inherited or sporadic condition called transthyretin amyloidosis, Kelly notes.
Scripps Research President and CEO Peter Schultz, PhD, says Kelly has been a leader in the field of protein misfolding and aggregation, helping illuminate its role in neurodegenerative disease progression.
“His breakthrough was in translating our knowledge of protein folding diseases to a human therapeutic through rational, structure-based drug design to prevent protein aggregation,” Schultz says. “The impact of this work on patients with transthyretin cardiomyopathy has been transformational.”
Kelly is now teaming with other experts at Scripps Research on a strategy to aid early diagnosis and improve the body’s ability to clear toxic protein clumps.
“Through the fashioning of small molecule drugs that can stabilize proteins against abnormal protein aggregation, I think we learned two really important things,” Kelly says. “One is the process of protein aggregation really does drive neurodegeneration. The second is that treating these patients early is absolutely critical.”
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