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Kelly Lab Discovery Leads to European Approval of the Drug Tafamidis

Vyndaqel® (tafamidis)—a drug discovered in the laboratory of Jeffery W. Kelly, chair of The Scripps Research Institute’s Department of Molecular and Experimental Medicine—has been approved by the European Medicines Agency for the treatment of familial amyloid polyneuropathy associated with transthyretin aggregation (TTR-FAP).

The initial symptoms of this inherited, and ultimately fatal protein misfolding / aggregation disease include loss of sensation, muscle weakness, and autonomic nerve dysfunction (including sexual dysfunction, gastrointestinal disorders and urinary problems) progressing to cardiac failure and loss of the ability to walk. The only previously known treatment available has been liver transplantation.

The European Commission approved Vyndagel® to treat TTR-FAP in adult patients with stage 1 symptomatic polyneuropathy. The drug’s clinical studies demonstrated it significantly halted disease progression and reduced the burden of disease after 18 months, compared to placebo. Since transthyretin tetramer dissociation, monomer misfolding and aggregation (prevented by tafamidis) also causes cardiomyopathies, affecting hundreds of thousands of patients, there is reason to be optimistic that tafamidis will be useful for ameliorating senile systemic amyloidosis associated with wild type transthyretin aggregation (leads to congestive heart failure) and familial amyloid cardiomyopathy associated with mutant transthyretin amyloid fibril formation.

Tafamidis was developed by the company FoldRx Pharmaceuticals cofounded by Kelly, and acquired by Pfizer in 2010. For more information on earlier work leading to the drug, see For Pfizer’s press release on European approval, see

Jeffery W. Kelly Wins ACS Hirschmann Award in Peptide Chemistry

Jeffery W. Kelly, chair of the Department of Molecular and Experimental Medicine, has won the American Chemical Society’s (ACS) 2012 Ralph F. Hirschmann in Peptide Chemistry. Sponsored by the Merck Research Laboratories, the award was established in 1988 to recognize and encourage outstanding achievements in the chemistry, biochemistry, and biophysics of peptides.

Kelly’s laboratory focuses on the principles of protein folding and employs a multidisciplinary approach to understand the chemistry and biology of protein folding, misfolding and aggregation (leading to diseases such as Alzheimer’s) with the goal of developing novel therapeutic strategies for the latter.

Scripps Research Graduate Students Named ARCS Scholars

arcs students
Robin Luby (front left), president of the Achievement Rewards for College Students (ARCS) Foundation San Diego chapter, presents Kellogg School Dean Jamie Williamson with a check for the ARCS 2011-2012 Scholars program. This year’s Scripps Research ARCS Scholars are (back row, left to right) Crystal Moran Gutierrez, Joseph Nagano, Joshua Silverman, Aaron Sather, Christopher Martin, Laurie Gay, Katie Duncan, and Erin Anderson. ARCS awards recognize outstanding students in science, engineering, and medical research. The ARCS Foundation has awarded $845,000 to Scripps Research students since 1997.

Alex Price Wins Hall Memorial Travel Award

The Scripps Florida Society of Research Fellows (SRF) presented its second Mark A. Hall Memorial Travel Award to Alex Price, research associate in the Paegel lab. Established earlier this year, SRF travel grants are awarded twice a year.

Named in honor of Mark Hall, a Scripps Research postdoctoral fellow in cancer biology, the award aims to support postdoctoral fellows pursuing advanced training by offering the opportunity to participate in a premier scientific meeting. 

Further information on the award program is available on the SRF webpage.

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A drug discovered in the laboratory of Jeffery W. Kelly, chair of The Scripps Research Institute’s Department of Molecular and Experimental Medicine and recent winner of the ACS Hirschmann Award in Peptide Chemistry, has been approved by the European Medicines Agency.