NEWS OF THE WEEK
ACS HONORS LEADERSHIP PUBLIC SERVICE AWARDS: Society
recognizes congressmen and National Academies' president
T Cicerone (left), Hobson, Hunt, and Hinojosa.
HE AMERICAN CHEMICAL Society bestowed its prestigious Public Service Award upon Reps. Ruben Hinojosa (D-Texas), David L. Hobson (R-Ohio), and National Academies President Ralph J. Cicerone during a Capitol Hill ceremony on April 25. The awards recognize people who have made outstanding contributions to the development of public policy that benefits chemistry and the sciences. "The awards are our way of thanking those who share our passion for science and of recognizing that science and technology are critical to advancing our nation/' said ACS President Catherine (Katie) T. Hunt during opening remarks at the ceremony. Hinojosa, who chairs the House
DRUG FOR GENETIC DISORDERS DRUG DEVELOPMENT: Compound
might treat conditions caused oy nonsense mutations
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ONSENSE genetic mutations, which result in shorter-than-normal proteins, cause 5-70% of cases of inherited disorders, including muscular dystrophy, cystic fibrosis, and cancer. A small molecule now in Phase II clinical trials might be capable of treating several of these types of cases. This treatment strategy—targeting a group of disease cases caused by one class of mutation rather than targeting individual diseases—represents a "paradigm shift," according to Stuart W. Peltz, president and CEO of PTC Therapeutics, in South Plainfield, N.J. The biopharmaceutical firm discovered the compound via highthroughput screening. A nonsense mutation in DNA introduces a code for a stop codon rather than for an amino acid. When the DNA is transcribed into messenger RNA (mRNA), this misplaced stop codon—also known as a premature termination WWW.CEN-0NLINE.ORG
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Education & Labor Committee's Subcommittee on Higher Education, Lifelong Learning & Competitiveness, introduced in 2006 the Partnerships for Access to Laboratory Science Act, which the House Science & Technology Committee recently passed. He thanked ACS for its leadership in promoting science, technology, engineering, and math education. Hobson, who serves as a ranking member of the Appropriations Subcommittee on Energy 8c Water and is a senior member of the Appropriations Subcommittee on Defense, oversaw the first maj or funding increase for basic science at the Department of Energy in a generation. "By making sound judgments today in research and development, we have an opportunity to transform our national economy and improve the quality of life for future generations," Hobson said. Cicerone, following a request from the U.S. Senate, led a panel that produced the report "Rising above the Gathering Storm: Energizing and Employing America for a Brighter Economic Future." The report provides a blueprint to increase the nation's ability to innovate. The awards ceremony was preceded by a poster session sponsored by the Council on Undergraduate Research. Students discussed their research projects with public officials and ACS staff and governance. The awards were established in 1997 under the leadership of former ACS president Ronald Breslow. —LINDA WANG
codon (PTC)—halts translation of the mRNA into a protein by a ribosome. The resulting truncated protein can cause disease. Some cases of Duchenne muscular dystrophy, for instance, are caused by nonsense mutations that yield malformed dystrophin, a protein necessary for maintaining the strength of muscle fibers. PTC Therapeutics wants to treat this fatal musclewasting disease with the small molecule it calls PTC124. Peltz, University of Pennsylvania physiologist H. Lee Sweeney, and colleagues report that the oral drug binds to ribosomes and allows them to bypass the mRNA site that interrupts dystrophin production. In a mouse model of the disease, the ribosomes then churn out normal, full-length dystrophin, thereby restoring muscle function (Nature, DOI: io.i038/natureo5756). PTC Therapeutics is now testing the compound's efficacy in patients with Duchenne muscular dystrophy and cystic fibrosis. In a commentary in Nature, Anton Schmitz and Michael Famulok of the University of Bonn, in Germany, point out PTC124/S "astonishing" selectivity for PTCs. The compound enables a ribosome to distinguish between a premature stop codon and a normal stop codon that indicates where protein production should properly cease. The antibiotic gentamycin also allows ribosomes to ignore PTCs, but its severe side effects and intravenous delivery method have limited its use. Furthermore, unlike PTC124, gentamycin increases the risk of producing mutated proteins.—SOPHIE ROVNER
APRIL30.2007
