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First drugs found to inhibit elusive cancer target

By Melinda Wenner Moyer Ever since scientists discovered the cancer-promoting gene MYC in the late 1970s, researchers have dreamt of developing drugs that inhibit its function. Yet efforts to target MYCactivity have proven unsuccessful, in part because the protein product encoded by the oncogene lacks an obvious target-binding site. Now, however, scientists from a handful of research groups have found a way to inhibit MYCindirectly—by preventing an upstream protein from instigating the expression of MYCand its downstream targets. Buoyed by the promising therapeutic effects that such experimental drugs have had in mice with several types of cancer, companies are racing  … Read more

Preemptive genotyping trialed to prevent adverse drug reactions

MONTREAL — In March 2010, the US Food and Drug Administration (FDA) added a ‘black box’ warning to the drug clopidogrel, noting the link between certain genotypes and reduced drug efficacy. Despite the label change, however, only around one in every 200 physicians actually orders a genetic test when prescribing clopidogrel — an antiplatelet medicine marketed by Sanofi and Bristol-Myers Squibb as Plavix — according to data from Medco Health Solutions, a New Jersey–based pharmacy services provider.  Read more

EDITORIAL: Risk-takers wanted

Treating costly conditions such as Alzheimer’s disease may soon collapse healthcare systems around the world, yet companies hesitate to invest in the long, large clinical trials required to discover disease-modifying therapies. New incentives are necessary to turn this tide.  Read more

New mouse models of autism highlight need for standardized tests

New mouse models of autism highlight need for standardized tests

By Sarah C P Williams Most laboratory mice, when meeting new cagemates, will sniff the strangers thoroughly. But the mice in Matthew Anderson’s lab instead sit alone, licking their paws repetitively. They ignore other mice, avoid new toys and rarely make noise. Taken together, the abnormalities closely resemble the behavioral symptoms seen in people with autism, a disorder that has been proven difficult to accurately recapitulate in animal models—until recently. “When I first started working on this, I really wondered whether we’d be able to study autism in a mouse,” says Anderson, a neuroscientist at the Beth Israel Deaconess Medical  … Read more

NEWS FEATURE: Autism, authenticated

NEWS FEATURE: Autism, authenticated

This past spring, Christian Schaaf sat back and watched seven-year-old Lily play in his office at the Baylor College of Medicine in Houston. She looked just like any other girl her age, he recalls, but she didn’t seek interaction or even eye contact in the way a child normally would. Instead, she communed with a corner of the room, excitably hopping and flapping her arms as if that spot held a treat too great to bear. Without peering into the file in front of him, Schaaf knew what afflicted Lily. “I’ve seen enough children that when I see someone with autism, I have a high suspicion for it,” he says.  Read more

Mysteries about drug metabolism in the obese weigh on doctors

By Alisa Opar The surgery was a success, but a question loomed after the procedure: given that the patient was obese, what was the right antibiotic dose? “The thought was, well, she’s twice as big as a normal person, so we’ll give her twice the dose,” says Aaron Cook, a clinical pharmacy specialist at the University of Kentucky in Lexington. “For that drug, levofloxacin, there’s just no information to go on, no dosage recommendation for obese patients.” The patient fared well, but such conundrums are becoming increasingly common as obesity rates rise around the globe. Just a month ago, researchers  … Read more

Childhood tuberculosis treatment remains imprecise science

Childhood tuberculosis treatment remains imprecise science

By Julie Manoharan Last year, the World Health Organization released updated procedures on how best to tackle the global scourge of tuberculosis. The fourth edition of the “Treatment of tuberculosis: Guidelines” recommended, among other changes, increasing the dosage of tuberculosis medication required to treat children. But, in a sense, the new guidance provided a destination without a map: it failed to address the larger problem of how to improve the accuracy of pediatric dosing. In recent months, researchers have pointed to a host of problems plaguing the diagnosis and treatment of tuberculosis in children, especially those younger than age 5.  Read more

New fee structure proposed by FDA might lead to more talk

Ever since 1992, when US lawmakers passed the Prescription Drug User Fee Act (PDUFA) to accelerate review of new drugs by the US Food and Drug Administration, industry money has had an increasingly important role in fueling the regulatory agency. In the program’s first year, drug companies paid less than $9 million total to the FDA through the initiative. But in the past two decades the amount has ballooned; this year, the agency anticipates receiving at least $619 million in user fees, composing roughly 65% of its budget for overseeing human drugs.  Read more

Ten years on from anthrax scare, analysis lags behind sequencing

Ten years on from anthrax scare, analysis lags behind sequencing

By Amber Dance A decade ago this month, a microbiologist at Northern Arizona University, in Flagstaff, took a special delivery from the US government. Federal investigators wanted the scientist, Paul Keim, to identify the anthrax that appeared in letters mailed to news organizations and US lawmakers. Overnight, he used PCR to determine that the anthrax sent was the Ames strain, commonly used in research—but that was just the beginning of a scientific investigation that would catapult the still wet-behind-the-ears science of microbial forensics to the forefront of the criminal inquiry. Ten years on, Keim’s PCR-based technique seems downright quaint in  … Read more

Businesses ready whole-genome analysis services for researchers

By Trevor Stokes The cost of sequencing an individual’s entire genome has fallen precipitously over the past five years, from around $100 million for the first personal genome to under $5,000 today when sequencing services are purchased in bulk. In response, a handful companies have started developing whole-genome annotation services that give clinical researchers lacking expertise in bioinformatics the ability to use genomic data for disease-discovery and drug-response testing. One company, Knome, based in Cambridge, Massachusetts, already offers a package deal. For about $5,000 it will sequence and annotate a genome—with a minimum order of ten genomes. Meanwhile, two California  … Read more