Improving risk/benefit estimates in new drug trials

Improving risk/benefit estimates in new drug trials

From the Science Daily News March 8 2011 news item

ScienceDaily (Mar. 8, 2011) — It’s all too familiar: researchers announce the discovery of a new drug that eradicates disease in animals. Then, a few years later, the drug bombs in human trials. In the latest issue of the journal PLoS Medicine, ethics experts Jonathan Kimmelman, associate professor at McGill’s Biomedical Ethics Unit and Department of Social Studies of Medicine, and Alex John London, associate professor of philosophy at Carnegie Mellon University, argue that this pattern of boom and bust may be related to the way researchers predict outcomes of their work in early stages of drug development.

 

The study suggests researchers focus too narrowly on pre-clinical data, which leads to overoptimistic predictions. It is also possible that drug bias is not as rigorous in animal testing than in human testing.

 

 

Structure of key molecule in immune system provides clues for designing drugs

Structure of key molecule in immune system provides clues for designing drugs

From the December 27, 2010 Eureka news alert

PHILADELPHIA – A team from the University of Pennsylvania School of Medicine and Utrecht University has deciphered a key step in an evolutionarily old branch of the immune response. This system, called complement, comprises a network of proteins that “complement” the work of antibodies in destroying foreign invaders. It serves as a rapid defense mechanism in most species from primitive sponges to humans.

In a study published in the December 24 issue of Science, the groups of John Lambris, PhD, the Dr. Ralph and Sally Weaver Professor of Research Medicine at Penn, and Piet Gros at Utrecht, detail the atomic structure of two key transient enzyme complexes in the human complement system.

Complement proteins mark both bacterial and dying host cells for elimination by the body’s cellular cleanup services and have been implicated in at least 30 diseases, including stroke, myocardial infarction, and age-related macular degeneration. The findings, Lambris says, provide a molecular scaffold for designing novel drug therapeutics….

…”Besides shedding light on a highly elegant mechanism of concerted activation and intrinsic regulation, this work also offers a detailed insight into one of the most important therapeutic targets within the complement network, which may facilitate rational drug development and could lead to novel drugs for treating complement-related diseases,” Lambris says….

Background material on complement proteins

• The complement system and innate immunity (via NCBI Bookshelf)
• The complement system (via NCBI Bookshelf in Immunology Review)
• The complement system (college lecture with flash animations)