Molecules that Block Protein Misfolding May Prevent Some Neurode
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Posted on Feb 10, 2003, 2 a.m.
By Bill Freeman
New research suggests that molecules that stop proteins from "misfolding" could prevent a number of diseases whose pathology involves abnormal protein formation, for example Alzheimer's, Parkinson's, and CJD - the human form of so-called 'mad cow' disease. Study leader, Dr Jeffery W Kelly, of the Scripps Research Institute in La Jolla, California, and colleagues studied experimental models of the inherited form of a disease called amyloidosis, which is caused by the misfolding of a liver protein called transthyretin.
New research suggests that molecules that stop proteins from "misfolding" could prevent a number of diseases whose pathology involves abnormal protein formation, for example Alzheimer's, Parkinson's, and CJD - the human form of so-called 'mad cow' disease. Study leader, Dr Jeffery W Kelly, of the Scripps Research Institute in La Jolla, California, and colleagues studied experimental models of the inherited form of a disease called amyloidosis, which is caused by the misfolding of a liver protein called transthyretin. Kelly found several small molecules that were able to prevent the misfolding of transthyretin, and he believes that the molecules will also prevent the disease. According to Kelly, "the next step is to test some of these compounds in humans with disease and others in animal models of these diseases." His team have already completed two human trials involving small molecules, and a third is underway, however the results, which Kelly describes as "promising", are yet to be published.
SOURCE/REFERENCE: Science 2003;299:713-716
