(Scientific American) Although clutter can be a nuisance, it does not typically pose a health threat—unless you’re an aging neuron. As brain cells get older, some proteins within and around the cell misfold. They twist into the wrong shape, unable to do their routine job. Then they glom together to form menacing clumps. If left to accumulate, this “junk” can overwhelm nerve cells’ quality control systems, triggering incurable brain disorders such as Alzheimer’s, Parkinson’s and Huntington’s.
So whereas these diseases produce distinct symptoms and billions of dollars have been spent researching potential drugs that target their unique molecular culprits, some scientists are placing their bets on cross-cutting approaches that might work across multiple disorders. Rather than going after proteins such as amyloid beta for Alzheimer’s or alpha-synuclein for Parkinson’s, one researcher has set on a different approach: “I settled on the idea that perhaps we should just get rid of as many abnormally folded, nasty-looking proteins as possible,” says Karen Duff, a neuroscientist at Columbia University.
Strategies that boost the cell’s quality control programs, rather than disarm specific pathologic proteins, have looked promising in lab animals that serve as models for human neurodegenerative disorders including Alzheimer’s, Parkinson’s, Huntington’s, amyotrophic lateral sclerosis (ALS) and frontotemporal dementia. Several molecules have entered human testing. It is still a long road to approved therapies but a growing body of basic research is fueling a search for drugs that interact with cellular cleanup processes to provide one-size-fits-all approaches for treating a megaclass of brain disorders.[Click the title, above, to post a comment.]