Potential new treatment for Huntington and Parkinson patients

A potential new treatment for neurodegenerative disorders, which seems to be able to reduce the toxic protein aggregates characteristic of many of these diseases, is published online this week in the journal Proceedings of the National Academy of Sciences.

Since it is believed that in most neurodegenerative disorders, like Parkinson and Huntington's disease (PD and HD respectively), abnormal protein aggregates are major culprits associated with neurodegeneration, this research may have important implications for the lives of thousands of neurodegenerative patients all over the world.

Parkinson's and Huntington's diseases are incurable devastating brain disorders that result from the death of brain cells associated with muscle control. Both illnesses, like many other neurodegenerative diseases, result from the formation of misshaped/incorrectly folded versions of normal proteins (all proteins have a specific shape/folding associated with their normal function) that tend to clump together leading to the death of the cells in the neighbourhood.

But the formation of these abnormal proteins is usually not a problem as two effective mechanisms exist to eliminate them: proteasomes, which are multi-protein complexes capable of digesting/cutting the toxic proteins into small pieces to be easily disposed and molecular chaperones, proteins capable of assisting other proteins to "move" into their correct shape/fold. In the cases where abnormal proteins are allowed to accumulate, as it happens in many neurodegenerative diseases, it is believed that these protective systems are malfunctioning. The fact that HD patients are known to have problems with faulty proteasomes supports this hypothesis.

Furthermore, cells with high accumulation of misshaped proteins, including those from PD and HD patients, can also present a cellular structure called inclusion body, which is known to accumulate high quantities of the altered aggregated protein. The role of these inclusions is not clear, and while for a long time they were believed to be toxic and part of the disease process, in recent years, this has been challenged and some researchers now believe that inclusion bodies (also called inclusions) might have a role eliminating the aggregates of toxic proteins. The fact that inclusion bodies are known to contain high quantities of proteasomes further supports this hypothesis. Nevertheless, research in therapies for neurodegenerative diseases seems to continue orientated towards drugs capable of diminish these cellular structures

But Ruth A. Bodner, Tiago Fleming Outeiro, a Portuguese scientist, Aleksey G. Kazantsev and colleagues from the Center for Cancer Research, Massachusetts Institute of Technology, Cambridge and the Mass General Institute for Neurodegenerative Disease, Massachusetts General Hospital, and Harvard Medical School, US had a different idea.