Last month, researchers at the University of Pennsylvania published new evidence pointing to the role of protein misfolding in Parkinsonís disease. Today the Wall Street Journal reports on the emerging hypothesis that Parkinsonís and other neurodegenerative diseases may be caused at least in part by misfolded proteins that spread from one cell to another. Known to researchers as prion disease, this type of cellular dysfunction is best known to the public for its role in ďmad-cow disease,Ē or Variant Creutzfeldt-Jakob disease, which infects cattle and can be transmitted to humans.†Cattle donít get Parkinsonís disease, and people canít get Parkinsonís or Alzheimerís from eating meat. Nonetheless, what scientists are learning about protein misfolding in mad cow disease may well have relevance for drug development in human brain diseases.
Reporter Amy Dockser Marcus writes:
While the human variant of mad-cow disease isn't normally lumped together with Alzheimer's, Parkinson's or Lou Gehrig's disease, which affect millions of mostly older people world-wide, the conditions share the ability to spread and wreak havoc through the body. And although there isn't evidence that these more common neurological disorders are transmissible to people, researchers are finding that each condition is associated with a similar deformation in the structure of particular proteins needed for normal healthy functioning. [Ö]
Deformed proteins can't be mended, but stopping cell-to-cell spread provides a new therapeutic target. "Arrest it and we can potentially stop the disease," says Neil R. Cashman, a neurologist in the Brain Research Centre at University of British Columbia, who conducts research on amyotrophic lateral sclerosis, also known as ALS or Lou Gehrig's disease. Dr. Cashman also works as chief scientific officer of a biotech company developing possible ALS therapies based on this method.
Marcus also spoke to our own Todd Sherer about The Michael J. Fox Foundationís efforts to drive this work forward in Parkinsonís disease:
This fall, Todd Sherer, chief executive of The Michael J. Fox Foundation for Parkinson's Research, gathered scientists researching protein misfolding in a number of neurodegenerative conditions to explore ways to stop aberrant proteins from spreading. He says the latest papers led the foundation to "increase its support for this theory" in Parkinson's disease. The foundation is helping to fund a trial of a vaccine developed by Austrian biotech Affiris AG aimed at getting the immune system to produce antibodies that bind to and clear the toxic alpha-synuclein protein. The foundation is also considering funding other projects to stop cell-to-cell spread.
Image via Max-Planck-Gesellschaft