Parkinson’s disease is a central nervous system disorder in which patients suffer from rigid muscles, gait and speech challenges, and slower movements. Eventually, this disorder leads to dementia and then death. The exact cause is not known, but the symptoms arise due to failing brain nerve cells responsible for the production of dopamine, a chemical that, among other roles, helps our muscles move. Although most patients are over the age of 60 at the time of diagnosis, 10% are only between 21 and 50, which is referred to as young-onset Parkinson’s. Researchers from Cedars-Sinai have been investigating such patients, and now have reason to believe that they were already born with problematic brain cells.
In this study, the researchers took blood cells from young-onset patients and converted them into a type of stem cell called induced pluripotent stem cells, or iPSCs. In embryos, stem cells can grow into any cell type of the body. These iPSCs are special because even though they are generated from adult blood cells, they can now grow into any of the patient’s other cell types, just like the stem cells the patients had while they were embryos. This gives researchers a glimpse into the past: by producing new brain nerve cells from iPSCs, they can see how well the nerve cells worked in very early life.
The results showed that these brain nerve cells did not function correctly. In particular, the cell structures responsible for getting rid of excess proteins were instead allowing a protein called alpha-synuclein to build up. This protein is known to accumulate in Parkinson’s disease, which suggests that in these young-onset patients, brain nerve cells might have had problems as early as birth. This insight may lead to improved screenings and potentially treatments for young-onset patients – understanding a disease is a big first step towards a cure.
Managing Correspondent: Isabella Grabski
Press Articles: Parkinson’s disease may start before birth. Medical Xpress.
Original Scientific Article: iPSC modeling of young-onset Parkinson’s disease reveals a molecular signature of disease and novel therapeutic candidates. Nature Medicine.