People who develop Parkinson’s disease before age 50 may have been born with disordered brain cells that went undetected for decades, according to new Cedars-Sinai research. The research points to a drug that potentially might help correct these disease processes.
Parkinson’s occurs when brain neurons that make dopamine, a substance that helps coordinate muscle movement, become impaired or die. Symptoms, which get worse over time, include slowness of movement, rigid muscles, tremors and loss of balance. In most cases, the exact cause of neuron failure is unclear, and there is no known cure.
At least 500,000 people in the U.S. are diagnosed with Parkinson’s each year, and the incidence is rising. Although most patients are 60 or older when they are diagnosed, about 10% are between 21 and 50 years old.
“Young-onset Parkinson’s is especially heartbreaking because it strikes people at the prime of life,” said Michele Tagliati, MD, professor in the Department of Neurology at Cedars-Sinai. “This exciting new research provides hope that one day we may be able to detect and take early action to prevent this disease in at-risk individuals.”
“What we are seeing using this new model are the very first signs of young-onset Parkinson’s,” said Svendsen. “It appears that dopamine neurons in these individuals may continue to mishandle alpha-synuclein over a period of 20 or 30 years, causing Parkinson’s symptoms to emerge.”
The investigators also used their model to test a number of drugs that might reverse the abnormalities they had observed. They found that one drug, PEP005, which is already approved by the Food and Drug Administration for treating precancers of the skin, may be effective.
The drug also countered another abnormality they found in the patients’ dopamine neurons — elevated levels of an active version of an enzyme called protein kinase C — although the role of this enzyme version in Parkinson’s is not clear.
For the next steps, Tagliati said the team plans to investigate how PEP005, currently available in gel form, might be delivered to the brain to potentially treat or prevent young-onset Parkinson’s. The team also plans more research to determine whether the abnormalities the study found in neurons of young-onset Parkinson’s patients also exist in other forms of Parkinson’s.