Study Suggests that Parkinson’s may be Present in the Brain Before Birth
Wednesday, 29th January 2020

A new study by Cedars-Sinai research suggests that if you were diagnosed with Parkinson’s before age 50, you might have been born with it.

The study was reported in the journal Nature Medicine this week where researchers used a specific type of stem cell called iPSC, from the blood cells of patients with Young Onset Parkinson’s. This process takes adult cells “back in time” to a primitive embryonic state. Acting as the body’s master cells, the iPSC can go on to develop into any cell type of the human body, while remaining genetically identical to the patient’s own cells.

“Our technique gave us a window back in time to see how well the dopamine neurons might have functioned from the very start of a patient’s life,” said Clive Svendsen, PhD, director of the Cedars-Sinai Board of Governors Regenerative Medicine Institute and professor of Biomedical Sciences and Medicine at Cedars-Sinai.

“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.”

“Young-onset Parkinson’s is especially heartbreaking because it strikes people at the prime of life,” said Michele Tagliati, MD, director of the Movement Disorders Program, vice chair and 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.” Tagliati was a co-author of the study.

The investigators also used their iPSC model to test a number of drugs that might reverse the abnormalities they had observed. They found that that one drug, PEP005, which is already approved by the Food and Drug Administration for treating precancers of the skin, reduced the elevated levels of alpha-synuclein in both the dopamine neurons in the dish and in laboratory mice.

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.