Image: Nur Yucer, PhD, a project scientist, and Clive Svendsen, PhD, director of the Cedars-Sinai Board of Governors Regenerative Medicine Institute and Professor of Biomedical Sciences and Medicine at Cedars-Sinai. Photo by Cedars-Sinai.

Parkinson's disease is a neurodegenerative disorder that affects predominately dopamine-producing neurons in the brain. Nearly one million will be living with Parkinson's disease in the US this year, according to the Parkinson's Foundation. This is more than the number of people diagnosed with multiple sclerosis, muscular dystrophy and Lou Gehrig's diseasecombined.

About 60,000 Americans are diagnosed with Parkinson's disease each year, and more than 10 million people worldwide are living with it. Incidence of Parkinsons disease increases with age, but an estimated 10 percent of people with Parkinson's disease are diagnosed before age 50. This is called young-onset Parkinson's.

Researchers at Cedars-Sinai, led by Clive Svendsen, PhD, director of the Cedars-Sinai Board of Governors Regenerative Medicine Institute and Professor of Biomedical Sciences and Medicine at Cedars-Sinai, reported in a study published in Nature Medicine that they found that patients who develop young-onset Parkinsons disease may have been born with dysfunctional brain cells that go undetected for decades.

The research team generated special stem cells, known as induced pluripotent stem cells (iPSCs), from cells of patients suffering from young-onset Parkinsons disease. These iPSCswhich can produce any cell type of the human body, all genetically identical to the patients own cellswere used to produce dopamine neurons from each patient to analyze their functions.

Two key abnormalities were observed in these neurons:

- Dr. Clive Svendsen

After testing a number of drugs on the abnormal dopamine neurons, the researchers discovered that a drug called PEP005 (ingenol mebutate) reduced the elevated levels of alpha-synuclein in both the dopamine neurons in the dish and in laboratory mice. A gel formulation of PEP005 is marketed by LEO Pharma as Picato and is FDA-approved for the treatment of actinic keratosis, a scaly skin patch that develops from years of exposure to the sun. According to the Mayo Clinic, a small percentage of actinic keratosis lesions can eventually become skin cancer.

Michele Tagliati, PhD, Director of the Movement Disorders Program and Vice Chair and Professor in the Department of Neurology at Cedars-Sinai, said the research team next will study how PEP005 might be delivered to the brain and whether or not the abnormalities found in young-onset Parkinson's patients also exist in other forms of Parkinsons.

- Dr. Michele Tagliati.

Edward Kim is Managing Editor of Equities.com.

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Sources: Equities News, Cedars-Sinai

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