FEB 13, 2020 7:40 PM PST

Study of Early-Onset Parkinson's Reveals Potential Therapeutic

WRITTEN BY: Carmen Leitch

Around 500,000 Americans are diagnosed with Parkinson's disease every year, and the rate of the disease is rising. Symptoms include tremors, rigid muscles, slow movement, and loss of balance, which all get worse over time. The neurodegenerative disorder happens when neurons in the brain that make a signaling molecule called dopamine start malfunctioning or dying. The reason for that neuronal dysfunction and death is still unknown.

Image credit: Pixabay

New research reported in Nature Medicine has focused on the ten percent of Parkinson's patients that are diagnosed from the ages of 21 to 50, which is younger than typical patients, who tend to be diagnosed when they are over 60.

"Young-onset Parkinson's is especially heartbreaking because it strikes people at the prime of life," said study co-author 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."

The researchers applied the Nobel-prize winning technique for generating stem cells from adult blood cells for this work. They obtained the cells from the early-onset Parkinson's patients, then used the patient-derived induced pluripotent stem cells they created to make neurons that produce dopamine. They were then able to study these patient-derived cells in the lab to assess their physiology.

"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," noted the senior author of the study Clive Svendsen, Ph.D., director of the Cedars-Sinai Board of Governors Regenerative Medicine Institute and a professor of Biomedical Sciences and Medicine at Cedars-Sinai.

Two critical cellular abnormalities were identified by the scientists; a protein called alpha-synuclein was building up, which is similar to what's seen in Parkinson's disease, and cellular recycling centers known as lysosomes were not functioning properly. This lysosomal dysfunction may be causing alpha-synuclein accumulation.

"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 researchers also tested drugs using the cells they engineered to see if any could stop or reverse the dysfunctions. One drug called PEP005 that is already used to treat skin precancers was able to lower the high alpha-synuclein levels in the cells as well as in a mouse model.

The team wants to learn more about how this drug might be used to prevent or treat early-onset Parkinson's disease, and whether the abnormalities they identified hold true for other types of Parkinson's.

Sources: Science Daily via Cedars-Sinai Medical Center, Nature Medicine

About the Author
  • Experienced research scientist and technical expert with authorships on over 30 peer-reviewed publications, traveler to over 70 countries, published photographer and internationally-exhibited painter, volunteer trained in disaster-response, CPR and DV counseling.
You May Also Like
SEP 14, 2020
Health & Medicine
Direct Amplification: Rapid, Extraction-Free RT-qPCR Results
SEP 14, 2020
Direct Amplification: Rapid, Extraction-Free RT-qPCR Results
As the SARS-CoV-2 pandemic continues to rage across the United States and around the globe, the demand for COVID-19 test ...
SEP 13, 2020
Genetics & Genomics
Men and Women Express Many Genes at Different Levels
SEP 13, 2020
Men and Women Express Many Genes at Different Levels
Most humans carry the same genes in their genome, but how are genes expressed differently in men and women?
SEP 14, 2020
Microbiology
The Immune System Can Kill HIV with a Helper Molecule
SEP 14, 2020
The Immune System Can Kill HIV with a Helper Molecule
HIV attacks the human immune system's CD4 cells, a major player in the body's defense against pathogens.
OCT 01, 2020
Cell & Molecular Biology
Signaling Waves Help Guide Cells to Heal Wounds
OCT 01, 2020
Signaling Waves Help Guide Cells to Heal Wounds
During development and throughout our lives, cells have to 'know' where to go to form the right structures and properly ...
OCT 26, 2020
Cancer
Investigating the Receptor Protein FPR1 in Brain Cancer
OCT 26, 2020
Investigating the Receptor Protein FPR1 in Brain Cancer
Amongst the more common targets for cancer therapies are cell surface receptors. These receptors are proteins – us ...
NOV 02, 2020
Cell & Molecular Biology
Catching Cells in the Act of Self-Repair
NOV 02, 2020
Catching Cells in the Act of Self-Repair
Cells have to be flexible and move with each other and our bodies. When cells get overstretched, they have to be able to ...
Loading Comments...