Parkinson’s Disease

 

Parkinson’s disease is a progressive neurodegenerative disease. Parkinson’s causes the nerve cells that generate dopamine to degenerate and die, leaving patients with limited muscle control.

Parkinson's disease results from the accelerated loss of dopamine neurons in the brain. These neurons control the activity of the circuits that initiate movement. Once these neurons fail, it becomes impossible to initiate movement. The brain does not replace dopaminergic neurons and, prior to 2002, there was no proven way to generate replacement neurons other than isolating them from human fetal tissue.

Parkinson's disease is a slow disease leading to years, often a decade or more, of declining quality of life. The disease is lethal. It affects more than one million individuals in the US alone. There is no cure.

 

Human and Social Costs

  • Afflicts more than 1 million people in the United States, with over 60,000 new cases being diagnosed each year
  • 2nd most common neurological disorder, and the No.14 leading cause of death in the U.S.
  • Studies suggest average American has a 1 in 100 risk of developing Parkinson’s over his or her lifetime

 

Potential for Cures

Regenerative medicine is the most promising opportunity for curing or dramatically reducing the impact of Parkinson’s and other chronic diseases –  programs are investigating the use of neural stem cells, mesenchymal cells, or bone marrow derived multipotent stem cells to repair, regenerate or replace damaged or injured neurons.

A significant reason for drug failure when transitioning from animal to human models is the subtle variations in the genetic make up that defines mice and humans.  Drugs that work in mice have different effects in humans, sometimes to unpredictable consequence. These screening paradigms are critical for all phases of discovery, validation and development.  Embryonic stem cells are a renewable laboratory resource capable of producing authentic human midbrain dopamine neurons in numbers useful for high throughput drug screening.

Stem cell technologies promise significant advances that are not available in any other context or form.

 

Recent Advances

Scientists at the New York Stem Cell Foundation modified the somatic cell nuclear transfer (SCNT) technique, combining the DNA of an adult human cell with the genetic material of an egg — rather than replacing the egg's DNA. Next, the researchers hope to find a way to silence or eliminate the extra set of DNA. The process is promising because it can potentially yield stem cells — which may one day treat diseases such as spinal cord injury and Parkinson's. [ TIME: Top 10 Medical Breakthroughs, Dec. 7, 2011 ]

 

A new method of synthesizing dopamine-producing neurons, the predominant type of brain cell destroyed in Parkinson's, offers hope for creating cell-replacement therapies that reverse the damage. The finding brings researchers a step closer to testing a stem-cell-derived therapy in patients with this disorder.  Cells seemed to survive and function and the cell source is one can easily scale up.   [ Read more about Parkinson’s disease and regenerative medicine.