Providing resources and ideas for cures for Parkinson's disease:


Parkinson's Disease Research and Development:

There is a lot of research on mice or test hosts which has not reached the level required for FDA human drug trials, but we have found to be noteworthy as it shows promise for future drug development. This research is the domain of university labs and sponsored research in lab rats and controlled laboratory environments. While research on rats has offerred much hope and mice and humans share 95% of the same genes, not all treatments in mice translate to humans, in fact 90% of drugs that show benefit in laboratory animals fail in human trials with only 9.6% making it to market (ref). This has led to the development of transgenic mice who have had human DNA spliced into the mouse DNA so that experiments are more representative of human responses.

Targeting Mitochondria to Slow the Loss of Brain Cells in Parkinson's:

This approach seeks to slow the progression of Parkinson's disease by targeting problems with energy-producing mitochondria. It is hoped that a molecule can be found to protect mitochondria within dopamine-producing cells.

This research is being performed by:

Targeting Nurr1 Protein:

The molecular pair: Prostaglandin E1 (PGE1), a hormone and Prostaglandin A1 (PGA1), have been found to bind to Nurr1, a class of proteins involved in the development and maintenance of dopamine in the brain. Their binding causes Nurr1 to be activated, resulting in a marked increase in dopamine production while preventing dopamine-producing brain cells from dying. Treatment of Parkinson's mice models showed significant improvements in their motor functions.
Also see inflammation and Nurr1

This research performed by: Nanyang Technological University (NTU), Singapore and Harvard University. Research is continuing as a funded study by the MJF Foundation.

Growth Factors:

There has been some interesting developments in the use of Glial Cell Line Derived Neurotrophic Factor (GDNF), a naturally occurring protein found in our brains, to regenerate dopamine producing neuron cells in patients with Parkinson's to reverse their condition. The experimental procedure required robot assisted brain surgery to install a Convection-Enhanced Delivery (CED) drug delivery pump and four tubes, to supply GDNF to the damaged area of the brain over a period of months. The study was performed at Southmead Hospital, North Bristol UK in affiliation with the Bristol Medical School, University of Bristol, on human patients and funded by Parkinson's UK. The result was 95% meaningful clinical improvement in one or more of the core outcome measures affected by Parkinson's, for the group receiving GDNF for 80 weeks.

Reference: Extended Treatment with Glial Cell Line-Derived Neurotrophic Factor in Parkinson’s Disease (2019) DOI: 10.3233/JPD-191576

Gene Therapy:

Chemistry - Creative Commons