All people with Parkinson’s know that in 1817 Dr. James Parkinson noticed tremor, rigidity, slow movements and stooped gait in some of his fellow Londoners which caused him to write his “Essay on the Shaking Palsy”. Being a good doctor and believing in the power of medicine he predicted “Although, at present, uninformed as to the precise nature of the disease, still it ought not to be considered as one against which there exists no countervailing remedy. On the contrary, there appears to be sufficient reason for hoping that some remedial process may ere long be discovered, by which, at least, the progress of the disease may be stopped.” It’s been 200 years and we are still uninformed as to the precise nature of the disease, there still is no cure and nothing stops the progression of the disease. The best thing you can do is be healthy, take you meds as prescribed, exercise, and live your life.
I wanted to write about the most promising advancements in the fight to prevent and cure Parkinson’s but realize that I am unable to. The first reason is because there is so much research going on that I can’t decide what is the most promising. Another reason is that a lot of it is beyond my technical understanding. “Can gene delivery of microRNA’s 7 and 153 protect the nigrostriatal system from MPTP toxicity.” Instead, I’ve chosen five areas to highlight: early detection, improved medicine delivery systems, stem cells, gene research, and alpha-synuclein. Most research is directed at finding the causes of PD and preventing, curing, and stoping the progression of the disease. You can find other areas. My neurologist said there is so much top notch research taking place that he believes there will be a major breakthrough within 10 years. Let’s hope he isn’t off by as much as Dr. Parkinson was 200 years ago.
I started writing this post a while ago and it is probably about 75% accurate at this time. I was diagnosed in 2012 and have witnessed a lot of changes in PD research since then. Some things my first neurologist told me are no longer believed to be true so read the following with caution and be aware of the use by or best by date. A final thought. This has been a century of science and amazing medical miracles causing us to expect cures for all diseases but what if PD cannot be prevented, the progression cannot be controlled, and maybe there is no cure.
Early Detection: Parkinson’s symptoms do not usually appear until about 80% of the dopamine producing cells are gone. The presence of symptoms is what makes diagnosis possible. Treatment options therefore cannot be preventive and are limited to managing the disease after the damage is done. Researchers are looking for early indicators of PD, possibly through blood or urine tests, so they can develop therapies that can stop the disease before symptoms develop.
Medicine Delivery Systems: Drugs and DBS are how we treat PD now. The drugs have serious side effects and DBS is invasive. Sinemet (levodopa and carbidopa) is still king. A concern regarding levodopa is that it is believed to cause serious side effects after long-term use. The pulsating effect of taking a number of levodopa pills every day which have an initial strong impact on the brain followed by a short half life is thought to be one cause of the problem. A dermal patch, a subcutaneous pump, and controlled release tablets have been developed to deliver levodopa in a continuous regulated way without jolting the brain. Providing a more continuous and regulated supply of dopamine to the brain may result in improved control of PD symptoms and lessen side effects.
Currently most PD medications are taken orally. However, it takes time for oral medications to be absorbed by the body before they start to work. An inhaled powder form of levodopa, Inbrija, passed clinical trials and is available. Self administered with an inhaler it reaches the brain faster than orally administered levodopa and provides rapid improvement of motor function to significantly reduce OFF time.
Stem Cells: Stem cell treatment is designed to target damaged dopamine neurons and help create new dopamine producing neurons. Unfortunately PD stem cell therapy clinical trials have not been productive so far but in 2016 a California based company, International Stem Cell Corporation (ISCO), was given permission to start clinical trials on humans for the first time. Doctors implanted replacement brain cells, called neural precursor cells, into the brains of 12 people with moderate to severe PD. It was hoped these cells will finish maturing into the kind of neurons which are destroyed by PD. 12 participants were injected with neural stem cells directly into the striatum and substantial migration ares of the brain and monitored for a year to see how their brains and bodies reacted with the main goal of assuring that the transplants were safe and well tolerated. The stem cells were well tolerated and some improvement in symptoms was noted so a second group of 12 PWP was injected in 2018.
Gene Research: There are two areas of PD gene research: the identification of genes associated with PD and the manipulation of genes to treat PD. Researchers are identifying the genes that cause and contribute to PD. This will allow for the early identification and treatment of those at risk with customized targeting of individual genes associated with PD.
A good and understandable explanation of Parkinson’s gene therapy can be found at http://pdcenter.neurology.ucsf.edu/professionals-guide/gene-therapy-pd
Alpha-synuclein Research: The Michael J. Fox Foundation has donated more than $50 Million to alpha-synuclein research. The alpha-synuclein protein is a major component of Lewy bodies, toxic clumps of protein that damage dopamine neurons. Researchers believe that PD is caused by an increase of synuclein in the cell due to genetic causes. They are devising strategies to lessen the amount of synuclein in the cell which may prevent and possibly reverse PD. There are currently at least six sanctioned clinical alpha-synuclein trails under way utilizing vaccines that bind to alpha-synuclein and then clear it from the brain.
A good, but slightly technical, explanation of the importance of alpha-synuclein research and its application to PD can be found at:
