A new therapy for Parkinson’s disease has now entered testing in human patients. The Austrian company AFFiRiS A.G. launched a two year long clinical trial of a vaccine designed to stop Parkinson’s disease progression. In this month’s What’s Hot Column we will explore the rationale for such an approach, and what patients need to know.
Parkinson’s disease is a neurodegenerative disorder associated with deposition of a brain protein known as alpha-synuclein. This protein clumps and seems to spread throughout the brain as Parkinson’s disease progresses. Many experts believe that much of the damage in Parkinson’s disease traces to the failure for the brain to process and to clear these proteins.
The idea underpinning the Parkinson’s vaccine is simple. Patients will receive four injections with the hope that these injections will stimulate an immune system response against alpha-synuclein, and that antibodies will attack these brain proteins and ultimately clear them. Thirty-two human Parkinson’s disease patients will be part of a two year safety and tolerability study which has been named the PD01A project. The study is now underway in Vienna, and it aims to modify disease progression in Parkinson’s disease.
It is important to keep in mind that not all experts believe that removal of these brain proteins will result in clinically meaningful changes and/or disease modification. Additionally, we must keep in mind that one highly publicized attempt to remove the Tau protein in Alzheimer’s patients led to serious safety concerns, and termination of a vaccine study known as AN1792 because several patients developed a meningoencephalitis.
What patients need to know about the vaccine is that it is still in the very early stages of testing, but that the idea is novel and the approach is promising. Safety, tolerability, and clinical efficacy will need to be demonstrated before the vaccine can move to the next phase of clinical testing. Our hope is that clearance of the Parkinson’s associated brain proteins will translate into disease modification. A similar approach is also being tested in other diseases such as Alzheimer's disease, diabetes, and atherosclerosis.
You can find out more about our National Medical Director, Dr. Michael S. Okun, by also visiting the Center of Excellence, University of Florida Health Center for Movement Disorders and Neurorestoration. Dr. Okun is also the author of the Amazon #1 Parkinson's Best Seller 10 Secrets to a Happier Life and 10 Breakthrough Therapies for Parkinson's Disease.