How will we create a world without Parkinson’s? On June 1, the Parkinson’s Foundation, is asking scientists to answer this question at our cutting-edge scientific event, “World Without Parkinson’s.” Our team predicts that scientific progress in the next 20 years outpace progress of the last 200 since James Parkinson published, “An Essay on the Shaking Palsy.”
How will we get there? Hear answers from one of our speakers Laura A. Volpicelli-Daley, Ph.D., of the University of Alabama School of Medicine.
Q. Why are you a part of this event? What excites you about it?
A. I am honored to be part of this event. Even though this disease was described thousands of years ago in ancient Sanskrit texts from India, 200 years ago, Dr. James Parkinson described and defined the disease. A little over 100 years ago, Dr. Franz Heinrich Lewy described one of the primary pathologic characteristics of PD, clumps of alpha-synuclein called Lewy bodies and Lewy neurites. Twenty years ago it was discovered that alpha-synuclein plays a key role in PD.
Looking at these developments, it’s clear that the pace of research is progressing.
I am excited that my work on alpha-synuclein could somehow contribute to stopping PD. I am also honored to be speaking alongside outstanding scientists who are also studying how to stop the disease and determining novel ways to diagnose PD early on so we can stop it in its tracks.
Q. You will be discussing alpha-synuclein and how you think the protein might help us to end Parkinson’s. Can you share a preview?
A. I believe that alpha-synuclein, a protein, plays a key role in the progression of Parkinson’s disease and if we better understand it, may help us to stop the disease.
Current treatments for Parkinson’s disease such as levodopa, deep brain stimulation and dopamine agonists are very effective at alleviating the motor symptoms of PD such as rigidity, slowness of movement, and tremor. However, we currently have no way of stopping the progression of the disease. Symptoms such as gait disturbance, falls and dementia are a major source of disability in the advanced stages of the disease. People with Parkinson’s often say they can live with some of their minor motor symptoms if we can stop disease progression.
Scientists have looked at alpha-synuclein as a possible culprit for a number of reasons. First, in the Parkinson’s brain, blobs of protein that characterize the disease are made mostly of the alpha-synuclein protein. In addition, a very small percentage of people with Parkinson’s have either mutations in the gene alpha-synuclein or extra copies, which causes their disease.
Not to mention, the appearance of blobs or “aggregates” of alpha-synuclein in particular brain areas (called Lewy bodies) correlate with appearance of PD symptoms: in the gut, they correlate with the appearance of gastrointestinal symptoms, in the olfactory bulb, they correlate with loss of smell; and in the cortex (a brain area important for cognition) correlates with dementia.
I think if we can prevent formation of these aggregates of alpha-synuclein from forming or if we can somehow clear them from the brain, we can prevent disease progression.
Q. What do you hope to see as an outcome of the World Without Parkinson’s event?
A. I hope that people with PD and caregivers have renewed hope that we can find ways to prevent the progression of this disease. I also hope that the community realizes that careful, controlled, scientific research is absolutely necessary and that it can be really difficult and seem to be slow sometimes. But, researchers are working diligently and are making major advances to understand the disease and are discovering new treatment strategies.
Q. Why should the PD community be hopeful about science?
There are several reasons to be hopeful. First, there has been a lot of progress made in identifying novel strategies for Parkinson’s therapies. Second, diagnosis of PD is becoming more precise with the discovery of biomarkers, molecular signatures in the blood, urine or cerebrospinal fluid. In fact, novel imaging techniques are also being developed to help diagnose PD and understand disease progression.
Lastly, scientists and pharmaceutical companies are developing new compounds that show great promise for treating PD and are even developing “personalized medicine” strategies for treating subtypes of PD- such as treating people who have a mutation in a certain gene like LRRK2 or GBA, as an example. Promising therapeutics targeting alpha-synuclein are also in early clinical trials which may help prevent disease progression.
Are you a scientist or health innovator? Learn about World Without Parkinson’s by visiting www.parkinsonsfoundation.org/wwp