August 2011 Monthly Message
Parkinson's disease and Parkinsonism: Are they the same thing? 
Federal Update: What's Going on in Washington 
Branded vs. Generic Medications in Parkinson's Disease: Does it Matter? 
Hettie Molvang is a Support Group Champion 
The Future of Translational Research at the National Institutes of Health:
NIH Director Francis Collins Releases Commentary Paper about New Research Center 
Parkinson's Action Network Welcomes Former Congressman John Spratt to its Board of Directors 
PAN joins the Alliance for Regenerative Medicine 
Join us for the 2011 Morris K. Udall Awards Dinner 
PAN CEO Amy Comstock Rick Featured on Comcast Newsmakers 
Parkinson's in the Media 
Parkinson’s disease and Parkinsonism: Are they the same?
Parkinson’s Action Network (PAN) CEO Amy Comstock Rick recently gave a presentation about Parkinson’s advocacy to the Board of Directors of CurePSP , an organization devoted to raising awareness about progressive supranuclear palsy  (PSP), corticobasal degeneration  (CBD), multiple system atrophy  (MSA, also known as Shy-Drager syndrome), and other rare neurodegenerative disorders  that are often classified as “Parkinsonsisms.”
“At CurePSP, we fund research that we hope will someday unlock the mystery behind the development of PSP, CBD, MSA and other rare, related brain disorders,” said Dr. Richard Gordon Zyne, President-CEO of CurePSP. “Because the patient population for these diseases is so small in relation to others and because PSP, CBD, and MSA progress more rapidly over 7-9 years on average, it’s important that we band together to have a greater impact both in terms of education and awareness to secure funding for research. We are thrilled to be able to work more closely with the Parkinson’s Action Network so that our patients’ voices are heard in the bigger fight for research funding and supportive policies.”
According to CurePSP, five or six out of every 100,000 people will develop PSP; and, it is estimated that a similar number of people in America have MSA. CBD is very rare: only 500-750 have ever been diagnosed with CBD in the U.S., but it is believed it may affect up to 2,000-3,000 people.
PAN has been hearing from its advocates across the country that there are people with “Parkinsonisms” in their local support groups and who are active advocates, so we wanted to provide an overview of the similarities and differences between Parkinson’s disease and Parkinsonisms. We are excited to begin working with CurePSP and their advocates on funding and policy issues to further strengthen our unified voice in Washington.
Our thanks to Dr. Dennis Dickson, neuro-pathologist and researcher in the Neuropathology Laboratory at the Mayo Clinic in Jacksonville, Florida, who was the primary source for this information.
What is a “Parkinsonism?”
Parkinsonism is the clinical definition of a variety of different underlying pathologies that can cause Parkinson’s-like symptoms such as slowing of movement, tremor, rigidity or stiffness, and balance problems. There are a number of disorders that can produce the symptoms referred to as Parkinsonisms; Parkinson’s disease is just one of them.
So, what’s the difference between being diagnosed with Parkinson’s disease and having what are known as Parkinsonisms? According to Dr. Dennis Dickson, a neuro-pathologist and researcher in the Neuropathology Laboratory at the Mayo Clinic in Jacksonville, Florida, the typical Parkinson’s patient has Lewy bodies (aggregates of a protein called alpha-synuclein) in the brain’s neurons. When that patient is given dopamine replacement therapy (e.g., Sinemet), those symptoms go away.
By contrast, Parkinsonisms – also sometimes referred to as atypical Parkinson’s –have features in addition to typical Parkinson’s disease symptoms, and those symptoms do not respond to dopamine replacement therapy.
Parkinsonisms fall into two classes:
One type – of which MSA is an example -- that produces alpha-synuclein clusters in the brain’s glial cells (the cells that protect the neurons in the brain) instead of in the neurons, as is the case with Parkinson’s disease; and,
A second type that involves defective tau proteins which act as a binder and stabilizer of microtubules of cells (microtubules are like the skeleton of neurons). A dysfunction in the tau proteins is what causes PSP and CBD.
So, why do PSP, CBD, and MSA– despite their different origins in the brain – get grouped together under the category of Parkinsonisms? According to Dr. Dickson, “They’re Parkinsonisms because they have overlapping clinical symptoms of typical Parkinson’s disease, and because dopamine-producing cells in the brain are affected in all these disorders.”
Therein lies the distinction: if a person’s symptoms do not respond to dopamine replacement therapy, or only do so for a very short period of time, then, it is believed they do not have typical Parkinson’s, and instead may have PSP, CBD, or MSA.
Dickson believes physicians have been “really good” at getting the diagnosis right when it comes to PSP, CBD, and MSA. He says, “It’s not 100%, though. Movement disorder experts get it right 80-90% of the time. Neurologists get it right maybe 75% of the time. Those doctors who’ve seen a lot of PSP patients get it right 90% or more of the time. Sadly, CBD only has a 50% diagnosis rate.”
Just as with Parkinson’s disease, there are no clear, obvious biomarkers or tests for PSP, CBD, and MSA. Right now, the only way to diagnose a Parkinsonism is to assess response to dopamine replacement therapy. And, just like Parkinson’s disease, the only way to diagnose with absolute certainty PSP, CBD, or MSA is after an autopsy of the brain.
What are some other symptoms associated with Parkinsonisms?
When it comes to looking at the spectrum of symptoms, there are some differences that might help a clinician know if a patient has a particular form of Parkinsonism:
Is it possible that Parkinson’s is not only a disease, but rather a broad spectrum of disorders?
Dr. Dickson says “yes.” He explained that for any category of disease, researchers do what’s called “lumping and splitting,” meaning you can split diseases into various subtypes – in fact, Dickson added, “researchers and pathologists already see differences in PSPs, so we know there are various types of PSP.”
With regard to lumping, that means researchers drill down to the molecular level and study what molecular links there might be between Parkinson’s disease and Parkinsonisms. That’s why these diseases are often grouped, or lumped, together.
What treatments are available to people with PSP, CBD, or MSA?
Right now, the only treatment is first to see if the patient responds to dopamine replacement therapy. If not, then the clinician knows it’s not Parkinson’s disease. According to Dickson, it’s only non-pharmacologic treatments that might be helpful in managing Parkinsonism disorders such as PSP and MSA. These include:
But, to reiterate, these are all symptomatic treatments. Just as there are no disease-modifying therapies for Parkinson’s disease, there also are none for Parkinsonisms.
“New drugs are now being developed, which are currently in clinical trials, and may have an effect on the prevention of tau protein aggregation,” added Dr. Zyne. “If these drugs prove to be successful, they may have a significant impact on the lives of people with Parkinsonisms.”
What causes Parkinsonisms, and what research is being done?
Like Parkinson’s disease, it is believed that there are both genetic and environmental risk factors for Parkinsonisms, but no one is certain. While government funding for research for Parkinsonisms has been very modest over the years, CurePSP has been the leader in raising new funds for innovative research projects – including a genetics whole genome study which has implicated several new gene loci. Since 1997, when CurePSP began its research program, more than $10 million has been raised for various basic research and clinical projects.
What is PAN doing to help?
The Parkinson’s Action Network strives to maintain a balance between advocating in support of policies and funding to support people who already have Parkinson’s disease, as well as funding and policies to shape a brighter future. There certainly are research overlaps between Parkinson’s disease and Parkinsonisms, as well as overlap in policies, such as Social Security Disability Insurance and Medicare, that impact both populations.
“Is it possible that as we learn more about Parkinson’s disease, what will really happen is that the list of Parkinsonisms will just get longer and longer? We don’t know,” said Amy Comstock Rick, CEO of the Parkinson’s Action Network. “But what is important to us is educating and raising awareness so that we can secure federal funding for research – because we believe studies around Parkinson’s disease and Parkinsonisms can inform each other and get us closer to better treatments and, hopefully, cures. It’s important to have federal funding and NIH oversight for this research, because that kind of focus helps engage private-sector research – and we need both.”
Many PAN advocates coordinate or attend support group meetings attended by people who have Parkinsonisms, and because diseases like PSP, CBD, and MSA are not as well-known and have fewer people with the disease, there aren’t specific, dedicated support networks available. PAN looks forward to reaching out to this community through CurePSP to be even more inclusive in our advocacy efforts.
“Just like we welcome people with MS and ALS into our Parkinson’s support groups, we certainly welcome people with Parkinsonisms, as well,” said Michelle Lane, PAN’s Louisiana State Coordinator. “Especially in rural areas where resources are scarce, it’s important to work together and support each other.”
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