Category Archives: MSA

Caution against unproven stem cell therapies (Parkinson’s, MSA, etc)

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This may be of interest to everyone in our support group, regardless of diagnosis.

The Movement Disorder Society (MDS) issued a position paper this week on the use of stem cell therapies for Parkinson’s Disease (PD).  The lay summary of the position paper concludes as follows:

“[Until] such treatments are proven to be of benefit and published in recognized scientific journals that objectively scrutinize their procedures, the Society encourages patients to participate only in cell therapy studies that are part of a research program affiliated with a recognized academic institution.”

A section of the paper was about stem cell treatment for “parkinsonian syndromes.” This term applies to PD, MSA, PSP, CBD, and DLB.  That section reads:

“Present publicly offered stem cell therapies for clinical use
There are several organizations world-wide which offer stem cell therapy for clinical application in patients. There is no detailed scientific information available on the outcome of these therapies. In a recent case series, data from patients with parkinsonism who underwent these procedures were collected retrospectively(31). … The report describes 17 patients with Parkinsonian syndromes who received intrathecal application of autologous unsorted bone marrow cells. There were no changes in motor function, activities of daily living, global clinical impression or antiparkinsonian medication after a median observation period of 10 months. Two patients (12%) reported a worsening of Parkinsonian symptoms, but the intervention was otherwise safe and well tolerated. Intrathecal application of autologous bone marrow cells in such uncontrolled conditions did not produce clinical benefit in these patients.”

I’ve copied the abstract to reference #31 below.

Of the 17 patients studied, 7 have a clinical diagnosis of PD, 7 have a clinical diagnosis of MSA, and 3 have other clinical diagnoses.  This break-down fits with my experience as well:  I’ve heard more about MSA patients trying out the various stem cell treatments available around the world than I’ve heard about those with PSP, CBD, or DLB diagnoses.  The reason may be that those with MSA are younger than those with the other diagnoses, on average, so there may be more desperation on the part of the patient and family.

We have had at least one person with MSA in our local support group who went to China for stem cell treatment.  And the medical advisor to our support group, Dr. Neng Huang, also reports that he knows of someone who went to China for stem cell treatment.  Neither patient seemed to have benefitted from the treatment.

I haven’t read the full article but presumably the 7 MSA patients don’t include those who participated in a South Korean study of autologous bone marrow cells (since clinical data was available in that case).

The MDS position paper can be found here:

www.movementdisorders.org/about/committees/stem-cell.php

There is a short lay summary in the position paper, and then a scientific summary that is three times as long.  I think most of you won’t have a problem reading the scientific summary!

Finally, here’s a link to the Movement Disorder Society’s short press release about the position paper:

www.movementdisorders.org/about/newsroom/release.php?contentid=1157

Robin

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Abstract

Movement Disorders. 2012 Oct;27(12):1552-5.  Epub 2012 Feb 23.

Intrathecal application of autologous bone marrow cell preparations in Parkinsonian syndromes.

Storch A, Csoti I, Eggert K, Henriksen T, Plate A, Lorrain M, Oertel WH, Antonini A.
Division of Neurodegenerative Diseases, Department of Neurology, Dresden University of Technology, Dresden, Germany.

BACKGROUND:
A growing number of patients is treated with intrathecal application of autologous bone marrow cells (aBMCs), but clinical data are completely lacking in movement disorders. We provide first clinical data on efficacy and safety of this highly experimental treatment approach in parkinsonian syndromes.

METHODS:
Retrospective data collection from patients with parkinsonism who spontaneously sought cell treatment. The application procedure was neither recommended nor performed by the authors.

RESULTS:
We report 17 patients with parkinsonian syndromes (Parkinson’s disease [PD], n = 7; multiple system atrophy [MSA], n = 7; various, n = 3) who received intrathecal application of aBMCs. We did not observe any changes in motor function, activities of daily living, global clinical impression, or antiparkinsonian medication after a median observation period of 10 months. Two patients reported a worsening of parkinsonian symptoms, but the intervention was otherwise safe and well-tolerated.

CONCLUSIONS:
Intrathecal application of aBMCs in uncontrolled conditions produces no clinical benefit in parkinsonian syndromes.

Copyright 2012 Movement Disorder Society.

PubMed ID#: 22362657

Dr. Wenning on MSA (informal video, June 2013, Australia)

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Gregor Wenning, MD, is an MSA specialist who lives in Austria.  He met around a table with MSA families in Sydney, Australia in June 2013.  The 80-minute informal discussion and presentation were recorded and are posted on Vimeo:

vimeo.com/relivetheday/review/68744603/81811a9e7d

Note that this is not a formal presentation or a professional video-recording.

The slides haven’t been posted yet but I imagine they will be.  Pam Bower posted the Vimeo link recently to the ShyDrager Yahoo!Group.

At our Sunday caregiver support group meeting, our MSA group leader, Candy Welch, told me she watched the video and thought it was worth watching.  Plus she said that Dr. Wenning is obviously a very kind and caring physician.

Robin

“Many Faces, Same Disease” – MSA Patient Primer (Univ of Florida)

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This MSA primer is posted to a webpage at the University of Florida (Gainesville) Center for Movement Disorders and Neurorestoration.  I’m not sure when it was published.  This is a well-written resource.

The author, who seems to be neurologist Nikolaus McFarland, MD, notes:  “As a multisystem disorder Multiple System Atrophy has many ‘faces’ and results in varied symptoms that require an interdisciplinary approach. Patient care begins with proper diagnosis and then focused, and even specialized, treatment of the various symptoms of MSA.”

Copied below are excerpts from the primer.  See the University of Florida webpage for the full primer:

mdc.mbi.ufl.edu/education/multiple-system-atrophy-primer-many-faces-same-disease

Vera James posted this today to the ShyDrager Yahoo!Group.

Robin

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mdc.mbi.ufl.edu/education/multiple-system-atrophy-primer-many-faces-same-disease

Excerpts from:

Multiple System Atrophy (MSA): Many Faces, Same Disease

A patient primer from the University of Florida Center for Movement Disorders & Neurorestoration

What is MSA?

MSA, or Multiple System Atrophy, is a form of parkinsonism with many features that overlap with those of classic Parkinson disease and make it confusing to diagnose clinically. In fact, early symptoms may appear just like Parkinson disease (PD) and standard treatments, such as carbidopa/levodopa (Sinemet), can initially be helpful only to wane later in effectiveness as the disease progresses. As the disorder’s name alludes, MSA is a multisystem neurodegenerative disease that is characterized by a combination of symptoms including parkinsonism, cerebellar and pyramidal tract signs, and autonomic dysfunction. Parkinsonism is a descriptive term and includes tremor (usually resting), stiffness or rigidity, bradykinesia (slowed movements), and postural and gait instability. Cerebellar signs in contrast refer to problems with coordination and may include tremor with activity, past pointing (when reaching for objects), slurred speech, and an unstable “drunk-like” gait. Autonomic problems are non-motor features that involve failure of the automatic nervous system which controls things like heart rate, blood pressure, bowel, bladder and sexual functions. These are things you don’t often think about and are “automatically” controlled by the brain. In MSA people develop problems with this system and may present with lightheadedness/dizziness (related to changes in position, such as standing, and referred to as orthostatic hypotension), fainting spells, urinary retention or urgency (even incontinence), erectile dysfunction in men, constipation, and abnormal heat/cold intolerance and problems with sweat production. Additional features that are closely linked to MSA (and other parkinsonian syndromes) include REM sleep behavior disorder — characterized by yelling or thrashing about during dream sleep — periodic limb movements or restless legs, and respiratory stridor (harsh, strangled breathing). Importantly, it is possible for these other symptoms to precede motor symptoms by months to years.

What are the “faces” of MSA?

Given the multitude of features in MSA, clinical presentation is varied resulting in many “faces” of the disease. Since its first description in the 1960s the disease has been given different names — Shy-Drager syndrome, striatonigral degeneration (SND), and olivopontocerebellar atrophy (OPCA) — depending on the predominant presenting symptoms. However, it was later discovered that each of these syndromes shared a similar pathology characterized by the presence of abnormal protein deposits in the brain, called glial cytoplasmic inclusions or GCI’s. These inclusions are similar to “Lewy bodies” seen in PD, but unlike PD they are not found in neurons but rather in supporting glial cells. In the brain these glial cells are important for making myelin, a substance that “insulates” nerve fibers. The finding of this common pathological feature regardless of the clinical presentation (or “face” of the disease) led to the terminology used today, multiple system atrophy or MSA. Two clinical variants are generally still distinguished by clinicians based on the predominant presenting symptoms: parkinsonism (MSA-P, also known as Shy Drager or SND) or cerebellar ataxia (MSA-C, or OPCA). Both variants include autonomic dysfunction and as the disease progresses the features of these variants increasingly overlap.

Who does MSA affect?

MSA affects men and women equally and median age of onset is about 58 years-old. Although not as prevalent as PD, MSA affects about 4 per 100,000 individuals. … Disease progression unfortunately is usually more rapid than in PD and reflects a more widespread neurodegeneration in the brain. The combination of autonomic and motor symptoms, particularly if early in onset, can be fraught with more complications and shorter survival. Therefore early disease identification is critical. Referral to a movement disorders neurologist is recommended for proper diagnosis and symptom management.

How is MSA treated?

Treatment of MSA remains largely supportive. About 30-60% of patients respond to typical Parkinson’s medications such as carbidopa/levodopa (Sinemet), and dose trial of up to 1 gram/day of levodopa for a few months is recommended. Benefit seen early in disease often fades though, or becomes fraught with complications. Two major complications include exacerbation of orthostatic symptoms (lightheadedness, dizziness, or fainting on standing) and dyskinesias, or abnormal involuntary movements that often involve the jaw or face. These symptoms can result in limiting the dose of medication tolerated, and thus also the effectiveness of drug treatment. More advanced motor symptoms, such as muscle spasm or fixed postures (dystonia), can be treated with “muscle relaxants” and sometimes by injection of botulinum toxin (i.e., Botox). Deep brain stimulation (DBS), however, is generally not recommended as poor outcomes have been reported. Physical and occupational therapies for gait and balance, range of motion and mobility, and help with activities of daily living are critical and require staff familiar with Parkinsonian patient needs. For those with progressive speech and swallowing issues therapy with a specialist is also highly recommended and includes regular swallow testing. Prevention of falls and aspiration (pneumonia) are major goals as these frequently lead to worsening disability and even death.

Although there is often focus on motor symptoms, the non-motor symptoms of MSA can also be just as disabling. Orthostatic symptoms, dizziness and even fainting, can become very limiting and lead to the wheelchair or recliner-bound patient. Treatment initially begins with “conservative” therapies including increased fluid intake, salt in diet (considering of course any concomitant heart disease), and wearing pressure stockings or binder. If these are not enough, drug treatment may be necessary. Options include “blood volume increasers” (fludrocortisone) or “pressor agents” (e.g., midodrine). Both increase BP thereby reducing episodes of low BP, but the later can also cause excessive high BP usually when lying down. Careful monitoring of BP is needed, avoiding ups and downs, and a happy medium found for each individual. Urinary and bowel symptoms likewise can be treated with select agents depending on the issue. Sleep disturbance can also be successfully treated depending on cause.

PSP on “The Doctors” (3 minutes), CBD and MSA mentioned (7-11-13)

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This email will mostly be of interest to the PSP folks, and maybe of passing interest to the CBD and MSA folks.

Local support group member Phil Myers sent me this info today.

Patricia Richardson, the actress who was on a show called “Home Improvement,” is the national spokesperson for CurePSP.  She was on “The Doctors” today (July 11, 2013).  “The Doctors” had segments today with actresses who played mothers on TV shows.

She talked about PSP, which is a disease her father had.  She mentioned CBD and MSA.  She said that the only person with PSP that we’ve heard of is Dudley Moore, and she said that Johnny Cash had MSA.  (I thought that the MSA community did NOT think Johnny Cash had MSA.)

When describing PSP, she said that it was a tauopathy, like traumatic brain injury that returning vets get and like football players.  I’m not aware that TBI injuries in war zones are connected with the protein tau.  Certainly, chronic traumatic encephalopathy (CTE) is a tauopathy that many athletes have been found to have on post-mortem brain examination.  CTE is caused in some way by repeated head trauma.

There was a graphic that came up when she talked that said 5 out of 100,000 people have PSP.  Ms. Richardson made the point that only one in three are diagnosed with PSP.  PSP is as common as ALS or Lou Gehrig’s Disease but people have heard of ALS.

Here’s a link to “The Doctors” webpage on today’s segment about health secrets from TV moms:

www.thedoctorstv.com/main/show_synopsis/1255?section=synopsis

On that webpage, scroll down to find a summary of info on PSP and a link to three short clips — 90-second PSP overview, 90-second comparison of PSP and Parkinson’s, and 50-second caregiver advice from Patricia Richardson.

I’ve copied the info from “The Doctors” website on PSP below.  (The details on the “synopsis” page were not all discussed in the segment with Patricia Richardson.)

Robin

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www.thedoctorstv.com/main/show_synopsis/1255?section=synopsis

Patricia Richardson on PSP Awareness
As the Home Improvement mom America could relate to, actress Patricia Richardson went from the TV limelight to being a national spokesperson for Cure PSP — an organization combating a rare and often misdiagnosed brain disease that claimed the life of her father and fuels her fight to find a cure.

“He probably had the disease [for] five or six years before we knew he did,” Patricia says. “We’re only really catching 1 in probably 3 people with it.”

PSP, or progressive supranuclear palsy, is a degenerative neurological disorder that causes abnormal movement, similar to that of Parkinson’s disease. People suffering from PSP develop sluggish body movement and stiffness, as well as balance problems and difficulty with eye and neck motility. PSP is caused by an inability to process natural waste from the cells in the brain. Most people can process this waste but for those with PSP, the waste remains in the brain and creates a protein that attacks the brain cells. Oftentimes, people are misdiagnosed with Parkinson’s and prescribed medication. If the medication proves ineffective, it signals that PSP may be the cause, as opposed to other brain diseases.

Approximately 5 people in every 100,000 will develop PSP, but only a third are diagnosed. Unfortunately, an official diagnosis usually occurs after death, when doctors can examine the brain during an autopsy to verify if the proteins that cause PSP are present. The best living diagnosis is if patients are not reacting well to their Parkinson’s medication. PSP usually develops in people in their 50s and 60s — not in the later years. The disease tends to last between five and 10 years, before patients die from related health complications, such as aspirational pneumonia and brain ulcers. Researchers speculate that there may be a genetic component to PSP, but the specific gene has yet to be identified.

(Note:  there’s a short ad before these video clips)

PSP Symptoms:
http://www.thedoctorstv.com/videolib/init/9405
This video is just over 3 minutes.  Caregiving and PSP are first raised about 1:33.  CBD and MSA are mentioned shortly thereafter.

PSP Explained:
http://www.thedoctorstv.com/videolib/init/9406
This clip is about a minute and a half.  It features Dr. Carlos Portera-Cailliau.  PSP is compared to Parkinson’s Disease.

Patricia Richardson’s Advice for Caregivers
http://www.thedoctorstv.com/videolib/init/9407
This clip is about 50 seconds long.  Patricia Richardson advises people to get into a patient advocacy group.  Caregivers are told to take care of themselves.  

“What is atypical parkinsonism and how does it differ from PD?”

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This blog post from the Northwest Parkinson’s Foundation (nwpf.org) is a short overview of atypical parkinsonism disorders.

The author, movement disorder specialist Monique Giroux, MD, uses the term “Lewy body disease,” rather than “Lewy body dementia.”  Multiple system atrophy and progressive supranuclear palsy are included as well but corticobasal degeneration is excluded.  Normal pressure hydrocephalus and Wilson’s disease are included on the list of neurodegenerative disorders that can cause parkinsonism.

The blog post is copied below.

Robin

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nwpf.org/stay-informed/blog/2013/06/what-is-atypical-parkinsonism-and-how-does-it-differ-from-pd/

PD Community Blog
What is atypical parkinsonism and how does it differ from PD?
by Monique L. Giroux, MD, Former Medical Director of NWPF
Northwest Parkinson’s Foundation
Monday, June 24, 2013

Atypical parkinson’s, parkinsonism, and parkinson’s plus are all terms used to describe syndromes that share features similar to Parkinson’s disease but are different conditions. These conditions are described below.

Just like Parkinson’s disease, the diagnosis is often a clinical one relying on an examination by a neurologist knowledgeable in these conditions. Because of this, the diagnosis may not be obvious at your first doctor’s visit and an accurate diagnosis may take time.

Common features of atypical parkinsonism that differentiate it from Parkinson’s disease are:
– Symptoms present on both sides of the body at onset.
– Early cognitive problems.
– Early problems with balance, falls and/or freezing of gait.
– Early problems with autonomic function such as orthostatic hypotension (lightheadedness when standing from low blood pressure.)
– Earlier speech and swallowing problems.
– Faster progression
– Limited improvement with medicine.
– Significant visual problems such as double vision, trouble focusing while reading.

Specific conditions include (note:  this list does not include all disorders)

Neurodegenerative Conditions. These conditions are associated with degeneration or nerve cell loss over time.

* Lewy Body Disease. Cognitive problems, hallucinations and fluctuations in levels of alertness are present within the first year of movement problems. Motor symptoms can otherwise mimic Parkinsons disease.

* Multiple System Atrophy (MSA): Slowness, walking problems, imbalance, and early autonomic nervous system problems (Orthostatic hypotension, constipation, bladder control) predominant in this disorder.

* Progressive Supernuclear [sic] Palsy  (PSP): Slowness, walking problems, imbalance, eye movement problems, speech and thinking problems predominate in this disorder.

* Normal Pressure Hydrocephalus (NPH): Early walking, thinking and bladder control problems predominate in this disorder. Brain MRI reveals enlarged ventricles and therefore is helpful in detecting this condition.

* Wilson’s disease: A genetic condition with personality changes, thinking problems, dystonia and other movement problems.  Brain imaging, blood and urine copper testing can aid in making this diagnosis especially in young people.

Secondary Parkinsonism. These conditions are caused by other problems.

* Drug induced parkinsonism.  Many antipsychotic medicines and anti-nausea medicines can cause symptoms of parkinsonism or even worsen movement problems when given to people with Parkinson’s disease.

* Vascular parkinsonism. Can cause problems with slowness, shuffling gait and thinking problems.  Head CT or MRI may be helpful in determining this.  Treatment includes careful control and treatment of cardiovascular and stroke risk factors such as smoking, high blood pressure, high cholesterol, diabetes, depression and sedentary lifestyle.

* Strokes

* Brain injury. Repeated brain trauma with concussion (ie. Boxing) and injury from lack of oxygen such as after cardiac arrest.

* Toxin exposure such as carbon monoxide poisoning, heavy metal exposure (industrial exposure to manganese, lead, cobalt or mercury), agent orange.