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Q: EXPERIMENTAL TREATMENTS FOR MULTI SYSTEM ATROPHY ( Answered 5 out of 5 stars,   1 Comment )
Category: Health > Conditions and Diseases
Asked by: brad1969-ga
List Price: $50.00
Posted: 01 Aug 2005 16:02 PDT
Expires: 31 Aug 2005 16:02 PDT
Question ID: 550572
Hello. My father has a rare neurological disorder called Multi System
Atrophy. I wanted to know if there was anyplace in the world that is
conducting experimental research for treatment and/or cure of this
disease. We are willing to travel anywhere in the world, if necessary,
for any experimental treatment. (We are located in Florida.)
Thank you and I look forward to hearing from you soon!
Answered By: welte-ga on 11 Aug 2005 18:19 PDT
Rated:5 out of 5 stars
Hi Brad1969-ga, and thanks for your question.  My condolences on your
father's unfortunate diagnosis.  You may already be familiar with the
details of MSA itself, but I will give you some resources in addition
to answering your primary question.

MSA actually comprises a group of rare multisystem neurological
disorders.  The three diseases that fall under the umbrella of MSA are
Shy-Drager Syndrome, Striatonigral Degeneration, and
Olivopontocerebellar Atrophy (OPCA).  The common features of these
diseases are Parkinsonism, autonomic failure (dysfunction of the
sympathetic and parasympathetic nervous system, which controls blood
pressure, sexual function, etc.), and cerebellar ataxia (failure of
muscular coordination).  Each of the three diseases above exhibit each
of these features to a greater or lesser extent, which is what
differentiates them from one another.   MSA is generally a
progressive, severe disease.  While one of the primary features is
often Parkinsonism, the current medical therapy for Parkinsonism is
largely ineffective.  Here's what the Neurology Channel has to say
about the matter:

"MSA is generally more severe than Parkinson?s. More than 40% of MSA
patients are confined to a wheelchair or otherwise severely disabled
within 5 years of initial diagnosis. Except in a few cases, the drug
of choice for Parkinson?s patients ? levedopa ? has no effect and may
even make the symptoms worse."

The Neurology Channel also has numerous links to resources and well
summarized information regarding each subtype of MSA, which are
written at a very accessible level, with explanations of medical

Although this site doesn't emphasize it, severe depression has also
been reported with MSA, particularly with Shy-Drager Syndrome.  The
treatment for MSA at this point is limited to levodopa in certain
patients, and experimental therapies.

One review article from Italy discusses the current, primarily
symptomatic, treatment of MSA:
"Multiple system atrophy (MSA) is a neurodegenerative disease of
undetermined aetiology that occurs sporadically and manifests itself
as a combination of parkinsonian, autonomic, cerebellar and pyramidal
signs. Despite the lack of any effective therapy to reverse this
condition, some of the symptoms may be, at least temporarily, improved
with adequate symptomatic therapies. Medical treatment is largely
aimed at mitigating the parkinsonian and autonomic features. The
therapeutic results of levodopa therapy in cases of MSA are difficult
to interpret because of their variability. Nevertheless, the statement
that patients with MSA are non or poorly levodopa-responsive is
misleading. Clinical and pathologically proven series document about
40-60% levodopa efficacy in patients with MSA presenting with
predominant parkinsonian features. Unfortunately, other
antiparkinsonian compounds (dopamine agonists, amantadine) are not
more effective than levodopa. Orthostatic hypotension (OH) can be
suspected from the patient's history and subsequently documented in
the clinic by measuring lying and standing blood pressure. The
diagnosis ideally should be confirmed in the laboratory with
additional tests to determine the cause and evaluate the functional
deficit, so as to aid treatment. A variety of pharmacological agents
with different mechanisms of action have been used in MSA to reduce OH
when this is symptomatic. OH can also be alleviated by avoiding
aggravating factors, such as the effects of food, micturition,
exposure to a warm environment and physiological diurnal changes and
by using other non-pharmacological strategies. The treatment of the
very common genito-urinary symptoms (incontinence, retention,
impotence) should also be considered in order to improve the quality
of life of these patients."

Colosimo C. Pezzella FR.  The symptomatic treatment of multiple system
atrophy.  European Journal of Neurology. 9(3):195-9, 2002 May.

If you are interested, you can request a reprint of this article from Dr. Colosimo:

Experimental Therapies

The National Institutes of Health has a good site discussing MSA,
complete with links to support organizations for each of the
subcategories mentioned above.

The NINDS is also an excellent source for finding reliable information
about ongoing clinical trials for this and other neurological
disorders.  Here is a link to ongoing clinical trials for MSA across
the country:

You can check this page periodically for new trials.

Here is a summary of the experimental clinical trials listed through
the National Institutes of Health:

Most of the current ongoing studies are trying to better understand
the pathogenesis of the disease and how to treat it's associated
hypotension (due to autonomic dysfunction), which is often a
predominant feature of this disorder.  Not all of the trials tracked
by the NIH are in the US.  The most relevant to your question, based
in the US, are outlined below.  To obtain more information about these
trials, I suggest visiting the site above (the links
to the specific trials expire) and visit each individual trial's
information page, as well as contact each trial's patient recruiting
office, listed with each trial below. At the bottom of each trial's
web page there is a link to "Detailed Information."  Clicking this
will take you to a page that gives more detailed information about the
design of the study, inclusion and exclusion criteria, etc.


Tests to Evaluate Primary Chronic Autonomic Failure (Trial #NCT00059033)
This trial, based in Maryland, is trying to understand the causes of
autonomic dysfunction, which often results in hypotension in patients
with MSA and other disorders.

For participation information, contact:
Patient Recruitment and Public Liaison Office


Droxidopa in Treating Patients With Neurogenic Hypotension (Trial #NCT00004478)
This trial, also exploring possible a possible treatment for
hypotension, is specifically studying droxidopa.  The trial is being
run by the Mount Sinai School of Medicine in New York City.

You can read more information about droxidopa here:

Recruiting information:
Horacio Kaufmann   212-241-7315  


A Phase IV Study in Subjects with Neurogenic Orthostatic Hypotension
(Trial #NCT00046163, NCT00046475)
A Phase IV trial means that a medication has been through initial
toxicity and efficacy testing and is in the final stages before being
introduced on the market.  This particular trial is run by a
pharmaceutical company (Shire Pharmaceutical Development).  They are
investigating treatments for hypotension due to multiple neurological
conditions, including MSA.  They are testing ProAmatine (midodrine
hydrochloride) to determine how effective it may be.  Of particular
interest, they have a study group in Florida (see below).


      Suncoast Neuroscience Associates, Inc., St. Petersburg, Florida
Suzanne Lash   727-824-7135  

      Dr. Harry Pepe & Associates, Inc., Miramar,   Florida,   33023
Donna Regula   954-981-4811  

      Neurological Associates of Delaware Valley, Upland, Pennsylvania
Barbara Dick, RN   610-876-4800  

      Diabetes & Glandular Disease Research Associates, PA,, San Antonio, Texas
Greg or Laurie   210-615-5565   Ext. 1077 or 1454  

      North Alabama Neuroscience Research, Huntsville,   Alabama,  
35801,   United States; Recruiting
George C. Morgan, MD, PhD   256-533-3552  

      Economou & Associates, LTD, Chicago,   Illinois,   60612,  
United States; Recruiting
Study Coordinator   312-829-3532  

      Johns Hopkins Hospital, Baltimore,   Maryland,   United States; Recruiting
Janice Stack   410-614-4576  

      Michigan Pain and Neurological Institute, Ann Arbor,   Michigan,
  United States; Recruiting
Study Coordinator   734-677-6000   Ext. 4  

New Hampshire
      Dartmouth Hitchcock Medical Center, Lebanon,   New Hampshire,  
03756,   United States; Recruiting
Pauline LeBlanc   603-650-4165      Pauline.R.LeBlanc@Hitchcock.Org  

New York
      NY Presbyterian Hospital, New York,   New York,   10032,  
United States; Recruiting
Daniel Bloomfield, MD   212-305-6634  

      Medical College of Ohio, Toledo,   Ohio,   43614,   United States; Recruiting
Dyan Serna   419-383-3697  

      COR Clinical Research, LLC, Oklahoma City,   Oklahoma,   73103, 
 United States; Recruiting
Clinton Corder, PhD, MD   405-272-8481  

      Neurological Associates of Delaware Valley, Upland,  
Pennsylvania,   19013,   United States; Recruiting
Barbara Dick, RN   610-876-4800  

      Westmoreland Neurology Associates Inc., Greensburg,  
Pennsylvania,   15601,   United States; Recruiting
Norma Skillings, Carol Clayton or Dr. Michael Sauter   724-835-1921 

      Monarch Medical Research, Norfolk,   Virginia,   23502,   United
States; Recruiting
David Vendt, RN   757-466-7263  

West Virginia
      West Virginia University, Morgantown,   West Virginia,   26506, 
 United States; Recruiting
Connie S. Bolyard, RN   304-293-4117  


Study of Inherited Neurological Disorders (Trial #NCT00004568)
This trial is not looking at therapies, but rather trying to better
understand the genetics of the disease, which may benefit future
generations, including yourself.  Here's their study objective:
"Information from this study may provide a better understanding of the
genetic underpinnings of these disorders, contributing  to improved
diagnosis, treatment, and genetic counseling, and perhaps leading to
additional studies in these areas."

      National Institute of Neurological Disorders and Stroke (NINDS),
9000 Rockville Pike,   Bethesda,   Maryland,   20892,   United States;
Patient Recruitment and Public Liaison Office   1-800-411-1222 

Detailed information:


The NINDS has no clinical trials listed for the study of potential
cures of MSA, only for symptomatic treatment as listed above.

You can check for clinical trials in the future at this site:


On the cutting edge, so to speak, of experimental therapy is surgical
intervention for Parkinsonian disorders such as MSA.  There are a
smattering of articles with very mixed results on the efficacy of both
deep brain stimulation and ablation of a portion of the globus
pallidus (or subthalamic nucleus).  Most of the small studies have
been discouraging for MSA, although promising for Parkinson's Disease.

Neuronal transplantation is also being investigated, primarily in
Europe, and appears to only be in the animal model testing phase at
this point.


One group at the Beth Israel Hospital in New York City examined
electroconvulsive (shock) therapy (ECT).  The rationale for this was
that ECT improves symptoms in some cases of Parkinson's Disease.  ECT
gets some bad press, but is very helpful for many people with
refractory depression and other disorders. You can read more about ECT
in the NIH Concensus Statement, located here:

Here is the reference to the study above:

Roane DM. Rogers JD. Helew L. Zarate J.  Electroconvulsive therapy for
elderly patients with multiple system atrophy: a case series. 
American Journal of Geriatric Psychiatry. 8(2):171-4, 2000.

In Germany, one group is looking at 

Here is an excerpt:

"Parkinson?s first, later multisystem atrophy

Ulm University is the German organisation coordinating a large
European drug study on 800 patients (Neuroprotection and Natural
History in Parkinson Plus Syndrome, NNIPPS), which focuses on two
atypical Parkinson?s diseases: PSP (progressive supranuclear palsy)
and MSA (multisystem atrophy, with a prevalence of 4.4/100,000). Both
disorders are more severe than Parkinson?s disease and on average lead
to death after six to eight years. The study, which is to date the
most comprehensive attempt to clarify the natural course of the
disease, is intended to clarify whether the drug of a French
pharmaceutical company can slow down the progression of the two
diseases. Final results are expected in 2006."

The study looking at the efficacy of a medication known as riluzole is
being performed by the Neuroprotection and Natural History in
Parkinson Plus Syndrome (NNIPPS) throughout Europe.  There will be 250
patients from France, 300 from the UK, and 250 from Germany, for a
total of 800.

You can read more about this study at these sites:

Also, Dr. Nigel Leigh at Kings College in the UK is a member of the
NNIPPS Study Group.  You can read more about his work here:

____________________________ is an excellent resource for all types of movement
disorders, including MSA.  The site below lists some additional

Of particular note is one study at the Massachusetts General Hospital
(MGH), which is trying to better understand various types of movement
disorders, including MSA.

Jeremy D. Schmahmann, M.D.
Associate Professor of Neurology
Harvard Medical School
Director, Ataxia Unit;
Cognitive/Behavioral Neurology Unit
Massachusetts General Hospital VBK
Massachusetts General Hospital
915 Fruit Street
Boston, MA 02114
Tel: (617) 726-3216
Fax: (617) 726-2353


Another study, at UCSD, is also trying to better understand the causes
of MSA.  Here is more information:


You should also keep an eye on this Yahoo newsgroup:

They list clinical trials along with contact information and maintain
a support group.  The trials they mention at this point I have
discussed above, but they sometimes have additional information, such
as the MGH trial listed above.

The journal Advances in Clinical Neuroscience and Rehabilitation in
the UK published an excellent 6 page article outlining the current
state of the art of the understanding and treatment of MSA, including
symptomatic treatment for it's various components.  I highly recommend
reading this article and sharing it with your physician.

Unfortunately, this is the sum of the most reliable trials available. 
I recommend checking back frequently at the NIH / NINDS websites, as
these will be frequently updated as new trials open.  They are the
most reliable sources of information on the subject.


I hope this information was helpful.  Best of luck on your family's
difficult course with this illness.  Please feel free to request any

brad1969-ga rated this answer:5 out of 5 stars and gave an additional tip of: $5.00

From: pinkfreud-ga on 01 Aug 2005 16:19 PDT
This website may be of interest to you:

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