1996 Shy-Drager Conference Transcripts - 3 of 4

[Guest guest]

Q: Can you tell us where we are currently in research?

A: (Dr. Benarroch) There is a lot of interest in this disease even

though unfortunately we lag behind Parkinson disease in the interest

in finding out what the mechanism is that produces the symptoms. MSA

is much more complicated than Parkinson's disease. Many different

parts of the brain, not only the part that has movement or autonomic

control, but the areas of respiration, sleep, etc. are involved. So

this is a disease which it is important to put a very wide

multidisciplinary approach to understanding this problem, not only

from a clinical or pharmacology research, but also from the stand

point of autonomic research on what parts of the brain are affected

and how and what type of chemicals and substances are affected. We

know in Parkinson's disease what chemicals are affected. We don't

know what's going on in multiple system atrophy and Shy-Drager

syndrome. We know that it is a different disease that affects many

different areas, very variable, one patient compared to another. No

two patients are identical. We need to understand at least what is

the common, if any, biochemical basis of this disorder. Much more of

the research now is looking at not just what part of the brain is

affected but what specific chemical is affected. Because I think that

treatment will come from that area of research.

Q: Are there any studies going on looking at nerve regrowth, or things

like that?

A: (Dr. Chokroverty) Not specifically yet for Shy-Drager syndrome

like there is with ALS, Alzheimer, and peripheral neuropathy. We do

know that there are particular groups of cells that are responsible

called growth factors that are accessible to specification because

there are receptacles on them. We have a lot of advantages for

example, with diseases like ALS or Alzheimer or Parkinson's because we

know the cell groups that are the most susceptible to the disease. In

MSA it is not at the same level of research, that knowledge of the

specific mechanism of disease. The very important step before that

would be to define what cell group is susceptible because different

cells in the brain work on different functions. You would want to

find the cell group that works with low blood pressure. So, there is

a lot of research going on in terms of use growth factors for other

disorders and I am sure this is going to be an advantage.

(Dr. Low to questioner) I think your interest in growth factors is

honorable. It is a natural sort of reaction that if something isn't

working properly why don't we give something to stimulate it and make

it grow and if you think about the number of disorders where there is

an interest; neuropathy, ALS. I just want to interject a note of

caution about growth factors, its fair to say that in none of the

areas where it is being studied at the moment, is it looking

particularly promising. A lot of the ways it is being done, it is

being given in what's called pharmacologic doses. Think of it like a

fertilizer type hypothesis, where the argument is that if you use

enough of it something will happen. When actually in fact, a lot of

these effects have been nonspecific and the likelihood is that the

answer is not going to come in a broad or nonspecific use approach.

(Dr. Schatz) I wonder if I can change direction a little bit. It is

not usual that we have

so many patients with this problem in one room since it is a very rare

disease. So I would like to ask you some questions. How many of you

can differentiate the trouble you have with blood pressure from the

trouble you have with balance or walking? (A show of hands was given)

So some of you can tell; your blood pressure is under reasonable good

control but you are still having some difficulty. When did your

symptoms first start? (Three, four, eight, and twelve years were

mentioned by patients in the room) So I get the sense, from this

small sample, that in retrospect the symptoms started longer ago than

we normally think of in this disorder.

(Caregiver) Can I speak to that and ask you guys to look at something?

The very first symptom, in retrospect, for my wife was a sleep

disorder.

(Dr. Chokroverty) And what was the nature of that sleep disorder?

(Caregiver) Absolutely crazy, screaming, flailing nightmares. The

kind of nightmare that raises the listener well off the bed. A scream

that starts at high C and ends at...

(Dr.Chokroverty) Did others of you have that?

(Several in the audience nod or make comments about their screaming

nightmares)

(Dr. Chokroverty) Does your wife still have that?

(Caregiver) No, they went away prior to diagnosis.

(Dr. Chokroverty) What time of the night did the nightmares occur?

(Caregiver) About two hours into sleep. (Others mentioned various

times of the night it occurred.)

(Dr. Chokroverty) There is one condition which is called REM varied

disorder which is seen in Shy-Drager/multiple system atrophy. That

particular type of sleep comes later, normally comes up to 80-90

minutes after the onset of sleep for a short period, then reappears

later and later during the night and REM sleep becomes longer and

longer so the more intense REM sleep usually in the last third of the

night. So that would solve for some of you what might have been the

REM varied disorder type of sleep hallucinations, where they might

think that someone is chasing them in their sleep and they scream.

Occasionally we can treat that with a small dose of medication. But

we have to be careful that the other condition sleep apnea, a

breathing disorder which is very common in the middle and late stage,

is not made worse by this drug. So you have to be careful about that.

There is a condition of sleep apnea where there is upper airway

obstruction and the person stops breathing for a few seconds and wakes

up with a snore or a scream. Now there are clues. In the day time if

you find you are nodding off, that is one of the clues that your sleep

is disturbed. You should have a sleep study to see if you are having

sleep apnea if you are dozing off in the daytime. If that is so, we

can symptomatically treat that by putting on a mask of continuous

positive airway pressure (CPAP) which keeps the upper airway open and

that will help.

(Dr. Schatz) I have another question for the group. We could talk

forever but I want to hear from you folks cause you have the problem

and I want to learn. How many of you have Shy-Drager support groups

in your own geographic locality? (Laughter from the group.) What's

so funny?

(Caregiver) We have one support group.

(Dorothy Trainor-Kingsbury) We are separate and apart but we are so

damn close it hurts.

(Dr. Schatz) I understand.

Q: We were talking before about when symptoms started. Would you

consider sleep disorder that we were just talking about, as the

beginning?

(Dr. Schatz) I would but I'm not sure if the classical symptom of

neurogenic orthostatic hypotension...

(Caregiver) Because when we went through this period and now it is

gone. That's another question as to why it is now gone? Where is the

beginning? There must be a life path to this condition. I would like

to know when did it start? Where does it end? And it is hard to ask

where it ends and it is hard to answer where it ends when you don't

know where it started. To have the time, to have x amount of

years...does someone usually go through all the symptoms till they get

to where they cannot function anymore or... What is the end? When do

you know when you are close to the end? They are ugly questions to

ask but when we went to the physician and he diagnosed my husband the

doctor was saying " get your insurance papers together, get yourself

organized. " Just how much of that should we have organized to date?

(Dr. Schatz) That's a very important question that most physicians

have difficulty in answering. First of all my own experience with

this disease over the past thirty years, is that it lasts much longer

than the ones described in the text books. And I think we heard that

today and that reinforces my clinical impression. I have one patient

who clearly had problems when she was a teenager. She is now forty

four years old and she is disabled. She is in a wheelchair but she is

still functioning. So it can last a very, very long time. I have to

say that I am not sure that control of the blood pressure, although it

helps you function, makes much difference in the progression of the

disease. But to answer your final question, how you know when it is

going to end; I don't think any of us can answer that accurately. I

think it is much too variable for us to be able to give a specific

response. Maybe ...

(Dr. on) I don't think I have an answer either. I will just

say that seeing the level of recognition on so many of your faces when

discussing the sleep problem tells me that even if we didn't know it

before, we know now that it is part of this illness I am quite

convinced. I think Dr. Chokroverty is agreeing with us on that. You

would think that we would be able to observe a patients progress and

understand with some precision when it was going to be difficult for

you to continue to maintain viability and to continue to live. We

aren't generally able to do that. I think that we are all a little

surprised by the end when it comes. But certainly if patients have

very prolonged apneic spells. Many are warned that apnea is not a

good sign. A lot of patients will have apneic spells that are of a

short duration where you stop breathing, very early or even as an

initial presentation of the illness but when those get very long, that

I think it is a difficult time. Maybe Dr. Chokroverty would wish to

comment on that.

(Dr. Chokroverty) The question you ask is extremely difficult. When

the patient bedridden and the breathing of course, as said, when

in the day time there are more and more groggy periods, a drifting

off, that shows that there is a serious breathing problem. And that

serious breathing problem usually comes middle to late stage of the

disease. We can give symptomatic relief if it is upper airway

obstruction by using the CPAP machine as previously mentioned. It can

be a very difficult problem particularly if it is related to blood

pressure.

Q: I think we have a good neurologist in our area, but how important

is it for us to differentiate between multiple system atrophy and

OPCA? Would that help us in treating his symptoms?

A: (Dr. on) It is important to get a good diagnosis, I believe.

It is important to know that it is multiple system atrophy instead of

some of the other rare neurological diseases or peripheral nerve

problems that could mimic it. If you know that, then I think that

just being well educated and having a physician who is very attentive

and reads what is written about this. And who is willing to call Dr.

Chokroverty or whoever to get advice when he needs it. We are

entering an era of primary care medicine so I think that ultimately

your local doctor is going to be the most important advocate. But, it

would be good for him and you to be at the end of the telephone line

from the mail list or other doctors like the ones here to help things

along.

(Caregiver) But there is no question that the local neurologist is not

nearly as informed or knowledgeable about this disease as you are.

(Dr. on) Yes, but on the other hand, you might be surprised.

He may have very good practical sense about the kind of things we do.

Now there are also research protocols at a number of institutions and

so it's good for mankind and the patients, in some cases, to come to

research centers where while they are teaching you about this illness

and learning about it, to learn something for future patients. So

that is a different situation.

(Dorothy) I want to just answer Dr. Schatz there on not having

physical support groups. We are very generous and I think that we

don't want that many people to having what we have to make up more

support groups. We'll settle for what we have got right now, OK? In

the mean time you find a cure so we won't have it anymore. Thank you

and we will be seeing some of you again and again. Thank you,

doctors.