Kennedy's Disease Association

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"Surfing the internet I found the KDA and for me it was like an island for a castaway. Thanks to them, I have a lighthouse to help find my way."

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Kennedy's Disease Chat Transcript  06-02-2007

Topic:  Special Guest - Dr. Diane Merry Ph.D.

Host: Terry Waite



TerryW: Hello

Alexandre entered the room.

Dart! entered the room.

doughboy entered the room.

doughboy: Good morning to All

Dart!: Greetings All

doughboy: Hi Dart

Dart!: Another grand day, Folks - good to be alive, right?

doughboy: Right on, hey Dart, refresh my memory, who you are and where you live, if you don't mind!

craig_h entered the room.

doughboy: Hey Terry, how are you

doughboy: Hi, Craig

Dart!: I am in British Columbia, Canada, just west of the Rockies

ARofer entered the room.

doughboy: How's the weather up there?

TerryW: I am back

doughboy: Terry, can you see my Text?

craig_h: hello, could someone send me today's chat? My father had a stroke last week. I need to go down there. But was interested in today's chat also

Dart!: And Terry, I hope that you are having a better week. The weather here couldn't be better- an almost clear sky.

doughboy: Sure is your email listed?

craig_h: yes it is

TerryW: I am in the midst of passing a kidney stone

TerryW: been on my back since yesterday morning

Gary_KS entered the room.

doughboy: Go take care of your Pop, we will make sure you get it!

TerryW: Hi Gary

craig_h: thank you

TerryW: Sorry to hear that craig

Gary_KS: Good morning, all. Gary Uchiyama from Kansas City.

robazbob entered the room.

craig_h has left the room.

Butch entered the room.

doughboy: Hi, Gary, TJ from Texas

TerryW: Hi everyone that just joined

ARofer: Good morning, Gary. I am Arofer from Felton, California

robazbob: hi terry

Dart!: What a joyous occasion, Terry., ans sorry about your Pa, Doughboy, a tough  break, but they can do great things inthe medical field.

Alexandre: Good morning from Brazil to all folks!

Butch: Hi you'all. How is everyone doing?

TerryW: Hi butch

ARofer: Hola, Alexandre

doughboy: Terry, hope your not in too much pain, I've had them and had to go into the hospitial to have them remmoved. Also can you save the chat and send it to Craig?

TerryW: Dr. merry said she may be a few minutes late

TerryW: Doughboy.  I have been in the kind of pain that is so bad it makes you throw up

doughboy: Hi, Alexandre, glad to have you, I'm from Texas and my name is T. J.!  Hi too everyone!

TerryW: it comes and goes

Butch: Hi Alexandre--good to have you in the chat.

TerryW: I kate these Kidney stones

TerryW: hate

Dart!: Sorry about that, Terry!

TerryW: Alexandre,  Brazil the land of cheap Fuel

doughboy: That's how I was Terry and then I had to go in the Hospital so they could take them out, I had 6 stones!

Butch: TJ     What happened to your Pa? Did you finally get your computer set up.

TerryW: Pa?

doughboy: Murf should be on any minute, he got him a new chair yesterday!  Yes my CPU is up but I don't think I like this new Microsoft Vista!

TerryW: Murf gets all the cool toys

doughboy: I'm suppose to go over his house today for bar be cue Ribs!

TerryW: Nice

Dr__M entered the room.

doughboy: Hey Murf's ordered his new swimming pool to be installed

TerryW: Hello Dr, Merry

Alexandre: Yes we have a none poluted fuel from the sugar cane, I have a flex car with both gasoline and ethanol

doughboy: Hello Dr.

Dr__M: Good morning everyone.  Sorry I'm a few minutes late.

TerryW: No Problem

Dart!: ANOTHER new chair for Murf? He must put on many miles!

Butch: I'm getting ready to go to NIH for Mon. and Tues. the 4th and 5th. Looking forward to it.

TerryW: Dr. Merry give us brief statement of what you will be chatting on today

Dart!: Good to have you aboard Doctor!

doughboy: Dr., right off the bat, I'm going to ask you if KD effects your bowel movements as far as them being uncontrolled at times?

TerryW: LOL TJ. Hold that movement for later if you can LOL

Butch: LOL  LOL

Dr__M: I'd be happy to answer any questions you might have about current research.  In addition, I just returned from chairing a conference on polyglutamine diseases and there was some exciting new information and certainly lots of efforts to identify new therapies.

LSimonis has left the room.

Alexandre: My question is. Are there any people being testing with some medicine against KDA?

Dr__M: Unfortunately, since I'm not a physician, I can't speak to medical issues (e.g., particularly non-neurological complaints).  Sorry!

mobiusloop entered the room.

mobiusloop: good morning

doughboy: Does KD effect the nerves in the back, neck, arms and legs?

Dr__M: Alexandre, yes there are currently two clinical trials going on.  One is in Japan (Leuprorelin) and one at the NIH (dutasteride).  We should have some results from the Japan trial this summer.  There are some early data that I know of as well.

Alexandre: What about the new therapies in USA?

mobiusloop: Is there any treatment yet for pins and needles in the feet?

Dr__M: KD does affect the motor nerves (and to some extent the sensory nerves as well) coming from the length of the spinal cord.  These control the muscles in the back, neck, arms and legs.

Murf entered the room.

Murf: Morning

Murf: Sorry I'm late

doughboy: Morning lazy bones

Murf: I was out walking the dog

ARofer: Dr_M, any testing of drugs on the mice?

TerryW: Dr. Merry, are there any labs working with stem cells inrelation to our disease?

Butch: That's OK Murf--I know you were riding your new chair.LOL

Dr__M: The dutasteride trial is too early for us to know any results from KD patients.  We did a dutasteride trial in mice and unfortunately, it wasn't successful.   However, there is a caveat to that statement, which is that is seems that mice are different from humans in how they metabolize male hormones.  We're still trying to sort out how we can make a mouse more like a man.

Murf: New chair is in the car

TerryW: Give the mouse an attitude LOL

doughboy: If it didn't work on mice, why then try it on us?

Murf: Dr_M will you make another movie for us this year?

Alexandre: Good question Terry!

Dart!: That is very good, Doctor - making a mouse to be like a man!

doughboy: Have the researchers evewr tried Chimps?

Dr__M: Ok, let's see.  I will apologize up front if I miss any questions.  Please repeat if I don't answer.  My typing is slower than I would like!  There are a number of compounds being tested in KD mice.  We are in the process of testing a number of compounds in motor neurons that come from the mice, but we must wait for the NIH funding to continue our clinical trials in the mice.

mobiusloop: There's a Movie?

ARofer: Whom do we harrass at NIH to get you the funding?

Murf: Dr_M and her lab made a movie for us at the conference last year. I was very good and won lots of awards //1

TerryW: Mobius, Dr. Merry made a short video presentation at the last KDA conferrence

doughboy: Dr, is there a corelation between my KD and type 2 diabetes?

TerryW: Dr merry, are there any labs that you know of working with Stem Cells in relation to our disease

Dr__M: So about the dutasteride in you versus the mice.  Dr. Fischbeck and I discussed this for a very long time, believe me.  We did the trial in the mice, even though we knew that a negative result would not fully answer the question, since a positive answer would have been so encouraging.  Let me explain.  Dutasteride prevents the conversion of testosterone to the more potent hormone dihydrotestosterone (DHT).  Motor neurons do this conversion very efficiently.  We thought that if we blocked the conversion to this more potent hormone we could ameliorate disease symptoms.  But it turns out that mice have additional ways to handle testosterone and there is some evidence that when you block this conversion, they have a lot more testosterone.  That doesn't seem to happen in hum

Dr__M: So that doesn't seem to happen in humans, and we both agreed that a  negative result in the mice should not prevent the test in KD patients.  I hope that explains.

mobiusloop: Yes, thank you

Dr__M: So funding is so tight these days that even when the grants are well received, there is just not enough money to go around.  I'm very optimistic, however, and it looks good that we will get the money.  I'll know in about a week.

Alexandre: Are there any trial about stem cells in relation to KD disease?

doughboy: Good Luck on that Dr. Merry!

kellyC_ entered the room.

Dr__M: I don't know of any trials currently using stem cells in KD.  In fact, there is one trial that I am aware of in France using stem cells in Huntington's disease.  I think that most researchers, including myself, feel that small molecule (drug) therapies are much more promising for KD.  We have ways to help the neurons get rid of the bad protein, and it's a matter of getting the drug to do its job in humans.

ARofer: Is anyone using microarrays for the drug testing on KD?

TerryW: I have been watching the Discovery Health channel and they had a guy on talking on timelines for sures for neuromuscular diseases and he saif that within the next 10 years he expects great strides and bounds in research and treatments/cures

doughboy: My arms are getting weaker and weaker all the way to my wrist my neurologist says my muscles are shrinking and that I'm laying my arms on my nerves, is that consistant with your studies, Dr. Merry

doughboy: Hope i'm still here in ten years!

Dr__M: There are currently no KD studies in monkeys that I know of.  It's a great question, though, since one would think that monkeys would best model a human nervous system.  It's really a trade-off on cost, time, drug design.  If we can identify drugs that work in mice and other species as well ( for example, flies, fish, etc.) then they are most likely to have effects in human KD patients.

doughboy: Well and I guess chimps would cost a lot more also!

doughboy: Hi, Kelly!

TerryW: Dr. Merry,  Thank you for joining us and giving us these updates on the research that is going on. it is most helpful

Alexandre has left the room.

kellyC_: Hi Doughboy

Murf: We had a study on monkeys but TJ stoped going//2

TerryW: Bad Murf  "Slap"

Dr__M: I agree with the optimism of what the next 10 years will bring for neuromuscular diseases in general.  I am even more optimistic about KD, since we have very good models and a lot of people putting a lot of effort into it.  I think there is beginning to be more synergy between the Huntington's field and the KD  field, with drugs being designed that could benefit both.

Murf: my bad//4

doughboy: Is Huntington's closely related to KD or similar?

doughboy: Murf's a converted Texan, we get all the Canadian rejects! LOL!

mobiusloop: Dr. M .  Is there an info center that I could refer My doctor to  bring him up o speed?

Murf: yup!

robazbob has left the room.

doughboy: But that converted Texan can really bar b que!

Dr__M: I'm not sure what you mean about "laying your arms on your nerves" and whether that's consistent with our studies; sorry!  It is true that the nerves shrink with time and goal is to 1-prevent that and 2-enhance the health of the motor neuron cell bodies in the spinal cord.  We have found some drugs that make KD motor neurons in a dish survive.  The question for us now is whether these prevent symptoms.  We'll be pushing hard on this in the next 2 years.  In fact, this summer we will be testing 4 compounds that look promising for HD in these motor neurons.  Hopefully we can find one or more that we can then test in the mice.

Butch: Dr Merry--I have gotten much weaker and lost muscle lately. I am in the clinical test at NIH. Hoping this is only temporary.

Alexandre entered the room.

Murf: I'll volunteer for the next study for monkeys or man

doughboy: How come I was told testosterone treatments were bad for KD people?  My level is 150 and I have no energy at all!

ARofer: Are there any big or small pharma companies sniffing around or doing research in KD?

kellyC_: //6

Dr__M: Huntington's disease is caused by the same gene mutation (mistake) that causes KD.  It affects a different protein and different neurons, but there are many, many similarities between them.  For example, the gene mutation makes the protein sticky in KD and in HD, and the efforts in common between the two areas of research are to identify drugs that will have the neurons re-fold the sticky protein and get rid of it through the cellular "trash trucks."

TerryW: We need a bigger truck

Murf: ARofer: No money in curing 330 guys

doughboy: Could it be possible to be diagnosed with KD but in reality it could be Huntington's?  Does it have the same side effects

Dr__M: Regarding an info center for KD, I think the best would be the KDA site, the MDA site and a site at the NINDS (part of the NIH).  Send me an e-mail separately and I'll give your more detailed URLs (This email address is being protected from spambots. You need JavaScript enabled to view it.).

Butch: 330 known--don't know just how many haven't found the KDA or been diagnosed.

TerryW: Huntingtons gives you much more feminine features

TerryW: I think

Dart!: A valid point, Murf - we aren't popular (nor populated) enough

ARofer: So how many people have restless legs syndrome? I see ads for this all the time.

doughboy: Such as?

Murf: Dart - we're one in a million

TerryW: I will let Dr. Merry explain

doughboy: I mean Murf's started carrying a purse lately! LOL!

Dr__M: Testosterone treatments are considered to be bad because we now know that testosterone is the initiator in the disease process.  I guess it's similar to the starter on a car.  You can have bad gasoline in the tank, but unless you start your engine, it won't ever gum anything up.  Well, I'm not much of a car mechanic so maybe I should have chosen a different analogy!

doughboy: Sorry Murf, don't posion my bar b que today!!

Murf: Care full I carry a 6 gun in it

Alexandre: What does mean LOL? Sorry...

doughboy: Lots of Love

Dr__M: No chance of misdiagnosing Huntington's for KD.  The clinical symptoms are completely different, despite the fact that the type of gene change is the same.  It affects different proteins and different cells.

Butch: Laugh out loud.

kellyC_: Good Point Dr-M

doughboy: OK

jameson411 entered the room.

jameson411: morning everyone

Butch: Hi James.

Dart!: jameson411:

Murf: hey James

Dart!: You have  missed so much good stuff this morning, Jameson!

jameson411: :-(

jameson411: when will the chat log be up?

Dr__M: About big or small pharma.  There is a lot of interest of pharma in polyglutamine disease in general.  This include KD, Huntington's, and several forms of ataxia.  I chaired the recent conference in France and was able to secure very generous funding, not only from the KDA (Thanks again very much for this!!) but from big and small pharma as well.  Some sent representatives (e.g., Merck, Novartis) and several from small biotech companies participated as well. I think there is really growing interest.

TerryW: I will try to get this posted by Monday

TerryW: maybe sooner

Alexandre: Are there any news being prepared to be presented at the KDA Conference in November?

TerryW: There is always ne news and reasearch updates presented at the conferences

Murf: ANgela will have a presentation on the trial. Will you have something Dr_M?

MICH entered the room.

kellyC_: Ataxia.? there seem's to be kinda associated with KD.. sytoms wise hey? I know i have iot in my Hands @ Times to.

Dr__M: I'm not sure about restless legs syndrome and its frequency.  I am in the process of finding out more, though, because I believe my father has it.

TerryW: I have it

kellyC_: yup me to Terry

TerryW: I think many KDers do from what I have heard

jameson411: i have a mild form of the dancing legs thing

Murf: Me too

kellyC_: ooooooooooooooooo/.............ok

jameson411: hmmmm

ARofer: Are med schools producing students who will take up the KD burdens?

Gary_KS has left the room.

TerryW: KD Burdens?

ARofer: Research burdens

Dr__M: Absolutely, I will update you on our new data in the fall.  I hope that we will have some new data on a number of drugs by then.  I hope that the summer is a really productive one, and expect it to be if I don't have to spend my time grant-writing or teaching this summer!!

Gary_KS entered the room.

jameson411: uhg... teaching.  What a drag ;-)

Gary_KS: I am back.

Murf: Does that mean I can put you down for a presentation?

TerryW: welcome back Gary

Murf: Diane?

doughboy: I have Restless leg syndrome, type 2 diabetes, severe sleep apnea and KD!

Murf: Lucky man

jameson411: you win

TerryW: Could be worse, ALS

Dr__M: About med schools teaching about KD.  Your question is well-timed, since I just taught 270 med students yesterday about the pathology of KD and how we identify treatments.  You can believe that the students at Thomas Jefferson University learn a lot about it!  Also, others in the KD (and HD) research field are teachers in medical schools and I know it is well-taught to med students.  We should see the fruits of this down the road.

Murf: Yup

kellyC_: Yeah thats for sure!

MICH: I seem to match with resless leg and sleep apnea but not much muscle left in legs might be the reason

Murf: We're lucky I figure

Dr__M: Yes, you can definitely put me down for a presentation if you'd like to hear from me!!

TerryW: yes!

ARofer: Dr_M, are you using microarrays and/or other automation techniques in your drug testing?

doughboy: Sure we would Dr. Thank you

Murf: Wahoooo Done!!!!!!!!!!!!!!!

Butch: Dr Merry will you talk at the conference ---Good move Murf. Strike while the fire is hot.

kellyC_: I often wonder if they teach the Med students here in Canada on this topic as well? would be nice to hear, that they do.!

Dean entered the room.

TerryW: Dr. Merry is always so helpful at our KDA conferences

Dr__M: I didn't realize so many with KD had restless legs syndrome.  I think that a better understanding of this will definitely come out of Dr. Fischbeck's clinical trial.  He is doing a good job of putting together a natural history of the disease and this includes other symptoms, I believe.

mobiusloop: Thank you Dr. Merry, I learned a lot this morning.

doughboy: Kelly are you coming to the conference? Are you Arofer? AROFER, where are you from?

Alexandre: What is Dr. Merry specialty?

ARofer: Allan Rofer from Felton, CA

TerryW: I think my mom had Restless leg syndrome. Because she kicked me out of the house

TerryW: Kidding

TerryW: LOL

jameson411: I have had the rls (restless legs) for over 10 years.   (i am 34 yoa).

jameson411: lol

jameson411: good one

Murf: LOL

Butch: LOL Terry---

Dr__M: I know that in Montreal and in Vancouver there are large groups studying KD and HD.  I'm sure how well it's taught but I would assume it is.

jameson411: other rls sufferers?   How long?

doughboy: Thank you Allen, TJ Noack, AKA; Mountain Man

kellyC_: Really thats great to hear Dr-M

Butch: I have RBS-- Restless butt syndrome--hate sitting so much.

TerryW: any last questions for Dr. Merry, we have her for 5 more min

kellyC_: No Doughboy.I cant make the conferance this yr..... Cant afford it! but thanks anyways

Dr__M: My PhD is in molecular genetics, but my training includes neuroscience, biochemistry, cell biology and pathology.  We use a multidisciplinary approach to study KD.  I'm in the Biochemistry and Molecular Biology Department, but am also heavily involved with the neuroscience and genetics communities.

Butch: Very impressssive Dr M.

Dr__M: Yikes, this has gone too quickly!!  I have really enjoyed spending this morning with you all. We'll have to do it again soon.

Murf: And A Great Lady

ARofer: Dr Merry rocks!

jameson411: Thank you.

TerryW: Thank you very much for joining us

MICH: Speaking of chemisty are there medicines a person with KD definitely shouldn't take,,, say Zicor.... or Viagra?

Dr__M: Thanks so much to all of you!!  I look forward to seeing you that can attend in Las Vegas.

jameson411: oh, nice question.

Alexandre: Thank you Dr. Merry for your nice explanation. I hope can meet you all at the KDA Conference. Greetings from Brazil!

Murf: We always love hearing from you

Dr__M: I'm not sure about these specific drugs, but will forward your question to Dr. Fischbeck.  I will see him in about a week at Jefferson.

doughboy: Aleandre, are you coming to the conference this year?

Alexandre: Yes I intend to be there, if God permits me...

TerryW: If there are no further ?'s for Dr. Merry we can let her go

Murf: Thank you Dr_M for everything you do

MICH: Dr. Merry, thank you for all your info

mobiusloop: Thanks again Dr M

TerryW: Dr. Merry, Thank you very much again. See you ar the KDA conference