Kennedy's Disease Association

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Kennedy's Disease Chat Transcript  03-07-2009

Topic:  Special Guest - Dr. Kenneth Fischbeck NIH/NINDS

Host: Mike Goynes


TerryW: Hello
loshimo11: Good morning Terry. This is Luis Shimomura joining in from a sunny San Francisco.
TerryW: Hi mike
MikeG: morning Terry & loshimo11
loshimo11: Hello Mike. How are you this morning?
MikeG: ok, it's finally warming up here in FL
MikeG: been a bad winter here!
loshimo11: Will be right back... gottta get some caffeine.
MikeG: although nothing like up north...
MikeG: thanks for getting the word out about the chat today Terry. I hope it wasn't too late for everyone.
TerryW: we will see, no problem
TerryW: hello Bob, Bill
loshimo11: M back
billeric: Bood morning fellows
MikeG: morning guys
billeric: good!
loshimo11: Good morning everyone.
MikeG: morning Ron
Ron: Good morning to all. Just a beautiful day here in Penna.
MikeG: morning Bruce
Bruce: Morning
MikeG: I think spring has sprung...
MikeG: morning Lou
loutudor: Good morning!
Bruce: Yes, the birds are building nests and the trees are budding and blooming. That is a good sign.
Bruce: Morning, Lou
MikeG: good morning Dr Fischbeck!
MikeG: we're so glad you could make it this morning.
fischbeck: Godd
MikeG: morning Gary
gary_kc: Good morning. This is Gary joining from Kansas City. It is warm days in mid-west this week.
gary_kc: Hi mike
MikeG: so, the one thing that everyone is chomping at the bit to hear is: Has anything positive come outt of the Deutasteride trial?
Bruce: Kurt, one of our associates in the UK said that he has been having Electrical Muscle/Nerve Stimulation to treat the Muscle Wasting caused by Kennedys Disease. He has seen some positive results. Have you heard of any success with this typw program?
TerryW: I am not sure whats going on with Kurts connection
CER: Good morning from WV - beautiful day here also.
MikeG: Kurt, are you having connection problems?
loshimo11: I saw some type of gizzmo on HSN last week which they were advertising as a nerve stimulation. Kinda caught my eye. May not be the same.
Bruce: I will resend this earlier question. Kurt, one of our associates in the UK said that he has been having Electrical Muscle/Nerve Stimulation to treat the Muscle Wasting caused by Kennedys Disease. He has seen some positive results. Have you heard of any success with this typw program?
Dart!: Now THAT'S a different welcome!! G'morning ALL!
TerryW: I am not onmy PC, I am on hte laptop so I have no acces to e-mail right now. fyi
rcborden: To all, if you are having trouble figuring out how to type into this system, the place where you enter is the light blue bar at the very bottom of the screen, immediately to the left of the 'send' button.
rcborden: Bob
TerryW: ty bob
loshimo11: Are we having problems?
fischbeck: I 'm writing now. Sorry, I couldn't fuigure out the new set up until Mike called & explained it to me. KF
MikeG: morning Ed
Ed M: Hello everyone
Bruce: Morning Ed
fischbeck: We're still in the midst of analyzing the data for the dutasteride trial.
fischbeck: I haven't heard of other experience with the electrical stimulation. I think at this point it's fair to say it's untested.
Bruce: Is there any other KD research taking place right now that looks interesting?
TerryW: Dr. Fischbeck, Gary_kc sent me a link to some posted results that were posted on 3 mar. do you know of them?
TerryW: Gary, do you have that link available
fischbeck: maria pennuto and Isabella palazzolo in our grouop have some nice results with IGF-1 in mice.
MikeG: morning Paul
PA-PAUL: Morning mikeg
fischbeck: What are the posted results from mar 3?
gary_kc: My friend who has SBMA and participated in the trail sent me the Phase 2 result published by Nagoya university as following link. Please type the following search key in the search field: Phase 2 trial of leuprorelin in patients with spinal and bulbar muscular atrophy You will see the abstract.
Bruce: The mighty mouse (IGF-1) sure sounds encouraging.
MikeG: How about J9, do you think it will be possible to set up a trial for it? Is it as good as is reported?
dsproul: What?
Bruce: Morning Murray
fischbeck: Ah, yes, I know of the Nagoya results. They found an effect on swallow time in the randomized phase of their trial, but it's not clear that it is clinically meaningful
MikeG: morning Murray
murf: morning
fischbeck: Hi, Murray
murf: morning Kurt
fischbeck: ASC-J9 still needs more animal testing for safety before it can be used in patients.
dsproul: At 82 years I'm running out of time.
Ron: What kind of animal will you do testing on. I'm a dumb ass. LOL
Bruce: The hype on ASC-J9 has been pretty big and we seem to be receiving an email a week by someone asking for where they can buy it and what dosage to use.
MikeG: :D
TerryW: My wife calls mme an animalsomtimes, so I am ready :)
MikeG: good one Ron
TerryW: darn laptop keyboards
fischbeck: at the same link above you can find an editorial we wrote that is being published with the Nagoya study. In the editorial we tried to put the study in perspective. I think it's an important step.
PA-PAUL: Dr Fischbeck,, correct me please Did Japanese trial of castration prove sucessful ???
fischbeck: We have several other ideas for clinical trials: 17-DMAG or 17-AAG, IGF-1, idebenone, exercise ...
MikeG: that's a bad word Paul!
Ron: Just to let you know, I was on the dusteride during the clinical trial. I definitely got alot weaker after going off the dusteride. Anyone else notice the same?
dsproul: Doctor Fishbeck, Is there any way to separate symptoms of severe lumbar disc degeneration from KD symptoms?
fischbeck: The japanese trial used leuprorelin (Lupron). It's a drug that reduces testosterone, not quite the same as castration! I'd say thay found evidence of an effect, but not yet convincing proof of benefit.
loshimo11: Were the official results from the Japanese Leuprorelin ever published?
fischbeck: Lumbar disc disease usually causes more pain than KD, and it's more localized, i.e., it just affects the loweer back and one or sometimes both legs whereas KD also affects the arms and face & throat muscles.
MikeG: Exercise is something we can all do now thanks to Bruce's wonderful Exercise Guide!
fischbeck: the official results of the japanese trial are just out as an advance electronic publication in Annals of neurology at the link above (search on Sobue) as is our accompanyiong editorial (search on Fischbeck). If you have trouible accessing it, send me an email at This email address is being protected from spambots. You need JavaScript enabled to view it., and I'll send you a pdf.
Bruce: When Maria and Isabella reported about IGF-1, it sounded very promising for slowing the progression if started early in the mouse's life. Is that still correct?
rcborden: Dr. F, do you have an idea of when the dutasteride results wll be released?
dsproul: Thanks, that would indicate that my left leg and foot weakness is back related. But what about foot and leg nuropathy-tingling-burming-cold-numness episodes in both legs?
gary_kc: loshimo11, the following is the link:
gary_kc: and then, type in Phase 2 trial of leuprorelin in patients with spinal and bulbar muscular atrophy in the search key field.
gary_kc: You will see the abstract.
loshimo11: Thanks Gary
fischbeck: Yes, the IGF-1 effect looks good. Mara & Isa are doing a few additional exoperiments requested by reviewers for the journal Neuron.
Bruce: Kurt, you mentioned the following possible trials: 17-DMAG or 17-AAG, and idebenone. Can you explain these in layman's terms?
TerryW: I just changed health care plans and got a new Doctor which I saw for the first time yesterday. I started telling them about KD and they looked at me llike I was an alien. They did not have a clue :) training now begins again
fischbeck: We hope to get the dutasteride results out to you all pretty soon, probably in the next few weeks.
rcborden: thanks
Bruce: The expense of a trial must be staggering. Will the current economy and NIH funding cuts constrain the number of trails on rare disorders?
fischbeck: You can direct your doctor to the KDA website, or have him or her get in touch with me with any questions. Neurologists & particularly neuromuscular specialists (e.g., MDA clinic docs) should know about it.
Ron: My experience with the dusteride was that it helped me from getting any worse. Not necessarily making things any better.
TerryW: I took them a brochure
dsproul: I just got kicked off site!
fischbeck: KD can cause numbness and tingling in the feet.
TerryW: if you click on hte link while in chat it will take you away from chat
Ed M: I was kicked off as well, and I had not click anything
TerryW: hmmmm
loshimo11: I was also kicked off.
TerryW: technology, you gotta love it
fischbeck: Other questions?
loshimo11: Dr. Fischbeck... I just recently began experiencing both numbness and tingling in the feet at nightime.
Bruce: The expense of a trial must be staggering. Will the current economy and NIH funding cuts constrain the number of trails on rare disorders?
fischbeck: Yes, KD affects the sensory nerves. Over 895% of the patients in our study had abnormal sensory nerve conduction on electrical testing, but most people don't notice it, or at least don't seem to be troubled by it.
loshimo11: I went to see my neurologist a few weeks ago and mentioned the expense of trials. He suggested tapping on the resources of the MDA. He mentioned his clinic had just received a sizeable grant without too much pain.
Ed M: The soles of my feet have been numb for several years and the numbness is spreading.
fischbeck: The funding for our trial came through the NIH, and the drug was provided free by GSK. In Japan, the patients had to pay for the drug themselves
Ed M: I wondered if the numbness might have been due to running.
Bruce: I love the pin prick tests. They can do a lot of sticking without much impact.
billeric: I have numbness in my feet after standing for awhile. Didn't realize it was KD related.
loshimo11: Bruce... That kinda sounds painful.
fischbeck: Yes, the MDA provides funding for KD research. Mara Pennuto has a grant from them, as have others.
Bruce: Not if you have KD and lost most of your sensations.
fischbeck: That should be over 95% (not 895%) of KD patients have sensory nerve involvement.
Bruce: Kurt, you mentioned the following possible trials: 17-DMAG or 17-AAG, and idebenone. Can you explain these in layman's terms?
dsproul: I'm scheduled for a Rhizotomy on the 16th Will see how that works out.
loshimo11: Dr. Fischbeck... Is there anything from an individual effort that we can do to get financial assistance to fund these trails from the MDA?
dsproul: I'm hoping that at least the back pain will be controlled
Ron: I must go for now. Stay healthy till next we chat.
fischbeck: 17-DMAG and 17-AAG have been shown by the Japanese group to be benficial in KD mice. When last I checked with them, they weren't planning to pursue these drugs with clinical trials, because they are focusing on leuprorelin. The drugs are made be the National Cancer institute here at the NIH, and have been used in cancer patients, so we are thinking of using one or the other in a KD trial. 17-AAG is given by injection and 17-DMAG can be taken orally.
dsproul: studys should lead to treatment---?
fischbeck: It's good to support the MDA and encourage them to support KD research. KDA funding has been put to particularly good use over the years in helping researchers get started. And of course we appreciate your supporting the NIH with your tax money!
fischbeck: It's our hope that the animal and clinical studies will lead to safe and effective treatmnet. The evidence so far indicate that anti-androgen treatment may help at least a little.
Bruce: We are learning that Research is a long process and those of us that are a little older are a little less patient.
dsproul: A little is allI need to spend a year or two with my great grand children
dsproul: Twins born yesterday!
Bruce: Congratulations!
MikeG: anti-androgen meaning deutasteride or J9?
fischbeck: It does take a lot of time and effort to develop new treatments, but there are good people working on it. A little benefit is good, more would be better!
Bruce: Well, I cannot say it often enough. We are very appreciative of all that you and the entire research community do for us.
fischbeck: Anti-androgen means leuprorelin/leuprolide and dutasteride.
loutudor: :} Is the anti androgen easily available?
fischbeck: These drugs are avilable by prescription, but we still can't say that they have been shown to be effective.
dsproul: accross the board effective or selectiely effective?
loutudor: Why are neurologists in this field not current in treatments?
dsproul: selectively
fischbeck: In our data analysis we are looking to see whether dutasteride had an effect on all patients, and if not whether a subset may have been more responsive (e.g., patients who are more or less severely affected). The Japanese have done a similar analysis with leuprorelin.
rcborden: It appears that Obama will allow stem-cell research. What does this mean for us? Is there any interest in this for KD?
dsproul: My brother and I have late-onset KD. Can that be a sub-set?
Dart!: I appreciate, Doctor, your candid opinion regarding the effectiveness of drugs being used, not holdding out questionable hopes!
Bruce: Looking for commonalities in the patients must be difficult. Does it rely heavily on captured background data?
fischbeck: Sometimes it's hard for neurologists and other docs to stay current on treatment. There are information sources available, and it's good to encourage us all to look things up or get advice from experts if we don't know.
Dart!: What do you make of the harvesting of stem cells from the patient's skin?
loutudor: Our neurologist appears to be interested only in what takes place at his university research hospital. He asks me to keep him informed on anything else.
fischbeck: I think it'sd good to encourage stem cell research, but this is more of a long term than a short term remedy. Hard as it is to dvelop drugs, at least what needs to be done is clear. Gene therapy is another possibility to pursue.
fischbeck: Late-onset KD is a subset that may respond better to treatnment. We'll look at that.
dsproul: My eurologist seems to take a well youv'e got it and You are doing better that I expected I'll see you next tyear
Dart!: Do you reckon that stem cells harvested from the skin would have positive effects?
loutudor: I am hopefull for gene much going on for KD?
fischbeck: i think the results with deriving stem cells from skin are encouraging. It's getting the cells to replace nerve or muscle cells that's difficult.
CER: Dr. Fischbecl and your staff - This is Charlie from WV. I know all of us KDer's are so grateful and thankful to have you working so hard trying to find a way help us. We surely appreciate your efforts....
MikeG: Thank you so much Dr Fischbeck for taking the time to come chat with us on your Saturday. We really appreciate you and all that you are doing.
dsproul: Yes' thank youfor the up-dates on research programs
Bruce: Amen to that!
fischbeck: The approach to gene therapy is to knock down the mutant gene with RNA interference. The results with this approach look good for related diseases (ALS, Huntington's disease) in animal, and human trials are now planned for these diseases
billeric: Well said CER. We are lucky to have dedicated folks like Dr. Fishbeck.
fischbeck: it'
fischbeck: It'
loshimo11: Gots to go. Thank you doctor and to everyone please stay healthy and vertical.
MikeG: cya loshimo11
loutudor: I learned alot this morning...Thank You
fischbeck: It's good to talk with you guys. Now that the NIH computer security system has allowed me to hook up, I'd like to do it again soon, maybe with the final results of the dutasteride trial. And our plans for the next one.
Bruce: You can count on it Kurt
MikeG: that would be great! THANKS!
Dart!: T'is good to have you aboard and on our side, Dr. F. Thanks for your dedicatrion and time!
fischbeck: OK. Closing time. Talk to you all later!
murf: Thanks Kurt I'll have to read the transcript
MikeG: gotta run too - take care everybody! Until next time - stay vertical!!!
Bruce: Take care everyone
gary_kc: Thank you very much Fr. Fishbeck.
CER: Bye
gary_kc: Bye
murf: cya later guys