Kennedy's Disease Association

A Public Benefit, Non-Profit Organization

"As a mother with a son who has KD, I appreciate those of you who are so dedicated to informing the public of this rare and difficult disease."

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Kennedy's Disease Chat Transcript  07-18-2008

Topic:  Special Guest - Dr. J. Paul Taylor

Host: Mike Goynes


BEGIN CHAT

MikeG : morning Dr. Taylor
TerryW : "Most of you regulars know KellyC in here,  His twin that had KD passed away this week"
jpaultaylor : "good mornig all, thanks for inviting me"
MikeG : you're early as several others...
TerryW : Paul remember to use the drag & drop test feature if you are on a MAC to copy large amounts of text into the test box
poohsdaddy : Was his passing related to KD orr another condition?
MikeG : "well, it's close enough to 10:30 for me... we are very pleased to welcome Dr. J Paul Taylor to the chat this morning."
TerryW : "Pooh,  Kelly thinks so but the toxicology report won't be back for 5 weeks , he found him alrady passed away in his apartment"
MikeG : "Dr. Taylor was raised in California and graduated from U.C Davis. He went to Philadelphia in 1987 for MD/PhD training. He trained in Neurology at the Hospital of the University of Pennsylvania and in Neurogenetics with Dr. Kurt Fischbeck at the NINDS. In 2003, he returned to join the Neurology faculty at Penn and start his own research lab. He's been a neurologist for ~10 years, and has run the neurogenetics clinic at Penn for the past 5 years where he frequently saw patients with Kennedy's disease.  He also supervises a research lab studying neurodegeneration where one of the main focuses is Kennedy's disease.  Two weeks ago he moved his lab to St. Jude Children's Research Hospital in Memphis, TN.  The focus of his lab remains the same and with the resources provided by St. Jude they will be able to redouble their efforts."
jpaultaylor : "My family is tryingto adjust to the heat down here in Memphis, but I'm doing very well thanks. "
Ron : Dr.  Taylor--it sounds that your move to Memphis is a very positive move. Congratulations.
MikeG : "Dr Taylor, could you briefly describe how you will be using resources at St. Jude to continue your work with KD?"
jpaultaylor : "thanks, yes, I think it's a good thing. I was very happy at Penn, but the resources at St. Jude cannot be matched."
PA-PAUL : Dr Taylor Is someone else picking up your KD patients here
Gary_KC : Good morning. This is Gary Uchiyama joined from Kansas City. It is a nice weather today.
craig_h : "oh, so your move did not mean you are leaving KD research?"
jpaultaylor : "Yes, Dr. Lauren Elman is picking up my KD patients at Penn. She is a fantastic young doctor and was eager to take this on."
jpaultaylor : Moving to St. Jude will enhance our research in several ways...
PA-PAUL : Dr Taylor what is the best way to contact her?
jpaultaylor : "First, they are in the financial position to support our research whereas at Penn my lab was run 100% by outside grants..."
jpaultaylor : I  can help put you in touch with Dr. Elman. Just contact me at This email address is being protected from spambots. You need JavaScript enabled to view it.
Gopher : Dr. Taylor can you refressh our memories on your research work?
jpaultaylor : "the resources at St. Jude will allow us to grow bigger, to more personnel..."
jpaultaylor : projects should move faster...
MikeG : great!
jpaultaylor : I will spendless time fundraising
craig_h : should we all plan Memphis vacations now ?
jpaultaylor : To give you some insight into what it's like to runa medium size research lab in these funding times: I submitted 46 grants in the last 48 months
MikeG : wow!  That's wild...
PA-PAUL : were all 48 to do with kd
MikeG : "well, the KDA granted you at least 2... :)"
MikeG : the transgenic flies were really great!!!  Quite the video!
jpaultaylor : the other big change at St. Jude is some of the technical capabilties here. I really want to push translational work - finding drugs - and they have an entire department devoted to this caled chemical biology. The chair of that department is an expert in finding drugs that alter the function of the androgen receptor. I found this very appealing.
Bruce : "Paul, this all sounds like a huge step forward for your research and staffing.  Again, congratulations."
jpaultaylor : "Yes, the KDA grants were essential, especially the first one...without that grant I would have had to let Udai go and we never would hav e finished that Nature paper on HDAC6"
jpaultaylor : You all ought to know the payoff on that grant has been huge in two ways...
jpaultaylor : "first, that grant allowed us to complete the Nature paper which led to NIH funding of about $2 million for furher work"
jpaultaylor : "second, it established the importance of HDAC6 as a therapeutic target that we hope to exploit"
Dart! : That is SOOOOOOO inappropriate that concientious researchers have to grovel for needed funds!!!
jpaultaylor : "there is also a ripple effect...after that paper came out, several labs contacted us to request KD flies so they could work on them too, so now the effort has grown larger"
Bruce : We are pleased it worked out for you and for the KDA. 
murf : There you go ... no one should call us a small association. We pack a big punch
PA-PAUL : Dr Taylor is that theraputic target the same as the androgen receptor
jpaultaylor : ...and many labs have started working on HDAC6 too so the understanding of that gene will go faster
jpaultaylor : "I shouldn't be so oblique...in that paper we showed our fruit fly model of KD. Basically, flies expressing androgen receptor (AR) develop KD when fed testosterone. We also identified  gene (HDAC6) that regulates the degradation of toxic AR. Thus, we could rescue the flies by increasing HDAC6."
poohsdaddy : What does/will this mean to KD patients?  a reason for greater expectations??
MikeG : "Yes, it was a very revealing study to see how the eye of a fly could manifest results of KD - we could see KD for the first time!!!"
craig_h : "by escue"" the flies?   cure? help ? what ?"""
jpaultaylor : i feel like we're making incremental progress towards therapy...it's frustrating because everything just takes so long...
Ron : "I don't know just how many of us are in the clinical trial but, I suggested to Angela that they continue the daily log for several weeks after the end of the two year study. I have been feeling weaker since no longer taking the test drug."
craig_h : "Ron, I've actually felt much better after I finished the trial"
murf : Terry could you let me know when this chat is posted? I'd like to send out a note to Halliburton letting them know where their was used.
Darwyn : "using on screen keyboard, still having trouble withit."
murf : cool
jpaultaylor : but we have been using the flies to identify promising compounds and we have found several that reduce toxicity in this model
poohsdaddy : "Ron, sounds like a good idea to continue diary"
jpaultaylor : we've also identified several gene targets to exploit for developing drigs
Ron : Angela was going to talk with Dr. Fischbeck to see how he felt about it.
Gopher : For those of us not in Trial...what kinds of things were kept in diary...good tracker...
jpaultaylor : "by escue"" I mean greatly reducing the degeneration. ook at this page: http://www.med.upenn.edu/taylor/autophagy%20and%20the%20UPS.html"""
poohsdaddy : I have tried to include more information than just the NIH pills.
murf : every thing we took every time we fell how we felt etc etc
TerryW : I am away from keyboard
PA-PAUL : thanks Dr Taylor i save the page for after chat
Gopher : Good idea for everyone...Thanks Murf
Bruce : "Paul, how is Udai's project coming along?"
Gopher : Dr. any ideas for KD patients that may be helpful
Bruce : "For those that aren't aware, the KDA gave Udai a grant last year for his KD research."
jpaultaylor : "Udai's project has come along well. In fact, we just combined his latest data with a collaborator and submitted it for publication."
Bruce : Great!
Bruce : "Paul, will Maria be moving with you?"
poohsdaddy : "I kept a brief record of all daily meds, pain level, weakness, falls, dr. appts, etc."
TerryW : I need to run - talk with you all later - thank you Paul and everyone for helping support the KDA.
murf : don't fall
Ron : Dr. Taylor--It is so great that you and other Dr. researchers are still pushing for the cure for KD. You don't know just how much it means to us knowing that a cure is just around the nexzt corner. THANKS!
PA-PAUL : Amen yes thanks
Gopher : "Yes, dr. Taylor thanks!! "
jpaultaylor : "I woud love for MAra to come, but she is staying at Penn for the next year. I am maintaining a lab at Penn with a small staff for those who couldn't move for personal reasons. Mara is one of the 3. I expect she will get a faculty job in the next year."
jpaultaylor : Mara is working closely on a project with Natalia that I am very excited about
jpaultaylor : they are getting down to the nitty gritty of how AR causes toxicity
poohsdaddy : " I'm not expecting a cure, but a combination of diet, exercise and meds to cope with eeffects of KD."
jpaultaylor : I really admire the effort these two are putting in and how well they work together ego-free
Ron : So long Terry--glad that you can still RUN! Go for it. Stay healthy.
Bruce : That is great to hear
MikeG : that's important!
jpaultaylor : "withut getting too deep in the weeds, the data developed by Mara and Natalia indicates that only a very minor alteration in AR function is responsible for all the problems (i.e. KD)"
Bruce : "Paul, at an earlier chat, we spent a little time discussing the sensory (touch, feel, etc.) problems that many of us are experiencing with KD.  Have you seen any of this in any of your patients?"
Gopher : Is alteration in AR function related to repeat number?
jpaultaylor : "yes, definitely, it became a regular question I asked after the chat, and every one of my patients reports this"
Ron : Dr. Paul--What are your thoughts on stem cell?
Bruce : "To me, this is one of the major issues with standing ... not receiving the signal that I am falling or losing my balance quick enough for the muscles to react."
jpaultaylor : "Gopher, we think expanded repeat number causes a sloght shift in AR function, yes"
MikeG : "That's exactly right, Bruce."
MikeG : Balance!
MikeG : and sensory feedback.
jpaultaylor : "yes, Bruce, I agree, the loss of somatosensory information complicates activities we take for granted like maintaining balace while standing or walking"
Bruce : I have burned myself when picking up something very hot because I didn't know it until it was already in the air.
MikeG : "Paul, do you think that the ASC-J9 compound will have any positive results with KD?"
jpaultaylor : I'm prettty excited about ASC-J9...
jpaultaylor : Last we chatted I was lamenting the fact that another researcher was unwilling to share the compound with us...
jpaultaylor : "well, we found a work-around..."
MikeG : GREAT!
Bruce : Wonderful!
MikeG : the ego thing was defeated?
jpaultaylor : there is a company outside this country that makes the compound- I stumbled across the website one day while surfing - and we were ale to purchase it...
MikeG : FANTASTIC!!!
jpaultaylor : we have tried it on our KD flies and it works great
Bruce : Even better
PA-PAUL : can we purchase it????
Ron : Seems to me the more shared research may result in greater findings to help find the cure.
poohsdaddy : "Bruce, is there more information to explain what this all means? in layman's terms?  I don't understand a lot of the discussion."
jpaultaylor : this is definitely not a compound I would take without clinical trials preceding it
Bruce : Wasn't there an issue with the amount of the ASC-J9 that was expected to be needed by a person to be effective?
Darwyn : I find my touch sense is exagerated. i.e. slight rub on feet hurts.
jpaultaylor : we're just beginning to understand how it works and I'm concerned about possible toxicities
murf : and the FDA might have something to say too
Bruce : "Yes, look on our web site for the Dr. Chang KDA grant information.  It will explain it.  There has been several reports on the web since then explaining the possibilities of using this for KD."
MikeG : Do you think a clinical trial is on the horizon for it?
Bruce : "Just Google ASC-J9"""""
poohsdaddy : Thanks!  I'll have to check it out later.
Bruce : "As you can tell, Paul, everyone is our boat is looking for a quick solution to this age-old problem."
jpaultaylor : I am not aware of a trial for asc-j9
Bruce : "The last we heard, it was still some time off because of several issues."
MikeG : how about HDAC6?
jpaultaylor : HDAC6-based therapy is even further off than asc-j9. we are setting up an assay to find drugs that target it
Bruce : Is this where the new lab at St. Judes will help with your research?
jpaultaylor : "we are getting to a an interesting point, however..."
Ron : Sorry guys. I must go for now. Stay healthy till next we chat.
MikeG : "Ok, thanks.  We know that things are moving... just seems like we're all too anxious to see a cure... :)"
jpaultaylor : "we (the research community) have developed multiple animal models in cells, fies, and mice..."
jpaultaylor : this has led to significant insight into the molecular mechanism of KD degeneration...
jpaultaylor : and will lead to an increasing diverse stockpile of promising strategies and even promising compounds like asc-j9...
jpaultaylor : then we are gonna get stuck.
jpaultaylor : because further throughput requires human data...
MikeG : everything takes time...
jpaultaylor : and at present we do not have an organized approach to prioritize clinical research in KD
PA-PAUL : shouldn't be a problem I know where plenty of human subjectsa are....
murf : that's what I'm here for ... human ginepig
Bruce : "Paul, do you see that changing in the near future (organizaed approach)?"
Gopher : The thing to remember is You have come a long way with the research and we are grateful
poohsdaddy : "Are you looking for patients to participate in your research? or limited to flies, mice, etc."
PA-PAUL : what can we do to help it change faster
jpaultaylor : "this has been appreciated in efforts to treat Huntington's disease, Parkinson's disease, ALS and those rganizations have established mechanisms to prioritize promising therapies and advance them through cinical trials..."
jpaultaylor : this needs to be a grass roots effort
jpaultaylor : with cooperation from reseachers
jpaultaylor : "if the researchers initiate it, turf wars develop...I've seen this"
Bruce : "In other words, it needs to be led by someone else ... perhaps NIH, NORD???"
jpaultaylor : although KD is a bit different for several reasons
PA-PAUL : "can we patients"" develope it"""
murf : most of you came from Kurt's lab
jpaultaylor : smaller demographics and more collegial community
poohsdaddy : "I agree with Murf--as to being a human ginea pig for continued research,"
jpaultaylor : I encourage you to look at the model developed by some other groups like FSMA
Bruce : "Okay, Paul, can you send me some more info on this and we, the board, will look into it."
jpaultaylor : ok
PA-PAUL : "model for \"" established mechanisms""""  ??"""
PA-PAUL : yes i will volunteer to help
Gopher : Thanks Bruce
PA-PAUL : yes thanks Bruce and Dr Taylor
Bruce : "Paul, as usual, your chats are always wonderful.  You give us hope and share your knowledge. "
Bruce : We all appreciate what you and your lab does.
jpaultaylor : "no problem, thanks for having me"
Bruce : "Of course, we wish you, your lab, and your family the very best in your move."
MikeG : "Thank you so much for being our guest on this chat, Dr. Taylor.  We really do appreciate you taking time out of your weekend to be here with us!"
jpaultaylor : thanks for that too
jpaultaylor : bye everyone
Dart! : GOOD JOB ALL!! Thaks for being here.
PA-PAUL : good by
Bruce : I will share some of your thoughts today with the board.
PA-PAUL : bruce if you need help Holler
Dart! : 'Thanks'!
Bruce : We are always looking for new and better ways to move this process along.
Gopher : Thanks Board for all you do!!!
Bruce : "Thanks, PA-Paul"
UTE : "Yes, Dr Taylor and the Board your great!"
Bruce : "Paul, we'll be in touch.  Thanks again for everything today.  I have to run to prepare for the meeting in 25 minutes."
poohsdaddy : Thanks to all; hope to be back in two weeks. Bye.
MikeG : me too - thanks again!!!  cya
Gary_KC : Thanks Dr. Taylor and the KDA board
END CHAT