Kennedy's Disease Association

A Public Benefit, Non-Profit Organization

"Back in the late 1990s, I felt alone. I had just been diagnosed with Kennedy's Disease. I came across the KDA and attended a Saturday chat. What an eye opener! There were two dozen others on the chat that were going through what I was experiencing."

Archives - 2015 and Older Transcripts

Topic: Novaritis Clinical Trial
Guest: Kurt Fischbeck, M.D.
Host: Ron Moffett  
     
Hugo Rodriguez Good morning Paul, how do you doing?
Dan Brindle   Hi everyone
Hugo Rodriguez Hi Han
Hugo Rodriguez Hi }Dan
bcpaul   Morning to you too Hugo, and Dan; only half hour early, but I'm almost awake
Dan Brindle   Almost awake, eh?  What is your time zone?
bcpaul   i
bcpaul   I'm on the west coast, just after 7 here
Ron M   Good morning everyone
bcpaul   first time i've slept through in months (naturally) had to be woken up
BruceG   Morning Everyone
Hugo Rodriguez In Queretaro we have the Center Time
Ron M   morning Bruce
Ron M   Hugo, hate to ask but how is the weather where you are at
Hugo Rodriguez No problem, right now the "summer" is starting. Well not the summer but the hot season is coming.
Ron M   Thats what I though. In Chicago, the weather has been very cold and snowy this year.
Hugo Rodriguez Yeah, we see impacting photos ath
Ron M   Bruce, how is Georgia? I know you guys have had some recent weather?
Hugo Rodriguez at the news, all covered with snow.
bcpaul   we reached +9C here ystrdy, -1C now and threatening snow
Ron M   Yes, not sure if the snow is going to melt anytime soon
Ron M   bcpaul, where you located at?
Hugo Rodriguez I hadn't used the celsius scale, but as Paul did, I will say that almost all January we had temperatures close to 0 C, as the minima and not higher than 19 C.
bcpaul   Vancouver island, 100 mi north and alittle west of Seattle
BruceG   Vancouver area was one of our favorite places to visit.  We loved sailing the San Juan Islands.
Ron M   Very nice. bcpaul, I image you are used to winters then
bcpaul   Out here every winters different, this has bee longest cod dry stretch in 30yrs, ending in much needed rain and snow
bcpaul   local mountains got 3ft in 24 hrs this week
BruceG   Morning Kurt
Ron M   That is a lot of snow. Do you ski bcpaul
BruceG   Thank you for joining our chatroom today.
fischbeck   Am I late/ Thought we were going to start at 10:30 ET.
BruceG   No, not late.  Some just sign in early.
bcpaul   Does sliding on you butt count :)
Ron M   Good morning Kurt, thanks for taking the time to join us
BruceG   We officially start at 10:30 EST
fischbeck   Glad to be here
Ron M   no, that would not count. that is what i do
BruceG   We have all been discussing this winter  -  how different it has been.
fischbeck   It's cold enough here in Bethesda.
Ron M   Kurt, glad you were able to get on the chat.
fischbeck   Any early bird questions? We are just starting an NIH trial with the drug from Novartis
BruceG   Yes, can you give us a background on the Novartis trial?
Ron M   Good morning UTE
bcpaul   how far are you casting your net for participants?
Gary_KC   Good morning. This is Gary joining in from near Kansas City. It is 4 degrees with cloudy this morning.
BruceG   Morning Gary
Ron M   Good Morning Gary
Gary_KC   Hi Bruce and Ron.
Ron M   Good morning David
david   Good Maorning all
BruceG   Morning Mike
UTE   Good Morning all
Ron M   Good Morning Mike, glad you could join us
MikeG   howdy from Daytona Beach
fischbeck   Sure. Our trial is with a compond called BVS857 that Novartris has under development that activates the IGF-1 pathway. It fits with studies in our group at the NIH that have shown a benefit of IGF-1 in mouse versions of KD.
Ron M   Mike - don't rub it in.
Ron M   Plenty of us are freezing
MikeG   howdy from Daytona Beach
Ron M   Good morning jdfish
fischbeck   We are just starting with 2 KD patients from the area to make sure it's safe. The first is due toi get the first dose on Tue
BruceG   I guess it is that time of year, Mike
MikeG   i'm on my mobile laptop in the car heading to Tampa... gonna be a challenge
Ron M   Good morning sfurman
fischbeck   We'll be recruiting 6 more in July (if all goes well) and another 30 next January.
BruceG   So, is BVS857 a IGF for muscles or something different?
sfurman   Goodmorning
fischbeck   BVS is a version of IGF-1 that should work on the muscles and also should help to protect motor neurons.
BruceG   Got it.  Good to hear this is moving forward.
jdfish   Good morning
Hugo Rodriguez IGF-1 trial, not BVS, is still on planning, is not?
BruceG   So the trial has 2 or 3 phases?  Is it a double blind trial?
fischbeck   It's pretty exciting for us to get to this stage, the result of several years of work with the mice in our lab and over a year & a half of working on it with the folks at Novartis.
Ron M   Dr. Fischbeck - when you say work on the muscles. Can you expand on that.
MikeG   how often do the patients get injections?
BruceG   It was exciting to read of the cooperation with Novartis.  That has to help move things forward more effectively and efficiently.
sfurman   is this drug expected to repair old damage or prevent new damage
fischbeck   The trial has 2 parts. Part A tests increasing doses for safety & tolerability. Part B tests the hiughest tolerated dose for efficacy oiver a 12 week period.
fischbeck   IGF-1 (and we expect BVS) helps to stimulate muscle growth. It also should help by increasing the breakdown of the mutant androgen receptor protein.
BruceG   What are the known side effects?
fischbeck   In Part A the patients will get injuectiuons with gradually increasing doses every 2 weeks. We currently plan to give injections once a week for Part B.
bcpaul   Will you be using thigh MRI's and liver studies to judge success?
Ron M   Good morning Gopher
fischbeck   Side effects are low blood sugar (like with insulin) at high doses. Also, IGF-1 (and BVS) at high doses have caused facial wekaness (Bell's palsy). These are two things we are trying to be carefulk about by keeping the doses low and advancing slowly. Also, we are only giving the drug to people with low IGF-1 to begin with. Most KD partients have low IGF-1.
BruceG   After you have a proper dosage, you expect to have 30 people on the trial.  Will that be 15 taking the drug and 15 taking the placebo?
fischbeck   Yes, in part B we will be using thigh muscle MRI as a way to confirm that the drug is increasing muscle growth. We'll also be testing muscle strength & function, and having patients fill out questionnaires to see how strong they feel.
bcpaul   interesting study out of Japan using tongue pressure as biomarker for deteriorating bulbar symptoms, as it is non invasive, developing a data base may be useful in your study too
fischbeck   We will randomize part B 2:1, that is, 20 on drug and 10 on placebo. The placebo is needed for comparison to test whether the drug is really working.
Hugo Rodriguez I was not weak enough to  participate in the Exercise Trial. In this condition, Am I able to participate in the BVS trial?
fischbeck   We are woking with the Japanese on developing a new outcome measure for KD, a functional rating scale.
jdfish   What do we need to do to see if we are able to participate in this trial?
fischbeck   For the BVS study we are taking patients who have some weakness and are still able to walk for at least 2 minutes, with or without a cane.
fischbeck   To see if you are eligible, you can contact Angela Kokkinis at the NIH.
bcpaul   Great Britain is also starting a clinical trial involving MRI and has just received permission to recruit through social media
Hugo Rodriguez I'm still able to walk a lot and for long time
Hugo Rodriguez I'll contact Angela
BruceG   Is there an expectation that if it is successful, weekly injections will be needed to keep BVS working?
fischbeck   Hmm. I don't know about the trial in Great Britain. Is it specifically for KD? The BVS trial will include several sites in Europe , in Italy, Germany, and Denmark.
BruceG   And a followup, can a person give himself injections (like a diabetic)?
fischbeck   Weekly injections is our best guess at present. We'll have toi see how long the drug stays in the bloodstream in this trial. We may wind up giving it more or less oftyen than weekly if it works.
BruceG   Thanks
fischbeck   Yes, the idea is that it would be given by injection, like insulin.
bcpaul   Yes the GB trial is KD specific, Team GB has been working with the researchers to allow its members to participate without having their neurologists or GP's submit names
fischbeck   I'd be interested to find out more about the GB trial. Is it being done by Mike Hanna at UCL?
BruceG   A selfish question, is it expected to work well for those of us more advanced ... and how about those just beginning to show symptoms?
Ron M   Good morning Stewart
BruceG   I will try to find out more, but what I have here is:  I have some GREAT news. I had an e mail today from Dr Helen Devine at UCL. The Ethics issues about Patient Selection for KD Research and Clinical Trials at University College London (UCL) have been resolved and we can now proceed to forward our Postal details to Dr Devine so she can send us the Preliminary/ Patient Consent Forms. If you wish to take part please send your name and address to.. This email address is being protected from spambots. You need JavaScript enabled to view it. ...See More
fischbeck   We decided to test BVs first in the range of patients who are weak but still able to walk, but the hope is that it would also help patients who are weaker and perhaps those who do not yet have muscle weakness (to slow or prevent the onset).
Ron M   Thank you Bruce
fischbeck   I'll heck with Dr. Devine. Does the GB study involve a drug? If so, do you know what it is?
jdfish   I'm sorry if you've already addressed this but what is the timeline for the study?
fischbeck   Sorry, I meant I'll check with Dr. Devine. I'm not a great typer!
UTE   Will it be helpful for people who can no longer walk without another persons assistance and use an elevated powerchair
Ron M   Good morning Connie
bcpaul   IGF has been used (abused) by body builders for some time, but my understanding is that it required substantial exercise to be effective to bulk up, is it suggested that BSV will stabilize existing muscle (neurons) without having to do constant strength training?
fischbeck   Yes, our hope is that BVS will also help patients who are wheelchair bound, but we'll have to see whether it works in patients who are able to walk first.
Gary_KC   bcpaul, you have a good question. :-)
fischbeck   Here's contact info for Angela: This email address is being protected from spambots. You need JavaScript enabled to view it., tel 1-301-451-8146.
BruceG   Kurt, it appears the UCL is a study of KD and not a trial:  "There is research being done at UCL. Two years ago a letter was issued to all the GPs in the land asking for KD patient details. Sadly there was no response and so there was no Patient register created. Prof. Greensmith was very suprised to learn of our existence on FB and equally suprised to learn we have such a collective of KDrs, Carriers, and Carers. Anyway to cut a long story short, Prof. Greensmith is going to speak with her colleagues to help us set up a registry and hopes that we may be able to start a research programme involving some of us over an initial 12 month period. This will involve attending UCL for evaluation and a full body tissue scan. After 12 months a second scan to see what changes and wasting have occured. Also if we arrange the venue, either herself or a representative from her Lab could attend two conferences a year to update us on their research and to answer any questions. She suggested one in the North of England and one in the South to avoid long journeys for us. I told her that we are involved in fundraising now and that I would be speaking to our commitee about raising funds to help support this research. No figures were mentioned at this stage. So thats the latest to date."
sfurman   Will participants have to travel to NIH for each injection during the study or can it be done locally?
fischbeck   Ah, OK, I have heard of this UCL study.
fischbeck   For safety reasons, we are having the patients in the US come to the NIH weekly for the injections and follow-up (we cover the travel expenses).
BruceG   You might have mentioned it, but ref. the 30, how long do you expect this trial to last?
BruceG   Oh, is BVS already FDA approved for other uses?
fischbeck   Re: the comment on IGF-1 in body-builders, we expect that exercise may help. We finished our exercise trial at the NIH in january, and we are still getting all the data together for analysis. We hope to have word on this soon.
fischbeck   Part B, with the 30 patients, will involve 12 weeks of treatment.
BruceG   So there is an expectation that improvement could be shown withing three months?
fischbeck   BVS is a new drug that has not yet been approved by the FDA.
Hugo Rodriguez As livng outside the U. S. do you think Dr Fischbeck that I could try to ask to my genetist to control and appliying the doses here, if I were included in the trial?, just to reduce expenses.
Ron M   When do you think you might have some preliminary results?
fischbeck   Yes, that is our hope, that we will see a benefit in 3 months.
BruceG   Great!
fischbeck   Outside the US, the best bet is to contact one of the sites in Europe, which should be coming online in the next few months.
Hugo Rodriguez Thanks for increase our hope!
BruceG   If BVS is effective, can I assume a person could go off Avodart at that time?
Hugo Rodriguez Wll
Hugo Rodriguez Well, I also live outside Europe. I live in Mexico.
BruceG   A followup ref. FDA approval.  Will the 12 week trial be enough to move forward with approval?
fischbeck   Our time frame is to finish Part A (which is just starting now) by the end of this year, and part B by the end of 2015.
BruceG   Kurt, can we use the information you provided today on the trial (on the KDA website under Research, for example)?
fischbeck   From Mexico, it would be difficult. If the current trial is successful, then there would probably be follow up studies in more places. There is a small chance the FDA would approve the drug if it has a strong effect in this study, but they usually like to have at least 2 positive studies for approval.
BruceG   Thanks
Connie A   have all of the particpants been selected
Hugo Rodriguez Thanks a lot for your info Dr. Fischbeck.
Connie A   Thank you, Dr. Fishbeck. This is very exciting and hopeful news.
BruceG   Besides the BVS study, is there other studies you are working on that you would care to share information about?
fischbeck   Yes, the trial has been posted publically on the clinicaltrials.gov website (you can search under "BVS857" or "Kennedy's disease") and on the NIH Clinical Center website. The company has not yet made public the details of the drugs composition, penbding a patent application.
MikeG   how do you stand on the ASC-JM17 study?
fischbeck   We have only selected the first two subjects for the BVS trial. There will be plenty of opportunity for patients to join in later this year and next, for those who are able to get to the NIH or one of the Europeanm sites on a weekly basis.
MikeG   soounds like it would be better for us to plan on taking a 12-week vacation in Bethesda...  :)
fischbeck   Laura Bott and Cartlo Rinaldi in our lab have been working on ASC-JM17 and ASC-J9 in mice, nad they have some interesting results. They were able to confirm that both drugs have a benefit in mice, although not as strons an effect as reported previously from Rochester. They are working out the mechanism of action, which should help in devloping a more potent drug.
MikeG   that's great news!
fischbeck   The weather here in Bethesda is nice in the spring, which hopefully will start sometime soon!
fischbeck   Sorry for my sloppy typing!
Ron M   Not a problem at all
BruceG   You are doing great, Kurt.  We appreciate you taking the time to bring us up to date.
Ron M   Any last questions for the Dr Fischbeck?
fischbeck   If anyone has further questions later, they can email me at This email address is being protected from spambots. You need JavaScript enabled to view it..
MikeG   I also understand that one may not be taking Avodart to be accepted in the study.
bcpaul   Please say Hi and thank your staff for all of their hard work too Kurt ,  it is appreciated
jdfish   Thank you for your time.  I'll be calling Angela on Monday!
Gary_KC   Dr. Fischback, thank you very much for your time this moring.
fischbeck   Ah, yes, a good point about Avodart (dutasteride). The rules of the trial require that the patiuents be off androgens & anti-androgens such as dutateride for at least 3 months before starting.  The other inclusion & exclusion criteria are listed on the clinicaltrials.gov website, or you can ask Angela.
UTE   Having just become a greatgrandfather this week your info gives me great hope for the future
Hugo Rodriguez Dr. Fischbeck, please say Hi to Angela and thanks for participate in this chat. Thanks to all and keep the hope high!
fischbeck   Congratulations on becoming a great-grandfather. I'd be happy to be a grandfather, but my wife says not to push it!
fischbeck   I'll apss on greetings to the restr of the team.
BruceG   :-)
Ron M   Thank you so much Dr. Fischbeck and everyone today who joined us. Great information
Hugo Rodriguez Bye to all. Have a nice weekend!
MikeG   Thanks so much for being our guest today, Kurt.  You really give us hope!!!
BruceG   I will also brag and say I am a great grandfather.  It is wonderful!
UTE   Thank you doctor
david   Thanks for the information it is good news indeed.  Hope to able to participate.
Ron M   Congrats Bruce - enjoy.
Stewart   Great thanks
Connie A   Bye everyone. Thanks Dr. Fishbeck
BruceG   Kurt, we cannot say it enough, we appreciate all that you and your staff do for us.
fischbeck   I appreciate evryone's interest & support!
BruceG   Let's hope for an early Spring!  We are all a little tired of this winter.
fischbeck   I'm signing off now. Thanks.
Ron M   Yes - I am worn out. Hoping for a warm March.
MikeG   it was even cold in Daytona yesterday for the NORD Rare Disease Day event!
BruceG   Mike, I hope Paula was driving while you attended the chat. :-)
Ron M   Wow. Didn't think that happens down there
MikeG   :)
MikeG   yes, but it's about my time to take over now...
BruceG   Thanks Ron for hosting the chat.  Mike, you need to set Ron up as a chat room leader.
Ron M   You are welcome. Enjoyed it. Good information today.
MikeG   this worked out really well - I still have 2 hours left on the laptop battery and it's charging my cellphone that is acting as the Intenet connection.
BruceG   Stay upright and safe
Ron M   Will try. take care of yourself Bruce
MikeG   I'm clad the chat website worked today - we don't need any repeat performanc of last month...
Ron M   Yes, I had my concerns. But it went smooth
MikeG   so long everyone - stay safe!
Ron M   Take Care
Ron M   I need to transition. Be well everyone
bcpaul   I had to update Java, but this was much better than my first time on, when |I couldnt see what I was typing, well done
UTE   My wife had to get rid of the old java...been gone a while
bcpaul   Time for breakfast, hope you all have a great weekend
UTE   you too
frabayrui   hola desde Sevilla, Spain