Kennedy's Disease Association

A Public Benefit, Non-Profit Organization

"Surfing the internet I found the KDA and for me it was like an island for a castaway."

Archives - 2015 and Older Transcripts

Topic: Open Forum  
Host: Ed Meyertholen
Jim Jelbert   Hi is anyoune out there today?
robazbob   I just got here. Good morning
TedA   Good morning from Richmond, VA
David   Goo0d Morning all from the very wet southwestern Ohio
GregF   Howdy from eastern CO.
robazbob   good morning from Arizona
David   Hope you all have a good Thanksgiving we have a great time with family in Chicago
David   Good Morning Ed
edM   Good Morning, everyone
GregF   ours was great family came to our house so we didnt have to travel 
Gary_KC   Good moring. This is Gary joining in from Kansas City.
David   Good Morning Gary
edM   We went to Cincinnati to see our new grand son
edM   'moring, Gary
Gary_KC   Hi David and edM
David   EdM Cincinnati is my hometown hope you enjoyed the Queen City
edM   e did, of course we did a lot of babysitting though
David   Of course a new baby boy is a joy all its own 
edM   ... and football watching
carofer   good morning
carofer   Early in the morning in california -- i was just looking at a rainbow created by the recent rains
David   Is there a topic for this morning? or is it open for any thing that is on our minds
Gary_KC   Did anyone attend the KDA conference? Any new information shared with us? 
David   carofer What a great way to start the day!:} 
TedA   Any coments on the KDA conference?   It was great and good to see people again and meet new people.
carofer   yes david, as long as i have my coffee
TedA   Yes
edM   I am not sure there is anything new but I would say that there were several items that I found interesting
robazbob   I would like to know how everyone is dealing with this disease? How and where did you find help if needed? My mom her husband and my sister all went and vollentered to help all three days
David   Yes I attended the conference and it seemed to me EdM that this years research discussions showed some significant promise, 
edM   In the past few years, there has been an increase in the researchers teaming up with different drug companies
edM   It is interesting, imo, that the companies are willing to support research, I think this is a good thing for us.
edM   Another change is that there is growing evidence that we can treat the muscles directly.
TedA   With 2 drug companies there it is obvious that they wae involved
David   However I know that there are likely years more research before any potential treatment is identified
edM   As you may know, the traditional description of KD is that it is a motor neuron disease and therapies must act on these cells which are in the brain and spinal cord.
edM   Recent papers have shown good results with just treating the muscles and not the brain/sc.
edM   This is significant as it is difficult to get drugs from the blood to the brains cells - muslce are easier to target as they do not have this difficulty
edM   TedA, there are at least three companies involved - 
David   EdM I found very interesting the discussion on using skin cells collected at a KD conference about 4 or 5 years ago and the development of stem cells to create motorneuron and muscle cells with the KD mutation. Do I have the right?
Gary_KC   edM, it is wonderful. Thanks.
frabayrui   buenas tardes desde España
frabayrui   goog morning
bartfarkel   edM: when you say these new approaches "treat" the muscles, how is the muscle being treated--by removing the "garbage" protien that is building up in our KD muscles?
edM   David, yep, that is correct - although these are not made to inject but to study, NIH has also done this but with other donor cells.
David   So they will use these stem cells lines to run test at the cell level?
edM   bartfarkel - the recent papers used genetic means to reduce the production of the adnrogne receptor (the mutated protein) in muscle alone and saw improvement in mice models.  There are techniques that may to able to be done in humans - but it is years away.  
TedA   By treating the muscles does that have any possibilities in the treatment of ALS?
edM   Also, NIH indicates that it is moving forward on the IGF1 trial in the next year - and this also treats muscles.
edM   TEDA, maybe - it is a different disease with different causes - without research, can't really predict if it is effective
carofer   for us west-coasters, there may be a trial site in Orange County, California for the IGF1 testing
bartfarkel   Thanks, edM. It is exciting to know that there are a variety of companies looking at different techniques for treatment
edM   David, yes - they have published one paper using our cells.
bartfarkel   Carofer--do you have more details on the possiblity of a trial site in Calif? 
edM   And they have research plans for future studies.  The advantage is that they get to utilize human cells
carofer   Not much -- it would be a place to go to participate in the trial, jointly run by NIH and the drug company that is running the trial. Saves 5 hours each way on the airplane for us  -- they hope to increase the participation of us left-coasters and I think it is near the drug company headquarters
edM   I think the plans are two US sites - east an west coast and several European sites.  I hope I remember this correctly
carofer   Yes europe too
TedA   Is the BVS87 trial for the muscles?
edM   BVS87?
bartfarkel   I would think that's a good sign if they are expanding their trial locations. Was there any mention at the conference of how the initial safety and tolerability testing of the IGF-1 treatment is going/went?
carofer   i think it was Novartis doing this igf1 trial -- is that right ED?
TedA   The current C T at NIH
edM   carofer - correct
edM   is that the name of the IFF1 varient?  If so, I believe that it is injected into the blood and thus can only affect the muscles directly
edM   IGF1 variant. i mean
TedA   BVS87 is what I was told in convesation with the nurse.
edM   If it was at NIH, then that is it - I do not know of any other drug trials going on there.  
David   I think that is the code given as a way to refer to the drug being tested in the NIH Clinical Trial
bartfarkel   I agree carofer--a west coast site would be nice. I'm in Montana and California is much easier to get to than Marlyand!
frabayrui   I want to participate in USA in BVS857 trial, traveling from Spain
edM   Frabayrui,
frabayrui   yes
TedA   David, I think you are correct about it being the code
carofer   I think the only trial at NIH is fatty liver trial right now.
edM   I beleive that they are hoping to have a site in Padua, much closer than Bethesda;-)
frabayrui   my name is Francisco Javier Bayona
TedA   Fatty liver is also going on now so there are 2 CT
edM   carofer - yes and that is purely a descriptive trail, no drugs 
bartfarkel   FYI...I had my annual blood work/health screen done through my employer and my liver enzymes were mildly elevated, which they have been for years. I' guesing that's a pretty consistent sign/symptom for us KDers?
edM   that is what the study at NIHis trying to determine
edM   They noticed that a good number of the exercise trial patients had high liver enzymes in blood
frabayrui   is that I contacted gianni soraru and still has not answered me, and there is only room for 6 patients
David   Yes there are two KD related clinical trails open at the NIH currently one for the fatty liver and the other for a drug test
carofer   Also called steatosis, the fatty liver may not produce unusual enzyme tests immediately
bartfarkel   I'm happy NIH is doig the liver study--who knows what it may reveal! 
edM   Have any of you participated in the liver trial (mine was normal)
carofer   so they do an mri and ultrasound to get a better look at the liver. It may be followed up with a biopsy,
bartfarkel   I have not, but if they are still recruiting for the liver study, maybe I should consider it. 
carofer   it's only one day in and out
frabayrui   You need treatment for patients with liver Kennedy?
carofer   i just got back from NIH yesterday
edM   As a side event,they found that I had kidney cancer during my MRI - pure luck - had it removed and am not cancer free.
edM   now!!! cancer free (I wish I could type)
carofer   Great to hear, EdM
bartfarkel   Great news edM! Good luck and timing are a beautiful thing!
David   frabayrui - they noticed that some of the KD men who participated in the exercise trial at the NIH had fatty livers and decided to do a controlled study of KD men for that condition to determine if there is any clear link between having KD and fatty liver
TedA   Type like me;  Columbus method ...   find a key and land on it!
frabayrui   can someone explain me briefly CT protocol BVS857?
frabayrui   if I have fatty liver also
carofer   lots of info here:
frabayrui   ok
bartfarkel   Anything else of note at the conference for those that attended? Did the KDA award any research grants?
carofer   Which drug companies attended?
edM   Yes, two - one to a researcher in Diane Merry's lab and one to a resercher in Al LaSpada's lab, both for $50000
frabayrui   There is someone here who has already been recruited for the CT on BVS857? 
bartfarkel   That
edM   They also raised over $35,000 at the meeting 
edM   There was a rep from Novartis there
bartfarkel   That's awesome! It's nice to see KDA contributing directly to help research
frabayrui   can take 3 months off and make me test Bestheda, traveling from Spain
David   One of the most exciting things to happen at this year's conference was meeting and having a presentation by Dr Kennedy.  What an amazing individual and he is still a very active researcher 
frabayrui   vey interesting
frabayrui   very
David   EdM I cannot remember what he is currently studying do you?
edM   He was interested in tests for early symptom detection, iirc
TedA   Dr Kennedy is 87 years young!    Met hin 10 years ago at conference in San Diego.
carofer   EdM, do you know if there will be parts of the conference published online?
Gary_KC   edM, It is wonderful if you can find KD by early symptiom detection.
frabayrui   I'm taking trehalose and I think that is helping me. There is a CT  provided by Bioblast  FARMA
edM   It was not just KD - looking for sensory changes that one can quantify to follow the progession of sensory loss in any disease like KD
edM   carofer - do not know
frabayrui   Today was scheduled to speak to RYCALS?
frabayrui   It no so?
edM   frabayrui - I am not sure what you are asking?  
David   I enjoyed our chat time today, but must move on to my next task.  Thanks all and I wish for you a blessed and happy Christmas season.
carofer   en espanol, qual es la pregunta, frabayrui?
frabayrui   ok
Gary_KC   Thanks lots for great information about the KDA conference. I got to go. Stay warm, please.
frabayrui   pregunto si hoy se iba a hablar del compuesto RYCALS?
edM   Thanks for coming - see (hear?) y'all next month
frabayrui   no se si hay noticias sobre ese compuesto
carofer   Que significa compuesto RYCALS?
frabayrui   eres hispano?
frabayrui   o español?
carofer   mi esposa es de peru
frabayrui   ahhhhh
frabayrui   okkk
frabayrui   Rycals es una droga en experimentacion
frabayrui   para los enfermos de Kennedy
frabayrui   y pensaba que hoy iba a venir
carofer   ah no sabia
frabayrui   una persona que hablara sobre esa droga prometedora
frabayrui   perdona pero no se mucho ingles
frabayrui   carofer eres enfermo de Kennedy?
carofer   he is asking about RYCALs and if anyone is going to talk about it today. I don't know what RYCALs is, but it is some kind of drug for KD. Anyone hear anything about that?
frabayrui   he viajado ya 2 veces a USA
carofer   frabayrui yes i have been diagnosed with KD for more than 20 years
frabayrui   waiting
carofer   si, frabayrui, tengo KD
frabayrui   i am 53 years old
frabayrui   i have also
frabayrui   53 Cag
frabayrui   vivo en Sevilla
frabayrui   España
frabayrui   soy arquitecto
frabayrui   hice un viaje a CHICAGO
carofer   mi edad es 66
frabayrui   en 2009
frabayrui   ok
frabayrui   camina bien?
frabayrui   también hice un viaje a Los Angeles, Phoenix, Gran Cañon y atravesamos hasta Yelowstone
carofer    no puedo caminar muy bien y ahora hago uso de un andador con ruedas por si me canso
frabayrui   y regresamos por Salk Lake City y Las Vegas
frabayrui   hasta Los Angeles
frabayrui   21 dias
frabayrui   Va a participar en el ensayo clinico?
frabayrui   del bvs857? 
carofer   has viajado mucho en Los Estados Unidos
frabayrui   SIIIII
frabayrui   yes
frabayrui   jajaa
frabayrui   me encanta viajar
frabayrui   me gusto mucho Chicago
frabayrui   y tambien el oeste
carofer   pues ahora es cuando debes de viajar mientras que puedas hacerlo.
carofer   para mi ahora es muy dificil viajar.Viajao con la asistencia d emi esposa pero es un dificil para mi
frabayrui   me canso enseguida
frabayrui   tambien
frabayrui   necesito parar mucho
carofer   asi es
frabayrui   tengo 4 primos que tambien tienen
frabayrui   lo mismo
frabayrui   estoy tomando trehalose
frabayrui   y pienso que funciona
frabayrui   pero ando poco
carofer   hace unos meses mis piernas colapsaron y me cai asi derrepente y me  fracture el dedo de mi pie
frabayrui   ya no trabajo
frabayrui   estoy jubilado por la enfermedad
frabayrui   tambien
frabayrui   si yo me he caido algunas veces
carofer   como este medicamento que estas tomando te ayuda?
frabayrui   pienso q si
frabayrui   no es medicamento
frabayrui   es un tipo de azucar
frabayrui   en estudio en la ELA
frabayrui   y en SBMA
frabayrui   en Israel
carofer   ah interesante
frabayrui   siiiii
frabayrui   Sabes Carofer
frabayrui   tengo un saco de 10 kgs y viene de Japon
frabayrui   lo he comprado aqui en España
frabayrui   es un edulcorante
frabayrui   simple
frabayrui   se esta probando en Huttington
frabayrui   tambien
frabayrui   con experiencia positiva
carofer   gracias -- la voy a probar
frabayrui   limpia las celulas
frabayrui   de basura
frabayrui   esa es su mision
frabayrui   no se si escribo rapido
carofer   quiero participar en CT por BVS857
frabayrui   3 cucharas de cafe 3 veces al dia
frabayrui   ya te ha llamado Angela?
carofer   si escribes un poquito rapido y yo me demoro en escribir encastellano
frabayrui   i am sorry
carofer   porque no recuerdo muy bien las palabras
frabayrui   yo tengo el traductor de google a mano
frabayrui   si quieres me hablas ingles
carofer   Si, Angela nos ayudo en NIH 
frabayrui   cuando comienza?
frabayrui   febrero 2015?
frabayrui   has sido ya seleccionado?
frabayrui   Carofer mi email es frabayrui@movistar.
carofer   no se -- pronto. Estoy esperando los resultados de  los examenes y segun eso para ver si soy un buencandidato para CT
frabayrui   PERDONA
frabayrui   This email address is being protected from spambots. You need JavaScript enabled to view it.
carofer   mi email es This email address is being protected from spambots. You need JavaScript enabled to view it.
frabayrui   ese es mi email
frabayrui   muchas gracias
frabayrui   los examenes los hace el NIH?
carofer   Quiero ir a Espana ...
frabayrui   Angela esta en contacto conmigo tambien
frabayrui   venga usted
carofer   si, en NIH
frabayrui   sera bien acogido
frabayrui   aqui en el sur de España el clima es mejor
carofer   si pero sera muy dificil para mi poder despalazarme hasta alla
frabayrui   mas calido
frabayrui   noooooo
frabayrui   es facil 
frabayrui   el avion y luego hay vehiculos adaptados
frabayrui   le espero
frabayrui   aqui sin problemas
carofer   hay ayuda alla en Espana a las personas discapacitadas como aqui en US?
frabayrui   siiiiiiiiii
frabayrui   en el sur mas aun
carofer   fantastico!
frabayrui   el gobierno de Andalucia apuesta por las personas discapacitadas
frabayrui   hay muchas ayudas
frabayrui   tenemos un gobierno en cada region
frabayrui   con competencias transferidas desde Madrid
frabayrui   y hay rtaxis adaptados
frabayrui   Sevilla es una ciudad sin obstaculos
carofer   mi cunada vive en Cannes, Francia
frabayrui   es plana
frabayrui   llana
frabayrui   ah si'
frabayrui   he pasado una vez por alli
frabayrui   aqui ahora hay 10 grados
frabayrui   en el norte estan en 3 o 4
carofer   y me gusataria visitarla otra vez pero la ultima vez que lo hice fue en el 2007 y fue dificil
frabayrui   es una ciudad de la costa claro
frabayrui   Sevilla es muy bonita
carofer   10 grados centigradoos?
frabayrui   si
frabayrui   cuanto hace alli ahora
frabayrui   hace frio pero no tanto como en el norte
frabayrui   aqui no suele bajar de 4
carofer   tengo que bajar 10 kilos para que me sea mas facil viajar.
carofer   estoy en ello
frabayrui   eso es otra cosa
carofer   aqui en aptos CA es acerca de 50 F
frabayrui   a cuanto equivale
frabayrui   en centigrados
frabayrui   aqui va a entrar el invierno ya
carofer   10 centigradosa
frabayrui   pero no es muy frio comparado con USA
carofer   10C
frabayrui   sobretodo la costa este
frabayrui   igual
frabayrui   entonces que aqui
frabayrui   en verano si suele llegar a los 40 y mas
carofer   En la costa este es acerca de 0C
frabayrui   exacatamente
frabayrui   estamos cerca de Africa
frabayrui   y se nota
frabayrui   la calidez del clima
frabayrui   estoy a 90 kms de la playa
carofer   suena fabuloso
frabayrui   Conoce algo de Sevilla?
frabayrui   conocera el flamenco?
frabayrui   la Giralda?
carofer   nosotros estamos como  2 dos kilometros de la playa
frabayrui   que cerca....
frabayrui   estoy casado y tengo un bebe de 2 años
carofer   no se Giralda, pero me gusta el baile flamenco
frabayrui   la Giralda es una torre que hicieron los musulmanes aqui
frabayrui   de 90 metros
frabayrui   de altura
frabayrui   busque en google
carofer   Felicidades por le bebe --- tengo dos hijos y 2 nietos 
frabayrui   aqui vivieron 600 años los arabes
frabayrui   gracias
frabayrui   y enhorabuena
frabayrui   Sevilla tiene 1000000 hab
frabayrui   es la 3 ciudad
frabayrui   Madrid, Barcelona y Sevilla
carofer   yo apuesto la comida es deliciosa
frabayrui   siii
frabayrui   lo es
carofer   y el vino tambien
frabayrui   es mediterranea
frabayrui   sii
frabayrui   esto lo llaman la California del sur
frabayrui   de Europa
frabayrui   el clima
frabayrui   sobretodo
frabayrui   como se llama usted
frabayrui   bueno quiero participar en el ensayo en Washington
frabayrui   tambien
carofer   si aqui hay bonitos lugares pero no creo que sean tan bonitos como alla sobre todo LA COMIDA. mi nombre es Allan
frabayrui   cuantos dias hay q estar en el hospital a la semana
frabayrui   encantado aLLAN
frabayrui   Allan
carofer   depende a vces pueden ser dos o tres dias.ellos pagan por boletos de avion inside de US
carofer   Oh cual es tu nombre?
frabayrui   no me importaria residir 3 meses
frabayrui   alli
frabayrui   Francisco Javier
carofer   oh okay. 
frabayrui   y cada semana 2 o 3 dias?
frabayrui   en Washington
frabayrui   me quedaria a vivir alli mientas dure el ensayo
frabayrui   he hablado con Angela
carofer   bueno en mi caso yo fui 2 y luego tres dias en total. esto fue un viaje pqueno, claro que de ahi yo me fui a New York por una semana a Vacacuinar
carofer   vacacionar
frabayrui   pero usted ha empezado ya el ensayo?
frabayrui   la fASE inicial ?
frabayrui   que eran 2 pacientes creo
carofer   ah si? seria fabuloso que vinieras por buen tiempo
frabayrui   no se si me pagaran la estancia los 3 meses?
carofer   no todavia no he empezados este ultimo ensayo pero he estado en el pasado en dos trials. uno fue de ejercicios y el otro fue de drugs
carofer   con el de ejercicios me ayudo a sentirme un poc mas fuerte. 
frabayrui   si?
frabayrui   y continua usted con los ejercicios?
frabayrui   se sabe algo sobre su beneficio definitivamente?
carofer   bueno tal vez si porque estarias viniendo desde muy lejos . Conversalo con Angela
frabayrui   si a unos 5000 kms
frabayrui   creo que casi igual que usted pero en otro sentido
carofer   puedes usar AirBnb. tu puedes conseguir estadia mas barato. 
frabayrui   AirBnb?
frabayrui   no se que es
carofer   yo pieso que tal vez Angela puede darte mas opciones
frabayrui   ok
carofer en internet
frabayrui   gracias
frabayrui   pero ha empezado a llamar ya?
frabayrui   para reclutar pacientes?
frabayrui   me dijo en diciembre
carofer   todavia no
frabayrui   se esta retrasando entonces
carofer   Angela dijo que van a empezar pronto, tal vez dos meses
frabayrui   si pero entonces tendrian que estar llamando antes para hacer la seleccion
frabayrui   de pacientes no?
carofer   si claro
frabayrui   o es que empiezan los ensayos mas tarde
frabayrui   creo que empezaban en febrero
carofer   puedes email a Angela
frabayrui   si
frabayrui   eso hare
frabayrui   hay resultados ya sobre los ejercicios?
frabayrui   digo resultados publicados
carofer   Si, estoy me ayudo a sentirme mas fuerte
frabayrui   entonces seguira practicando 
carofer   y hasta ahora practico los ejercicios cada manana
frabayrui   eso es
frabayrui   a ver si por email puedes enviarme los ejercicios
frabayrui   si puedes claro
frabayrui   los que habria que hacer
frabayrui   solo si puedes
carofer   tambien estuve con una terapista y ella me ayudo a practicar moviminetos por si akgun dia me caigo y estoy solo en casa.
frabayrui   estupendo
frabayrui   solo hay q levantar la cabeza
frabayrui   y para arriba
frabayrui   es broma
carofer   Si, yo voy a  enviarte una informacion d elos ejercicios. unos son de brazos y los otros son de piernas.. pararse y sentarse que en este caso es lo mas dificil para mi.
carofer   oh hahaha
carofer   Espero que tu esposa puede ir contigo...
frabayrui   siiiiii
frabayrui   y el bebe
carofer   mi esposa me ayudo MUCHO!!
frabayrui   no vive?
frabayrui   perdona si..
frabayrui   a mi me cuesta andar bastante
carofer   si dime?
carofer   si ami tambien
frabayrui   su esposa
frabayrui   vive vive
frabayrui   si?
frabayrui   como dijo en pasado me ayudo
carofer   mi primera esposa murio hace 14 anos y mi segunda esposa si vive yesat conmigo apoyandome
frabayrui   ok
frabayrui   y es de Peru?
carofer   si, de Lima. Ella esta muy bonita
frabayrui   debe serlo para vivir con usted
frabayrui   que me esta cayendo muy bien
frabayrui   los hispanos somos muy emocionales
frabayrui   y usted lo esta siendo conmigo
carofer   siiii a veces ella esta muy enojada
frabayrui   siiiiiiiiiiiii
frabayrui   correcto
frabayrui   es el alma pasional española
frabayrui   que lleva dentro
frabayrui   jajjaja
frabayrui   yo parezco ingles
frabayrui   soy menos pasional
frabayrui   y ademas por el fisico
carofer   oh osea tu eres como yo
carofer   adios.
carofer   Thank you EdM for all the great info -- I need to leave now. See you at the next chat
wpawula   good afternoon
wpawula   :? 8) 8) 
wpawula   bye