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Mary Ann Great you have such close family!! Medical clinic sounds interesting. I haven't down such a good job of posting since my research buddy, Diane, died over two years ago. also my computer does some strange things when I move the mouse and "click". Its harder to cut and paste now, but I hope I won't miss real news. -----Original Message----- From: Mary Ann Ryan Sent: Friday, January 24, 2014 7:46 AM To: [log in to unmask] Subject: Re: ProSavin gene therapy Ray, life is good here. Jamie continues to be a part of our lives - my grandkids sit in his recliner more than in any other furniture in the house so I think they feel his sweet presence. I volunteer once a week at a homeless shelter - I run a medical clinic there. We still have the farm and continue to plant a huge garden every year. My daughter and her husband plus their two kids live with me so I never feel alone. My son and daughter-in-law visit frequently. It's good to see that you still are active in informing PWP about studies and the need for advocacy. Thanks for all that you do. -------- Mary Ann ----- Original Message ----- From: "Rayilyn Brown" <[log in to unmask]> To: <[log in to unmask]> Sent: Thursday, January 23, 2014 2:20 PM Subject: Re: ProSavin gene therapy > you're welcome, Mary Ann. How are you doing? > > -----Original Message----- > From: Mary Ann Ryan > Sent: Wednesday, January 22, 2014 7:31 AM > To: [log in to unmask] > Subject: Re: ProSavin gene therapy > > Thanks, Ray. This is great information and I plan on saving it for future > use. It is frustrating that a miracle therapy is not on the horizon but > at > least you got the attention of this doctor so that the PD community can be > better informed. Thank you so much for your effort. > ------- > Mary Ann (CG Jamie 68/28 with PD, died 11/20/07) > > > >> Dear Rayilyn, >> >> I perfectly understand your current situation. Although I have been >> trained as MD, I have never treated patients since I am engaged 100% in >> PD research. I am in close contact with the Spanish association of >> Parkinson's disease, and I strongly feel that one of my obligations is to >> explain the patients what's going on in PD research. I know that it is >> fustrating to see that anything new has arrived in the past few years, >> besides some minor improvements in dopamine-replacement therapies (if I >> am right, a phase 2 trial testing an inhaled preparation of levodopa is >> going to start soon in US). When attending scientific meetings, the >> overall feeling is that everybody is always moving around the same >> topics, without any real new improvement or any real new ideas to follow. >> In this regard, gene-based therapies such as ProSavin at least represent >> something different. I cannot anticipate how long will it take to see >> these new arrivals fully available in the market as a therapeutic tool. >> Nevertheless, I still thinking that approaches this kind at least >> represent something different, and I am sure that this is a right step in >> the right direction. In the past two years my group has received some EU >> funding to start testing different approaches of gene therapy for >> manipulating basal ganglia circuits. We are using MPTP-treated monkeys >> since these represent the best animal model available so far, and working >> with monkeys speeds up the time required to start testing new tools in >> humans suffering from PD. I can give you my word that we are working very >> hard trying to generate the expected results asap. We have just started >> several experiments, all very risky approaches, and therefore I cannot >> anticipate any final result. Nevertheless, you can be sure that the >> situation of PD patients always is very well represented in my mind at >> all times. >> >> I know that it is not easy to be optimistic in the current scenario. >> However, my personal feeling is that testing some new research avenues >> has just been started. There is too much pressure to go traslational, and >> to start testing new approaches in patients asap. This pressure implies >> that some promising things enter clinical trials too early, and therefore >> it is fustrating to see that most of these new therapies failed in >> clinical trials. Just simply remember the failures of GDNF and nurturin. >> These are very good examples of promising things that failed because by >> the time these get tested in humans, we knew very little about their >> function. For instance, by the time in which GDNF entered phases 1-2 (the >> "Kentucky" and "Bristol" trials), we didn't knew too much about the >> neurobiology of GDNF. Researches we have to deal with this pressure, >> that's life. However, my feeling is that we cannot generate any solid >> expectances for PD patients without a clear proof-of-principle. As I have >> mentioned before, some failures we have seen in the past easily rank >> within this category of things being tested too early. >> >> According to the published results from ProSavin, this might not be seen >> as the way to go. My opinion is just the opposite. Although I will have >> been delighted to see a better clinical outcome, the important argument >> of ProSavin is that approaches this kind at least opens new vistas in PD >> research. I can perfectly understand that you are tired of getting worse >> and waiting. I just can tell you that everything that me -and many >> others- we are currently doing is done bearing in mind at all times the >> clinical situation of thousands of PD patients. You are our main >> motivation, and this is how things should be. Of course I cannot promise >> any miracle, just a tireless effort in PD research, every day. >> >> All the best and have a nice day, Jose L. Lanciego >> >> Rayilyn Brown escribió: >>> Jose >>> >>> I've had PD 18 years, have gotten worse, am 78 years old and fear at the >>> present pace of research will not live long enough to benefit from any >>> of the 'promising" treatments on the horizon. I'm tired of getting >>> worse and waiting. But then I'm anemic, maybe that’s it. >>> >>> -----Original Message----- From: José Luis Lanciego >>> Sent: Tuesday, January 21, 2014 3:06 AM >>> To: [log in to unmask] >>> Subject: Re: ProSavin gene therapy >>> >>> Dear Rayilyn, >>> >>> I guess this is not discouraging at all. Although this is a preliminary >>> study, my feeling is that ProSavin represents a right step in the right >>> direction. A number of gene therapy experiments are currently undergoing >>> and we will see what's coming out in the next few years. Indeed, this >>> approach -and few more under experimental implementation- is something >>> different, e.g., it is not based on dopamine-replacement strategies. My >>> laboratory -as many others- strongly feels that many new arrivals can be >>> expected from gene therapy experiments, with the ultimate goal of using >>> gene therapy tools to manipulate basal ganglia circuits. Compared to >>> cell therapies, gene therapy seems to be a much more feasible and >>> realistic strategy. >>> >>> All the best and have a nice day, Jose L. Lanciego >>> >>> Rayilyn Brown escribió: >>>> discouraging isn't it? >>>> >>>> -----Original Message----- From: Nic Marais >>>> Sent: Saturday, January 18, 2014 12:29 AM >>>> To: [log in to unmask] >>>> Subject: Re: ProSavin gene therapy >>>> >>>> When you read the Viartis notification, it does not look as positive as >>>> this article... >>>> >>>> http://www.viartis.net/parkinsons.disease/news/140113.htm >>>> >>>> Nic 61/19 >>>> >>>> >>>> On 17 January 2014 21:18, Rayilyn Brown <[log in to unmask]> wrote: >>>> >>>>> http://www.domain-b.com/technology/Health_Medicine/20140115_patients.html >>>>> >>>>> Ray >>>>> Rayilyn >>>>> Past Director AZNPF >>>>> Arizona Chapter National Parkinson Foundation >>>>> >>>>> ---------------------------------------------------------------------- >>>>> To sign-off Parkinsn send a message to: mailto: >>>>> [log in to unmask] >>>>> In the body of the message put: signoff parkinsn >>>>> >>>> >>>> ---------------------------------------------------------------------- >>>> To sign-off Parkinsn send a message to: >>>> mailto:[log in to unmask] >>>> In the body of the message put: signoff parkinsn >>>> ---------------------------------------------------------------------- >>>> To sign-off Parkinsn send a message to: >>>> mailto:[log in to unmask] >>>> In the body of the message put: signoff parkinsn >>>> >>> >>> ---------------------------------------------------------------------- >>> To sign-off Parkinsn send a message to: >>> mailto:[log in to unmask] >>> In the body of the message put: signoff parkinsn >>> ---------------------------------------------------------------------- >>> To sign-off Parkinsn send a message to: >>> mailto:[log in to unmask] >>> In the body of the message put: signoff parkinsn >>> >> >> ---------------------------------------------------------------------- >> To sign-off Parkinsn send a message to: >> mailto:[log in to unmask] >> In the body of the message put: signoff parkinsn > > ---------------------------------------------------------------------- > To sign-off Parkinsn send a message to: > mailto:[log in to unmask] > In the body of the message put: signoff parkinsn > ---------------------------------------------------------------------- > To sign-off Parkinsn send a message to: > mailto:[log in to unmask] > In the body of the message put: signoff parkinsn ---------------------------------------------------------------------- To sign-off Parkinsn send a message to: mailto:[log in to unmask] In the body of the message put: signoff parkinsn ---------------------------------------------------------------------- To sign-off Parkinsn send a message to: mailto:[log in to unmask] In the body of the message put: signoff parkinsn