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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