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Dear Rayilyn, thanks a lot for your kindest message. I will keep you informed on what's going on in PD research, that's for sure. All the best and have a nice day, Jose L. Rayilyn Brown escribió: > thank you for this explanation about what is going on in research. > I think that knowledge eases the pain. > > when I was diagnosed in 1996 I had never heard of DBS,yet in 2003 it > stopped my tremors, so I don't completely give up hope. > > -----Original Message----- From: José Luis Lanciego > Sent: Wednesday, January 22, 2014 12:49 AM > To: [log in to unmask] > Subject: Re: ProSavin gene therapy > > 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