Jose -
Please keep us informed - you can't imagine how keen we are for news.  
Even negative news - and don't worry about intimidating jargon; we 
can always look it up.

 
> Date: Wed, 22 Jan 2014 08:49:04 +0100
> From: [log in to unmask]
> Subject: Re: ProSavin gene therapy
> To: [log in to unmask]
> 
> 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