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
>>>>
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