Hi Rayilyn,

for those of you interested on ProSavin and gene therapy approaches, the 
following link provides some useful explanations: 
blogs.plos.org/dnascience/2014/01/23/gene-therapy-news-brain-skin-eye#.UuF5rgnETNs.twitter 
<http://blogs.plos.org/dnascience/2014/01/23/gene-therapy-news-brain-skin-eye/#.UuF5rgnETNs.twitter> 


Best, Jose L.

Rayilyn Brown escribió:
> THANK YOU!!!  Jose, medical researchers have the most important jobs 
> on the planet.   Without you we'd have no hope at all.   You are 
> appreciated.
>
> -----Original Message----- From: José Luis Lanciego
> Sent: Thursday, January 23, 2014 12:03 AM
> To: [log in to unmask]
> Subject: Re: Fw: ProSavin gene therapy
>
> 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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