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-   -   Dramatic improvement in Parkinson disease symptoms following intranasal delivery of s (https://www.neurotalk.org/parkinson-s-disease/211969-dramatic-improvement-parkinson-disease-symptoms-following-intranasal-delivery.html)

zanpar321 11-09-2014 06:01 PM

Dramatic improvement in Parkinson disease symptoms following intranasal delivery of s
 
Looks like intranasal delivery of Stem Cells works great!

The promising findings, reported in Rejuvenation Research, a peer-reviewed journal published by Mary Ann Liebert, Inc. highlight the potential for a noninvasive approach to cell therapy delivery in Parkinson disease-a safer and effective alternative to surgical transplantation of stem cells.

www.sciencedaily.com/releases/2011/02/110208163504.htm

lurkingforacure 11-09-2014 08:02 PM

dated
 
This article is dated almost three years ago, in February of 2011. I wonder why there does not seem to be any follow up or newer work in this?

girija 11-09-2014 09:56 PM

Quote:

Originally Posted by lurkingforacure (Post 1107094)
This article is dated almost three years ago, in February of 2011. I wonder why there does not seem to be any follow up or newer work in this?

It is always the same reason, MONEY. Too many promising projects stop at preclinical studies because it is difficult to get funds for clinical studies.

beeks3010 11-10-2014 08:56 AM

Intranasal adm of GDNF
 
Is it because they've run out of money, or might it be because the drug companies do not want cures to be discovered for some diseases????? When something looks to be as promising as this intranasal GDNF, if it truly were the money... what about a foundation such as the Michael J. Fox Foundation picking up where the study left off?? I am very apprehensive... think there may just be a whole lot more to promising studies dead ends that what the general public is aware...

soccertese 11-10-2014 10:58 AM

Quote:

Originally Posted by beeks3010 (Post 1107149)
Is it because they've run out of money, or might it be because the drug companies do not want cures to be discovered for some diseases????? When something looks to be as promising as this intranasal GDNF, if it truly were the money... what about a foundation such as the Michael J. Fox Foundation picking up where the study left off?? I am very apprehensive... think there may just be a whole lot more to promising studies dead ends that what the general public is aware...


there are 2 or more GDNF trials in progress, there has been a huge amount of research on gdnf and some very large and expensive trials in the past that failed treating pd, one funded by AMGEN, search on GDNF here. everything seems to cure/prevent pd in rats but fails miserably in humans. money is an issue but so is finding enough volunteers willing to risk introducing drugs and/or genes into the brain when pd isn't fatal, that is also slowing down the pace of research.
every gene therapy trial in humans has failed yet worked great in rodents and primates. gotta keep things in perspective.
if you want to get involved join PAN or some other advocacy group.

zanpar321 11-10-2014 11:09 AM

Quote:

Originally Posted by beeks3010 (Post 1107149)
Is it because they've run out of money, or might it be because the drug companies do not want cures to be discovered for some diseases????? When something looks to be as promising as this intranasal GDNF, if it truly were the money... what about a foundation such as the Michael J. Fox Foundation picking up where the study left off?? I am very apprehensive... think there may just be a whole lot more to promising studies dead ends that what the general public is aware...

It seems quite evident that what we need is a patient led organization vs a money focused behemoth that we presently have in the US. The Cure Parkinson's Trust UK seems to be a patient led research team. Face it folks, money drives the research in the US and the FDA only approves research that has big money behind it! The end result seems to be that research that may be quite promising is all to often left to die on the vine if Big Pharma doesn't see a big payoff down the road. The Michael Fox Foundation may be sort of a patient run organization, but I'm not sure who is on their board. Does anyone know who makes the decisions there regarding which studies to fund? No one is more motivated than a Parkinson's patient (or a Parkinson's patient's family member), to cure Parkinson's. The MJF Foundation seems to be funding some good research but maybe adding another patient led organization in the US would heat up the research. I've had Parkinson's now for 4 years and I'm beginning to realize that waiting for Big Pharma to help me is a fruitless effort!

zanpar321 11-10-2014 11:39 AM

Quote:

Originally Posted by soccertese (Post 1107163)
there are 2 or more GDNF trials in progress, there has been a huge amount of research on gdnf and some very large and expensive trials in the past that failed treating pd, one funded by AMGEN, search on GDNF here. everything seems to cure/prevent pd in rats but fails miserably in humans. money is an issue but so is finding enough volunteers willing to risk introducing drugs and/or genes into the brain when pd isn't fatal, that is also slowing down the pace of research.
every gene therapy trial in humans has failed yet worked great in rodents and primates. gotta keep things in perspective.
if you want to get involved join PAN or some other advocacy group.

If I understand the earlier AMGEN GDNF study, far from a miserable failure, many of the quite human patients, were very pleased with how GDNF gave them their lives back, so much so that they sued AMGEN to get them to continue giving them GDNF, but sadly they were refused (see article below).

Amgen sued for Parkinson's drug

http://www.bizjournals.com/losangele...5/daily29.html

zanpar321 11-10-2014 11:51 AM

Quote:

Originally Posted by lurkingforacure (Post 1107094)
This article is dated almost three years ago, in February of 2011. I wonder why there does not seem to be any follow up or newer work in this?

Hi Lurking,

I sent you a message.

Tupelo3 11-10-2014 12:03 PM

Quote:

Originally Posted by zanpar321 (Post 1107165)
It seems quite evident that what we need is a patient led organization vs a money focused behemoth that we presently have in the US. The Cure Parkinson's Trust UK seems to be a patient led research team. Face it folks, money drives the research in the US and the FDA only approves research that has big money behind it! The end result seems to be that research that may be quite promising is all to often left to die on the vine if Big Pharma doesn't see a big payoff down the road. The Michael Fox Foundation may be such an organization but I'm not sure who is on their board. Does anyone know who makes the decisions there regarding which studies to fund? They seem to be funding some good research but maybe adding another patient led organization in the US would heat up the research. I've had Parkinson's now for 4 years and I'm beginning to realize that waiting for Big Pharma to help me is a fruitless effort!

Zanpar you're right, the Cure Parkinson's Trust is a great organization. Maybe we need another patient led org and maybe not. And, money is clearly a driving force in drug research. However, I'm not sure I agree with you about Big Pharma letting drugs "die on the vine". Other than the Amgen - GDNF controversy, what other drug has been left out to die by Big Pharma? The real issue is more that Big Pharma does little research these days on any disease or illness that doesn't represent hundreds of millions of patients (e.g. cancer, heart, diabetes). They would rather spend time and money on me-too drugs that they know will work for millions rather than risk funds on diseases that are harder to treat and cure. I read recently that there are now 24 treatments available for erectile dysfunction. I know this is a rising problem, but how many more treatments do we actually need? Or drugs for cholesterol, hypertension, etc.

So, instead, we have the research for PD and other less prevalent diseases left to smaller biotech companies, and there are actually many of them doing great research. However, their funds tend to be limited and this leads to extended timelines for getting drugs approved. Ultimately, we see Big Pharma stepping in at the end, when the drug is close to approval and has been de-risked, and just buying out the smaller biotech. It's a lot simpler model for Pharma than doing their own research.

Still, with all of the above being said, the number one reason for delays in the clinical trial process is finding participants to volunteer. If PWP want to make a real difference, they need to be more willing to step into the line of fire and participate in trials.

zanpar321 11-10-2014 12:22 PM

Quote:

Originally Posted by Tupelo3 (Post 1107169)
Zanpar you're right, the Cure Parkinson's Trust is a great organization. Maybe we need another patient led org and maybe not. And, money is clearly a driving force in drug research. However, I'm not sure I agree with you about Big Pharma letting drugs "die on the vine". Other than the Amgen - GDNF controversy, what other drug has been left out to die by Big Pharma? The real issue is more that Big Pharma does little research these days on any disease or illness that doesn't represent hundreds of millions of patients (e.g. cancer, heart, diabetes). They would rather spend time and money on me-too drugs that they know will work for millions rather than risk funds on diseases that are harder to treat and cure. I read recently that there are now 24 treatments available for erectile dysfunction. I know this is a rising problem, but how many more treatments do we actually need? Or drugs for cholesterol, hypertension, etc.

So, instead, we have the research for PD and other less prevalent diseases left to smaller biotech companies, and there are actually many of them doing great research. However, their funds tend to be limited and this leads to extended timelines for getting drugs approved. Ultimately, we see Big Pharma stepping in at the end, when the drug is close to approval and has been de-risked, and just buying out the smaller biotech. It's a lot simpler model for Pharma than doing their own research.

Still, with all of the above being said, the number one reason for delays in the clinical trial process is finding participants to volunteer. If PWP want to make a real difference, they need to be more willing to step into the line of fire and participate in trials.

Excellent points Tupelo. We PWP do need to volunteer more. I've volunteered once so far and am looking for studies that I qualify for now. Regarding the funding, I still think it would be better if patients had more input into which studies are funded. Nine years or so have gone by since the AMGEN GDNF trial and now 3 years have gone by since the successful intranasal stem cell study and no one knows when a human study will even start! It just seems like perhaps PWP need to step up and do more to help coordinate research or at least set up an organization to track studies and provide better communication as to the status of studies!


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