[lurkingforacure]

Hi everyone,

I found this tiny tidbit buried in a newslink and find it encouraging...here's to hope:

Up at Oxford
Oxford Biomedica, the FTSE-listed biopharmaceutical firm, saw its shares rise nearly 6% to 23.25p after encouraging news in the development of its gene therapy treatment for Parkinson's disease. The aim of its ProSavin treatment is to introduce genes responsible for dopamine production into cells that populate the area of the brain that is dopamine deficient. Analysts at Panmure Gordon noted that a second patient has been "dosed" on ProSavin. "The importance of the second patient being dosed is that it comes a full month after the first patient, which implies that no issues were raised on safety," Panmure said, adding that the company remains "well capitalised". It repeated its buy recommendation on Oxford Biomedica and its 69p price target

[aftermathman]

posting about Prosavin, the investor boards are oozing optimism, reminds me of GDNF !!

Long way to go here.

Neil.

[lurkingforacure]

But don't you think, given the GDNF history in the big pharma world, that surely no one would go down that path again? Not to say that if there are issues, like it doesn't work, they will stop the trial(s), but otherwise I sure would like to believe that they would have thought of GDNF before the human trial started. For example, wouldn't a company have already thought of, and addressed, delivery mechanisms (apparently was a big issue in GDNF?), and either else resigned themselves to the product being delivered by brain surgery and therefore on a one-at-a-time basis (and thus, much less profitable than the en masse take-a-pill one sixe fits all approach)? It's wayyyy too early on a saturday morning to try to articulate what I'm trying to say, but I guess the point is, given GDNF, I would hope they would have worked those kinks out before proceeding this far. But then again...

[Fiona]

yes, but then again. It's true stocks are going up for Prosavin - my sister just bought some - and while the news is very sanguine on these gene therapies, I think they are very serious (obviously) things with very serious risks. After listening to the researchers speak about another genetic therapy being developed here in New York, which i think has a very similar delivery system to ProSavin - well, it sounds very promising. But the part that freaks me out is the part about "the main question we have is whether or not the virus that is engineered specifically for insertion into the brain will at some point - and who knows when - decide to mutate randomly and uncontrollably." I really don't want to be the person that that happens in...

[paula_w]

It appears to have been the delivery system with GDNF, and the deliveries above and below are not going to be ready for years; Thus, GDNF has not gone quietly into the night...p

Posted April 24, 2008

Stem cell experts: Results take time

By Robert Mentzer
Wausau Daily Herald
rmentzer@wdhprint.com


The science of stem cell research holds real promise, but many practical applications remain far off, scientists said this week at a Wausau conference on stem cell ethics.

Clive Svendsen, a professor of anatomy and neurology at the University of Wisconsin-Madison, conducts research on Parkinson's disease and other neurological disorders.

One promising technique, he said, is the use of stem cells that generate a protein known as GDNF, which can limit degeneration of neurons. Svendsen has conducted clinical tests on rats, and has a new academic paper showing that the same techniques work in the brains of primates. This treatment, he said, could be used to dramatically slow the progress of diseases such as Parkinson's, Alzheimer's and Lou Gehrig's or ALS.

"Cure is a very strong word," Svendsen said. "But I think there are very interesting implications for treatment."
Full article

paula

[1990nyboy]

Over the past several years I have tried to gain as much information on gene therapy for Parkinson's as I could. I don't believe that gene therapy is another GDNF. I am a medical scientist and work extensively with the pharmaceutical industry. (My research is not involved with PD, but I know enough about science and clinical trials to be an informed consumer of data on gene therapy.)

There are three companies that are working hard in this area with slightly different approaches: (1) Oxfordshire Bioscience-in the UK; (2) Ceregene and (3) Neurologix in the US. (If there are others that I am not informed about please let me know.)

As far as I can tell Oxfordshire is actually behind the other two companies in their research. Ceregene's CERE 120 has completed Phase I and will complete Phase II and report results this year in Q4. (Enrollment for Phase II is now complete and patients are still being followed from Phase I as well.) Ceregene entered into a development deal with Genzyme last year that is quite substantial. In addition, Michael J Fox Foundation has contributed funds to Ceregene's research. The results from their Phase I trial are outstanding. Some patients are getting close to 3 years post surgery with no adverse events and continued improvement on standard measures at each follow-up point! This is following a single treatment. PET scans are showing cell regrowth. The cell growth is not out of control in any way.

The Neurologix approach is similar to the Ceregene approach. They have had a similar number of Phase I patients and the findings are very much like the Ceregene findings. Their study was unique in that the patients int he Neurologix study got treatment to only one side of their brains. The results were consistent with this test in that improvement generally occurred on the side of the body that would be predicted by the treatment. The Neurologix patients also showed significant improvement with continued improvement for the study participants who are about 2 years post initial treatment.

Neurologix has just initiated its Phase II study. It is also important to note that the FDA gave Neurologix a Fast Track designation to facilitate their research and potentially bring the drug to market as quickly as possible. (My company works closely with the FDA and Fast Track designation is not easy to come by.)

My wife has had Parkinson's for nearly 5 years now and I know that people with PD have been frustrated in their desire for a cure for years. I don't see gene therapy for PD as a cure, however, the impact looks to be greater than anything else that is in the pipeline at present. For people in the studies that I have read the improvements were in the range of 25%-60%+. Again, please remember that people are continuing to get better over time.

With stem cell research set back by at least a decade by an anti-science Bush administration, I believe that it is far more likely that gene therapy will affect the national and international population of people with this terrible disease. My guess is that (as long as there is no terrible unforeseen event for participants in the clinical trials--over the next year or so) we could see a commercially available gene therapy in the next 3 to 5 years.

That would be very exciting for all of us. Let's hope.

[paula_w]

Thanks for a really clear summary! I am wonderng if the companies share data with each other to create a more complete picture. They are each studying a separate aspect. That would give me reason to hope.


paula

[Howardh]

share your view as too where we are at in terms of a cure for Parkinson's 1990NYboy. Gene Therapy appears to be the next step along the way too a cure, as we approach the Embryonic and Nano Medicine era. Fiona raised a concern I hadn't thought about (visions of raving mutant organisms rebelling against what they were designated to do and ending up as FDA agents or cloned Hugo Chavez socialists.)

Neurologix is one I have been following closely since their success with Nathan Klein and others in 2003. And those trials only involved one side of the brain. The fact that their research team had a New Zealand scientist involved at the top level was exciting, and too hear Neurologix had been granted fast track status in March 08 means their is much too look forward to.

[aftermathman]

that ProSavin was making claims of great efficacy and that there was a lot of optimistic "noise" surrounding the product. I wasn't comparing the two products.

1990nyboy, great summary, ProSavin is behind other gene therapies in terms of clinical trial progress. However its effects are claimed to be obvious within weeks and (as already mentioned), these effects are hoped to be substantial. As a result it appears to be more black and white than the others, it will either crash and burn spectacularly or be very exciting all within a matter of weeks.

On the other hand it may be hideously over hyped in order to gain investment

Fingers crossed,
Neil.

p.s. 1990, there is another trial, the old Avigen now genzyme product, AV201. Seems to be struggling to get interest though and may well have been dropped now that Genzyme are working with Ceregene.

[lurkingforacure]

I can't thank you enough for this, what an excellent and informative summary of information I did not have. Even though it is pouring the rain here, and I mean a gulley-washer as they they, this is such good news I don't even care! Makes me want to go run out and wash the car in the rain!

Thanks for sharing.

Originally Posted by 1990nyboy

Over the past several years I have tried to gain as much information on gene therapy for Parkinson's as I could. I don't believe that gene therapy is another GDNF. I am a medical scientist and work extensively with the pharmaceutical industry. (My research is not involved with PD, but I know enough about science and clinical trials to be an informed consumer of data on gene therapy.)

There are three companies that are working hard in this area with slightly different approaches: (1) Oxfordshire Bioscience-in the UK; (2) Ceregene and (3) Neurologix in the US. (If there are others that I am not informed about please let me know.)

As far as I can tell Oxfordshire is actually behind the other two companies in their research. Ceregene's CERE 120 has completed Phase I and will complete Phase II and report results this year in Q4. (Enrollment for Phase II is now complete and patients are still being followed from Phase I as well.) Ceregene entered into a development deal with Genzyme last year that is quite substantial. In addition, Michael J Fox Foundation has contributed funds to Ceregene's research. The results from their Phase I trial are outstanding. Some patients are getting close to 3 years post surgery with no adverse events and continued improvement on standard measures at each follow-up point! This is following a single treatment. PET scans are showing cell regrowth. The cell growth is not out of control in any way.

The Neurologix approach is similar to the Ceregene approach. They have had a similar number of Phase I patients and the findings are very much like the Ceregene findings. Their study was unique in that the patients int he Neurologix study got treatment to only one side of their brains. The results were consistent with this test in that improvement generally occurred on the side of the body that would be predicted by the treatment. The Neurologix patients also showed significant improvement with continued improvement for the study participants who are about 2 years post initial treatment.

Neurologix has just initiated its Phase II study. It is also important to note that the FDA gave Neurologix a Fast Track designation to facilitate their research and potentially bring the drug to market as quickly as possible. (My company works closely with the FDA and Fast Track designation is not easy to come by.)

My wife has had Parkinson's for nearly 5 years now and I know that people with PD have been frustrated in their desire for a cure for years. I don't see gene therapy for PD as a cure, however, the impact looks to be greater than anything else that is in the pipeline at present. For people in the studies that I have read the improvements were in the range of 25%-60%+. Again, please remember that people are continuing to get better over time.

With stem cell research set back by at least a decade by an anti-science Bush administration, I believe that it is far more likely that gene therapy will affect the national and international population of people with this terrible disease. My guess is that (as long as there is no terrible unforeseen event for participants in the clinical trials--over the next year or so) we could see a commercially available gene therapy in the next 3 to 5 years.

That would be very exciting for all of us. Let's hope.