[Conductor71]

Funny, I was noting how much I liked Rick's thought in his Stress post:
"There is more in the world than dopamine" When lo and behold, I stumbled upon this little gem reported early this spring.

Apparently, scientists have discovered a novel form of stem cell treatment involving embryonic medial ganglionic eminence (MGE) cells. This type of treatment is unique because it completely bypasses the lack of dopamine. Given that with traditional stem cell treatment, the aim is to restore dopamine...only to have the new dopamine cells killed off all over again.

With the MGE cells, the GABA neurons are inhibited and it slows down the excitatory process that takes place in PD. Better yet, this may treat more than just PD:

The results, the scientists say, demonstrate that the transplanted cells, known as embryonic medial ganglionic eminence (MGE) cells, can very precisely modify the balance of excitation and inhibition in neural circuits to influence behavior. As overactive neural circuits are associated with other neurodegenerative diseases -- a result of nonfunctioning or missing cells or abnormal synaptic transmission -- the finding may have broad implications.


The Full Monty

I must say things are looking a little brighter these last few days.

Laura

[lurkingforacure]

Laura,

Wow, this is interesting because there is a professor I believe in Ohio, During or Durig is his last name, who also works in New Zealand. He splits his time between the two. He is the guy who is developing a GABA-based therapy for PD. I cannot remember the name of it but it doesn't involve dopamine, rather, like your article, it works on calming things down.

We mentioned this to our neuro who is at the forefront of PD stuff and he could have cared less. My take was that he is on the dopamine bandwagon and any treatment that does not target that is not worth looking at. But we are.

So interesting to read this, thanks!

[caldeerster]

working with Kaplitt and the Neurologix team?

Cal

Originally Posted by lurkingforacure

Laura,

Wow, this is interesting because there is a professor I believe in Ohio, During or Durig is his last name, who also works in New Zealand. He splits his time between the two. He is the guy who is developing a GABA-based therapy for PD. I cannot remember the name of it but it doesn't involve dopamine, rather, like your article, it works on calming things down.

We mentioned this to our neuro who is at the forefront of PD stuff and he could have cared less. My take was that he is on the dopamine bandwagon and any treatment that does not target that is not worth looking at. But we are.

So interesting to read this, thanks!

[lurkingforacure]

An interview with Durig:

http://www.medscape.com/viewarticle/460396

and in the news more:

http://www.cornellneurosurgery.org/a...nsDisease.html

http://tvnz.co.nz/content/1195234/425826.html

and most recently, in the news because it needs funding for a phase III trial (wonder why Fox Foundation is not assisting here?...)

http://seekingalpha.com/article/2246...-1-big-concern

[caldeerster]

If the results from Phase II turn out to be as successful as anticipated, hopefully someone will step up to the plate.

Cal

Originally Posted by lurkingforacure

An interview with Durig:



http://www.medscape.com/viewarticle/460396

and in the news more:

http://www.cornellneurosurgery.org/a...nsDisease.html

http://tvnz.co.nz/content/1195234/425826.html

and most recently, in the news because it needs funding for a phase III trial (wonder why Fox Foundation is not assisting here?...)

http://seekingalpha.com/article/2246...-1-big-concern

[Conductor71]

Originally Posted by caldeerster

If the results from Phase II turn out to be as successful as anticipated, hopefully someone will step up to the plate.

Cal

It looks like Phase II was successful, so I wonder why this is stalled out. I wonder sometimes if alternative ideas or potential therapies languish because so much more press is given to something else? In this case, I am thinking this GAD approach is overshadowed by GDNF? A lot of us know Ceregene, but I hadn't heard of Neurologix until now.

Anyway, looks like MJFF did fund Phase II. They post a very positive outcome.

http://www.michaeljfox.org/newsEvent...cle.cfm?ID=640

Lurking, thanks for the clinical trial history review! Looks like Dr. During and Dr. Kaplitts may be on to something big.

This statement from MJFF really hits home for all of us, doesn't it?

"While dopamine clearly plays a role in Parkinson's disease, dopamine levels in the brain are inherently difficult to control, resulting in sub-optimal treatment outcomes for patients. We believe that by altering chemical targets further downstream in the brain's network that regulates movement, we have the potential to help improve outcomes and restore motor function for patients with advanced Parkinson's disease," added Matthew J. During, MD, DSc, scientific co-founder of Neurologix, Inc

[caldeerster]

I'm reading your link and trying to find confirmation of that.

In one of Lurking's links, the financial writer mentions that perhaps Medtronics will partner up with Neurologix.

Since Neurologix' aim with this breakthrough medicine is to make DBS obsolete, what financial incentive would Medtronix have to shoot itself in the foot?

Originally Posted by Conductor71

It looks like Phase II was successful, so I wonder why this is stalled out. I wonder sometimes if alternative ideas or potential therapies languish because so much more press is given to something else? In this case, I am thinking this GAD approach is overshadowed by GDNF? A lot of us know Ceregene, but I hadn't heard of Neurologix until now.

Anyway, looks like MJFF did fund Phase II. They post a very positive outcome.

http://www.michaeljfox.org/newsEvent...cle.cfm?ID=640

Lurking, thanks for the clinical trial history review! Looks like Dr. During and Dr. Kaplitts may be on to something big.

This statement from MJFF really hits home for all of us, doesn't it?

"While dopamine clearly plays a role in Parkinson's disease, dopamine levels in the brain are inherently difficult to control, resulting in sub-optimal treatment outcomes for patients. We believe that by altering chemical targets further downstream in the brain's network that regulates movement, we have the potential to help improve outcomes and restore motor function for patients with advanced Parkinson's disease," added Matthew J. During, MD, DSc, scientific co-founder of Neurologix, Inc

[soccertese]

supposedly they will present results sept 28, got this off another message board but no link to verify.

phase3 depends on funding but phase2 results just came out. medtronic supplies the catheters for infusing the virus/gene mixture.

Under a manufacturing and development agreement, the Company and Medtronic, Inc. (“Medtronic”) have co-developed a new catheter infusion device to infuse the Company’s gene transfer product into the brain with respect to the treatment of Parkinson’s disease. (See “– Manufacturing”). The FDA reviewed and approved the use of this device in connection with the Company’s Phase 2 clinical trial for its Parkinson’s disease product under the Company’s investigational new drug application (“IND”). The use of such a catheter facilitates the delivery of the Company’s gene transfer treatment by neurosurgeons and simplifies the procedures for infusing the gene product into the brain. In order for the Company to market its products, Medtronic must obtain the FDA’s approval for the commercialization of such catheter infusion device, and the Company must obtain sufficient quantities of the catheter infusion device whether from Medtronic or another manufacturer. (See “Risk Factors”).

The Company is currently taking steps to move toward a pivotal trial for the treatment of Parkinson’s disease, and hopes to be in a position to file its protocol with the FDA in 2010 or 2011. The Company’s conduct of such a trial will require, among other things, approval by the FDA and adequate funding. Currently, the Company estimates that the pivotal trial could be completed in 2013 and the estimated total direct costs to reach that milestone are expected to be between $20 million and $40 million. (See “Risk Factors” and Item 7 – “Management’s Discussion and Analysis of Financial Condition and Results of Operations”).

http://www.sec.gov/Archives/edgar/da...03249e10vk.htm

they are getting help looking for phase3 financing, doesn't mean they will get it or FDA will approve phase3.
http://phx.corporate-ir.net/phoenix....437&highlight=

[imark3000]

Is this a break through? Stage 2 successful tryal on humans !? A feasible alternative to DBS but much simpler !
What are the chances of its becoming a commercially available treatment in 2-5 years?

Imad

[Conductor71]

Originally Posted by caldeerster

I'm reading your link and trying to find confirmation of that.

In one of Lurking's links, the financial writer mentions that perhaps Medtronics will partner up with Neurologix.

Since Neurologix' aim with this breakthrough medicine is to make DBS obsolete, what financial incentive would Medtronix have to shoot itself in the foot?

Maybe we can make this a little cause celebre and make enough noise to help get them the funds they need to proceed.

Caldersteer,

Oops, I had always thought that MJFF funded the potential newer therapies they reviewed? If they have not, I apologize for the misinformation. Maybe Debi Brooks can verify?

I don't see how Medtronic is at odds with developing and funding the infusion catheter. It's actually a smart move. Yes, DBS may be a cash cow, but sooner or later less invasive treatments will be taking over DBS. If this gene delivery system is a success, it'll be a few (ahem) years before we see any benefit. They are still taking in the profits from DBS and positioning themselves for next the next treatment paradigm. If Medtronic did not fund/back it then some other company would be making loads of money. In fact, just the idea of Medtronic funding it means it is HUGE and that they believe it will be available as a treatment that makes DBS obsolete. Besides, they can still gank up the prices as much as they want.

Soccertese,

Yes, they do have to prove themselves to the FDA, but if you read Lurking's history of their research and trials, it looks like they have really learned from other studies where people were harmed and have redressed the problem.

My main concern is in looking over the company's web site, it is clear they think they are onto something big in treating PD. The same doctors pioneering the method are also founding members of Neurologix. They are trying a business start up to capitalize on what they have done; can't blame them, but they are essentially a penny stock. They are trading at $1.27. I may actually look into purchasing some. Better now than when they release results of Phase II trials on 9/28. I just wish they could get some real venture capital going in to keep them afloat. I hate to see the whole thing stalled because they can't raise capital. Calder is right, we need to get the word out. They need a lot more publicity.

Laura