A stem cell treatment that looks beyond dopamine

caldeerster · 09-19-2010, 08:14 PM

might be political and it might be related to what Conductor is talking about. The Doctors who have worked on this for years stand to profit, since they are also the owners of the company. This seems to irk people. I have actually had this discussion with my MDS, who was more hopeful for the Ceregene trials (a privately held for-profit company I believe).

Well, the facts are the Ceregene Phase II trials failed (for whatever reasons - and yes I know they're redoing them) while the Neurologix Phase II trial looks to be a huge success.

The #1 priority here should be fast-tracking therapies that work to the patient - to us. I quite frankly don't care who gets rich in the process as long as we get the new treatments we need.

If politics are playing a part in this, then it's up to all of us to start speaking up VERY LOUDLY about this.

(Conductor - you're absolutely right about the catheter. I hadn't realized Medtronics was the manufacturer.)

Quote:

Originally Posted by Conductor71

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

paula_w · 09-19-2010, 09:39 PM

just a reminder that these doctors and neurologix were the first to bring back gene therapy [at least for pd ] since the death of the boy in PA.

Secondly, remember levesque attributed his patient's improvement to "probably gaba" as his dopamine levels were not elevated by his own stem cells."

lurkingforacure · 09-19-2010, 09:39 PM

My personal thoughts are that with everything offered to us, SOMEONE makes money and I don't really give a rip who or how much, within reason, if the thing being offered is better than what we currently have available. No one can expect someone to spend several decades of his or her life developing something only to give it away. Look how much we spend right now on drugs that provide, at best, marginal benefit? And let's not even talk about those pesky side effects which in many cases almost negate the slight therapeutic benefit the drugs provide.

I dont' get the lackluster excitement either. I think part of it may be the folks I lovingly refer to as The Dopaminers, and there are a LOT of big names in that group, not wanting to admit that maybe, just maybe, there is more to PD than just dopamine.

If this were to go forward in a phase III and really do well, as the prior trials indicate it can, well, then, what does one do about all those dopamine-based treatments and therapies and research out there? This poses quite a threat, I think, to that establishment. Not that the two cannot co-exist together, because that way we learn the most. I hope that can happen, it would only benefit everyone. Heaven knows the PD market is enormous enough to accomodate several platforms of therapy/treatment, and getting bigger every day.

caldeerster · 09-19-2010, 10:29 PM

I wasn't aware of that info from Levesque. So his thoughts were that his adult stem cell transplantation worked (for a few years) due to circuitry repair which normalized GABA rather than dopamine?

Quote:

Originally Posted by paula_w

just a reminder that these doctors and neurologix were the first to bring back gene therapy [at least for pd ] since the death of the boy in PA.

Secondly, remember levesque attributed his patient's improvement to "probably gaba" as his dopamine levels were not elevated by his own stem cells."

imark3000 · 09-20-2010, 01:36 AM

Is any body following up this? It seems to me that this must be related to the discussed stem cell therapy?

The epilepsy drug zonisamide may help curb the tremors and other movement problems of Parkinson's, a Japanese study shows.
Zonisamide is a sulfonamide anticonvulsant approved for use as an adjunctive therapy in adults with partial-onset seizures. Zonisamide may be a carbonic anhydrase inhibitor although this is not one of the primary mechanisms of action. Zonisamide may act by blocking repetitive firing of voltage-gated sodium channels leading to a reduction of T-type calcium channel currents, or by binding allosterically to GABA receptors. This latter action may inhibit the uptake of the inhibitory neurotransmitter GABA while enhancing the uptake of the excitatory neurotransmitter glutamate

Imad

soccertese · 09-20-2010, 02:18 AM

conductor,
you posted
"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."

if the results are good they'll have no problem raising money, getting a deep pocketed partner or getting bought out. they have had no problem raising money.
General Electric Pension Trust controls 32.8% of the company, they'll want to help them find money if there is potential.
http://www.sec.gov/Archives/edgar/da...3g-a_16300.htm
Chrysler LLC Master Retirement Trust controls 15.5% of the company.

management has no problem finding those who have money including medtronic. it will only fail if the results aren't good enough.

Conductor71 · 09-20-2010, 04:40 AM

Quote:

Originally Posted by caldeerster

The #1 priority here should be fast-tracking therapies that work to the patient - to us. I quite frankly don't care who gets rich in the process as long as we get the new treatments we need.

Agreed there should be no question on funding a Phase III given prior results. The focus should be fast-tracking, of course, with all safety parameters firmly in place. There are several celebrities with family members afflicted with PD; honestly, these doctors should not have any trouble finding venture capital. It's troubling that their peers would think less of them because they wish to profit from their own talent and ingenuity. That being said, if there is some sort of capitalist jockeying going on or ploy to delay the clinical trial process for profit, well than, that is another story.

Soccertese, I am quoting from the link Lurking provided on their difficulty in finding Phase III funding:

The problem here is that it lacks the financing to start of phase 3 trial and desperately needs a partner, would Medtronics being a possibility. This is a classic pink sheet stock at a buck and a quarter with very low volume. I'm playing this one like an option and buying the stock on any dips. Any stumble on the data front or the failure to find a partner will wipe this company out – but if all goes well, this is a potential 5 to 10 bagger. High risk, high reward.

Lurking mentioned the "dopaminers" (funny, btw). They should not be too afraid. I doubt that we would be able to entirely stop dopaminergic therapy, but as in DBS, the hope/goal is that we would need less of it.

-Laura

soccertese · 09-20-2010, 05:24 AM

Quote:

Originally Posted by Conductor71

Agreed there should be no question on funding a Phase III given prior results. The focus should be fast-tracking, of course, with all safety parameters firmly in place. There are several celebrities with family members afflicted with PD; honestly, these doctors should not have any trouble finding venture capital. It's troubling that their peers would think less of them because they wish to profit from their own talent and ingenuity. That being said, if there is some sort of capitalist jockeying going on or ploy to delay the clinical trial process for profit, well than, that is another story.

Soccertese, I am quoting from the link Lurking provided on their difficulty in finding Phase III funding:

The problem here is that it lacks the financing to start of phase 3 trial and desperately needs a partner, would Medtronics being a possibility. This is a classic pink sheet stock at a buck and a quarter with very low volume. I'm playing this one like an option and buying the stock on any dips. Any stumble on the data front or the failure to find a partner will wipe this company out – but if all goes well, this is a potential 5 to 10 bagger. High risk, high reward.

Lurking mentioned the "dopaminers" (funny, btw). They should not be too afraid. I doubt that we would be able to entirely stop dopaminergic therapy, but as in DBS, the hope/goal is that we would need less of it.

-Laura

the seekingalpha author is exaggerating, he's an unpaid investment blogger - you or i could write an article for that blog, nrgx has never stated they are desperate. seekingalpha is not the wall street journal. nrgx is not a pink sheet stock btw, it's otc, they have to file quarterly and undergo audits. it's common sense that phase3 will proceed if they get results as least as good as a DBS from phase2, $20-40mill is nuttin. if they do, the only questions left would be safety, cost and duration of benefit. i would think it would be 1/2 the cost.

Conductor71 · 09-20-2010, 07:13 AM

Quote:

Originally Posted by soccertese

the seekingalpha author is exaggerating, he's an unpaid investment blogger - you or i could write an article for that blog, nrgx has never stated they are desperate. seekingalpha is not the wall street journal.

Thanks for the clarification. I don't have a real understanding of how all the financials work or go for start ups.

I just happened to discover that the Movement Disorder Specialist that diagnosed me is involved in phase II of the Neurologix trial. That speaks volumes in that my former doc only gets involved in research that looks like it will make it into clinical practice as a treatment in the near future. He has a good track record. I could have participated in phase II had I known

Anyway, this prompted me to look into GABA a little more closely it looks like the GAD treatment was still in animal model phase just seven years ago, so in FDA time, they are cruising right along.

Turns out that GAD (glutaminergic acid decarboxylase) injected via viral vector has not only the potential to improve our motor symptoms by dampening the over firing circuits in our basal ganglia, it also works with our brain's inherent plasticity. I don't quite get the science, but apparently GAD can alter the normal excitatory glutamergic neurons into inhibitory ones expressing GABA. Within this shift, they have found that dopaminergic neurons are spared. In other words, it treats our symptoms and has the potential to arrest or slow down the loss of dopamine. (Luo 2003)

Funny, my original post is not related at all to Neurologix, but it just goes to show that there is even better potential for stem cell therapy if the embryonic cells can target and produce GABA, they think it might work for dopamine as well. Then we would have restorative therapy.

Laura

paula_w · 09-20-2010, 08:05 AM

Cal and all,

I was going to quote from this but thought you might find it interesting since it was in 2002. This is a patient conducted interview. Whatever else has been said of Dr. Levesque, he was always available for the patients.

http://web.archive.org/web/200610291...transcript.htm

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