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“Living Cell Technologies Parkinson’s disease treatment shows signs of efficacy in phase II study”

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https://smallcaps.com.au/living-cell-technologies-parkinsons-disease-treatment-efficacy-phase-ii-study

Over the past 2 years, NTCELL has undergone a phase 1/2a clinical trial for the treatment of Parkinson’s disease in New Zealand and has met the primary endpoint of safety whilst “halting the progression of the disease three years after implant”. Results from that trial were used to design the larger phase 2b trial which is still ongoing.”

 

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More detail on NTCELL here:

https://scienceofparkinsons.com/2017/02/23/how-pigs-are-helping-with-parkinsons-disease/

tl; dr:

The treatment involves injecting capsules of pig cells into the brains of Parkinson's patients.

Phase IIB trial was completed in May 2017. According to Living Cell Technologies website:

"At 26 weeks post treatment, the trial was unblinded. It showed that NTCELL was safe and well tolerated but at this stage, did not meet the primary efficacy endpoint. Patients will be assessed again at 52 weeks post implant and we are waiting for these follow up results."

More recently, the company posted:

"The one year follow up of the 18 patients in the Phase IIb study of NTCELL® for Parkinson’s disease shows a statistically significant improvement change in the Unified Parkinson’s Disease Rating Scale (UPDRS Part III in the off state) in the patients who received 40 or 80 NTCELL capsules implantation to the putamen on both sides of the brain as compared to the placebo group that received sham surgery."

As yet, no publications listed for any of these results.

A few questions:

What is 'statistically significant'? Is it a few points off the UPDRS, or a couple dozen points off? Is that in patients with relatively high UPDRS scores, or relatively low scores?

If Living Cell Technologies isn't bragging about the actual numbers, doesn't that suggest the numbers aren't very impressive?

I'm skeptical--Living Cell Technologies wants to bring this to market as soon as possible (even before Phase III), but it seems like a pretty invasive treatment for likely modest results.

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5 hours ago, PatriotM said:

I wonder if this is a one time treatment or if multiple surgeries would be required over time?

Not sure.  I know its been said before, but the next 5-10 years of Parkinson’s therapy will be groundbreaking.  In either direction.  Either they’ll have breakthroughs or find out there are no breakthroughs : (

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I know its been said before, but the next 5-10 years of Parkinson’s therapy will be groundbreaking.  In either direction.  Either they’ll have breakthroughs or find out there are no breakthroughs : (

For the reasons above, I'm a bit skeptical of Living Cell Technologies' drug, but actually quite optimistic about PD research over all.

There's a good argument that we're less than five years from a breakthrough (as in, announcement that a new treatment has shown significant results in Phase III trials), and availability to patients of new treatment just a few years after that.

It's been nearly 20 years since the last big breakthrough (DBS), and there's more research going on now that at any time in the past.

Immunotherapy approaches are currently very exciting--the potential is treatment that will dramatically slow PD progression, or stop it all together. Both active and passive approaches are showing encouraging results in Phase I, and at least two companies have started Phase II. We should hear about the first Phase II results by 2020 or 2021:

https://scienceofparkinsons.com/2018/05/19/affiris/#more-50669

https://scienceofparkinsons.com/2018/06/22/prothena/comment-page-1/#comment-3812

https://scienceofparkinsons.com/2018/04/25/biib054/

There's also a raft of repurposed drugs and OTC supplements in Phase II or III trials, including Isradipine, Inosine, Nilotinib, and Exenatide. If any of these show significantly better than placebo results, they could be available immediately (since already FDA-approved for other conditions). As you can see from my signature, I'm currently taking Isradipine off-label (with MDS supervision) on the chance it's shown to be disease-modifying.

A Japanese group is about to start Phase I trials with induced pluripotent stem cells. This is real-deal research, not the fly-by-night stem cell quackery that's making money for sketchy clinics and putting desperate people at risk:

https://scienceofparkinsons.com/2018/08/01/ips-cell-trial/

And I understand trials of sub-cutaneous levodopa pumps are going well, with availability expected in just a few years. This is the same idea as the Duopa pump, but much more convenient: a little needle under the skin (like an insulin pump), rather than a surgically implanted tube in the intestine.

 

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1 hour ago, ShopGuy said:

For the reasons above, I'm a bit skeptical of Living Cell Technologies' drug, but actually quite optimistic about PD research over all.

There's a good argument that we're less than five years from a breakthrough (as in, announcement that a new treatment has shown significant results in Phase III trials), and availability to patients of new treatment just a few years after that.

It's been nearly 20 years since the last big breakthrough (DBS), and there's more research going on now that at any time in the past.

Immunotherapy approaches are currently very exciting--the potential is treatment that will dramatically slow PD progression, or stop it all together. Both active and passive approaches are showing encouraging results in Phase I, and at least two companies have started Phase II. We should hear about the first Phase II results by 2020 or 2021:

https://scienceofparkinsons.com/2018/05/19/affiris/#more-50669

https://scienceofparkinsons.com/2018/06/22/prothena/comment-page-1/#comment-3812

https://scienceofparkinsons.com/2018/04/25/biib054/

There's also a raft of repurposed drugs and OTC supplements in Phase II or III trials, including Isradipine, Inosine, Nilotinib, and Exenatide. If any of these show significantly better than placebo results, they could be available immediately (since already FDA-approved for other conditions). As you can see from my signature, I'm currently taking Isradipine off-label (with MDS supervision) on the chance it's shown to be disease-modifying.

A Japanese group is about to start Phase I trials with induced pluripotent stem cells. This is real-deal research, not the fly-by-night stem cell quackery that's making money for sketchy clinics and putting desperate people at risk:

https://scienceofparkinsons.com/2018/08/01/ips-cell-trial/

And I understand trials of sub-cutaneous levodopa pumps are going well, with availability expected in just a few years. This is the same idea as the Duopa pump, but much more convenient: a little needle under the skin (like an insulin pump), rather than a surgically implanted tube in the intestine.

 

I hope you’re right.  My coworker has PD.  We just had coffee.  I want nothing more than for him to get well.  Great dude.  He’s 69 and I like to turn to him for advice here and there.

I have to agree about the Japanese research. When I read it it made total sense.  If PD is a relatively boutique disease, to which broad medication only has variable results in respect to the variable progression —but progression nonetheless— it makes sense that an individuate approach would be a necessary outcome at the genetic/cellular level.  

I sort of figured that national healthcare systems would be first to implement this.  After reading Dr Okun’s abstract on the future of PD disease, it is definitely headed towards pandemic levels, and unfortunately only states with national healthcare systems are prepared to handle it.  

Do you think, objectively, a cure is likely in your lifetime?  

Best,

Zer0

Edited by zerohours000

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1 hour ago, zerohours000 said:

Do you think, objectively, a cure is likely in your lifetime?  

Depends what you mean by 'cure.'

If 'cure' means: undergo some (short) course of treatment, or take some pills, or have a surgery, and boom! PD's all gone and everything's back to normal—I'm with Patriot on that: never going to happen. It's wishful thinking, as in wishing for magic.

In fact, I'd say for me at least, living my life in hope of such a cure would not only be enormous waste of time, it'd likely lead to depression and despair, as the years go by and the magic cure fails to materialize.

I'm optimistic about better forms of treatment (because I think the evidence supports optimism), but I never use the word 'cure' and if I had my way, PD organizations like MJFF and the Parkinson's Foundation wouldn't either. It's not realistic, and I expect it turns off more than a few donors who become cynical waiting for a promised cure that never arrives.

PD is a complex disease--anything as simplistic as what most folks seem to imagine when they say 'cure' is pretty unlikely. 

From the Science of Parkinson's Blog: 

Quote

As we have discussed before, any ‘cure’ for Parkinson’s requires 3 components:

1. A disease halting mechanism – slowing or stopping the progression of the condition

2. A neuroprotective agent – a treatment that will protect and support the remaining cells

3. Some form of cell replacement therapy – introducing new cells to replace the ones that have been lost

Now, the bad new is that there is no ‘silver bullet’ on the horizon that provides all three (for example, there is no neuroprotective agent that also replaces lost cells).

Regarding the Japanese stem cell trial: it's very interesting research but there's good reason not to hold your breath. If the hypothesis that PD is a prion-like disease is correct, then just replacing dead neurons with new ones won't prevent eventual death of the replacements--there's evidence from past transplant research that this may happen. I'm a lot more hopeful about numbers 1 & 2 on the SoP list (but then, I don't have advanced PD--yet--so would still see quite a bit of benefit even without cell replacement).

2 hours ago, zerohours000 said:

After reading Dr Okun’s abstract on the future of PD disease, it is definitely headed towards pandemic levels, and unfortunately only states with national healthcare systems are prepared to handle it.

Dr. Okun's notion of 'pandemic,' as I read it, doesn't mean the likelihood of getting PD is increasing, just that an aging population will have more total cases. World pop. in 2040 will be about 9 billion; if there's 14 million or so people with PD at that time (likely I'll still be one of them) that's still fewer than 1 in 600. The real problem is going to be finding enough people of working age to take care of all of us old folks (with or without PD), and I'm not sure any nation is prepared to handle that--Japan is already running into problems.

As far as I know, the various forms of national healthcare are all intended to provide access to existing forms of treatment, not develop new ones. That's the job of researchers in both public and private sectors, supported by public and private funding. Public funding usually goes to basic research, without which private research geared toward commercialization and profit would be impossible (and vice versa). I believe the US govt. is one of largest funders of medical research in the world, through the NIH; love 'em or hate 'em, research by private pharmaceutical companies (many based in countries with government-managed healthcare) is also critical. And in recent years, it's hard to overestimate the impact of the (private but nonprofit) MJFF.

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3 hours ago, ShopGuy said:

Depends what you mean by 'cure.'

If 'cure' means: undergo some (short) course of treatment, or take some pills, or have a surgery, and boom! PD's all gone and everything's back to normal—I'm with Patriot on that: never going to happen. It's wishful thinking, as in wishing for magic.

In fact, I'd say for me at least, living my life in hope of such a cure would not only be enormous waste of time, it'd likely lead to depression and despair, as the years go by and the magic cure fails to materialize.

I'm optimistic about better forms of treatment (because I think the evidence supports optimism), but I never use the word 'cure' and if I had my way, PD organizations like MJFF and the Parkinson's Foundation wouldn't either. It's not realistic, and I expect it turns off more than a few donors who become cynical waiting for a promised cure that never arrives.

PD is a complex disease--anything as simplistic as what most folks seem to imagine when they say 'cure' is pretty unlikely. 

From the Science of Parkinson's Blog: 

Regarding the Japanese stem cell trial: it's very interesting research but there's good reason not to hold your breath. If the hypothesis that PD is a prion-like disease is correct, then just replacing dead neurons with new ones won't prevent eventual death of the replacements--there's evidence from past transplant research that this may happen. I'm a lot more hopeful about numbers 1 & 2 on the SoP list (but then, I don't have advanced PD--yet--so would still see quite a bit of benefit even without cell replacement).

Dr. Okun's notion of 'pandemic,' as I read it, doesn't mean the likelihood of getting PD is increasing, just that an aging population will have more total cases. World pop. in 2040 will be about 9 billion; if there's 14 million or so people with PD at that time (likely I'll still be one of them) that's still fewer than 1 in 600. The real problem is going to be finding enough people of working age to take care of all of us old folks (with or without PD), and I'm not sure any nation is prepared to handle that--Japan is already running into problems.

As far as I know, the various forms of national healthcare are all intended to provide access to existing forms of treatment, not develop new ones. That's the job of researchers in both public and private sectors, supported by public and private funding. Public funding usually goes to basic research, without which private research geared toward commercialization and profit would be impossible (and vice versa). I believe the US govt. is one of largest funders of medical research in the world, through the NIH; love 'em or hate 'em, research by private pharmaceutical companies (many based in countries with government-managed healthcare) is also critical. And in recent years, it's hard to overestimate the impact of the (private but nonprofit) MJFF.

Thanks.  That was super informative.  I think you’re right about those first two.  Hopefully those two can improve quality of life.  I guess I was thinking more of in the line of quality of life that has improve for those with HIV, for example.  Different pathologies, I know, but I can’t think of anything similar.  Would finding biomarkers be of any use in this situation?  

Thanks again,

Zer0

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@ Zer0,

I'm not a doctor or a PD researcher, so anything I have to say is only an interpretation of what I've read. Maybe a misinterpretation.

That said, the guy who runs the Science of Parkinson's blog is a PD researcher--in my opinion, it's a great source of up-to-date, detailed information. The Michael J. Fox Foundation also regularly published updates on the current state of PD research. Here's something they linked to regarding alpha-synuclein that you might find interesting:

http://www.readcube.com/articles/10.1038/nrd.2017.95?shared_access_token=X_NzUw7vu4_cNX_TEJtn69RgN0jAjWel9jnR3ZoTv0MS9PnCoWe_UuQ5GUohdo8M22W0grgKbbY0Rjxk-Vz1YYT1UTtOMNWc3SksCsfsmTh4xco4oNZrI_E7NGyViQhYjiobKCbl_-7CDRPQM8QAJA%3D%3D

In my view, there's a number of reasons biomarkers for PD would be useful--one of those is the possibility of early intervention when (and if) a treatment to slow/halt progression becomes available. Actually, such a treatment exists now--it's called 'exercise,' which is more or less free from negative side effects and useful for all sorts of conditions. Tough for a lot of people to stick with, however, unless there's some strong incentive.

With regard to your friend with PD: it's natural to want to help, but my recommendation would be to resist making suggestions or trying to share ideas unless he specifically asks. Most folks, by the time they decide to go public with their condition, have a pretty good handle on where they're at and what treatment options look like.

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