LCT Gets Commercial Approval In Russia!

I had not planned to make another blog entry until Jan-2011, but I felt that the following news was important, so made an exception.   Happy Winter Solstice everyone!

The official sound track for this blog entry is The Blur's "Song 2":  Woooo-hooooo!
http://www.last.fm/music/Blur/+videos/+1-OSB3SUh3Xd8

LCT Gets Commercial Approval In Russia!

Background: LCT is developing an encapsulated pig beta cell cure for type-1 diabetes.
Called "Diabecell", it has pig beta cells encapsulated in a special coating.  The coating allows blood sugar in, and insulin out, but does not allow the body's immune system to attack the beta cells.  It also allows nutrients in and waste products out.  This allows the beta cells to naturally grow and to react to the body's sugar by generating insulin which goes into the body's blood system.  Meanwhile, the body's autoimmune attack can not target these beta cells, and you don't need to take any immunsuppression drugs (as you would for a normal beta cell transplantation).  Remember that for decades, diabetics injected pig and cow insulin every day, so the fact that they are transplanting pig beta cells instead of human beta cells should not make anyone nervous.  Different researchers have been working on this kind of system for decades, but LCT is the first group to get government approval for this sort of "bio-artificial" pancreas.

I completely understand that not everyone considers a bio-artificial pancreas to be a cure.  Some people think it is just a good treatment: a much better treatment that we have now.  I've written this blog entry from the point of view of someone who thinks that a bio-artificial pancreas that really works is a cure.  If you don't believe that, just replace the word "cure" with "better treatment" in your mind as you read this.

So What Does This News Mean: My understanding is this means LCT has approval to sell their Diabecells as a commercial medical treatment in Russia.  LCT already has a commercial presence in Russia, so I would expect that in the next few months we will see actual availability of the treatment there.  LCT has said that they are working with two different clinics in Russia. LCT thinks that in the second half of 2011, you will be able to fly to Russia (if you don't live there already) and get this treatment.

What can LCT's Diabecell Do Right Now: In my opinion, the current performance of Diabecell does not make it a cure.  They have reported on less than 16 people, and only 2 of them were insulin free for any length of time, and those two were insulin free for only a few months.  But don't underestimate LCT's Diabecell just because it is not a cure right now:  First, research is about doing more in the future than you are doing right now, and this may grow into a cure over time.  Second, this treatment almost eliminates very low blood glucose episodes, and that is a benefit even without being a cure.  For "brittle" type-1 diabetics, this might prove reason enough to get this treatment.

Discussion

Just about every one of these discussion points could be expanded to a full blog entry, all by themselves.  I'm just trying to "hit the high points" here.  As you read this discussion, don't forget the central point: these guys have gotten farther along the path of encapsulated beta cells as a future cure of type-1 diabetes than anyone else.  They are the first people to get government approval for something that might, in the future, with some more work, cure type-1 diabetes (at least by my definition of "cure").

Edmunton Protocol vs. Diabecell
In a previous blog I compared Diabecell with Burt's immune system reboot research, but that was unfair because Diabecell works on established diabetics, while the reboot research has only been tested on honeymoon diabetics.  However, a better comparison might be to compare the Edmunton protocol (for beta cell transplants) to Diabecell (for encapsulated beta cell transplants).  Both work on established type-1 diabetes.  Unfortunately, I don't know exactly how successful the Edmunton Protocol is, as used these days, so I can't compare it to Diabecell.  But it would be a great project: a useful head to head comparison of cure rates and durations of these two transplant techniques.

My best guess is that right now, the Edmunton protocol has a much higher success rate (in terms of % of people who don't need to use insulin for some period of time) and a much longer remission rate (length of time they don't need to use insulin).  However, it requires the person to take immune suppession drugs for the rest of their life.  Those drugs have serious side effects, and taking them for years or decades raises the chance of problems in the future (like cancer), and Diabecell doesn't have those side effects.

But I do think that the first commercial impact that Diabecell is going to have is on the Edmunton protocol clinics.  Right now, only a few diabetics get an Edmunton protocol beta cell transplant.  The ones who do are often "brittle" diabetics who experience seizures and are either worried about driving, or worried about "dead-in-bed" or both.  Those diabetics will now have an alternative to the Edmunton protocol, and we will see over the next few years how many of them take advantage of it.

From Here to a Cure
One obvious question is, if the current Diabecell is not a cure, can they make it into one?  And if so, how long will that take?  I think there are two steps needed for Diabecell to become a cure (by my definition):
First, it needs to work better.  Right now, about 90% of the people who get the treatment, continue to need to use insulin.  That's not a cure for me.  
Second, they need their treatment to last longer.  Since it requires an operation, I think it needs to last at least a couple of years.  Five or ten years (or longer) would be reasonable for a cure for me.

The big unknown for me is how hard will it be to improve Diabecell in these two ways.  If it is just an engineering issue, then that is great news: if they can just tinker with it and gradually improve both the success rate and the duration, that would be great.  They could tinker with it for 5 to 10 years and end up with a cure.  On the other hand, there might be a research issue in there that they need to solve.  That would require scientific research and a breakthrough of some kind to make it more successful and last longer.  Research breakthroughs are much harder to predict.  You might get it in a year, or maybe never.

Getting Approval Elsewhere
Another important question is, how quickly will LCT get approval in other places like the US and the EU?  I'm not an expert in government approval, and I know some of my readers know this area much better than I do.  However, I don't think getting approval in Russia is going to speed up the process for getting approval in the US.  I think they are still two or three large clinical trials away from US approval.  Remember, in the end, only 8 people have completed clinical trials with Diabecell, and that only lasted a year.  Eight or twelve more are in the middle of a second trial.  But even if you stretch this to the max, it is still just 20 people for a year or two each.  I'm not sure if that is enough data to even start a phase-III trial (as defined by the US FDA).

This is an important point: LCT got their approval in Russia, because the standards in Russia are much lower than the US and EU.  (I'm sure the LCT guys will have a much nicer way to phrase this.  And I'm sure they will be unhappy that I'm so blunt.)  But, my understanding is that the research they have done to date is no where near enough to get approval in the US or EU, and they are not trying to, right now.  (As I pointed out above, I'm not even sure it is enough to start a phase-III trial.)  Obviously, if you believe that the US FDA and the EU EMEA over regulate and are a bunch of "nervous Nellies" that tie up promising treatments in red tape, well here is your chance to get a treatment that hasn't yet gone though all that approval process.  To be a little crass: nothing bad happened in the first 20 people; your kid (or yourself) can be number 21.

The LCT press release talks about starting a pivotal study in New Zealand in 2011.  (Pivotal usually means the first phase-III study, designed to provide proof of efficiency and safety. Remember that even after this study, to get US approval they will need to do a second ("confirmatory") phase-III study and get marketing approval.)  So it still feels to me like they are 3-6 years away from approval in either the US or EU.  Although the Russian approval was quicker than I thought it would be, so maybe these guys are just faster than I expected.

Cost and Availability
Dr. Elliot at LCT estimated a cost of AU$ 150,000 per operation to start, which is pretty close to US$ 150,000.  There was no mention of exactly how soon the procedure would be started.  There are issues of training, and of transporting the cells from New Zealand to Russia.  At the start, there will be volume limitations, based on the size of LCT's herd of pigs. With that all said, I assume as more people get the procedure done, there will be economies of scale, and it will get cheaper.  An important part of the economic calculations is how long the implanted cells will work.  After all, $150k once is quite different than $150k every ten years, or every year!  And we don't know how long it will last. 

What's The Big Deal?
One way to look at this news is this: nothing has changed in the research.  The results LCT has today are the same as the results they had yesterday.  The only difference is Russian government approval.  So why is this important news?  I think that part of the answer involves the pace of progress in research vs. the commercial world.  Things happen more quickly in the commercial world.  Economic pressure and competition between companies do that.  This news marks part of the transition for LCT from the world of research to the world of commerce.  Not a complete, black and white conversion from one to the other, but a shifting of importance.  Another part of the answer involves availability.  Up until now, you could only get the treatment as part of a research study.  In the future, anyone with money and desire will be able to get it.  So availability will be controlled by the patients who choose to get the treatment, rather than researchers who choose to provide it. (That's over simplified a little, but you get the idea.)

Newspaper article: http://news.smh.com.au/breaking-news-national/animaltohuman-transplant-first-20101210-18sfk.html
Press release: http://www.lctglobal.com/html/blob.php/LCT%27s%20Diabecell%20Registered%20for%20Sale%20and%20Use%20in%20Russica_101210.pdf?attach=0&documentCode=2409&elementId=20084
Forum: http://islet.org/forum/messages/54131.htm
Previous blogging on LCT: http://cureresearch4type1diabetes.blogspot.com/search/label/LCT
Previous status on LCT: http://joshualevy.pbworks.com/w/page/13864073/DiabetesCureReadyForHumanTrials#DiabecellbyLivingCellTechnologies

Joshua Levy
All the views expressed here are those of Joshua Levy, and nothing here is official JDRF or JDCA news, views, policies or opinions. 
Blog: http://cureresearch4type1diabetes.blogspot.com
Web: http://joshualevy.pbworks.com/DiabetesCureReadyForHumanTrials
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