New T1 therapy with no shots, no testing, no carb counting goes to phase 1 trials

Saw this on diabetesmine and wanted to share. I dont know much about this therapy but just a little bit of reading it certainly looks interesting!

We’re making big strides towards a band-aid sized device implanted under the skin of people with diabetes that would release insulin as needed, potentially eliminating the need to check blood sugars, count carbs and dose for insulin through injections or a pump!

Yes, on Friday news broke that San Diego-based biotech company ViaCyte filed an Investigational New Drug (IND) application with the FDA to move forward with Phase I/II clinical trials in people with type 1 diabetes. The trial would evaluate the safety and efficacy of a new stem cell-derived encapsulated cell replacement therapy, known officially as VC-O1. Basically, that product uses pancreas endoderm cells derived from embryonic stem cells and would be put into the body using something called the Encaptra delivery system.

You may remember from our past coverage that Encaptra is a flat device about the width of a credit card. It would be implanted in under the skin (possibly in the upper back) through a simple outpatient surgical procedure and designed to last at least a year, possibly up to five years, before it would need to be replaced.

This device would be loaded with insulin-producing cells before implantation, and contains pores that allow glucose and insulin to be transferred through, but not antibodies — meaning insulin would be released as needed in response to the varying glucose levels, but no immuno-suppression drugs will be necessary because the device is protected from autoimmune attack by the sheet’s membrane. Very cool!

More on the original article:

<serious comment>Wow... that's awesome!!</serious comment>

Next is testing the T2 version: A little bigger (2 ft x 2 ft), and is worn via adhesive on the back for up to 5 years. You give up any flexibility, but they're talking about a program to sell advertising space on the large square as a means to help cover the cost. Patients would get credits for any activity, like the beach, that presents to a wider audience.

Rumor is the Mars candy company has secured the first slot for advertising.

(all a joke, for the humor impaired)

This may be the answer, but who knows? After the Phase1/2 trials that address safety and effectivity, there will probably follow a larger scale Phase 3 trial to see how it works in a much larger group.

Given all the attendant regulatory delays, if this is the real deal, if will be many years before we get a chance to try this therapy. Getting around the requirement for immune suppression therapy usually needed for any transplant, is a big deal, for me.

I like the idea that this is a more natural solution and eliminates/reduces the need for exogenous insulin. That will reduce or eliminate hypos, the bane of using insulin. It should also produce excellent BG control. I'm hopeful, but won't hold my breath. This will take many years, if it's successful at all.

Dave - All humor aside, if this therapy is successful for T1Ds, I can't help but think that the lure of the enormous T2D market will induce movement in that direction. It still doesn't address, however, the underlying insulin resistance that many T2Ds struggle with.

I believe that the ultimate cures for T1D and T2D will be different. And not in my lifetime. I hope I'm surprised!

I agree completely. And I hope you're wrong about the cure :-)

Follow / Show me the money... the T2 market is where the money is. In my view, as a T2, success with this will, as you say, spur much more research to get that 2' x 2' patch down to something manageable for us T2s :-)

While this is a promising direction, my favorite treatment research is Smart Insulin, and I'm hoping like mad that it succeeds.

The idea of taking an injection once a day, once a week, whatever schedule, that simply responds to BG directly is a dream I have every day.

Well, yes Terry as T1 and T2 are two completely different diseases ;)

Agreed ;-)

I was encouraged to read this clearly written position statement published by the American Diabetes Association recently. The opening paragraph included this:

Type 1 diabetes is characterized by an immune-mediated depletion of b-cells that results in lifelong dependence on exogenous insulin. While both type 1 and type 2 diabetes result in hyperglycemia, the pathophysiology and etiology of the diseases are distinct and require us to consider each type of diabetes independently.

I like the smart insulin concept as well but I don't think it's even started the regulatory review process.

Sounds good, is this the same as encapsulation where you won't need anti-rejection drugs? I guess it is since it says no imuno- suppression drugs needed...

Assuming (hoping) that this device proves its worth through all phases of clinical trials, would anyone care to speculate when it will finally be on the market?

I can't find any info about clinical trials at their site yet. I emailed to ask about this. My endo said, referring to encapsulation, that he doesn't think it will really happen for 10-15 years. I hope he is wrong. So the way to try this earlier would be to enter a clinical trial if possible.

this actually brought tears to my eyes. i dont care about having to wait 15 or 20 years for something like this. it sounds amazing and liberating! this or smart insulin-anything that hasnt got a gazillion pieces of hardware like a bionic pancreas!

I can not recall exactly how old this membrane idea really is now. Maybe 15 years +/- 5. First they wanted to encapsulate cells from other humans, then pig cells and now human embryonic stem cells. The autoimmune reaction can not penetrate the membrane. I do not get why embryonic stem cells have to be used? Why are ordinary donations not sufficient? Where are these embryonic lines from? Are there moral implications? What if the membrane malfunctions and multipotent unspecialized stem cells can be released?

I wish them to be successful in the following trial phases. In another 10 years we will know how this worked out...

Holger - I've read that it takes two cadaver donations for an islet cell transplant. I've also read that it is the supply that limits these procedures. I don't know a great deal about this science but I understand that embryonic stem cells can produce an inexhaustible supply. I can't answer your other questions but I do hold out hope that this line of research may produce a highly effective automatic treatment for diabetes. I fear that 10 years may be overly optimistic.

I read a similar account of the islet cell and stem cell applications which is why they switched to stem cells. I don't know because islet cell transplant is already done but doesn't last very long in most people if it is effective. Maybe, we can only hope, that it will be sooner than what is expected.

Just for clarification: for an islet cell transplant you will have to sign a paper that states you have been informed about the risks of this treatment. Risks that are higher than those of Diabetes Type 1 in my opinion. With the new method a tiny membrane will make the decision whether the risk applies or not. Not very comforting to know that a tiny accident could open the pandora. Well, you will have to sign off these risks as well.

Today all embyonic stem cells are from in vitro fertilized embryos that are not used for the generation of pregnancies. Actually this is so questionable that even the US legislation does not allow this method. All the lines we have are derived from lines that have been started in countries with less strict legislation like Sweden. Obviously this does not solve the moral issue. For future lines there are methods proposed that can derive the cell from embryos with 8 cells. The remaning 7 cells survive the procedure and can be refrigerated to become fully developed embryos later. Although this is better and the stage to derive the cell is very early this just moves the problem into the future. Now we have 7 cells left should we trash them or do we take another one out? At the end it still might be the decomposition of an embryo. Of course it is daily practice that fertilized embryos are not used / destroyed. It is the side effect of this kind of fertilization technique. Still the dilemma remains where life really begins. 8 cells does not sound much. But these are fully operational cells with all the inner workings. For instance the mechanism to copy the DNA has a countable number of atoms. In operation it acts very efficient and smart. The separation between matter and life can get very fuzzy on this level.

Holger, I was diagnosed with T1 diabetes in 1952.
Since then I must have been told at least twice per year that diabetes will be cured within five years.
If there even was a cure it would be 10 years before the FDA approved it. The Feds still maintain that a CGM is experimental.
Dr Denise Faustman of Mass. General is in stage two of a totally different cause and end of the condition known as Type 1 diabetes. She is really an impressive and accomplished woman. Has permanently cured T1 in rats.
If your not familiar with her work please check it out and please get back to me.

One correction:

I wrote: "for an islet cell transplant you will have to sign a paper that states you have been informed about the risks of this treatment. Risks that are higher than those of Diabetes Type 1 in my opinion."

But I meant: "for a stem cell transplant you will have to sign a paper that states you have been informed about the risks of this treatment. Risks that are higher than those of Diabetes Type 1 in my opinion."

I know Dr. Denise Faustman's work. The proposed moderation of the rogue behaving t-cells with BCG is indeed promising. Patience is all we need and your experience is the same than mine. It just takes another 10 years to get there...