Phase 1 Trial Celiac Treatment

[Claire]

Some of you may have seen this. It is in the Celiac.com newsletter under Celiac news items.

Alba Therapeutics Announces Phase I Trial for Zonulin Antagonist AT-1001

Alba Therapeutics Corp. today announced that it has begun first human dosing of AT-1001, an orally administered zonulin receptor antagonist that is being developed for the treatment of Celiac Disease (celiac disease). The double blind, placebo controlled dose escalation study will evaluate the safety, tolerability and pharmacokinetics of AT-1001 and is being conducted in twenty-four normal volunteers.

Claire

[debmidge]

I'd like to know more about this...how does it work in a person?

[FaithInScienceToo]

Is this the med the researchers think will prevent 'leaky gut'?

...the one that is supposed to allow the gluten to be digested before the immune system can detect it?

Gina

[Claire]

Is this the med the researchers think will prevent 'leaky gut'?

...the one that is supposed to allow the gluten to be digested before the immune system can detect it?

Gina

<{POST_SNAPBACK}>

I do not think this is the same one but I am not sure. I only found this last night and I will be looking into and get back to the thread if I find out anything more. Claire

[Claire]

I'd like to know more about this...how does it work in a person?

<{POST_SNAPBACK}>

Once upon a time I could have explained this far better but I am removed by years from the technicalities of things like this. Simply put there are receptors on the cells - little guys that are there in take in 'their' stuff when it arrives on the scene. If the receptor for gluten was somehow fixed so it didn't recognize gluten then 'perhaps' the reaction to it would be blocked. The drug somehow interferes with the receptor receiving what it is intended to receive. This is not very technical but generally correct I believe.

Obviously this is not ready for the public at this time. Claire

[Kasey'sMom]

Here's the link to the press release if anyone hasn't read it yet.

Open Original Shared Link

[FaithInScienceToo]

Thanks for the link -

this part of what was written about the med makes me think it is the one that stops the 'leaky gut' thing:

"zonulin appears to be involved in many disease states in which leakage occurs via paracellular transport across epithelial and endothelial tight junctions (tj), and thus may play an important potential role in the treatment of autoimmune diseases. "

Let's hope it works!!!!

Of course, it will take years before it can be determined if it truly works, and then years before the drug would get approved - and, from what I read about it before, it may turn out that it can only prevent the disease reaction in people with the gene who haven't already gotten the immune system disorder yet, since it is unlikely that one drug can prevent all leakage of gluten, before it is able to all get digested...but, let's hope it can!!!!

It's just SO nice to hear that there the drug trial is underway!

Gina

[skoki-mom]

If this stuff works, I'll meet you at the local pizza joint to celebrate! LOL

[FaithInScienceToo]

I'll have mine with pineapple, tomatoes, and green pepper...deep dish, too...with beer...

Oh, gee...am I actually allowing myself to think of ever being able to go to a pizza joint again!? (Pizza Hut...I DO miss you, after all.... )

And, I don't even have any real problem with the gluten-free diet, other than not being able to kiss my boyfriend without having to ask what he's recently eaten/had to drink! [That and the total inconvenience/fear connected with eating out! That part truly sucks... ]

UGH...that other thread about 'what would you eat if you could eat anything' is starting to creep into my consciousness, especially now that the thought of possibly being cured one day is in there too ;-)

I had better now go and make a lovely gluten-free dinner, and get back to my current reality...

...while still hoping for the best!

Maybe, just maybe, we all will one day get to say..."we WERE celiacs!"

Gina

[Claire]

Thanks for the link -

this part of what was written about the med makes me think it is the one that stops the 'leaky gut' thing:

"zonulin appears to be involved in many disease states in which leakage occurs via paracellular transport across epithelial and endothelial tight junctions (tj), and thus may play an important potential role in the treatment of autoimmune diseases. "

Let's hope it works!!!!

Of course, it will take years before it can be determined if it truly works, and then years before the drug would get approved - and, from what I read about it before, it may turn out that it can only prevent the disease reaction in people with the gene who haven't already gotten the immune system disorder yet, since it is unlikely that one drug can prevent all leakage of gluten, before it is able to all get digested...but, let's hope it can!!!!

It's just SO nice to hear that there the drug trial is underway!

Gina

<{POST_SNAPBACK}>

Hi again - think you are right. What I put up is a about the same 'drug' you are thinking of. Here are some interesting sites on the subject -- one is an earlier date and one is dated in the last few days.

RESEARCHERS FIND INCREASED ZONULIN LEVELS AMONG CELIAC DISEASE PATIENTS

Open Original Shared Link (several years old)

Some Random Thoughts on Zonulin

Open Original Shared Link

Study Findings Published in Proceedings of National Academy of Sciences

http://www.celiaccenter.orgpressrelease_diabetes.html

UMB Licenses Zonulin Technologies

Open Original Shared Link

Pharmacy News Article

 9/27/05 - Alba Therapeutics Announces Phase I Trial for Zonulin Antagonist AT-1001

Open Original Shared Link

My earlier remarks about how this would work stands modified! Same principal no doubt except it is zonulin that is being 'antagonized' not gluten. This will be interesting to watch. If you are young enough it may help you one of these days. Claire

[tiredofdoctors]

Hi Claire -- Any info with regard to whether the new med is for enteropathy only???

[Claire]

Hi Claire -- Any info with regard to whether the new med is for enteropathy only???

<{POST_SNAPBACK}>

It looks like celiac and type 1 diabetes are the initial candidates for study but the implications are related to all autoimmune diseases. Keep watch - I am posting additional information and comments. Claire

[Claire]

I am adding this excerpt to what I have already posted on this subject. This is a very good article - something to give to newly diagnosed celiac patients and to family members etc. The website source is listed below. Claire

Is AT1001 the Answer? (from Celiac Disease: Living Gluten Free)

Alessio Fasano, MD, was recently appointed director of the new Mucosal Biology Research Center at the University of Maryland, of which the university’s Center for Celiac Research (CFCR) is an integral part. Fasano, who also directs CFCR, reports that a multidisciplinary group of researchers at the two entities is collaborating to advance knowledge about autoimmunity within the context of celiac disease.

“Our team is convinced that celiac disease is the best disease model for studying basic autoimmunity because we have luxuries with celiac disease that we don’t have with other diseases,” Fasano says. “We know some of the genes involved; we know tissue transglutaminase is the antigen that is the object of the autoimmune response [the protein the body mistakes as foreign]; we know the small intestine is the primary target organ; and most importantly, we know the environmental trigger that leads to the autoimmune process is gluten.”

Fasano explains that under normal circumstances, large molecules like gluten are prevented from entering the body by a formidable barrier that covers the entire intestine. The barrier is a single layer of cells, and the spaces between the cells are tight junctions, dynamic structures that can be conceptualized as gates that open and close.

But what is the key, and when does the key open the gate, and why?

A few years ago, Fasano’s team discovered the key—the protein zonulin, which regulates the opening and closing of the gates and controls gut permeability. “Around the same time, we also determined that most of the autoimmune diseases are characterized by an extremely permeable intestinal wall and that individuals [with impaired immune function], particularly those with diabetes and celiac disease, have abnormally high zonulin levels,” Fasano explains. “Our next task, of course, was to find a way to inhibit the zonulin.”

It didn’t take long for the Fasano team to find an inhibitor and to develop an animal model for testing. Using diabetes-prone rats, the researchers evaluated the effectiveness of the zonulin peptide inhibitor AT1001. The rats were randomized to two groups—one group that received the inhibitor in their drinking water on a daily basis and an untreated control group. Eighty percent of the untreated rats developed diabetes, compared with only 26 percent of the treated rats.

“We know these animals developed diabetes because they leak in their gut, and they leak in their gut because of out-of-control zonulin,” Fasano says. “If you stop the leak by preventing the unusually high level of zonulin from interacting with its target receptor on intestinal cells, you will prevent diabetes.”

The plan now is to test the inhibitor in humans, Fasano states, specifically in individuals with celiac disease. “You would take a pill that contains the inhibitor and 15 minutes later eat some pizza or a Big Mac. The gluten in the food would cause a huge amount of zonulin to be released, but by the time it reached the target, the target would be blocked, and the intestine would not leak. The gluten would remain in the intestine until completely digested and would not have access to the immune system.”

Open Original Shared Link

[Claire]

The following is an excerpt - source is at the bottom. Claire

from:

Alba Therapeutics initiates trial in celiac disease

Alba Therapeutics has begun testing AT-1001, an orally administered zonulin receptor antagonist that is being developed for the treatment of celiac disease.

The double blind, placebo controlled dose escalation study will evaluate the safety, tolerability and pharmacokinetics of AT-1001 and is being conducted in twenty-four normal volunteers.

"The data we plan to generate from this and subsequent phase I studies will educate us on the safety and tolerability of AT-1001, in addition to quantifying the impact of this novel compound on intestinal permeability," stated Dr Blake Paterson, CEO of Alba.

Open Original Shared Link

[FaithInScienceToo]

Thanks, Claire!

I REALLY want to keep track of this now!

Gina