Wednesday, December 1, 2010

Possible Cures for Type-1 in the News (late Nov)

Here are various news items on possible cures for type-1 diabetes which are in human trials, and related items:

Diamyd Completes Enrollment of their Second Phase-III Trial

Since this study follows people for about 15 months, it is very reasonable to expect that it will complete in mid 2012.  Since this is the second phase-III trial, if successful, market approval might come in 2013 or 2014.  Diamyd is a vaccine like treatment designed to teach the body's own immune system to stop attacking it's own beta cells.  The company's description is this: "Diamyd® is thought to induce tolerance to GAD, thereby intervening in the autoimmune attack and preserving the capacity to produce insulin in patients with autoimmune diabetes".  Remember, the phase-III trials are only in honeymoon diabetics.

Press release:

Leptin Starts a Phase-I Clinical Trial, but as Treatment, not Cure

I have blogged in the past about Leptin:

And here is the press release for the new news:

Back in 2008 Leptin was presented (in the news, at least) as a possible cure, but by April 2010 it was presented as a possible new treatment.  This most recent news makes it clear that it is a possible treatment for type-1 diabetes, not a cure.  I don't expect to cover this research moving forward.  Remember that even as a treatment, it takes 10 years to move through the regulatory process and get approved.

Single case of Putting Type-1 into Remission for One Year
This is a single patient report (not a research study).  The patient had two diseases: ITS and type-1 diabetes.  To treat ITS, she was given Rituximab about 15 months after being diagnosed with type-1.  The result was that her type-1 diabetes went into remission for 11 months.  (No need for external insulin during that time).   This is reported here:

Obviously, this is interesting.  Especially since there has already completed a phase-II study on type-1 diabetics, and got good results there, too.  (Although I'm embarrassed to say that I've not blogged on the specific results of that study.)

Rituximab targets the CD20 part of the immune system's B cells (different from the pancreas's beta cells) to try to prevent the autoimmune attack. B cells are part of the body's immune system and communicate with the T cells, which actually attack the body's  beta cells in the pancreas.  By targeting the B cells, it is hoped this treatment will stop or lower the attack of the "bad" T cells.

Comment: Most treatments aimed at stopping the autoimmune attack are very focused on stopping the "bad" T cells which directly attack the beta cells in the pancreas.  This treatment (if successful) opens up a whole 'nother way to stop the attack: by targeting the immune systems communication and support system, the B cells.

I have previously blogged on Rituximab here:
And reported status here:

Cautionary Note: Possibly Faked Phased Clinical Trial Data
This news article made me very nervous; the impact could be to many other clinical trials. 
Here is the article:

The basic summary is this:  MannKind is developing an inhaled insulin doing several clinical trials in foreign countries, using contract clinical trial companies.  (These are companies that specialize in just doing clinical trials for other companies.  They don't develop drugs.  They do testing of other company's new drugs.)  A MannKind employee claims that some of the data reported by the contract clinical trial company in Russia is obviously false.  All patients had the exact same blood pressure.   Since FDA relies on these studies to do approvals, this is a big deal.  This one case impacts MannKind, but the general problem could effect others treatments as well.  Did that contract clinical trial company do other trials for other drugs?  Is the problem limited to one company, or does Russia have systematic problems that put all Russian clinical trials at risk?  You get the picture: it's bad.

Omission from Previous Blog Entry
In my last blog (comparing Burt's treatment with LCT's) I did not include the fact that LCT's clinical trials were all people with established type-1 diabetes, while Burt's were all honeymooners (people with recent onset type-1 diabetes).  This is a serious omission.  Working on established type-1 diabetes is a huge advantage over honeymoon type-1 diabetics, and that is a strong point in LCT's favor that was not included in the comparison.  I'm sorry for this omission.

General Background Information

All "Animal Models" are not Equal
This is an interesting blog entry about "animal models" used to test type-1 cures:

General Background for Understanding Type-1 Research
This article describes the immune system in general, what is meant by "honeymoon" and other useful words and ideas, but the Teplizumab clinical trial it talks about has since been stopped.  Also the phase-I/phase-II/phase-III description is for new drugs or treatment.  Already approved drugs or treatments can follow a slightly different path.

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.


Ilya said...

Rather interesting blog. Glad to subscribe to it. Do you have any info about the mentioned in the link below approach to DT1,namely, ketone bodies infusion? Tried to e-mail Dr. Pan to check if she carried out any controlled experiment but in vain for now. Thank you in advance and we'll be happy if you keep me updated.

Ilya said...

Just subscribed through e-mail to the comments in order to get them instantly. Sorry for this second post.

Joshua Levy said...

I looked through my "usual sources" but could not find a clinical trial run by Dr. Pan targeting type-1 diabetes. Her comments made it sound like there was one, but I could not find it. Ilya, if you find that study or any papers that Dr. Pan has published on human trials, please tell me about them! Thanks.