Saturday, September 21, 2013

Possible Cures for Type-1 in the News (Sept)

This blog posting is a collection of little news items, rather than a single large one.

Two Year Delay for LCT

In a very brief announcement, LCT said that it would delay general commercial availability of it's encapsulated pancreatic product (DIABCELL) from 2016 to 2018.  It sounds to me like they hit a problem in their phase-II study (an open label clinical trial in Argentina), and they need to fix that problem before continuing with their testing for approval.

LCT is developing an encapsulated islet cure for type-1 diabetes. Pig beta cells are wrapped in a coating and implanted into people.  The beta cells generate insulin in response to glucose, while the coating prevents the body's immune system from rejecting or attacking the new beta cells.  Several groups are developing technology like this, but LCT is the farthest along, as they are the only company with results from multiple human trials.

News: http://www.irasia.com/listco/au/lct/press/p130829.htm
Press release: http://www.lctglobal.com/html/blob.php/ASX_130829.pdf?attach=0&documentCode=5126&elementId=20084

ATG (Thymoglobulin) is Unsuccessful in a Phase-II Clinical Trial

ATG (non-human sourced, human T cell antibody infusion) is approved in the US for transplant rejection issues (not type-1 diabetes).  This was a phase-II clinical trial involving 58 people to test it's use for type-1 diabetes.  After one year, the C-peptide production in the treated group and the placebo group was about the same.  Here are two summaries of the results:
Our findings suggest that a brief course of ATG does not result in preservation of β-cell function 12 months later in patients with new-onset type 1 diabetes.

We recorded no between-group difference in the primary endpoint: participants in the ATG group had a mean change in C-peptide area under the curve ...
Press Release: http://www.sciencedaily.com/releases/2013/08/130827203920.htm
Summary: http://www.thelancet.com/journals/landia/article/PIIS2213-8587(13)70065-2/fulltext
Wikipedia information: http://en.wikipedia.org/wiki/Thymoglobulin

Phase-I Results of Dapagliflozin as a Treatment

Dapagliflozin is a pill used for treating type-2 diabetes.  It has been approved for use in the European Union, Japan, and elsewhere, but was rejected by the US FDA.  It has been resubmitted for approval in the US, and that second approval is still under consideration.  It is in a family of drugs called SGLT-2 inhibitors.  


Because the drug has already gone through phase-I, phase-II, and phase-III testing for type-2 diabetics, this early trial in type-1 diabetics was a phase-II study.  62 patients from 5 different locations were included.  Half got the drug, half got a placebo.  All had A1c numbers of 8.5% or higher and were treated for 3 months. 

The results were good, for a treatment.  A1c numbers dropped 0.7 to 1.0 for treated patients, and BG numbers were 30-40 points lower. 

Canagliflozin (Invokana) is a similar SGLT-2 inhibitor which is approved in the US, but I don't think it's been tested on type-1 diabetics as yet.

I don't plan to cover Dapagliflozin in the future, because it is a treatment not a cure, but I did think it was an interesting drug for type-1 diabetics who have trouble controlling their BG and have higher than desired A1c numbers.

Press release: http://www.clinicalendocrinologynews.com/news/top-news/single-article/dapagliflozin-explored-in-type-1-diabetes/5b2de5a29aa324691649e6a01768441b.html
Wiki: http://en.wikipedia.org/wiki/Dapagliflozin

Good Sources of News

I know that it is hard to find good news sources these days.  Two sources that I think are worth reading are:

DiaTribe newsletter: http://www.diatribe.org/ 
This is one of my favorite sources of information.  These guys understand type-1 diabetes and all of it's complexities.  They are not just cutting and pasting other people's press releases into their news articles, but actually applying their own knowledge and expertise to their reporting and analysis.

Ellen Ullman's Scoop.it page: http://www.scoop.it/t/diabetes-and-more   
This is a collection of media news stories, but it is a very good collection of media news stories. Unfortunately, the media does not do a good job of reporting on type-1 cures or treatments.  The reasons for this are too complex to discuss here.  But Ellen's page is my favorite way of reading about diabetes in the media.

Note that both of these sources cover both type-1 and type-2 diabetes, so be ready to mentally filter out the type-2 stories, if you only care about type-1.

Donating to Non-Animal Research

I was asked the following question: "I like to donate every year to help find a cure [for type-1 diabetes]. However, I don't want to support testing on animals. Have you come across any research groups that you think are effective but don't test on animals?"

I don't know of any research aimed at curing type-1 diabetes that has never used animal trials. Indeed, it is hard to see how such research could exist, given our current technology, and the technology we expect to have for the foreseeable future.  Curing type-1 diabetes is expected to require both changing the immune system and regrowing beta cells.  Both of these are complex interactions that occur only within actual animals.  There is no way to test either one of these effects in single cell organisms (which don't have separate immune systems or beta cells), or tissue samples, or in computer simulations.  So researchers either need to test on animals, or guess wildly, and then test in humans.  The second path is blocked by the FDA (because it's unsafe), but even if not, it's almost impossible that such guess work could lead to a cure.  

Now, if you want to only donate to research which is not currently using animals (which has already transitioned into human trials) then there are several research projects to choose from.  Any research in human trials would be a good target.  You can see a list from last September here:
http://cureresearch4type1diabetes.blogspot.com/2012/09/jdrf-funding-for-cure-2012.html
and I expect to post an updated list for this year in a few weeks.

But to put it bluntly: right now, if you are not willing to fund animal research, you are not going to fund a cure for type-1 diabetes.  For type-1 diabetes, cell cultures, tissue samples, and computer simulations are not good enough for research use.

Joshua Levy -- http://cureresearch4type1diabetes.blogspot.com
publicjoshualevy at gmail dot com
All the views expressed here are those of Joshua Levy, and nothing here is official JDRF or JDCA news, views, policies or opinions. My daughter has type-1 diabetes and participates in clinical trials, which might be discussed here. My blog contains a more complete non-conflict of interest statement. Thanks to everyone who helps with the blog.

3 comments:

Łukasz Moliński said...

Bad news about LCT. I hoped the sooner they hit the market the sooner this treatment would be available for an average diabetic as the costs would go down with time. I read somewhere this can oscillate around hundreds of thousands of dollars.

Neha Elecwire said...
This comment has been removed by a blog administrator.
Steve said...

Combs Lab is working on creating an oral non-insulin treatment that lowers glucose eliminating all of the injections, monitoring and side effects that comes from a daily insulin regimen. The brainchild of Dr. Terry Combs working out of the University of North Carolina, this new therapy is in its initial stages of development but holds terrific promise for all sufferers of this dreadful disease.

In order to help make this dream a reality we have started a crowdfunding campaign and would love your help in spreading the word. You can check it out here https://www.microryza.com/projects/can-we-eliminate-insulin-injections-for-patients-withtype-1-diabetes

If you’d like I can send you some additional information or write a short piece that you can re-post. I’ll look forward to discussing this with you more and I appreciate your help in letting us work with you to help inform all who are concerned with the advancement of diabetes treatment.

Thank you for your work and support.

Steve