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

Rilonacept Starts a phase-I Clinical Trial

About six months ago, I blogged about three clinical trials which had not yet started, but were expected to start "soon".  Now the Rilonacept study is actually starting. 

This drug has not previously been tested on type-1 diabetes, but is already approved for other diseases (since 2008 to treat CAPS) under the name Arcalyst.  It is currently in use in about 12 phase-II and phase-III clinical trials for several different inflammation based diseases, especially Gout. It is an IL-1 inhibitor, and IL-1 is known to be involved in the destruction of beta cells in people with type-1 diabetes.  The hope is that this treatment will result in a longer, stronger honeymoon, and may result in long term improvements in A1c (such improvements might lead to fewer serious complications years or decades down the road).  This is the third treatment targeting IL-1 that I'm following. Others include Anakinra and Canakinumab.

The study will enroll 15 patients, all of who will be treated with Rilonacept (no placebo group).  To enroll you must be over 16 years old, within 5 years of diagnosis, and still producing some C-peptides (> 0.2 nmol/L) in response to food. These people will be followed for six months, involving five clinic visits, and it is hoped the trial will be complete by June 2012.  The treatment is weekly injections (under the skin, like insulin).  They will be looking for general safety issues, as well as effectiveness (A1c, C-peptides, and insulin dose).  This is NOT a honeymoon only trial, by my definition.

This is being run out of Children's Medical Center (Dallas, Texas, USA):
Principal Investigator: Perrin C White, MD    perrin.white@utsouthwestern.edu

If you are interested in participating, send Dr. White some email.  Don't worry about your C-peptide production.  If you meet the other criteria, they will test for that.

It is interesting to me how much the design of this clinical trial has changed in the last six months.  You can compare what I'm writing now, to what I wrote back then:
http://cureresearch4type1diabetes.blogspot.com/2010/06/three-new-treatments-preparing-to-enter.html
to see the differences.

Clinical Trial Record: http://www.clinicaltrials.gov/ct2/show/NCT00962026

Thanks very much to Dr. White for providing information about this trial.

News on the Animas Artificial Pancreas

News article on Animas's Artificial Pancreas.  Obviously, I'll be a lot more interested when it actually starts a clinical trial:
http://www.bizjournals.com/philadelphia/print-edition/2011/01/21/artificial-pancreas-awaits-fda.html

Background reading on Artificial Pancreases in General

This paper is a little dry and a little dense, but it gives a good summary update of where we are, and the lead author (Hovorka) is actively involved in AP research:
http://www.touchendocrinology.com/articles/progress-closed-loop-systems-type-1-diabetes-0?page=0%2C0

Reminder About The Blog

I'm not a doctor or research professional of any kind.  I can not provide medical advice, because I lack the knowledge.  I don't recommend one clinical trial or another.  I think that any discussion about participating in a clinical trial must be held between you and your medical team.  I know not everyone has a doctor they trust, and I'm sorry about that, but I can't give you advice on which clinical trial you should be part of, or avoid. 

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
To Get as Email Join here: http://groups.google.com/group/type-1-diabetes-clinical-trials-news

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

MicroCHIPS Will Start phase-I Trial of new CGM (and gets 16+ million)

For people interested in the artificial pancreas project, MicroCHIPS just got about 16 million dollar investment, and one of the things they will do with that money is start a phase-I study later in 2010 on their CGM technology. Here is their description of why their technology is better than current technology:

MicroCHIPS hopes their product will last months, or even years, and their main innovation is to create the implant with a differently-built sensor. Current devices, Pax says, have a single sensor that can only detect glucose levels until it runs out of the sensing chemical. But the MicroCHIPS device employs a redundant array of sensors, with each one becoming active as needed.

And the part that interests me the most is this:

MicroCHIPS intends to conduct initial clinical testing in 2010 to advance its diabetes program.

Some Discussion

I think it is clear that CGM technology is the weakest component in current artificial pancreas research. However, a CGM technology that lasted 3 months or more in the body would be a huge improvement in this area. So I'm excited by this technology, and am looking forward to seeing how well it works. One of the new funders is Medtronic. Since Medtronic already has cutting edge commercial CGM technology, they are not expecting more of the same: they are expecting better than they have. So I think it is fair to expect that whatever MicroCHIPS is creating, it is a lot better than we have now.

Press Release: http://www.mchips.com/10_Jan_07_pr.html
News Article: http://www.dotmed.com/news/story/11267/

Novo starts phase-II Clinical Trial of oral GLP-1

Novo Nordisk (one of the largest pharma companies targeting type-1) is working with Hvidovre University Hospital in Denmark to test an pill version of Victoza (generic: Liraglutide) which is a GLP-1 receptor agonist. They are specifically targeting type-1 diabetics in this study. Technically a phase-II trial (because the drug is already approved), it will enroll about 30 people, and be completed by second half of 2010.

A Little History

Several treatments leveraging GLP-1 have been tried over the years as GLP-1 related drugs help the pancreas generate more insulin. They are usually targeted at type-2 diabetics, who have a lot more useful pancreas to work with. Byetta is the most famous GLP-1 analog used by type-2 diabetics. Of course, the question here is: will it help people with type-1 diabetes?

Some Discussion

This is right on the edge of what I consider a possible "cure". It seems unlikely to me that any treatment that works by causing the pancreas to generate more insulin will -- by itself -- result in a cure for type-1 diabetes. However, I'm reporting on this because it might turn out to be part of a cure. For example, by combining it with Diamyd's, ToleRx's or MacroGenic's treatments (all in phase-III clinical trials, and all of which prevent/weaken the autoimmune attack) .

The injected form of this drug (Victoza) was approved for use in the EU in 2009, and is marketed in at least three countries (UK, Denmark, and Germany). However it has not been approved in the US, because of worries about thyroid tumors. The US FDA review was in April 2009.

If this drug turns out to help type-1 diabetics, then (in addition to Byetta) there are two other similar drugs, each being developed by a different drug company, which may also help type-1 diabetics: albiglutide and taspoglutide.

Press Release: http://www.reuters.com/article/idUSLDE60C0CB20100113?type=swissMktRpt
Clinical Trial Record: http://www.clinicaltrials.gov/ct2/show/NCT00993720
Wikipedia: http://en.wikipedia.org/wiki/Liraglutide

News (but no Announcement) from Faustman

Although Faustman's team has not made an official announcement (at least not one that I've seen), they did reopen enrollment in their phase-I BCG clinical trial, after having closed it last February. Without information on why they did this, there is no way to know if this is good news or bad, or if it will cause a delay in reporting their phase-I results. Although you'd think if it was good news they would have had an announcement.

This is the timeline: In Feburary 2009 they updated their clinical trial record to show that they had finished enrollment in their phase-I trial. In mid 2009 they announced that their results would be published in early 2010, and specifically would be turned over to their internal statistics group in November of 2009. However, in October 2009 they updated their clinical trial record again to show that they were once again enrolling patients in the study. Right now, their web site says specifically they are NOT enrolling new patients, but their Clinical Trials web page says that they ARE enrolling new patients.

What might this mean? The best possible news would be that they are trying to do more with their phase-I trial than originally expected. LCT did this for their trial in Russia. Once it was underway, they updated it to include more people and bigger doses. If the BCG team is trying the same thing, that would be good news.

The worst news would be that they did some early data analysis of their results, and found they did not have statistically significant results. So they added more people to the study in the hopes of getting a statistically significant result. The DiaPep227 guys did this with their phase-III study.

Middle of the road news would be that some of their samples or data got lost, mislabeled, or a patient pulled out at the last minute or something. So they need some more patients to enroll, and might have a delay, but nothing really bad. Something like this happened to Diamyd's phase-II LADA trial.

Clinical Trial Record: http://www.clinicaltrials.gov/ct2/show/NCT00607230

JDRF, J and J's Animas Corp, and DexCom Start Joint AP Project with Clinical Trial this Year

For people interested in the artificial pancreas project, there was a big announce from JDRF of a joint project with Animas (pump makers, and a division of Johnson & Johnson), and DexCom (CGM makers). It's been widely publicized, so I won't go into details. To me, the most interesting part was this:

The first patient testing could begin in less than a year, Kowalski said.

and that is good news, especially since most phase-I AP testing can be done quickly. We should start seeing clinical trial results on this AP combination maybe by end of this year, but more likely sometime next year.

News Article: http://abcnews.go.com/Business/wireStory?id=9550823

Joshua Levy