Friday, May 19, 2017

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

Phase-I Study of Of Gluten Free Diet In Honeymoon Type 1 Diabetes (Diabglut)

This trial will recruit 160 children, aged 3 to 18.  It started in December 2015, and is expected to end in December 2020. Half of the people will be put on a gluten free diet within one month of diagnosis, and the other half will not (and will be the control group).

This study is being done in Skanes University Hospital, Lund, Region Skane, Sweden, 22185
Contact: Annelie Carlsson, MD PhD    +46768267170
Contact: Iren Tiberg, PhD, nurse

Clinical trial registry:


My first thoughts when I saw this study were: "Why do a study like this?  Does anyone really believe that a gluten free diet will cure/improve type-1 diabetes?"

The researchers included four previous studies as references for this work, but I think the one that mattered was this one:
This is the case study of a single patient (a five year old boy).  He was was put on a gluten free diet a few weeks after diagnosis, and remained off insulin for 20+ months.  That's an unusually strong honeymoon, and I think it is reasonable to say that the researchers are hoping that they can create that kind of honeymoon in other newly diagnosed people.

For me, case studies (like this one) represent a middle ground between anecdotes and scientific studies.  I don't think they are very strong by themselves, but I do think that following them up with a research study is a good way to proceed.

The other three studies cited by the researchers as background for this research were much less dramatic. One study tried delaying introduction of gluten in the diet of babies: no effect. Another tried putting people with two antibodies on a gluten free diet before diagnosis: no effect on eventual diagnosis, but might have a small effect on beta cell survival after diagnosis. The third study was population based, and suggested that introducing gluten earlier (at 4 months, rather than later) might lower the rate of celiac diagnosed at 12 years. As a population study, I don't put a lot of weight on it, and the impact was to celiac and not type-1, in any case.

Minimal Islet Transplant at Diabetes Onset (MITO)

This is mostly a transplant trial, so I don't expect to follow it moving forward, but it is a different type of transplant, so I'm describing it here.

Most transplants are done on the most seriously impacted people with type-1 diabetes.  These people often have a lot of trouble controlling their type-1, they are often already having serious complications, and are generally on the worst side of the type-1 spectrum.  These are the people who often volunteer for transplantation.

This study is targeting the opposite: people who within 6 months of type-1 diagnoses, and are still generating some residual insulin.  The idea is to transplant some islet cells in combination with ATG, G-CSF, and Rapamycin treatments. It's a "kitchen sink" approach.  All of those treatments are in active clinical trials right now, but none of them has been shown effective so far.

I do think that it will be valuable to get data from people who are not so extremely sick when they get their transplant, although I doubt this will lead to a cure in the short term.

They are recruiting 6 people in Italy:
IRCCS San Raffaele Scientific Institute   Milan, Italy, 20132
Contact: Lorenzo Piemonti, MD   0226432706 ext 39
Contact: Paola Maffi, MD
Contact: Emauele Bosi, MD 0226432818 ext 39

Clinical trial registry:

T-Rex Now Can Enroll People 8 Years Old, and Older

Previously, they could enroll people as young as 12, but they have enough safe results with those kids, so that the FDA will now allow kids as young as 8 to enroll. You can read all about the technique they are using and who is eligible in my previous blogging:

Also, the last time I blogged, this study was only recruiting in two locations, but now they are recruiting all over the US, from Oregon to Connecticut to Florida to UCSF, not to mention Tennessee, Missouri, Massachusetts, Indiana, and that is not a complete list.  (See the Clinical Trial Record, link below, to get a complete list.)

This is a phase-II trial which is a follow on to the previous "Polyclonal T-Reg" study, which I also blogged about here:
(Look at all the postings in this link, except the first one, in order to see the history of this treatment.)

Clinical Trial Record:

Joshua Levy
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.

Thursday, May 11, 2017

Update on the University of Jordan's Stem Cell Project

The University of Jordan recently announced the start of a phase-I trial of stem cells. While researching that, I found that they had already published results from a pilot study done at Stem Cells of Arabia (also in Jordan). So in this blog posting, I will first discuss the results of the pilot study, and then the phase-I study they are starting now. Even though these trials were sponsored by different organizations, I consider them part of the same research program.

What Is Being Tested?

These researchers are taking someone's own blood, and then process it before putting it back into the person's body.  I'm not sure the exact nature of this processing.  The pilot study makes it sound similar to Dr. Zhao's work (exposing the patient's cells to stem cells).  However, an interview in makes it sound like a filtering process.  It may be both.  The description in the FDA Clinical Trial record for their upcoming phase-I study talks about donors and "passaging" the cells, which might be a shorthand for the same process used in the pilot study.  Whatever their process is, it focuses on three types of immune cell markers: CD34+, CD133+, and CD271+. The process is similar to Dr. Zhao's and also to the T-Rex study. This is not a classic transplant, because the patient is getting their own cells back, and immune suppression drugs are not used.

Results from Stem Cells of Arabia's Pilot Trial of Stem Cells

The only information I have is from an abstract. I think it is from a talk given at the 2015 International Cord Blood Symposium conference, and was published in the journal Transfusion (abstract only).
The patients' average age was 35 years old, but ranged from 13-52, but there was no information on their honeymoon status.  The average A1c at start was 9.0, average Fasting Blood Sugar 350 (although I'm not sure what that means in a type-1 diabetic), C-peptide average was 0.06 (three people had 0.00 and one had 0.23.
Here is the complete results section of the abstract: 
There were no complications over the follow up period (14-51 months). Three out of four patients completely stopped their insulin requirement at 6 months. All 4 patients showed significant improvements in Fasting Blood Sugar (average 145), C-peptide (average 1.01), and HbA1c (average 7.0), during 12 months post transplantation.

This is a study where only having the abstract really hurts.  Obviously, these results look very good, but there are key details missing. If the reported C-peptide numbers are fasting, and measured in ng/ml, those are excellent, but if they are in response to eating, then they are not particularly strong. (The abstract does not say.) Having 3 out of 4 patients not using insulin at six months is great, but there is no information on how long each person did not need to use insulin.  The results are great for established type-1 diabetes, but the abstract doesn't say how many people are established vs. honeymooners.

Based on those results, it is easy to see why this group is excited about doing a larger study. I'm excited too!

   University of Jordan Starts a Phase 1 Trial of Stem Cells

This is the start of the study which was previously discussed on ASweetLIfe:
Now the study has officially started and there is a FDA Clinical Trial Record with the details of how the study will work.

This is a 20 person study, which will enroll adults (between 18 and 35) who have been diagnosed within 3 years, or are still generating C-peptide at a rate of 0.5 ng/ml or more. They expect to finish the study in July 2018.  Some people will get a small dose of stem cells, while others get a larger dose.  There is no control group.  Patients will be followed for 36 weeks.  The primary outcome is safety after 6 months.  No effectiveness outcomes are mentioned.

Cell Therapy Center,  Amman, Jordan, 11942
Contact: Abdalla Awidi, MD    0096265355000 ext 23960
(I think this phone number would be  +96265355000 ext 23960, where + means "whatever you need to do to get an international number".)

Web site: and
Clinical trial registry:


This is a quick study.  Expecting to have results in less than 18 months is much faster than most clinical trials, and from my point of view, that's a good thing.  On the other hand, there is no mention of reporting on the things that people with type-1 would care about.  No A1c data, no C-peptide data, and no insulin usage data is listed in the secondary outcomes.  Hopefully those things will be reported on, but most studies (even phase-I studies) list these as secondary outcomes.

This research is based on the same basic ideas which also led to Dr. Zhao's "Cell Educator" research, but the two approaches are not the same treatment.  Dr. Al-Zoubi's group is taking stem cells from a person with type-1 diabetes, treating them, and then injecting them back into that patient.  Dr. Zhao is taking stem cells from third parties, and then exposing people's immune cells to proteins made by those cells, but the stem cells themselves are never injected into the patient.   But both approaches are trying to harness proteins created by stem cells.

Joshua Levy 
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.