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

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

Afgun   January 07, 2019   Comment  

ViaCyte Starts A Three Year Follow-up Safety Study in Subjects Previously Implanted With VC-01™
This clinical trial is studying people who were part of ViaCyte's clinical trial of VC-01™, an encapsulated beta cell cure.  Once the device is removed at the end of the study, the patients can enroll in this follow on study which tracks them for three additional years, looking for adverse effects.
This clinical trial studies people who participated in the VC-01TM clinical trial of ViaCyte, an encapsulated beta cure. Once the device is removed at the end of the study, patients can participate in this study, which tracks them for an additional three years, in search of adverse results.

Discussion

I'm hopeful that this means that one person has finished the ViaCyte protocol, which is motivating them to start this follow on.  The other option is that they are just planning ahead.  Since there is no control group, the interim data could be published, if ViaCyte wanted.

Two Phase-I Studies Start with Umbilical Cord Treg Cells 
These two studies have a lot in common, so I'm going to discuss them together: first, their similarities, then their differences. Here are the similarities:

• They are both run by the same researcher (Dr. Zhiguang Zhou) at the same hospital (Second Xiangya Hospital of Central South University).
• They recruit at the same place: Institute of Metabolism and Endocrinology, Second Xiangya Hospital, Central South University, Changsha, Hunan, China, 410011
  Contact: Zhiguang Zhou, MD/PhD    86-731-85292154    zhouzg@hotmail.com
• They have already started recruiting 40 people, and expect to finish in 2019.
• These trials are open to people who have been diagnosed within 3 years and are between 6 and 60 years old.
• Each has a primary end point which is safety related, and secondary endpoints which are effectiveness related and include C-peptide, A1c, insulin usage, etc.
• Both studies will work with stem cells which have been harvested from umbilical blood, separated into components, and had the T-reg cells "grown out" for two weeks.  These enriched T-reg cells will be infused into patients.  T-reg cells are regulatory cells which are part of the immune system, and work by controlling other immune cells so that those other cells don't attack beta cells.

The first study is Safety Study and Therapeutic Effects of Umbilical Cord Blood Treg on Autoimmune Diabetes: This study will have two groups, one will get the treatment, and one will be a control group and will not get the treatment.

Clinical trial registry: https://clinicaltrials.gov/ct2/show/NCT02932826

The second study is Safety and Efficacy of Umbilical Cord Blood Regulatory T Cells Plus Liraglutide on Autoimmune Diabetes: This study has four groups.  One will get the treatment and also Liraglutide, another just the treatment, a third just Liraglutide, and a fourth will be a control group.  Liraglutide (sold as Victoza) is similar to exenatide (Byetta), which is a common type-2 medication, but is also sometimes used on type-1.  Victoza is a weekly injection. 

Clinical trial registry: https://clinicaltrials.gov/ct2/show/NCT03011021

Discussion

The researchers are not clear on why they are using Liraglutide, but site its "various benefits for beta cells". They expect it will increase the effectiveness of the new T-regs, possibly by encouraging beta cell growth.

JDCA's Update on Dr. Faustman's Research

An update on Dr. Faustman's BCG research by the JDCA (Juvenile Diabetes Cure Alliance) is here:
http://eepurl.com/cHrGqX

My key takeaway points are:
* The Phase-II trial should finish in 2023.
* They have enrolled 125 out of the 150 they need.
* An 8 year follow up from their Phase-I trial should be published by the end of 2017.

(Remember, I am a fellow of the JDCA and we regularly discuss various research programs, including this one.)
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.

ViaCyte Clinical Trial News: VC-01 is Fully Enrolled, VC-02 Starts

Encapsulated Transplant VC-02 ViaCyte

ViaCyte Clinical Trial News: VC-01 is Fully Enrolled, VC-02 Starts

Afgun   January 07, 2019   Comment  

This blog posting covers two important pieces of news from ViaCyte.

ViaCyte's Phase-II Trial of VC-01 is Not Recruiting
(But What does that mean?)

I missed this news, when it was announced back in May 2017, but ViaCyte's Phase-II clinical trial of VC-01 is not recruiting more people.  I'm not sure if this is a "pause" between their phase-I group and their phase-II group, or if they have finished recruiting all people for both groups.  That's important because they are gathering effectiveness data for six months.  Whichever group they recruited in May, data on efficiency should be available in November. Effectiveness data would be good for 15 people, but 65 of them would be better. Since this trial is not blinded (and does not have a control group), they can publish their data if they wish.

They also collect security data for two years and in May 2019 the same group will finish collecting safety data. The same " 15 people are good 65 people is better" and " they can publish if they want without a control group" also applies to these safety data.



Differences Between ViaCyte's VC-01 and VC-02 Treatments

ViaCyte has started a clinical trial for their VC-02 stem cell implantation product, but as this treatment is not a cure (by my definition of "cure"), I will not be following it.  Don't mix up VC-02 with their earlier VC-01 product.  VC-01 is a potential cure, and I will follow it moving forward.

I found this confusing: why would a company test a non-cure after they had a cure already in the pipeline, and ahead of the non-cure?  Why even bother with the non-cure?

While VC-01 and VC-02 are encapsulated stem cells, the nature of the encapsulation is completely different.  VC-01 uses a "strong" encapsulation which prevents the body's immune system from attacking the new cells. This prevents both the normal rejection (ie. the foreign organ reaction), and type-1 rejection (ie. the malfunctioning autoantibody reaction) from attacking the new cells.  Therefore, VC-01 does not require anti-rejection drugs.  VC-02 uses a "weak" encapsulation which holds the stem cells together and in one place, and encourages new blood vessels to integrate into the encapsulation, but provides no immune protection.  People getting VC-02 will need to take anti-rejection drugs [d1].

Both VC-01 and VC-02 have the same cells inside.  They both start with ViaCyte's PEC-01 cells. These are created by harvesting human embryonic stem cells from a long-existing culture and treating them so they differentiate into pancreatic cells.  The cells that are inside the devices are these pancreatic cells.   (There are no "raw" stem cells in the device.)  This process is described on

The obvious question is, why would any person with type-1 diabetes choose to be in the VC-02 trial, if they could be in the VC-01 trial?  The immunosuppression required by VC-02 has known bad side effects, and there are known risks in taking those drugs for decades.

First is that the VC-01 trial is not recruiting right now.  So if you want an encapsulated ViaCyte stem cell treatment right now, VC-02 is your only option.  I don't know if the VC-01 trial is completely enrolled, or if it will open up again, to gather a second group of patients.

Second is this: The ViaCyte team believes that the reason some implanted beta cells work and some fail is "vascularization".  Vascularization is the body's ability to grow blood vessels into the new beta cells so that they can get oxygen, remove waste products, and generally integrate with the host person.   ViaCyte believes that the encapsulation used in VC-01 will allow this vascularization and therefore be successful.   However, they also believe that VC-02 will have even better vascularization, and therefore an even higher chance of success.  So if someone currently has type-1 diabetes, they may choose to have VC-02 because it has a higher chance of success (even if this is a trade off against a known higher risk from the treatment).

The VC-02 Study

Although there is only one official clinical trial of VC-02, there are two patient groups within this trial (which they call "cohorts"), and these cohorts are really separate phase-I and phase-II trials. It's just that one clinical trial registry covers both. The first cohort will be 15 patients, all of whom will get a low dose version of the treatment. The second cohort will be 40 patients, all of whom will get a higher dose version of the treatment. There is no control group. They expect to finish the second cohort in Dec 2020.

They are collecting their primary safety data at 4 months post transplant, and primary effectiveness data (C-peptide data) at 6 months post transplant.

They are recruiting at two sites:

• University of California San Diego, San Diego, California, United States, 92121
  Contact: Study Coordinator    1-844-317-STEM (7836)    alphastemcellclinic@ucsd.edu
• University of Minnesota Recruiting, Minneapolis, Minnesota, United States, 55455
  Contact: Study Coordinator    612-626-4993    kreel001@umn.edu

5 Health Food In This Year

5 Health Food In This Year

Afgun   September 02, 2018   Comment  

These is about some good food for your healthy, there some recomended food to consumption every day.

As the new year approaches, everyone is looking for a healthy reset. Maybe you want to lose some of that holiday weight or just adopt healthier habits heading into the new year. And even if your resolution isn’t to shed pounds, it’s always a good idea to take a look at what you eat every day and make some healthy changes.

That’s why we’ve compiled a list of 20 foods you need to incorporate into your diet. And even if some of these foods are in your fridge, it’s worth eating even more of them. From gut-healing probiotics to heart-healthy omega-3s, antioxidants and essential vitamins, these are the healthiest foods you need to stock in your kitchen. Looking for even more healthy eating inspo? Check out our list of 40 Healthy Snack Ideas To Keep You Slim.

1 Green Teagreen tea Shutterstock

Green tea isn’t just good for a jolt of caffeine; this antioxidant-rich beverage can help you blast away belly fat. Researchers in The 7-Day Flat-Belly Tea Cleanse attribute the fat-burning properties of green tea to catechins, specifically EGCG—the name of a group of antioxidative compounds that blast adipose tissue by revving the metabolism, increasing the release of fat from fat cells (particularly in the belly), and then speeding up the liver’s fat burning capacity. It gets better: Research suggests that combining regular green-tea drinking with exercise may maximize the weight loss benefits. In one study, participants who combined a daily habit of 4-5 cups of green tea with a 25-minute workout lost 2 more pounds than the non-tea-drinking exercisers.

2 Kefirkefir Shutterstock
Although this smoothie-like dairy drink resides next to yogurt, if you have a dairy-intolerance, this might be your better pick. That’s because kefir has been found to counteract the effects of the milk’s stomach-irritating lactose: Ohio State University researchers found that knocking back this fermented drink can reduce bloating and gas brought on by lactose consumption by 70 percent. What’s even more promising about kefir is that its bacteria have been found to colonize the intestinal tract, which makes them more likely to confer their healing benefits to your gut.

3 Kombuchakombucha Christina Stiehl
You’ve probably seen bottles of kombucha lining your grocery store refrigerator section but may not know why it’s a healthy choice. Kombucha is a slightly effervescent fermented drink made with black or green tea and a symbiotic culture of bacteria and yeast, known as a SCOBY. This fermented tea is filled with gut-healthy probiotics which can help balance good gut bacteria and help boost your immune system. Plus, kombucha still has the healthy properties of tea, including antioxidants.


4 Chlorophyll WaterChlorophyll Water Shutterstock
Chlorophyll water is a big trend popping up in health food stores and juice bars, and it’s exactly what it sounds like—water infused with chlorophyll, the pigment that makes plants green and helps them create energy from sunlight. Although there may be a health halo around this beverage, there are some healthy benefits. Chlorophyll water contains vitamins A, C, and E, and it could help keep you healthy; a study in The Journal of Surgery found supplementing with chlorophyll may serve as a powerful immunity boost, able to increase wound healing time by up to 25 percent. Just be sure to check with your doctor before drinking it, especially if you’re pregnant or breastfeeding.

5 Bone BrothBone broth in bowl Shutterstock
Bone broth may seem like another health food craze, but there’s ample evidence to back up sipping on this warm beverage. The broth is made when animal bones (usually beef or chicken) are left to simmer in water for an extended period of time, which breaks down their collagen and other nutrients. Some of that broken down material from the cartilage and tendons is glucosamine (which you may have seen sold as a supplement for arthritis and joint pain). According to a study published in the journal PLOS One, when overweight, middle-aged adults took a glucosamine supplement, they were able to decrease serum CRP (inflammation biomarker) levels by 23 percent more than those who didn’t take a supplement. The stock is also full of anti-inflammatory amino acids (glycine and proline), and the ample levels of gelatin will help rebuild your gut lining to further assist with your anti-inflammatory gut microbes.

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

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

Afgun   November 19, 2017   Comment  

Merck Cancels "Smart Insulin" (MK-2640) After Unsuccessful Phase-I Trial

This is the news everyone hoped we would not get.  After completing a phase-I trial of "Smart Insulin" (also known as MK-2640), Merck has decided not to move forward with it.  They have not published the results, but have published this: "MK-2640 was discontinued due to lack of efficacy".

News report:  http://www.pharmaceutical-journal.com/news-and-analysis/features/towards-a-smarter-insulin/20203828.article

Discussion

This was the first (and so far only) glucose responsive insulin to be tested in humans.  However, there are several "smart insulins" being tested in animals, not to mention "smart artificial cells" and "smart membrane" based technologies, all aimed at automatically regulating the amount of insulin in the blood stream.  I expect some of these to enter human trials in the next few years.  So "smart insulin" may yet be a trail blazer, even if it itself was unsuccessful.

If results from this study are ever published, I'll blog on them here.  However, there is nothing forcing Merck to publish these results if they don't want to.

Phase-II T-Rex Trial Update

Caladrius Biosciences recently announced that they had enrolled the 70th patient (out of 111) in their clinical trial of CLBS03 for T1D.  So it has taken them roughly 18 months to recruit 2/3 of the patients they need.  If they can keep up that pace, they will finish recruiting in the third quarter of 2018, and finish collecting data in the third quarter of 2019, and publish in 2020.

Previous Blogging: http://cureresearch4type1diabetes.blogspot.com/search/label/Polyclonal%20Tregs

A quick summary of this treatment is as follows: remove one specific type of T regulator cell (called "CD4(+)CD25(+)CD127(lo)") from a person with type-1 diabetes.  Grow them out so you have about 500 times more, and then put them back in the same person.  Since regulatory T cells naturally regulate the body's immune system, and the patient now has more of them, the hope is that they will prevent the autoimmune attack which causes type-1 diabetes.

They are recruiting patients in about 15 locations all over the US, so read the clinical trial registry to get a complete list and some contact information.

News Report: https://www.caladrius.com/press-release/caladrius-biosciences-announces-enrollment-of-the-70th-subject-in-the-phase-2-t-rex-clinical-trial-of-clbs03-for-type-1-diabetes/
Clinical Trial Registry: https://clinicaltrials.gov/ct2/show/NCT02691247

SIMPONI (Golimumab) Starts A Phase-I Trial In Pre-Symptomatics

Golimumab (sold as Simponi) is an immune system modulator, which has been approved in the United States and many other countries for treatment of several autoimmune diseases, so testing it on type-1 diabetes makes a lot of sense.  This is the second trial underway for this drug.  (The other one is called T1GER, and is for honeymooners.)  Simponi has already been approved to treat rheumatoid arthritis, psoriatic arthritis, ulcerative colitis, and ankylosing spondylitis.

This study is recruiting people who have two autoimmune antibodies (but no other symptoms of type-1 diabetes). They are recruiting 30 kids (aged 6-21), and will follow them for 17 months. Each kid will get 26 weekly injections.  Half the patients will get Simponi and the other half will get a placebo.  They hope to finish this study in mid 2021.

This study is current recruiting in Linkoping University Hospital, Linkoping, Sweden, SE 58185.
Study contact: 844-434-4210 JNJ.CT@sylogent.com
They hope to start recruiting at other locations in Sweden and Finland soon.

The unusual thing about this trial is that they will not measure any effectiveness data at all.  The only data gathered will be safety and side effect data.  No C-peptide data, no A1c, or insulin usage.  That's very unusual for a type-1 diabetes study.  In my experience, even the phase-I studies gather some effectiveness data.

Clinical Trial Record: https://clinicaltrials.gov/ct2/show/NCT03298542
Previous Clinical Trial Record: https://clinicaltrials.gov/ct2/show/NCT02846545

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.

Results From Phase-I Clinical Trial of Proinsulin Peptide Vaccine in Honeymooners (MonoPepT1De)

Results From Phase-I Clinical Trial of Proinsulin Peptide Vaccine in Honeymooners (MonoPepT1De)

Afgun   November 05, 2017   Comment  

For over 10 years there has been a research group at Cardiff University (UK) working on creating vaccine-like treatments to cure or prevent type-1 diabetes, and this blog posting covers their recent results.

As background: one of the autoantibodies that is associated with type-1 diabetes targets insulin molecules [d1]. Therefore, there is a theory that giving people with T1D a protein fragment from insulin might prevent or delay the onset of type-1 diabetes.  It would train the body not to produce this autoantibody. The process is vaguely similar to giving small amounts of peanut proteins to people with peanut allergies [d2].  These researchers are using a peptide (a small part of a protein) from proinsulin, which is a precursor of insulin.

Results From Phase-I Clinical Trial of Proinsulin Peptide Vaccine in Honeymooners

This trial involved 27 people divided into three groups.  One group got a placebo and was a control group, the other two groups got the Proinsulin Peptide injections for 6 months.  One group got the peptide every 2 weeks, the other every 4 weeks.  The people were adult, honeymoon diabetics (within 100 days of first insulin injection).  They will be followed for 3 years, although this publication only covers the first year after treatment.

The untreated group went through a normal honeymoon period, where over time they gradually generated less and less of their own insulin, and had to inject more and more.  However, the treated people (on average) held steady in their ability to generate their own insulin, and did not lose their ability to generate insulin for the time covered. So this meant that the treated group did better than the untreated group over time, and the difference was statistically significant.  Also, there were no safety issues.

JDRF funded this trial specifically, and this whole line of research, in general.

News Coverage:
http://www.medicalnewstoday.com/articles/318899.php
http://www.latimes.com/science/sciencenow/la-sci-sn-diabetes-immunotherapy-20170809-story.html
Abstract: http://stm.sciencemag.org/content/9/402/eaaf7779
Full Paper: http://stm.sciencemag.org/content/9/402/eaaf7779.full
Clinical Trial Record:  https://clinicaltrials.gov/ct2/show/NCT01536431

Discussion

I think there are three important points for this research:

First, these results are good enough to spur a phase-II trial, and the Cardiff researchers have already made it clear they hope to run a phase-II trial starting in 2018.  Two of the authors of this paper are consultants to UCB Pharma to help design that trial.  So that is good.

These results join a growing number of treatments with what I call "medium good" results in honeymooners.  "Medium good" means that the treated group did not get worse, but did not improve either.  This is in comparison to the untreated group which did get worse.  (During the honeymoon phase, people with type-1 diabetes get worse: they gradually lose the ability to generate insulin.  They go from generating a little insulin, to generating none.)  I think there are about half a dozen treatments which have phase-I or phase-II? results of this type.  So it's better than nothing, but because none of these treatments have moved forward to even better results, I'm not not particularly excited about them.  I'm still hoping for better results in future trials.

These "medium good" results may become even more valuable in the future, if they can be applied to people in the earliest stages of type-1 diabetes, when they have two autoantibodies, yet are not showing any other symptoms.  I'm hopeful that preserving insulin generation at that level could delay or prevent needing injections completely.

Second, this same research group is working on another clinical trial closely related to this one, but it has not gotten as far along the development path: https://clinicaltrials.gov/ct2/show/NCT02837094  (This is an 8 person clinical trial without a control group.)

Finally, this same research group is working on a similar treatment, but based on many peptides, rather than just one.  That trial is called MultiPepT1De:
https://clinicaltrials.gov/ct2/show/NCT02620332
They completed recruiting on 3 July 2017, so should finish gathering data about the end of 2017.

Details

[d1] Autoantibodies are the malfunctioning antibodies which cause the immune system to attack beta cells. There are five autoantibodies associated with type-1 diabetes, and there may be more that we haven't discovered yet. The five we know about are:
* micro insulin autoantibodies (mIAA or just IAA)
* islet-cell antibodies (ICA)
* glutamic acid decarboxylase (GAD) antibodies
* islet antigen-2 (IA-2) antibodies
* zinc-transporter 8 (ZnT8) autoantibodies

[d2] It is important to realize that type-1 diabetes is NOT a conventional allergy to insulin. It is similar to allergies in that it is the body's immune system overreacting to something that it should not react to, but other than that, is quite different. Allergies involve the immune system overproducing histamines. These histamines attempt to get physical irritants, like pollen, out of your body. You can counter this histamine reaction by taking antihistamines. Type-1 diabetes involves the immune system overproducing malfunctioning killer T-cells (or perhaps under producing regulatory T-cells). These malfunctioning killer T-cells mistakenly kill beta cells, thinking they are foreign cells (ie. living creatures like viruses, that have invaded the body). So the mechanism is different (histamines vs. T-cells), and the mistaken target is different (physical things, like pollen or wheat vs. living organisms, like viruses).

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.

JDRF Funding for a Cure 2017

JDRF Funding for a Cure 2017

Afgun   October 06, 2017   Comment  

In the US, we are in the "Walking Season" when JDRF asks us to walk to raise money for a cure. So I'd like to do my part, by reminding you all of how important JDRF is to the human trials of potential cures for type-1 diabetes, which I track.

Let me give you the punch line up front: 63% of the treatments currently in human trials have been funded by JDRF. (And the number is 81% for the later phase trials.) This is a strong impact; one that any non-profit should be proud of. This summary does not include Artificial Pancreas research or stem cell growth trials, because there are so many of those that it would be hard to include them all.

Below is a list of all the potential cures, grouped by phase of trial that they are currently in, and separated into potential cures that JDRF has funded, and those that JDRF has never funded.

This list is a list of treatments, and many of these are being tested in more than one clinical trial.  For example, the "ATG and autotransplant" treatment is actually running three trials, but since they are all testing the same treatment, it is only one item in the list. The list below uses the following marks to show the nature of the treatments:
    (Established) One or more trials are open to people who have had type-1 diabetes for over a year.
    (Presymptomatics) One more more trials are open to people who have 2 or more autoantibodies, but have not yet started showing symptoms of type-1 diabetes.
    (Prevention) This treatment is aimed at preventing type-1 diabetes, not curing it.

Also remember that I give an organization credit for funding a treatment if they funded it at any point in development; I don't limit it to the current trial. For example, JDRF is not funding the current trials for AAT, but they did fund earlier research into it, which helped it grow into human trials. I also include indirect funding of various kinds. For example, the JDRF funds nPOD,  ITN, and several other organizations, so I include research done by these other groups as well.

Starting Last Year: Phase-II? Trials
Starting last year, I divided Phase-II trials into two groups.  Phase-II trials are "classic" phase-II trials; they are done after a successful Phase-I trial in type-1 diabetes.  What I call Phase-II? trials are done with treatments which are known safe, so they don't need Phase-I trials, but have never been tested on type-1 diabetes before.  These Phase-II? trials might be Phase-II from the point of view of safety, but they are Phase-I in terms of effectiveness, so I'm putting them in their own category.

Cures in Phase-III Human Trials

Summary: currently there is only one treatment in a phase-III clinical trial, and that is aimed at prevention.  It is funded by JDRF.  While I see the benefit of prevention, this is the sixth year in a row there have been no phase-III trials aimed at curing existing type-1 diabetes, and it's not a good thing. Even worse, I don't see a phase-III study starting even next year.  Some people might be discouraged by that, but for me, it's a reason to donate.  Money is the thing that is going to move the Phase-II studies listed below into Phase-III studies, and the Phase-I studies to Phase-II, create more Phase-I studies, and so on.

• Oral Insulin (Preventative) 

Cures in Phase-II Human Trials

Summary: there are 22 trials in phase-II, and 17 of them have been funded by JDRF, while 5 have not. Here are the treatments that have been funded by JDRF:

• AAT (Alpha-1 Antitrypsin) by Grifols Therapeutics and also Kamada 
• ATG and GCSF by Haller at University of Florida (Established) 
• Abatacept by Orban at Joslin Diabetes Center 
• Abatacept by Skyler at University of Miami (Prevention) 
• Aldesleukin (Proleukin) at Addenbrooke’s Hospital, Cambridge, UK 
• Diabecell by Living Cell Technologies (Established) 
• Diamyd, Ibuprofen ("Advil"), and Vitamin D by Ludvigsson at Linköping University
• Diamyd, Etanercep, and Vitamin D  by Ludvigsson at Linköping University
• Diamyd and Vitamin D by Larsson at Lund University (Prevention)
• Gleevec by Gitelman at UCSF 
• Gluten Free Diet: Three Studies  (Preventative)
• Polyclonal Tregs by both Trzonkowski and Gitelman  
• Stem Cell Educator by Zhao (Established) 
• Teplizumab (AbATE study team) 
• Teplizumab by Herold/Skyler/Rafkin (Prevention)
• Tocilizumab by Greenbaum/Buckner at Benaroya Research Institute 
• Umbilical Cord Blood Infusion by Haller at University of Florida 
• Ustekinumab by University of British Columbia
• Verapamil by Shalev/Ovalle at University of Alabama at Birmingham

Not funded by JDRF:

• ATG and autotransplant by Burt, and also Snarski, and also Li 
• BCG by Faustman at MGH (Established) 
• Dual Stem Cell by Tan at Fuzhou General Hospital 
• Stem Cells of Arabia (Established)
• Vitamin D by Stephens at Nationwide Children's Hospital (Prevention)

Cures in Phase-II? Human Trials
Summary: there are 8 trials in phase-II, and 2 of them has been funded by JDRF, while 7 have not. Here are the treatments that have been funded by JDRF:

• Rituximab by Pescovitz at Indiana University
• Intranasal Insulin by Harrison at Melbourne Health (Prevention)

Not funded by JDRF:

• Albiglutide by GlaxoSmithKline
• Golimumab by Janssen
• Ladarixin by  Emanuele Bosi of Dompé Farmaceutici
• Liraglutid (Presymptomatics)
• NNC0114-0006 and Liraglutide by Novo-Norsk
• Rapamycin Vildagliptin Combo by IRCCS (Established)

Cures in Phase-I Human Trials
Summary: there are 24 trials in phase-I, and 15 of them are funded by JDRF, while 9 are not. Here is the list funded by JDRF:

• Alefacept by TrialNet 
• ßAir by Beta-O2's at Uppsala University Hospital in Sweden (Established) 
• TOL-3021 by Bayhill Therapeutics (Established) 
• CGSF by Haller at University of Florida 
• Trucco at Children’s Hospital of Pitt / Dendritic Cells (DV-0100) by DiaVacs (Established) 
• Exsulin and Ustekinumab by Rosenberg at Jewish General Hospital, Canada (Established) 
• IBC-VS01 by Orban at Joslin Diabetes Center  
• Metformin by Littleford at The University of Exeter (Prevention)
• MultiPepT1De (Multi Peptide Vaccine) by Powrie at King’s College London
• Nasal insulin by Harrison at Melbourne Health (Prevention)
• Smart Insulin (MK-2640) by Merck (Established) 
• Tauroursodeoxycholic Acid (TUDCA) by Goland at Columbia University
• Polyclonal Tregs by both Trzonkowski and Gitelman 
• Pro insulin peptide by Dayan at Cardiff University 
• VC-01 by Viacyte (Established)

Not funded by JDRF:

• CGSF and autotransplant by Esmatjes at Hospital Clinic of Barcelona (Established) 
• Encapsulated Islets at University clinical Hospital Saint-Luc (Established) 
• Gluten Free Diet by Carlsson at Lund University
• Mesenchymal Stromal Cell by Carlsson at Uppsala University
• Microvesicles (MVs) and Exosomes by Nassar at Sahel Teaching Hospital 
• Monolayer Cellular Device (Established) 
• Rilonacept by White at University of Texas 
• Substance P by Vanilloid Genetics at Hospital for Sick Children Toronto (Established)
• The Sydney Project, Encapsulated Stem Cells (Established) 

Summary of all Trials
55 in total
35 funded by JDRF
So 63% of the human trials currently underway are funded (either directly or indirectly) by JDRF. Everyone who donates to JDRF should be proud of this huge impact; and everyone who works for JDRF or volunteers for it, should be doubly proud.

Just Looking at Trials on Established Type-1 Diabetics
17 of these treatments (31%) are being tested on established type-1 diabetics.
Of these, 8 are funded by JDRF
So 47% of the trials recruiting established type-1 diabetics are funded by JDRF.

Compared to Last Year
In 2016 there were 42 treatments in clinical trials, in 2017 there are 55 (growth of 31%)
In 2016 there were no treatments in Phase-III trials, in 2017 there is one.
In 2016 there were 22 treatments in Phase-II and Phase-II? trials, in 2017 there are 30 (growth of 36%).
In 2016 there were 20 treatments in Phase-I trials, in 2017 there are 24 (growth of 20%).

A Little Discussion

Although the growth of 31% looks really good, I'm a little worried that that high growth number is because of a mistake on my part.  In previous years, I would review the research and remove everything that had failed or was going nowhere for too long.  However, this year, I was hit by some extra work at my real job, and so did not have time to check the older research to see if it was still active.  My guess is that some of the research still listed here really should be removed, and I'll do that over the next few months.  Still, I do think there was growth this year, just not 31% growth.

How I Count Trials for This Comparison

• I give an organization credit for funding a cure if it funded that cure at any point in it's development cycle. 
• I mark the start of a research trial when the researchers start recruiting patients (and if there is any uncertainty, when the first patient is dosed). Some researchers talk about starting a trial when they submit the paper work, which is usually months earlier. 
• If there are different clinical trials aimed at proving effectiveness as a cure and as a preventative, or effectiveness in honeymooners and established diabetics, then those are counted separately. 
• For trials which use combinations of two or more different treatments, I give funding credit, if the organization in the past funded any component of a combination treatment, or if they are funding the current combined treatment. Also, I list experiments separately if they use at least one different drug. 
• The ITN (Immune Tolerance Network) has JDRF as a major funder, so I count ITN as indirect JDRF funding. 
• I have made no attempt to find out how much funding different organizations gave to different research. This would be next to impossible for long research programs, anyway. 
• Funding of research is not my primary interest, so I don't spend a lot of time tracking down details in this area. I might be wrong on details. 
• I use the term "US Gov" for all the different branches and organizations within the United States of America's federal government (so includes NIDDK, NIAID, NICHD, etc.) 
• I don't work for the US Gov, JDRF, or any of the other organizations discussed here. I have a more complete non-conflict of interest statement on my web site. 

Some Specific Notes:

• Oral Insulin: This trial was a phase-III trial, meaning that it was large and designed to provide enough information so that if, if successful, the treatment could be widely used. However, as it turned out, only part was successful, and that part was phase-II sized, so I don't think we will see widespread use based on this trial alone. You can think of this as a phase-III trial with phase-II results.
• Serova's Cell Pouch and DRI's BioHub: These two clinical trials are both testing one piece of infrastructure which might be used later in a cure. They are testing a part of a potential cure. However, in both cases, the clinical trials being run now require immunosuppression for the rest of the patient's life, so I'm not counting them as testing a cure.
• Substance P at Hospital for Sick Children Toronto: This trial is avoiding the honeymoon period by testing for insulin production.  Patients must inject more than 1/2 unit/kg to be accepted, therefore they will accept recently diagnosed people, if they are injecting enough insulin to be passed the honeymoon.  I'm counting this as "Established".

This is an update and extension to blog postings that I've made for the previous seven years:

• http://cureresearch4type1diabetes.blogspot.com/2016/10/jdrf-funding-for-cure-2016.html
• http://cureresearch4type1diabetes.blogspot.com/2015/10/jdrf-funding-for-cure-2015.html
• http://cureresearch4type1diabetes.blogspot.com/2014/09/jdrf-funding-for-cure-2014.html
• http://cureresearch4type1diabetes.blogspot.com/2013/10/jdrf-funding-for-cure-2013.html
• http://cureresearch4type1diabetes.blogspot.com/2012/09/jdrf-funding-for-cure-2012.html
• http://cureresearch4type1diabetes.blogspot.com/2011/10/jdrf-funding-research-for-cure-2011.html
• http://cureresearch4type1diabetes.blogspot.com/2010/09/jdrf-funding-research-for-cure-2010.html
• http://cureresearch4type1diabetes.blogspot.com/2009/09/jdrf-funding-research-for-cure.html
• http://cureresearch4type1diabetes.blogspot.com/2008/10/jdrf-funding-of-cure-research-phases-ii.html

Finally, please remember that my blog (and therefore this posting) covers research aimed at curing or preventing type-1 diabetes that is currently being tested in humans. There is a lot more research going on, not covered here.

Please think of this posting as being my personal "thank you" note to all the JDRF staff, volunteers, and everyone who donates money to research a cure for type-1 diabetes:

Thank You!

Finally, if you see any mistakes or oversights in this posting, please tell me! There is a lot of information packed into this small posting, and I've made mistakes in the past.  As in previous years, I'll be at the Santa Clara (California) JDRF One Walk.  Come by and say "hi", or strike up a conversation about research.  I love to talk about research!

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.

Important Update For Stem Cells Arabia

Important Update For Stem Cells Arabia

Afgun   October 01, 2017   Comment  

If you are interested in potential cures for type-1 diabetes, then I urge you to read this report from the JDCA about Stem Cells Arabia:
http://thejdca.org/pc-2017-stem-cells-arabia
(Note that as a Fellow of the JDCA, I did contribute to their report.)

Stem Cells Arabia is in the middle of a clinical trial were they combine two stem cell procedures as a possible cure for type-1 diabetes.  However, the real excitement is fueled by the results of a very small pilot study they did previously.  The results from the pilot study were presented at a conference, but not published in a journal.  But those results are very strong: all four treated patients went months without needing to inject insulin and never needed anti-rejection drugs.  If you find that exciting (and I certainly do), then you'll want to read the JDCA report which contains more details, and you'll be looking forward to the results of their larger trial, which is expected to complete in early 2019.


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.