Showing posts with label ATG. Show all posts
Showing posts with label ATG. Show all posts

Sunday, May 20, 2018

Results from a Phase-II ATG and GCSF Combination Trial


I'm embarrassed about this blog posting, because it is very late.  It reports on news announced about 18 months ago.  I was digging through some old emails, and found it.

ATG is Anti-Thymocyte Globulin, a biological agent used to lower immune reactions.
GCSF is Granulocyte Colony-Stimulating Factor, a biological agent which causes bone marrow to generate more stem cells and more immune cells, and put them into the blood stream.

So the two of them together could make an effective combination therapy against type-1 diabetes. ATG would lower the autoimmune attack, and GCSF would help the body regrow beta cells. At least, that is the hope. I wrote about the start of this trial here, and it contains a lot of background material:
https://cureresearch4type1diabetes.blogspot.com/2010/08/atg-gcsf-starts-phase-i-clinical-trial.html

In brief: this study took 25 people, 2/3s got the treatment, and 1/3 got a placebo.  The treatment was spread out over 12 weeks.  The primary result was C-peptide results after 1 year, but this paper reported on extended results after 2 years.

Results from a Phase-II ATG and GCSF Combination Trial

The following graph is from the paper, and you can see that for the first year the treated group did noticeably better.  (Meaning they generated the same C-peptide at the end as at the beginning, while the placebo group dropped over time.)  The 3 to 6 month time period was particularly strong, with the treatment group generating more C-peptide while the control group generated less.  The first year change was statistically significant.  However, during the second year, C-peptide numbers continued to drop for both the treated and placebo groups, and the separation between the two of them shrunk, so that the difference was not statistically significant.



Research Paper:
http://diabetes.diabetesjournals.org/content/65/12/3765?etoc
http://diabetes.diabetesjournals.org/content/early/2016/09/26/db16-0823?papetoc

Clinical Trial Registration: https://clinicaltrials.gov/ct2/show/NCT01106157

Discussion

This is one of several studies which preserved beta cell function for a year during the honeymoon.  Early on, these studies gave me a lot of hope that they could be improved on, and eventually even lead to a cure.  However, so far I haven't seen any movement in that direction.  Treatments that preserved beta cells for one year, have (so far) not been extended to last longer.

Earlier results from this trial were strong enough so that this team started a follow on trial for honeymoon type-1s (people in their first 4 months after diagnosis):
Recruiting Site: http://www.diabetestrialnet.org/ATG-GCSF/index.htm
Clinical Trial Record: https://clinicaltrials.gov/ct2/show/NCT02215200

There are two obvious questions in this research:
1. If this treatment were started prior to diagnosis (in people who had several autoantibodies, for example, but were not showing any symptoms of type-1 diabetes), would it prevent the onset of symptoms?
2. If this treatment were repeated on a yearly, or every two year basis, could it prevent type-1 diabetes completely?  And would the hassle and side effects of the retreating on a regular basis be worth it?

Unfortunately, the new study (in honeymooners) will not answer these questions.  But it is three times as large, so it will confirm that this study was not a fluke, and the results should be available very soon.  Recruiting was completed before July 2016, so primary data should have been collected by July 2017, and publication should be imminent.

Since this study started, there is now a new hope for this level of result ("preserving beta cells for one year").  Those results could be applied to presymptomatics: people who had two or more autoantibodies, but no symptoms of type-1.  Preserving beta cells for these people would have the effect of delaying the diagnosis of type-1 diabetes.  That would be great.  However, identifying presymptomatics to test this on has just happened in the last few years, and few studies have looked into this.  But I will be looking forward to seeing the results of these studies (as prevention).

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.

Monday, July 4, 2016

Research In The News (July)

This is a combination of updates from the last month or two.

DILfrequency Completes Data Collection

In late may, the DILfrequency research team announced that they had finished collecting data, and were now starting to analyse that data.  Of course, that's great news, because it implies that results will be published in the next year or two.  (In my experience successful results are published within a year, unsuccessful results often take longer, if they are published at all.)

This study is testing Aldesleukin (also called Proleukin or IL-2) by giving it to adults within 5 years of diagnosis.  IL-2 is a component of the immune system, and they hope that more it will either improve or cure type-1 diabetes.  Are are looking for changes in the immune system which occurs quickly (ie. in a few weeks), or changes in insulin, A1c numbers, or C-peptide over a three month period.

I previously blogged on this study here:
http://cureresearch4type1diabetes.blogspot.com/2016/05/general-update-on-il-2-research.html

These researchers seem to be months ahead of schedule.  Previously, they expected to finish data collection by October 2016, but they have actually finished in May 2016, which is a testament to their ability to recruit people with type-1 diabetes.  Even better, they enrolled more people than expected: 36 expected vs. 41 actual, that's 14% bigger than planned for.  This group is very media-savvy, with lots of tweeting, facebook posting, a pinterest page, etc.  I suspect that this media focus helped them recruit, and I hope it represents the future of clinical trial recruiting techniques.

https://www.facebook.com/ClinicalTrialsType1Diabetes/?fref=nf
https://twitter.com/t1diabetestrial/status/735832240513515520/photo/1

New To Me: Cord Stem Cells In A Phase-II Clinical Trial

This trial started in 2009, but it registered with the US FDA's clinical trial registry in April 2016, and I did not know about it before then.  The basic plan is to give a total of 30 people a transplant of 3rd party umbilical cord stem cells (called allogeneic umbilical cord mesenchymal stem cells).  The people treated will be honeymooners (within 12 months of diagnosis), and must have had DKA when diagnosed.  There is no control group; everyone will get the treatment.  People will be followed for three years after transplant.  Primary outcome will be insulin usage, and secondary outcomes will be C-peptide, A1c, and autoantibody counts.  The researchers hope to finish collecting data in Dec 2019.

They are recruiting at one site:
Nanjing Drum Tower Hospital of Nanjing University Medical School. Nanjing, Jiangsu, China, 210008
Contact: Dalong Zhu, MD.PhD.    86-25-83106666 ext 61430    zhudalong@nju.edu.cn  
Contact: Jing Lu, PhD.    86-25-83106666 ext 61431    qiusnow16@hotmail.com  

Clinical Trial Registry: https://clinicaltrials.gov/ct2/show/NCT02763423

Discussion

This research is similar to Haller's work at the University of Florida, which completed a phase-I and started a phase-II clinical trial also in 2009.  I've blogged on that research here:
http://cureresearch4type1diabetes.blogspot.com/2009/05/haller-cord-blood-trial-results-from.html

Unfortunately, the phase-II study (which included a control group) completed in 2012 and was published in 2013, but was unsuccessful.  Exact quote was "Autologous UCB infusion followed by daily supplementation with vitamin D and DHA was safe but failed to preserve C-peptide.".

ATG Is Unsuccessful in a Phase-II Trial

58 people recently diagnosed with type-1 diabetes were given antithymocyte globulin (ATG) in the hopes that it would modulate the autoimmune attack on the pancreas's own beta cells.    The primary end point was C-peptide generation after 2 years (a measure of the body's ability to generate it's own insulin).  People who got the treatment did no better than people who did not.

Abstract: https://www.ncbi.nlm.nih.gov/pubmed/27053235
Trial Registration: https://clinicaltrials.gov/show/NCT00515099

Discussion

Although there was no improvement if the researchers looked at everyone in the study, if the researchers only looked at older patients (between 22 and 35 years old), then they did see a statistically significant improvement in C-peptide levels as compared to untreated patients of the same age.  The researchers were hopeful that future testing might show that ATG is helpful for older patients.

Diabetes Care Has A Section On Artificial Pancreas Papers

You can read 9 AP papers in one place:
http://care.diabetesjournals.org/content/39/7?etoc
Diabetes Care is published by the ADA.

Losing Autoantibodies: Does It Happen And What Does It Mean?

It is now well established that people test positive for autoantibodies before they are diagnosed with type-1 diabetes, and (statistically) more autoantibodies are detected over time as people move closer to diagnosis.  Also, there is a big difference between having one autoantibody and having more than one.  I'm sometimes asked: can someone lose an autoantibody that they previously had?  Put another way, are autoantibodies a one way progression to type-1 diabetes? Or can people move closer or farther away from diagnosis (at least as measured by number of autoantibodies).

The answer, according to this paper:
http://care.diabetesjournals.org/content/early/2016/06/13/dc16-0181?papetoc
is "its complicated".  Basically, some people do lose an autoantibody that they previously had and therefore, at least on paper, move away from type-1 diabetes.  However, in real life, most of the people who lose an autoantibody only had one to start with, and therefore were unlikely to ever be diagnosed with type-1 diabetes.  The people with more than one autoantibody (and who are likely to be diagnosed) rarely lose an autoantibody.

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.

Wednesday, June 22, 2016

Summary of ADA 2016

Every year the American Diabetes Association holds the largest diabetes conference of the year.  This year it was in New Orleans.  Attendance was over 16,000 people with 58% international participation. Although I did not attend, I did read (or at least skim) all the abstracts, and read all the tagged tweets that came out of the meeting.  This is my summary.  There were 100s of abstracts, scores of talks, and 1000s of tweets, so I'm only mentioning the most interesting items here.  I've divided this posting into four sections:

  • A very quick summary of the most important findings.
  • Coverage of clinical trials aimed at curing type-1 diabetes.
  • Coverage of research aimed at curing type-1 diabetes (but not yet in people).
  • Other items of interest.
A Very Quick Summary

The big type-1 news was all about artificial pancreas research.  The "Do It Yourself" artificial pancreas crew had a poster or two, plus a few meetings, but they dominated the buzz for the first day of the convention.  The commercial / professional artificial pancreas developers took over for the second day, with lots of published data.  

There was also more focus this year on "Quality of Life" issues.  Not just better numbers, but a better life.  More discussion of the whole person, and not just BGs and A1Cs.  Memorable quote was "The data alone can't be enough to make a decision", but I did not write down who said it.  There was also some discussion of using more patient friendly terminology (especially in the type-2 world).

In the world of type-2, the LEADER study showing several good outcomes from using liraglutide‎/Victoza and more data from EMPA-REG (both huge studies) made big news.  Everyone was talking about Metformin as though it was the next Vitamin-D (or the Vitamin-C of the 1970s...).

You'll notice I didn't mention much about clinical trials aimed at curing type-1 diabetes, or even curing type-1 at all.  There were two posters on clinical trials aimed at curing type-1 diabetes, and less than 10 talks aimed at curing type-1 diabetes, and that was about it.

This is DiabetesMine's summary of ADA 2016 (they cover a lot of topics which I do not):


Reports From Clinical Trials Aimed At Curing Type-1 Diabetes 

Combination Therapy with ATG + GCSF in Established Type 1 Diabetes: Two-Year Outcomes
Poster 1676-P:  
These researchers had previously reported beta cell preservation at 12 months.  (Meaning that most diabetics lost beta cells over time, but for those treated with ATG and GCSF their beta cell count remained constant.)  The result here is that after two years, treated and untreated people with type-1 diabetes had the same C-peptide numbers, so whatever advantage was seen after a year was not seen after two years.  (There were some immunological differences seen, but the C-peptide numbers, which are the most important in terms of a cure for type-1, were the same.)

Update on BCG Clinical Program for Reversal of Established Type 1 Diabetes


This is an update on Dr. Faustman's  phase-II study of BCG, which I've blogged on before.  The key new information is that they have recruited 125 patients out of the 150 people they need.  For one year of recruiting at one site, that is strong progress.  It suggests they will be fully enrolled by the end of the year.  Since the study runs for 5 years, we can expect completion in late 2021 and publication thereafter.  

Also, there is a single line on the poster about "Clinical Program 3" which is a new study.   It will give repeat doses of BCG to people who were already in the phase-I trial.  I think of it as a follow on study to the phase-I trial.  Since only three people got BCG in the phase-I trial, this clinical program will be tiny.

5-IT-SY03 - What Is the Future of Immunotherapy for Type 1 Diabetes?
This session contained about 5 talks which focused on using immunology to cure type-1 diabetes. Unfortunately, there were no abstracts for the talks, and no information about them at all.  So my only knowledge is a few tweets and web articles.
  1. Here is the official ADA preview of the session:
    http://www.diabetesdispatchextra.org/new-trials-testing-immunotherapy-for-type-1-diabetes/
  2. There was general optimism about IL-2 (which I just recently blogged about).
    https://twitter.com/em_saidwhat/status/742120883129593857
  3. There was pessimism about antigen-based therapies (ie. blocking the immune system's reaction to a specific target):  "Antigen-based new onset and Immunomodulatory onset studies have really not showed any positive substantial results.  --Jay Skyler MD"
    https://twitter.com/Diabetesdad/status/742109575868882944
Safety and Tolerability Results from a Phase-I study of Phizer's PF-06342674
PF-06342674 is a antibody that blocks a part of the immune system (IL-7 binding). This trial is testing physical properties of the treatment (how much ends up in the body, how quickly the body sheds it, any adverse effects, etc.) This is called "safety and tolerability". This trial is not in any way testing that PF-06342674 will treat or cure type-1 diabetes, but based on what is learned here, future studies could test this as either a treatment or a cure.

Optimistic Overview of Transplanting Pig Islet Cells Into People
http://www.ajmc.com/conferences/ada2016/islet-cell-transplantation-addressing-the-underlying-defect-in-type-1-diabetes
My comments: LCT is the company farthest along in transplanting pig islet cells.  They have done several phase-I and phase-II clinical trials, but the results have not led to a cure as yet.  As for islets from stem cells, Viacyte has started a phase-I trial.  Neither LCT nor Viacyte announced new data at ADA 2016 (that I know of).

Leptin (Metreleptin) In Patients With Type-1
We conclude that metreleptin was not efficacious in improving glycemic control in T1DM although it reduced body weight and daily insulin dose modestly.
My comments: In the past I have covered Leptin as a possible cure for type-1 diabetes.  However, this study suggests that, while it might lower insulin usage, it is not a cure.

Other Cure Research 
(I'm including a few AP papers here, but nowhere near all of them.)  From here down, this posting is mostly links to other sources of information.  Different people will be interested in different topics, so I encourage you to read the source material for the topics you care about.  My few comments are in italics.

Transplants  (But Still Need Lifetime Immunospressive Drugs)
http://www.abstractsonline.com/pp8/#!/4008/presentation/44271
My comments: one patient, but successful.

Tidepool
873-P / 873 - Pilot Study of Tidepool’s Blip Application for Data Visualization in Type 1 Diabetes (T1D)
http://www.abstractsonline.com/pp8/#!/4008/presentation/39609

AP Papers, Posters, Tweets, etc.

What do people think an AP is?  (It turns out to be totally different than what I think it is.)
http://www.abstractsonline.com/pp8/#!/4008/presentation/39746

More commerical AP links:



DiabetesMine @DiabetesMine
In designing AP pivotal trials, discussion is to make them 6-12 months to pursuade payers. Beyond FDA's 3-month requirement. #2016ADA
https://twitter.com/DiabetesMine/status/742167089453006848
My comment on this last tweet: The FDA has made it clear that they will approve APs based on 3 month clinical trials.  (I think this even covers bihormonal APs, which would include approving a lifetime of Glucagon micro doses with only 3 months of testing.)  However insurance companies may not pay for APs based on 3 months of data.  They might argue for more data to show it really is better.  Therefore there is pressure to run longer phase-III trials, so that one trial can lead to both FDA approval and insurance company payment.  But that would delay initial FDA approval while the longer trials completed.  There was a similar issue in CGMs.  They were approved by the FDA, but some insurance companies would not reimburse until the JDRF funded a longer study that showed benefits that the insurance companies could accept.


More "we are not waiting" (Homebrew AP) links:
While using #OpenAPS: self-reported outcome measures showed median HbA1c dropped from 7.1% (SD 0.8%) to 6.2% (SD 0.5%). #2016ADAWhile using #OpenAPS: self-reported outcome measures showed median HbA1c dropped from 7.1% (SD 0.8%) to 6.2% (SD 0.5%). #2016ADA
https://twitter.com/danamlewis/status/741671445911248896
#OpenAPS self-reported outcome measures showed median percent time in range (80-180 mg/dL) increased 58% (SD 14%) to 81% (SD 8%). #2016ADA
https://openaps.org/2016/06/11/real-world-use-of-open-source-artificial-pancreas-systems-poster-presented-at-american-diabetes-association-scientific-sessions/
Of note: user growth of #OpenAPS is doubling every 3 months, even though users must self-build these AP systems. #2016ADA
https://twitter.com/danamlewis/status/741671296245862401
My comment: that is a growth curve that any high-tech start up would be proud of.

Other Interesting Research
There were vast amounts of "my new insulin is better than your existing insulin" research.  None of that is included here.  If you care about the new insulins, I would search the ADA material directly, or wait for the press release announcing it is for sale in your country.

Easing the Child to Young Adult Transition
Two good tweets from Dan Browne:
Monaghan: for young adults, shared responsibility w/ parents for t1d care correlates with worse hbA1C. #2016ADA @collegediabetes
https://twitter.com/nakedsucrose/status/742719716632862721
#2016ADA Monaghan: protective factors for YA A1C: pers. responsibility, contact w providers, fear of hyperglyc. #2016ADA @collegediabetes
https://twitter.com/nakedsucrose/status/742719143649017856
My comment: what this seems to say is that giving young adults personal responsibility for their BG is a better strategy than shared responsibility.  I don't think that is conventional wisdom, however.  I do think this talk will be available on line in the future, and will be worth viewing when it is.

Systematic Population Screening, Using Biomarkers and Genetic Testing, Identifies 2.5% of the U.K. Pediatric Diabetes Population With Monogenic Diabetes.
http://www.ncbi.nlm.nih.gov/pubmed/27271189
via Eleen Ullman's tweet:
https://twitter.com/CureT1Diabetes/status/741676639096963073

CGMs Beat Diabetic Alert Dogs 
Basically, CGMs detected lows earlier than dogs, and dogs often alerted when there was not a low situation.  You can read more in this news article:
http://www.healio.com/endocrinology/diabetes/news/online/%7B7577bf07-3df7-4937-82fd-7bf741697f60%7D/service-dogs-can-detect-hypoglycemia-alert-companion
http://www.medpagetoday.com/clinical-context/Diabetes/58474
And three tweets on that talk:
https://twitter.com/dcarbohydrated/status/741645653969928192
https://twitter.com/KellyRawlings/status/741644965407789056
https://twitter.com/sstrumello/status/741649562847305728

Causes of Death for People With Type-1
http://www.abstractsonline.com/pp8/#!/4008/presentation/40218
1473-P / 1473 - Causes of Death in the First 100 Type 1 Diabetes (T1D) Donors in the Network for Pancreatic Organ Donors with Diabetes (nPOD)
My comment: the link above goes to an abstract.  It is tough to read but important.  Deaths directly caused by type-1 were very common.  So was suicide and drug use.  This is very cautionary data which should not be ignored.

Inhaled Insulin
MannKind announces more data (six posters) on their inhaled insulin: 


Dosing For Protein
Pratik Choudhary tweeted:
#2016ADA - preliminary results show 60 gms protein require 25% more insulin - practically - % increase of bolus with increased fat or prot
https://twitter.com/drpratikc/status/741760675622621184

Nasal Glucagon
https://twitter.com/SEDiabetes/status/742732325914562561

Type-1 Diabetes and Autism
http://www.abstractsonline.com/pp8/#!/4008/presentation/40873
http://www.abstractsonline.com/pp8/#!/4008/presentation/40901

Type-1 Diabetes and Sleep Apnea
http://www.abstractsonline.com/pp8/#!/4008/presentation/39594

Metformin (there was far more than this)
https://twitter.com/Doctor_Deena/status/741377801996009472
http://www.abstractsonline.com/pp8/#!/4008/presentation/39832

Type-2 
Shaming Is Common:
https://twitter.com/janespeight/status/742360128716984320
LEADER data:
https://twitter.com/kellyclose/status/742476794352144384
http://www.nejm.org/doi/full/10.1056/NEJMoa1603827#.V18nymZf1mY.twitter
My comments: If you have type-2, you may want to discuss this with your doctor.  (And even if you don't, your doctor may want to discuss it with you. :-)
EMPA-REG data:
https://twitter.com/RpratleyMD/status/742711726978699266
Position paper on terminology for "Diabetes"
https://static.diabetesaustralia.com.au/s/fileassets/diabetes-australia/9864613f-6bc0-4773-9337-751e953777cd.pdf
My comments: This statement misses what I consider the two most important rules:
Differentiate between type-1 and type-2 diabetes! (when appropriate)
Do not say "diabetes" when you mean "type-2 diabetes" or when you mean "type-1 diabetes".


More Interesting Tweets:

Doctor Deena @Doctor_Deena
Amazing #technology for #diabetes-- a skin patch using wavelengths to deliver #insulin into skin pores. #2016ada
https://twitter.com/Doctor_Deena/status/741745686526361600

Mark Harmel MPH, CDE @MarkHarmel
Results of DiaMonD study (CGM in MDI users) impressive. #2016ADA Fewer highs, lows and reduced variability. + Lower A1C by 0.9% at 24 weeks
https://twitter.com/MarkHarmel/status/742120533848936449
My comment: MDI is multiple daily injections.  What this study is showing is that even people who are not using a pump will benefit from using a CGM.  While I suspect this is true, I'm not sure it is useful, because I suspect these people don't want or cannot use a CGM for the same reason they don't want or cannot use a pump: they don't want an attachment or can not afford it.  Telling non-users "it is good for you" will not make them users: they know it is good for them.  They have other reasons for not using it.

Dr.Harsha Doddihal @Harshadod
Consuming #proteins followed by carbs could be beneficial in #glycemic control! Poster 62-LB
https://twitter.com/Harshadod/status/742123177766223872
My comment: is this news?  I always thought eating carbs after non-carbs led to smaller post meal BG spikes.


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.

Sunday, April 19, 2015

ATG and GCSF Combination Starts a Phase-II Trial


ATG is Anti-Thymocyte Globulin, a biological agent used to lower immune reactions.
GCSF is Granulocyte Colony-Stimulating Factor, a biological agent which causes bone marrow to generate more stem cells and more immune cells, and put them into the blood stream.  It is sometimes called G-CSF, and the exact product being used is Neulasta.

So the two of them together could make an effective combination therapy against type-1 diabetes. ATG would lower the autoimmune attack, and GCSF would help the body regrow beta cells.  At least, that is the hope. I wrote a more detailed blog entry on this combination four years ago, which you can read here:
http://cureresearch4type1diabetes.blogspot.com/2010/08/atg-gcsf-starts-phase-i-clinical-trial.html

ATG and GCSF Combination Starts a Phase-II Trial

This is a three armed study.  Each group will have at least 28 people.  The first will get ATG.  The second will get ATG and GCSF.  The third will get a placebo.  The ATG will be given twice, one day apart.  The GCSF will be given six times, two weeks apart. They started recruiting in December 2014, and hope to finish collecting their data in October 2016.  The primary outcome (the most important result they will measure) is C-peptide generation after a meal as measured a year after treatment.  There are no secondary outcomes listed, however I've been told they will track A1c, insulin usage, low BG, adverse effects, and infection rates as secondary outcomes.

They are recruiting in at least six different locations, all over the United States.  Although it's not listed on the clinical trial site, Stanford is recruiting for this trial, and they are planning on adding UCSF soon as well.  The list (with contact information) is in the Clinical Trial Record below. Patients must be between 12 and 46, and have been diagnosed within a 100 days,so this is a honeymoon trial.

Recruiting Site: http://www.diabetestrialnet.org/ATG-GCSF/index.htm
Clinical Trial Record: https://clinicaltrials.gov/ct2/show/NCT02215200
Press Release: http://jdrf.org/2015/01/unlocking-a-combination-therapy-for-new-onset-t1d/
Wikipedia: http://en.wikipedia.org/wiki/Anti-thymocyte_globulin http://en.wikipedia.org/wiki/Granulocyte_colony-stimulating_factor

Funding for this trial comes from several US government agencies, JDRF, ADA, Helmsley, and two commercial companies: Sanofi and Amgen.

Discussion About Previous Results

For me, the most important question in a phase-II trial is: "What happened in the phase-I trial?"  If there was a phase-I trial.  For this treatment, there was and it included a placebo group.  The results were good, but not great, from the point of view of curing type-1 diabetes.  Over the years of the trial, the placebo group had lower C-peptide levels (meaning they lost the ability to make their own insulin). This is what would be expected during the years after diagnosis.  The treated group's C-peptide levels stayed about the same.

Obviously, an optimist will look at those results, and say "they stopped the loss, this is good, and the earlier we can use this, the better it will be".  A pessimist would say "they had type-1 when the trial started; they had it when it was over; there was no real improvement".

My blog on their previous results is here:
http://cureresearch4type1diabetes.blogspot.com/2014/07/ada-2014-type-1-diabetes-cure-research_14.html

And here is the most important graph from those results, you can see that treated people stayed the same (dark line at top) while untreated people got worse (lighter line below):

The full paper is here: http://www.jci.org/articles/view/78492.
Thanks to the The Journal of Clinical Investigation for making the whole paper available on line.

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.

Monday, July 14, 2014

ADA 2014: Type-1 Diabetes Cure Research, Immunology

This posting discusses two treatments which have the potential to cure type-1 diabetes, which have been tested on people, and which were reported on at the ADA's 2014 Scientific Sessions.  They were both tested in honeymoon diabetics (as I define "honeymoon").

The soundtrack for this posting (in honor of the Ramones,  RIP):
http://grooveshark.com/#!/s/What+A+Wonderful+World/1XjvmQ?src=5

Expanded Polyclonal Tregs

There was a presentation of results from a clinical trial on Expanded Polyclonal Tregs, which I've blogged about before:
http://cureresearch4type1diabetes.blogspot.com/search/label/Polyclonal%20Tregs
Remember that this line of research is being pursued by two teams: the UCSF team (led by Dr. Gitelman) which is reported on here, and a team at Medical University of GdaÅ„sk lead by Dr. Trzonkowski.

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 body.  Since regulatory T cells naturally regulate the body's immune system, the hope is that they will prevent the autoimmune attack which causes type-1 diabetes.

The UCSF team ran a phase-I clinical trial with 14 people.  There was no placebo group and the patients had type-1 for between 3 and 24 months.  The basic results were that after two years, these patients continued to generate C-peptide at the same rate as when they started the trial.  There was no drop off in C-peptide production.  That means there was no drop off in insulin production.  Since all these people were already diagnosed with type-1 diabetes, they were not generating much insulin, however people with type-1 generally generate less and less insulin over time.  So these patients did better than would be expected of an untreated group.   (Although as a pilot study, there was not an untreated group here.)

Source: ADA 2014 Presentation 174-OR.

ATG and GCSF

There was presentation of results from a clinical trial on Antithymocyte Globulin (also called Thymoglobulin or ATG) and Granulocyte Colony Stimulating Factor (GCSF).  I've blogged on this trial before:
http://cureresearch4type1diabetes.blogspot.com/2012/08/possible-cures-for-type-1-in-news-early.html

The basic idea behind this research is that ATG modulates the body's immune system, while GCSG causes the body to generate more T-cells directly from it's own bone marrow.  So this therapy is a combo therapy with one treatment to stop the autoimmune attack, and another to restore beta cells.

This study had 17 patients who got the treatment, and a placebo group of 8 who did not.  People had type-1 diabetes for 4-24 months when they received the treatment.  Basically, the 8 untreated people lost C-peptide production (which means they lost insulin production), just as you would expect.  The 17 treated patients ended up, after one year, at about the same C-peptide level from where they started. So they did significantly better than the untreated group.

This news article covers this research as well:
http://www.gainesville.com/article/20140619/ARTICLES/140619588
But note that this story has some phrases like this "there was an increase in the insulin-producing beta cells in the pancreas" which is overselling, in my opinion.  This treatment preserved beta cell levels; I don't see evidence that it increased them.

The most interesting quote in this story is the following forward-looking view from the researcher:
[Dr.] Haller said the eventual goal, years down the road, is developing a therapy that first uses an IV infusion of Thymoglobulin and then a Neulasta [trade name of GCSF] treatment once every two weeks for three months to greatly reduce or eliminate the need for some Type 1 diabetes patients to take insulin injections.
Another interesting point, is that both ATG and GCSF are already FDA approved for other uses.  This makes them easier to use in clinical trials, and means they could be used "off label" for type-1 diabetes, if prescribed by a physician.

Source ADA 2014 Presentation 173-OR.

Discussion

First, these two studies highlight the lack of standardization in terminology used to describe "honeymoon" type-1 diabetes.  The first study enrolled people 3-24 months after diagnosis, and used the term "recent onset" (which I interpret as a more scientific way of saying "honeymoon").  The second study enrolled people 4-24 months after diagnosis, and used the term "established" (which I interpret to mean "non-honeymoon").

For my part, I'm considering both of these clinical trials to be "honeymoon" tests, because they included people who had been diagnosed for less than a year.  That's the dividing line I've used informally in the past, and I'm going to continue to use it, until I see a better definition.

Second, I view both of these results as honeymoon style results.  If they gave these treatments to people with long established type-1 diabetes, one would expect no improvement because the group would start out with no C-peptide production. On the other hand, these treatments have real benefits to recently diagnosed type-1s (who still have some insulin production), and could be even more beneficial to people who are losing insulin production, but have not yet been diagnosed. This is the hallmark of a honeymoon style result to me: it is highly dependant on how long a person has had type-1 diabetes.

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, JDCA, or Tidepool 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.

Sunday, July 6, 2014

ADA 2014: Type-1 Diabetes Cure Research, Artificial Pancreas


At the 2014 ADA Scientific Sessions, there were several reports on progress on artificial pancreas (sometimes called "closed loop").  Unfortunately, all of them were reported on a day that I was not at the convention, so the information below is mostly from the printed materials at the convention, news reports, and convention "buzz".

The "Bionic" Pancreas:
Bihormonal, Closed Loop, Artificial Pancreas Progress


This was clearly the big news of the scientific meeting.  Here is my previous coverage on this (and it includes links to DiaTribe's more complete coverage):

Bihormal refers to supplying both insulin and glucagon (so it can raise or lower a person's blood glucose).  Closed loop artificial pancreas refers to automatic dosing as needed with data from a CGM to a pump without human intervention.  Bionic is a marketing name used by Dr. Damiano's group at Boston University.

There were two big publications on the Bionic AP.  The first was in a scientific journal, published the month before the show, and the second was a presentation at the show.

First, I'l discuss the study published just before the show.  The basic set up was that people wore the devices for one day of calibration, and then two days of data collection.   Data was collected for four groups: adults and adolescents, and people who signaled when they were going to eat a meal, and those that didn't.  No one counted carbs or dosed in response to meals.  The signaling group just told the AP that they were about to eat a breakfast, lunch or dinner; nothing about the content.

Group
  Average BG  
  Estimated A1c  
  % in range  
(70-180)
Adults Before Treatment7.3
Adults who signaled meals1326.280
Adults without meal signaling1426.770
Adolescents Before Treatment7.9
Adolescents who signaled meals1627.368
Adolescents without meal signaling1757.760

What this means, is that for adults who did not signal when they were going to eat, they had an average BG level of 142, a likely A1c level of 6.7 (if they had done this for 3 months), and their BG levels were in range 70% of the time!  Now, that looks pretty good, but the news gets better.

Here are the results from the follow up study, done by the same researchers, and given as a scientific talk.  This study was "free range" adults who were free to roam over 3 square miles of Boston, staying in a hotel, working out at a gym, and eating mostly at restaurants, while the adolescents were attending camp.  For this study, no one signaled meals.  It included 20 adults and 32 adolescents, which makes it phase-II sized by my reckoning.

This study has only two data points that matter:

Average BG Number
  (for both adults and adolescents)  
  Estimated A1c  
138
6.4

There was slight complexity in the data.  That 138 number was the average over all five days of the test.  The researchers expected that the first day would be worse than the other four, because the unit was calibrating itself to the patient the most during that first day.  For adults, this worked out, the next four days average BG was 133 suggesting that long term use would result in an even lower number, and might even drop a few more points (over time, as the AP better learned how the person reacted to insulin, glucagon, and food).  But for adolescents, that's not what happened.  They averaged 147 over days 2-5.  Even if 147 (A1c of 6.7) is the long term number, that is still a complete success.   It is lower than the ADA standard of 7.5 for adolescents.  But it is a mystery to me why those days should average higher than the first day.

Summary of NEJM data: http://www.nejm.org/action/showImage?doi=10.1056%2FNEJMoa1314474&iid=t02

Note: information for this section came from an ADA abstract, a JCEM paper, and a NEJM abstract.   You can read the whole NEJM article here:
http://www.nejm.org/doi/full/10.1056/NEJMoa1314474#t=articleTop
JCEM abstract here:
http://www.ncbi.nlm.nih.gov/pubmed/24483160

Single Hormone vs Bihormonal Artificial Pancreas
A group from Canada gave a talk where they directly compared injected insulin, an insulin AP, and an insulin and glucagon AP.  For average BG numbers, they found that both types of APs were similar to each other (the dual pumps were only very slightly better), and that they were both significantly better than injections.  However, when they looked at low BG events, then the dual hormone APs had significantly fewer such events than single hormone APs.  This makes sense, since the dual hormone pumps can directly prevent lows by dosing glucagon.

So this Canadian trial suggests that a bihormonal AP might do a little better than a "classic" AP, but it should not do vastly better, if measured by average BG.  When I first saw that poster, I was a little dubious.  Two hormones seemed like much better technology than one.  But then I saw the results below.  One of the complexities, is how does one measure an AP?  Using average BG is easy and straightforward, but should we also measure low BG events and/or high BG events?  If you do (especially low BG events), then the dual hormone APs might look better in comparison.

The Cambridge Artificial Pancreas

With all the excitement about the bihormonal AP, it is important to remember that there are also several "classic" AP projects out there.  For example, the results from the Cambridge AP, a "classic" insulin-only AP, were almost as good as the bihormonal results.  There were something like 7 presentations on various aspects of this project, so it was very well represented.

The "24 hours a day" trial included 17 people, and ran for 16 days (8 days with AP and 8 days with regular treatment).  They also reported on a nighttime only trial, which ran for 90 days!  Again, half with AP and half with regular treatment.

It's big results that matter, from the 24 hour and day trial, are:

Average BG Number
  (for both adults and adolescents)  
  Estimated A1c  
146
6.7


MD-Logic Artificial Pancreas Project

What's better than two closed loop, artificial pancreas projects?  Three!  The MD-Logic project uses a "fuzzy logic theory algorithm" to predict insulin dosing.  The research group presented a poster, which showed that using the MD-Logic AP at night, improved BG numbers the next day.  This clinical trial included 24 people and lasted for 3 months (6 weeks using the AP, 6 weeks not, for comparison).

People who used the AP woke up about 15 points lower (on average) than people who did not use it. Looking at all the BG numbers the next day, people who used the AP the night before had an improvement of about 11 points on average.  People who did not use the AP were in range about 66% of the time, while those not using the AP were in range about 62% of the time.  (Range was 70-180).

Source is poster 949-P.

The Virginia Artificial Pancreas

This is another ongoing research project into a "classic" artificial pancreas.  In the trial reported on at ADA 2014, 13 people were tested for 42 hours: 14 hours "open loop" treatment, and 28 hours of "closed loop" treatment.  People in the trial could move about a hotel.  This same research group is planning a 2 month trial of the same AP.

Source is poster 954-P and 104-LB.

Direct Comparison

Group
Average BG
Estimated A1c
Size
Adolescents?
Duration
AP Use
Boston University138
6.4
53
Yes5 days24 Hours/Day
Cambridge
146
6.7
17
No
8 days24 Hours/Day
MD-Logic
24
Yes
90 days
Night Only
Virginia
135?
13
No
2 days
24 Hours/Day

When you look at that, you might say the two hormones are better than one.  But I would not read too much into that difference.  It's not huge (8 BG points and 0.3 A1c), and remember that the single hormone solution is simpler all the way around: only one hormone to buy and load into the pump, less moving parts on the device, and so on.  (Not to mention the fact that Glucagon hasn't yet been approved for this application, although that is expected.)  Of course, the comparison is based on average BG, so might miss extra low BG events in the single hormone APs.

None of this competition bothers me in the least.  I love the idea of having four closed loop systems getting to market at about the same time with slightly different feature sets.  Having a bihormonal AP with slightly better control competing against a single hormone AP which is slightly simpler, sounds like just the sort of competitive situation that feeds progress in a capitalist economy.

Other Bits and Pieces

Poster 75-LB compared CGM data from actual BG data (measured using laboratory grade equipment) from blood pulled directly from a vein. They found that CGM data was very similar to the actual BG data, and that even when different, the differences were small. The researchers conclude that existing CGM technology is not the "weakest link" of AP technology.

Poster 747-P asked people who were testing a closed loop AP, what they thought of it. They liked it. They liked it because it provided better BG control, reassured them that nothing bad would happen while they slept, and improved BG control the next day.  Poster 110-LB contained similar information, but focused on the remote monitoring of an AP in a family situation (ie. parents remotely monitoring children).  A major conclusion of this research was that families wanted the AP/remote monitoring combo being tested; it did not need any improvements at all, it just needed to be made available.

Poster 948-P tested a closed loop system using diluted insulin compared to regular insulin, for small children (aged 4-7).  They found that diluted insulin worked a little better.  Average BG levels were the same, but time spent in range was 8% higher when diluted insulin was used.

Poster 951-P tested a closed loop system which (in addition to BG data) also used energy expenditure and galvanic skin response data.  These are two measures of energy use.  The hope was that by using energy expenditure data, they could make a better AP.  However, the data showed very little difference between using this data and not, and even this little difference was only when BG was above 250.

Summary

My summary of closed loop, artificial pancreas research is this:  We are seeing cure level control in phase-II clinical trials and for several different AP systems.  This is great news, for several reasons. First, it means they "only" need to get through phase-III trials (and marketing approval) for these APs to be sold in the US. They don't need to do better than the results they already have, just produce the same results in larger trials. Second, it means that if one falls apart, there are others which can still get marketed. Third, it means that the technology is ready. When one AP is successful, that team might just be ahead of the rest, but if four groups can do it, that means the technology is here for all.

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, JDCA, or Tidepool 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.

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.

Monday, August 13, 2012

Possible Cures for Type-1 in the News (early Aug-2012)


Prologue: Why Completing Enrollment is an Important Milestone for Clinical Trials

For two reasons.  First, because at that point it is possible to predict when the researchers will eventually finish collecting data.  The experimental design will say how long data will be collected for each person, so you can just add that duration to the date they finished enrollment, and that tells you when the final data will be collected.  Second, because much of the uncertainty that surrounds clinical trials involves recruiting participants, it is often unclear how hard it will be to get people, and long it will take.   But at this point, all that cunertainty is behind the researchers.  Once enrollment is complete, it is just gathering data, then analyzing data, and then publishing the data.  Researchers have a lot more control over those later stages, than over recruiting people in the first place.

Put another way, a clinical trial is made up of five tasks:
1. Do research to figure out what clinical trial should be done.  During this phase, the researchers are at the mercy of the science.  If they science doesn't pan out, then the experiment never gets done.
2. Get FDA approval for the clinical trial.  During this phase, the researchers are at the mercy of the FDA (or similar regulatory agency).  If they don't get approval, the experiment never gets done.
3. Recruiting patients.  During this phase, the researchers are at the mercy of the patients (or their parents).  If they can't get enough people to enroll, the experiment fails.
4.  Gathering the data.  During this phase, the researchers pretty much control their own destiny, unless a huge number of people drop out, or there is some unexpected safety issue.
5. Writing and publishing the results.  During this phase, the researchers are subject to editors and peer review (to some degree).  However, there are so many journals and web sites, that the researchers have many choices; they can even self publish, if all else fails.  They are not at the mercy of any one person or committee.

Completing enrollment is the end of phase 3 / start of phase 4, and you can see that researchers have a lot more control of their own destiny in phases 4 and 5, then in 1, 2, or 3.  

"REPAIR-T1D" (Lansoprazole and Sitagliptin) phase-II Study Completes Enrollment

This study finished enrolling in May 2012 and they plan to gather data for two years, so we should have results soon after May 2014.  This trail gives two currently approved drugs (Januvia and Prevacid) to type-1 diabetics within six months of diagnosis.  The hope is that it will restore or at least preserve beta cell function.

Press Release: http://www.newswise.com/articles/the-sanford-project-reaches-milestone-in-quest-to-cure-type-1-diabetes
Clinical trial record: http://www.clinicaltrials.gov/ct2/show/NCT01155284

GCSF/Neulasta phase-II Study Completes Enrollment

Previous blogging here: http://cureresearch4type1diabetes.blogspot.com/search/label/Haller

This study finished enrolling in March 2012, and they plan to gather data for two years, so we should have results soon after March 2014.  (Their clinical trial record says December 2014.) GCSF, also known as Neulasta or Pegfilgrastim, causes the body to generate it's own stem cells from bone marrow. These might rebalence the immune system or they might retrain it, but in any case, the hope is they will help cure type-1.

Burt's trial (the most successful so far) uses GCSF, ATG, and CZ. CZ is, by far, the most toxic of the three, so this trial can be viewed as "Burt ultra-lite". GCSF and ATG together would be "Burt lite" and there is a trial for that also ongoing.
Clinical Trial Record:   http://www.clinicaltrials.gov/ct2/show/NCT00662519

Thymoglobulin (ATG) Phase-II Study Completes Enrollment

Please read about Thymoglobulin (ATG) here: http://cureresearch4type1diabetes.blogspot.com/p/drugs-and-treatments-in-clinical-trials.html (scroll down the page until you see the section on ATG)

This phase-II study of ATG has completed enrollment.   It took them about 2 years longer than expected, so that's not good, but we can expect data collection to be complete in 2 years, and hopefully results will be published soon there after.  The ATG link about has complete information on why the researchers hope ATG might cure type-1 diabetes or be part of a cure.

Clinical Trial Record:  http://www.clinicaltrials.gov/ct2/show/NCT00515099
Previous blogging here: http://cureresearch4type1diabetes.blogspot.com/search/label/Gitelman

Combo (Diamyd, Lansoprazole and Sitagliptin) Trial Fails

This trial has a long and not so good history, which you can read in my previous blogging here: http://cureresearch4type1diabetes.blogspot.com/search/label/Combo

This Phase II study combined regenerative agents (lansoprazole and sitagliptin) and also Diamyd. The regenerative agents are both marketed drugs in the US. Diamyd is a vaccine like drug which trains the body's autoimmune system not to attack it's own cells.

This study's clinical trial record updated to show that it was terminated on 20 March 2012, although I think it had actually ended earlier.  Dr. Harlan, who started this study, had left the NIH, and the phase-III studies of Diamyd alone had failed or been canceled, so they decided not to pursue it.  

Obviously, it's too bad that it was canceled.  This was the first study that I knew of that attempted to combine a drug to stop the autoimmune attack and drugs to encourage the regrowth of beta cells.      I think that combining treatments like that is still a good thing to try, even if this specific combination did not work out.

Clinical Trial Record:  http://www.clinicaltrials.gov/ct2/show/NCT00837759

More Stable Glucagon

This is only vaguely related to a cure, but I thought I'd mention it.  Xeris is trying to create a form of glucagon which can be stored at room temperature for long periods of time.  From a cure point of view this is useful for an artificial pancreas (if you consider that a cure).  Simple APs are based on insulin alone.  However, there are more complex APs, which are based on insulin and glucagon.  Having a glucagon which was approved to sit in the pump for days, maybe weeks, would be important to these "bihormonal" APs.

As a nice side effect, it would mean that emergency glucagon needles could be preloaded, just like insulin pens, which would make them much easier to actually use in an emergency.  It also would make mini-glucagon doses much easier to administer, for those who use them.

You can think of an insulin only AP sort of like a car with a break but no accelerator.  The engine "revs high" and you run along.  You use the break to slow up.  A bihormonal AP (insulin and glucagon) is more like a car with a break and an accelerator.  More control.  The analogy is not perfect, but that's the basic idea.

Press Release: http://www.marketwatch.com/story/xeris-pharmaceuticals-awarded-phase-i-ii-nih-sbir-fast-track-grant-to-advance-stable-non-aqueous-glucagon-for-bi-hormonal-artificial-pancreas-2012-07-31

Why Pharma/Med Device Co's Apply for Approval in Europe FIRST

If you haven't read this blog posting by Scott Strumello, you should!  It compares the approval process for medical devices in the EU to the same process in the US:
http://blog.sstrumello.com/2012/08/why-pharmamed-device-cos-apply-for.html

Next Up: Results from Dr. Faustman's Phase-I BCG Trial

She published in PLoS last week, so I hope to blog about it in a week or two.

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. My blog contains a more complete non-conflict of interest statement.
Clinical Trials Blog: http://cureresearch4type1diabetes.blogspot.com
Cured in Mice Blog: http://t1dcuredinmice.blogspot.com/

Saturday, August 14, 2010

ATG + GCSF Starts a Phase-I Clinical Trial

ATG + GCSF Starts a Phase-I Clinical Trial
This is a very interesting study, which is why I'm giving it, it's own blog posting.  The official title of this study says it all, optimistically: "Reversing Type 1 Diabetes After it is Established".  It builds on three separate lines of research: Burt's ATG/GCSF/cyclophosphamide trial, a separate line of ATG-only trials, and NOD mice trials on ATG and GCSF (all discussed more below).

The basic idea behind this research is that ATG modulates the body's immune system, while GCSG  causes the body to generate more t-cells directly from it's own bone marrow.

ATG is a mixture of different antibodies that target T cells, including the anti-CD3 antibody.  The hope is that this multi-faceted approach will be even more successful and have a longer lasting effect than with anti-CD3 alone, and may treat diabetes by several mechanisms. First, it lowers the number of T cells, so there are fewer to attack the beta cells. It also seems to alter the T cells remaining behind, rendering them less likely to be destructive. Following this depletion, the T cells that grow back in the following weeks may be reset and have a healthier balance (meaning that a special type of T cells, called regulatory T cells, will help keep the destructive T cells in check).

GCSG  is essentially a growth factor that helps the body repopulate the cells after ATG depletes them.  So, if ATG fixes the immune system by getting rid of the destructive cells, and the GCSG can cause the body to generate more t-cells afterwords, and presumably a healthier balance of these cells, then the two together could stop the autoimmune defect that causes type-1 diabetes.  (Regrowing new beta cells might still be required for some people.)  And, this process has already been used successfully to cure type-1 diabetes in mice, so that is always a good first step.

But the really positive thing about this research is it's relationship to previous research by Burt.  You can read my previous blog entries about Burt's research at the URL below, but the quick summary is this: Burt has -- by far -- the best results in people of any researcher.  That study used a combination of 3 drugs, ATG, GCSF, and cyclophosphamide.  Some patients went into remission (no need for injected insulin) for a period of years, others for months, and all of these people used much less insulin after the trial.  Only two patients in the study did not go insulin free for at least a few months.  This research also has a serious weakness, which is safety.  Although none of the people in Burt's had serious side effects, the process involves "rebooting" the immune system.  So while this is happening, the patients are in an isolation ward, because their immune system is highly compromised.  Also, there were "hints of toxicity" seen in the trial: a few people had unusual infections, and some of the males had reduced sperm counts afterward.  Burt's research was in honeymoon diabetics, only.

A second line of research that has been going for years uses ATG alone to try to preserve beta cell function in honeymoon type-1 diabetics.  This research has already completed a phase-I study, and a phase-II study is underway right now.  The phase-II study is called START and they hope to finish recruiting patients in 4-6 months.  ATG alone is very good in curing mice, but in combination with GCSF the success rate is nearly 90% for the mice, and hopefully that will translate to people.

Oversimplified a little: Burt's research involves using three drugs (ATG, GCSF, and cyclophosphamide), and reinjecting the patient's own precursor bone marrow cells (previously removed). Dosing with just ATG and GCSF can be viewed as a "kinder, gentler" Burt. Especially since the cyclophosphamide is the one that really hammers the immune system and requires the isolation ward. 

In the past, I've been asked who was following up Burt's research, and this is a pretty direct follow up. Most phase-I trials are aimed at minimum effect and maximum safety, so minimal doses are used.  The follow on research usually involves more drugs.  However, in this case the effectiveness is high, while the safety is the issue.  So the follow on research involves less drugs.  In this case it involves removing the most toxic drug from the treatment.

They are enrolling 25 people who have had type-1 diabetes for more than 4 months, but less than 2 years.  Planned completion date is April 2013.  They are recruiting at the University of Florida, which is in Gainsville.  UCSF (San Francisco, California, USA) and also the Davis Center (Denver, Colorado, USA) will be participating in this study, but they are not listed in the clinical trial record.  The coordination is through University of Florida by Dr. Michael Haller (who is running several other clinical trials aimed at type-1 as well).  The email contact is listed as hallemj@peds.ufl.edu, so I would start there if you are interested.


Previous blogging on Burt's research:  http://cureresearch4type1diabetes.blogspot.com/search/label/Burt
Previous blogging on ATG: http://cureresearch4type1diabetes.blogspot.com/search/label/ATG

Description of study: http://diabetes.ufl.edu/research/clinical-trials-and-studies/established-type-1-diabetes/atggcsf-clinical-trial/
Clinical Trial Record: http://www.clinicaltrials.gov/ct2/show/NCT01106157
Abstract of animal study: http://www.ncbi.nlm.nih.gov/pubmed/19628781?dopt=Abstract
Wikipedia for GCSF: http://en.wikipedia.org/wiki/Neulasta
Wikipedia for ATG: http://en.wikipedia.org/wiki/Anti-thymocyte_globulin

I'd like to thank Dr. Steve Gitelman (who is a Principal Investigator in the ATG START study) for his thoughts on this research.  All mistakes here are my own.


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

Saturday, August 29, 2009

Update on Gitelman's ATG Phase-II Clinical Trial

Dr. Gitelman at UCSF has started a Phase-II human trial aimed at using ATG to cure (or lessen) type-1 diabetes. The goal of this study is to preserve remaining beta cells for people within 100 days from diagnosis. This study started in August 2007, and is expected to run until June 2011. So far it has enrolled 11 out of an expected 66 patients. It's a four day treatment (in hospital), and 12 follow up visits over a 2 year period. They are recruiting at a total of 9 sites (growing to 13), which are listed in the clinical trial page linked below.

Here is a quick summary of how they hope ATG (Thymoglobulin) will work:

Type 1 diabetes is caused by destruction of insulin-producing beta cells by T cells, part of the immune system. Thymoglobulin is a mixture of different antibodies that target T cells. This mixture includes the anti-CD3 antibody (and anti-CD3 treatments are in Phase-III trials already, having completed successful Phase-I and II trials). The researchers hope that this multi-faceted approach will be even more successful and have a longer lasting effect than with anti-CD3 alone, and may treat diabetes by several mechanisms. First, it lowers the number of T cells, so there are fewer to attack the beta cells. It also seems to alter the T cells remaining behind, rendering them less likely to be destructive. Following this depletion, the T cells that grow back in the following weeks may be reset and have a healthier balance (meaning that a special type of T cells, called regulatory T cells, will help keep the destructive T cells in check).

Using ATG to cure type-1 diabetes is an idea that flows from two sources. The first source is a line of clinical trials aimed directly at curing type-1 with ATG. Eisenbarth's work of long ago, a Phase-I trial done in Europe, etc. But it also has another source, which is Burt's Brazilian research as described below.

Discussion: How to Follow Up Burt?

One of the questions that I'm occasionally asked is this: when will there be a follow up to Burt's Brazilian research? When will there be a Burt, phase-II? This is a natural question, because Burt has -- by far -- the best results of any one. Many patients on that trial went years without using external insulin. No other study comes close. So an obvious question is: how do you move forward with that research.

Burt's research is the opposite of most. Most researchers use the smallest possible doses during phase-I. Phase-I is targeted at safety, so they use small doses to assure safety even at the expense of effectiveness. So for most studies, for phase-II studies, they move forward by raising the dose to try to make a safe treatment a more effective one. But Burt's research is the opposite. The effectiveness is the strongest yet, but there are real questions about safety. So you would NOT follow it up by raising the doses!

One way to follow up Burt's relative success, is by turning down the dose. Trade off a little effectiveness for added safety. Oversimplified a little: Burt's research involves using three drugs (ATG, GCSF, and cyclophosphamide), and reinjecting the patient's own precursor bone marrow cells (previously removed). Dosing with just ATG can be viewed as a "kinder, gentler" Burt. Especially since the cyclophosphamide is the most risky drug of the bunch. (I'll be posting on two GCSF clinical trials in the coming weeks.)

Since I'm a software engineer, I'll use a software analogy: Burt is doing a "hard reboot", Gitelman is trying for a "soft reboot".

Notes

One of the complexities of following the research, is that ATG is referred to by many different names by different groups. For example: Thymoglobulin, rATG, hATG, ATGAM, antithymocyte globulin, etc.

The cells that Burt is removing and then reinjecting are sometimes called "precursor bone marrow cells" and other times "adult stem cells".

The lead investigator for this clinical trial is Dr. Gitelman at UCSF. For those of you at Bearskin Meadows: yes, this is the same "Dr. Steve" who is often up there. I'm indebted to Dr. Gitelman for his insights into Burt's research and how it relates to other research, including his own. All mistakes, opinions, and oversimplifications are my own.

Patient oriented information on this trial:
http://www.type1diabetestrial.org

Previous Blog postings on this
http://cureresearch4type1diabetes.blogspot.com/search/label/ATG (blog on ATG)
http://cureresearch4type1diabetes.blogspot.com/search/label/Burt (blog on Burt)
http://cureresearch4type1diabetes.blogspot.com/search/label/Gitelman (blog on Gitelman)

Web pages (but these need updating):
http://joshualevy.pbworks.com/DiabetesCureReadyForHumanTrials#ThymoglobulinalsoknownasATGbyGitelman (Gitelman)
http://joshualevy.pbworks.com/DiabetesCureReadyForHumanTrials#ATGandothersbyBurtatUniversityofSaoPaulo (Burt)

Clinical Trial records for this study:
http://www.clinicaltrials.gov/ct2/show/NCT00515099 (Phase-II ATG)
http://www.clinicaltrials.gov/ct2/show/NCT00190502 (Phase-I ATG)
http://www.clinicaltrials.gov/ct/show/NCT00315133 (Burt)

Joshua Levy