The full story on "Stem cells reverse woman’s diabetes — a world first"

If you've been reading news recently you might have seen headlines like these:

Stem cells reverse woman’s diabetes — a world first

Stem Cell  treatment reverses Type 1 Diabetes!

Chinese scientists have put a young woman’s type 1 diabetes into full remission using stem cell treatment.

And even if you didn't your friends and relatives might have seen them and asked you about them.

(And that first one is from the journal Nature, which is a major scientific publication.)

Unfortunately, those headlines are basically hype.  There is some news, and it is good news, showing important progress, but it is no where near as good as the headlines make it sound.

What Was Reported

The researchers took some adult stem cells from a person with type 1 diabetes, and treated those stem cells with a specific recipe of drugs, which caused them to change into beta cells.  They then implanted those cells back in the person who donated them.  The results were spectacular.  After about 2 months the person stopped injecting insulin, and then was followed for another 10 months without needing any injected insulin.

The woman was not your average person with T1D.  She had been diagnosed 11 years prior, so was well established.  However, she had previously had two liver transplants and one pancreas transplant (more on that below).  At the end of a year, her A1c was 5% and her blood glucose time-in-range was 96%, both great numbers.

Not A Cure

Before getting this transplant, this person had already had several transplants, and was therefore on immunosuppressive drugs and would need to stay on those drugs for the rest of her life.  Therefore, by my definition, she was not cured but rather traded one treatment (insulin for T1D) for another treatment (immune suppression for a transplant).  I don't consider this a cure.  Some people may prefer one drug regimen over another, but it is not clear to me that one is generally better than the other.  My understanding is that people who have whole life immune suppression generally have shorter life spans than people with T1D.  

But in any case, I don't consider this a cure.  Of course, you may, and if you do, then you should look into existing transplantation surgeries, because there are some available now that have similar results to what is seen here.

Shows Progress

The big headline "no insulin injections for almost a year" is misleading because those results have been seen previously in transplants when whole life immune suppression has been used.  The JDCA has published two great overviews in 2016 and 2022, which you can read here:

• https://www.thejdca.org/publications/report-library/archived-reports/2016-reports/islet-cell-transplantation-update-new-phase-iii-study-results-show-insulin-independence-unchanged.html
• https://www.thejdca.org/publications/report-library/archived-reports/2022-reports/pioneering-islet-cell-transplant-team-publishes-20-years-of-data.html

The bottom line is that no insulin injections for a year occurs in half the cases, and for two years in over 40% of the cases.  So the headlines are hyping something we can already do. (If you are willing to suppress your immune system for the rest of your life.)

If fact, there is an FDA approved transplant protocol, where over 2/3s of the people did not have to inject insulin for more than a year, but it does require immune suppression:

https://www.fda.gov/news-events/press-announcements/fda-approves-first-cellular-therapy-treat-patients-type-1-diabetes

However, the research reported here does show some specific progress, and some strong future possibilities.  The key improvements seen here are:

1. More stem cell availability.  Previous transplants have used beta cells from cadavers, pigs,  occasionally live doners, and just recently from the patient themselves.  This research sources beta cells from the patient, which means there will always be a strong supply.  The treatment that they use to mature the stem cells into beta cells is new and unique and the researchers claim it is much better than previously available techniques.  They think it gives them more control over the resultant beta cells and also is more effective.  It is this technique that is the real progress.

2. Using a person's own stem cells.  My memory is that this is not the first ever case where T1D  transplantation used cells from the person being treated.  However, this is a very recent and experimental technique.  The success here is important.  Most importantly, there is hope that these cells will not be automatically attacked by the person's immune system, because they are not foreign.  This is a serious issue with other transplants.  Because these cells are not foreign, this transplant may not require long term immune suppression.  That would be a huge breakthrough.

Unfortunately, since this person is already using long term immune suppression, there is no way to know from this trial if this kind of immune suppression is needed or not.

Furthermore, even if this procedure becomes immune suppression free in the future, it is not clear what will happen to the new beta cells long term without it.  Will the person's T1D immune system attack the new beta cells just like it attacked the original ones?   We don't know.  That is future research that must be done.

There are four hopeful possibilities here (lines of research which might prevent this attack).  They are theories held by some researchers.  The first two are active areas of research, with several research groups working on each one, and the second two are tested on occasion, but are less actively pursued:  

1. Several research groups are investigating encapsulation, so that the immune system cannot physically get to the new beta cells, which would protect them.
2. Other groups are developing the process of converting stem cells to beta cells so that the new cells were invisible to the immune system and protected that way.  The immune system tends to focus on very specific structures in the "skin" of the cell to identify what to attack.  If those structures are missing, then maybe the immune system would ignore the new cells.
3. The trigger of the autoimmune attack on beta cells might be time specific.  It occurs at a specific time for a specific reason.  So therefore, years later, when the new cells are transplanted, the trigger is no longer there and the cells will not be attacked.  
4. Finally, the immune attack on beta cells might be location specific.  It occurs because of the situation in the pancreas specifically.  Therefore beta cells not implanted in the pancreas won't trigger the immune response.

The researchers were waiting for results from the first person, but now that they have them, they will start up two more people.  It is a little hard to tell, but I suspect these two people will also have prior transplants and therefore long term immune suppression.

The study is ongoing and they are recruiting more people with T1D in Tianjin, China.  The study is described as "Phase 0", but I don't know what that means in China.  It is similar to an American phase-1 "pilot" study: no control group, no blinding, three people who will be followed for two years (I think) after transplantation.  Primary end point is A1c.  Secondary end points are C-Peptide and insulin use.

Contact: Wang Shusen        +86 136 1218 3907  shusen1976@126.com

               Shen Zhongyang  +86 138 0301 9898  zhongyangshen@vip.sina.com

More Information

This research was published in the journal Cell, which is a big name scientific journal:

https://www.cell.com/cell/abstract/S0092-8674(24)01022-5

JDCA's report: https://www.thejdca.org/publications/report-library/archived-reports/2024-reports/established-t1d-patient-insulin-independent-is-it-really-a-practical-cure.html

Chinese Clinical Trial Registry: https://www.chictr.org.cn/showproj.html?proj=192835

WHO Clinical Trial Registry: https://trialsearch.who.int/Trial2.aspx?TrialID=ChiCTR2300072200

Coverage: https://www.sciencedirect.com/science/article/abs/pii/S0092867424010225

General Article: https://stemcellres.biomedcentral.com/articles/10.1186/s13287-024-03636-0

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 kid has type-1 diabetes and has participated 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! 

Combo of Rituximab and Abatacept Starts a Phase-II In Honeymooners (T1D RELAY)

Rituximab is FDA-approved to treat several autoimmune diseases, including Rheumatoid Arthritis (RA).  About 10 years ago there were a couple of clinical trials testing it for T1D, and the results were medium-good. I blogged on those: https://cureresearch4type1diabetes.blogspot.com/search/label/Rituximab

Abatacept is FDA-approved to treat adult Rheumatoid Arthritis, as well as Juvenile Idiopathic Arthritis (JIA) in children as young as six.  I blogged on those: https://cureresearch4type1diabetes.blogspot.com/search/label/Abatacept but not on the most recent results, which are here: https://diabetesjournals.org/care/article/46/5/1005/148547/Abatacept-for-Delay-of-Type-1-Diabetes-Progression and were unsuccessful on the primary end point, but some interesting data on the lessor end points.

Rituximab suppresses CD20 cells, which are a subset of the immune system's B cells (different from the pancreas's beta cells).  Hopefully this will block the autoimmune attack. B cells communicate with the T cells, which actually attack the body's beta cells in the pancreas. By targeting the B cells, it is hoped this treatment will stop or lower the attack of the T cells.

Abatacept is a treatment that prevents T-cells from becoming activated.  Presumably, for type-1 diabetes, it works by blocking the "bad" killer T-cells from activating.  

So you can see how these two drugs can work together to have a stronger effect than either alone.

The Study

This study will enroll 76 honeymooners (within 100 days of diagnosis), between 8 and 45 years old.  Everyone will be treated with Rituximab once a week for 4 weeks.  This is an IV infusion which will take 3-8 hours. There is then a 3 month period without treatment, and then 2/3s of the people will get Abatacept.  That is a weekly injection (much like injecting insulin) which will go for 20 months.  1/3rd of the people will get a placebo and be the control group.  (So everyone gets Rituximab, but not everyone gets Abatacept.)

Everyone will then be followed for a total of 4 years, so 2 years after the last treatment.  The primary end point is C-peptides and secondary endpoints include immune measurements to see how the treatment effects people.

The study started in Oct-2023 and is expected to end between Oct-2027 and Oct-2029.  However, since they are still recruiting, and the study follows people for 4 years, I don't see how it can finish before mid-2028.

They are recruiting in 13 locations in the United States plus Melbourne, Australia.  The full list is in the clinical trial link below or you can get in touch with the study contacts:
Ariana Rojas  +1-813-974-682    ariana.rojas@epi.usf.edu
Melissa Parker  +1-813-396-9378   melissa.parker@epi.usf.edu 

Web Site: https://www.trialnet.org/our-research/newly-diagnosed-t1d/t1d-relay
Clinical Trial Registration: https://www.clinicaltrials.gov/study/NCT03929601

Discussion

This study tries to combine two treatments with lackluster results into something better.  Of course, I have no idea if it will work, but if it does, that is good news in two different ways.  First, as a promising honeymoon treatment to delay or even prevent T1D, that is the direct goal of this trial.  But second, many researchers believe that two different immune drugs (with two different mechanisms) might be a lot better than one immune drug alone.  They may interact synergistically.  A success here would show that two drugs together could be much more than either one alone, and that could open the flood doors of combination drug trials.

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 kid has type-1 diabetes and has participated 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!

 

Abrocitinib and Ritlecitinib Start A Phase-IIΔ Clinical Trail in Honeymooners (JAKPOT)

(Note: JAKPOT is a name of at least two different clinical trials.  If you are reading about a French clinical trial targeting Rheumatoid Arthritis, that is a different clinical trial.)

The site's web page describes the drugs involved this way:  Abrocitinib and Ritlecitinib are in a new class of autoimmune treatments called Janus kinase (JAK) inhibitors.  Abrocitinib is approved by the U.S. Food and Drug Administration (FDA) to treat eczema. Ritlecitinib is being studied as a treatment for several autoimmune diseases, including Alopecia, Ulcerative Colitis, Crohn’s Disease, Vitiligo, and Rheumatoid Arthritis.

Researchers believe Abrocitinb and Ritlecitinib may be able to calm the immune system response that harms beta cells.  Located in the pancreas, beta cells are responsible for making insulin.  Continuing to make even a small amount of insulin helps keep blood glucose levels in the normal range, lowering the risk of long-term complications.

The Study

This study enrolls 78 people in three groups: a placebo group, an Abrocitinib group and a Ritlecitinib group.  The drugs are in pill form and people will get them for a year and then be followed for another year.  The are recruiting Honeymooners (within 100 days of diagnosis) who are 12 to 25 years old, and will be measuring C-peptides as primary end point.  No secondary end points are listed (which is very unusual, but I don't know what it means).

The study started in October 2023 and is expected to finish in June 2026, but that is dependent on successful recruiting.  They are recruiting at a total of 15 locations in the United States, including both UCSF and Stanford.  Here is contact information: 

Jessica S Conaty  +1-813-396-9234  Jessica.Conaty@epi.usf.edu
Melissa Parker  +1-813-396-9378  Melissa.Parker@epi.usf.edu

Web Page: https://www.trialnet.org/our-research/newly-diagnosed-t1d/jakpot-t1d
Clinical Trial Registry: https://clinicaltrials.gov/study/NCT05743244

Discussion

I have previous reported on another JAK inhibitor (Baricitinib), which had medium-good results in a phase-IIΔ study:
https://cureresearch4type1diabetes.blogspot.com/2024/01/results-from-phase-ii-baricitinib.html
and there is a lot of interest in JAK inhibitors in general for a wide variety of immune and autoimmune diseases.  The American Diabetes Association scientific sessions (held last month) had several talks, papers, and posters on JAK inhibitors.

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 kid has type-1 diabetes and has participated 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.

 

BMF-219 Starts a Phase-IIΔ

BMF-219 is a drug that inhibits Menin, a protein.  Menin's functions in cells are not fully understood, and may be complex and contradictory.  For example, Menin is generally a tumor suppressor, but inhibiting Menin is a treatment for certain acute leukemias.  It is being developed by Biomea for T1D because it "is thought to act as a brake on beta-cell turnover and growth, supporting the notion that inhibition of Menin could lead to the regeneration of normal, healthy beta cells" according to the company's web site. 

The Study

The trial started in 2023, and is expected to finish in Sept-2025. It will enroll 150 people (mostly) within three years of diagnosis.  Everyone will get BMF-219 for 12 weeks and will be followed for one year.  The study design is a little complex, and the public description of the trial does not include how many people will be involved in each part, but the study has 3 parts, each with its own groups:

• The main study is blinded and has a total of three groups with different doses: 100mg, 200mg, and placebo.  These people will all be within 3 years of diagnoses.
• Substudy 1 is open label and has two dose groups: 100mg and 200mg, and also be within 3 years of diagnosis. 
• Substudy 2 is also open label with the same two dose groups, but includes people who were diagnosed between 3-15 years ago, but have slightly higher C-peptide generation than those in the main study or substudy 1.

For all parts, the primary end point is C-peptides, and the secondary end points include more C-peptide numbers, A1c numbers, insulin usage, and adverse events.  

The people to contact if you want to enroll in this study are Cristina Guzman and Michelle Stevens-Brogan, who are both at clinicaltrials@biomeafusion.com and +1-844-245-0490.  They are running the trial at a total of 11 locations in the US and 2 in Canada.  The list is in the clinical trial record (but none in California).

Clinical Trial Record: https://clinicaltrials.gov/study/NCT06152042

Discussion

My opinion is that the complex study design is to allow Biomea to have a small group where they can publish results relatively quickly (substudy 1) while also having a larger group (main study) which can deliver blinded (and therefore higher quality) results.  For me, this seems similar to how the company designed its T2D study, which I describe below.  The substudy 2 results can give them an early indication of effectiveness on people with established T1D, instead of honeymooners.

This strikes me as a very smart way to do a clinical trial if you have enough money. You file one set of paperwork and technically run one trial.  However, from that one trial you get quick data to use for marketing, slower but stronger data for eventual drug approval, and a little taste of data for a future development direction.  Very efficient.

Honeymoon or Established?

One question is, why recruit people within 3 years of diagnosis, when the honeymoon period is generally assumed to be a year or less?  Of course, I don't know the answer, but this is my best guess as to what is going on: The actual recruitment criteria include within 3 years of diagnosis and generating a little of your own insulin (and some other requirements).  This is pretty common.  But it may be that the researchers think that generating a little of your own insulin is the important part, and when that ends, the honeymoon ends, and the years matter much less.  As a practical mater, everyone will be within a year of diagnosis, because those are the only people who are generating enough insulin to enter the trial.

Another interesting question is: will a drug like this work better on honeymooners or established T1D?  The answer is not as simple as you would think.  Everyone agrees that T1D is caused by the immune system mistakenly attacking beta cells (which normally generate insulin).  When enough beta cells are destroyed, insulin production drops, and T1D is diagnosed.  But there are two open questions: (1) Is the immune attack a limited event that ends at some point in time?  And (2) does the body continue to naturally regenerate beta cells?

Most researchers believe that the immune attack continues for a person's whole life, and that natural beta cell regeneration is either nonexistent or really tiny.   If both of those assumptions are true, then this treatment is likely to have a stronger effect in extending the honeymoon, than curing established T1D.

But there are other scenarios: If the immune attack ends, but the body does not regenerate beta cells, then this treatment might cure established T1D, but not honeymoon T1D.  Because bMF-219 causes the beta cells to regrow after the immune attack has eneded.   On the other hand, if the immune attack is ongoing, then this probably will not cure established T1D, but might extend the honeymoon, depending on the relative strength of the immune attack vs. the treatment's effectiveness at regenerating beta cells.

Previous T2D Trial

Biomea had previously started a clinical trial on people with T2D, and published results from a very small (about 20 people) initial group.  Those results seemed very slightly positive to me, but I'm not used to evaluating T2D studies.  The entire trial (about 420 people) is expected to finish sometime between mid-2024 and mid-2025.

Of course, even if the results from a T2D trial are very positive, that does not mean it will work for T1D.  This is especially an issue for BMF-219, because if the T1D immune system attack on beta cells is still active, regenerating "normal, healthy beta cells" may have no effect as they are destroyed by the body's own immune system, same as the previous beta cells.  However it would tell us if the body naturally regenerated beta cells.

Biomea is also running two more clinical trials where BMF-219 is tested to treat cancers.

DiaTribe is a great source of information on diabetes research, and they covered both BMF-219 trials here, but focused on the T2D one:
https://diatribe.org/clinical-trial-tests-new-technique-stimulate-beta-cells

Corporate Web Site: https://biomeafusion.com/

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 kid has type-1 diabetes and has participated 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.

 

Fenofibrate Starts A Phase-IIΔ Trial

Fenofibrate (also spelled Phenofibrate) is commonly prescribed for high cholesterol and high triglycerides. In 2017, it was the 70th most commonly prescribed medication in the United States with more than eleven million prescriptions.  However, it is not approved for use in children under 18. It is also sometimes prescribed for diabetic retinopathy (eye damage from long term diabetes), and (off label) for gout.

However, it also showed some promise in NOD mice (an animal model for T1D), and so one person took it "off label" when they were diagnosed with T1D as a 19 year old.  They did not need to inject insulin for years after that, a huge result.  I blogged on this:
https://cureresearch4type1diabetes.blogspot.com/2020/06/strong-results-from-single-case-use-of.html
with an update:
https://cureresearch4type1diabetes.blogspot.com/2021/03/possible-cures-for-type-1-in-news-march.html

The Study 

The good news is that this study started in Sept-2022 and is scheduled to finish in July-2024.  However, the US registry lists them as still recruiting.  If this is true, completion will be delayed.  The European registry does not differentiate between recruiting and active, not recruiting.

The study includes about 100 children (10 to 17 years old) divided into two groups: one gets 160 mg Fenofibrate and one gets placebo.  This is a once per day pill given for a year.  The study is randomized and blinded.  The primary result is C-peptide and secondary results include more C-peptide numbers, insulin usage, adverse events, A1c, and several immunology measures.

European Clinical Trial Record: https://www.clinicaltrialsregister.eu/ctr-search/trial/2020-003916-28/PL
US Clinical Trial Record: https://clinicaltrials.gov/study/NCT05909800

Discussion

This is exactly the kind of study I want to see for Fenofibrate, and I'm very excited to see its results. 

There are two trials of Fenofibrate in people with T1D which are currently running.  The first is designed to prevent kidney function loss rather than cure/prevent/delay T1D itself and therefore I have not been following it.  It is NCT04929379.  They are enrolling 40 adults with T1D for at least 8 years and have diabetic kidney disease.  It started in 2022 and they hope to finish in 2025.  No intermediate results have been published, and I don't expect any.  But the bigger issue is that all their end points involve kidney function.  They are not measuring C-peptide, A1c, or insulin use at all.  So even if the medicine had a big impact, they would not see it.

The second is designed to prevent rather than cure/prevent/delay T1D itself, so I have not been following it, either.  It is NCT01320345.  They are enrolling 450 (!) adults with T1D and have eye problems.  It started in 2016 and they hope to finish in 2025.  No intermediate results have been published, and I don't expect any.  But the bigger issue is that all their end points involve eye function.  They are not measuring C-peptide, A1c, or insulin use at all.  So even if the medicine had a big impact, they would not see it.

Personal note: I try not to get excited about potential T1D cures.  I think treating all potential cures dispassionately is better for me and better for this blog.  (Plus, my child was diagnosed over 22 years ago, so getting excited would not have panned out.)  But I am human, and I do sometimes get excited.

I am optimistic about Fenofibrite potential to prevent T1D in the honeymoon phase.  I can't explain exactly why this honeymoon treatment and no other.  Maybe it is because it really seems to have worked for one person, for many years, and (as yet) there is no clinical trial results to dampen my enthusiasm. 

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 kid 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!

 

 

 

Siplizumab Starts Two Trials in Honeymoon T1D (DESIGNATE and STRIDE)

Siplizumab (MEDI-507, TCD601) is an monoclonal antibody which targets CD2 cells in the immune system.  It is being developed by ITB-MED.  It has an active research program for several different diseases with about 20 clinical trials in process.  There are currently two clinical trials targeting Type 1 Diabetes, so I'll discuss each in turn.

Part of the motivation to test Siplizumab was a previously good result from Alefacept.  I blogged about that result here:
https://cureresearch4type1diabetes.blogspot.com/2016/02/alefacept-reports-on-phase-ii-results.html
You can read my conclusions in the previous blog posting, but the company that made Alefacept stopped marketing the drug, which had been approved for Psoriasis, because of newer drugs in the field.  The hope is that Siplizumab will work in the same way and have similar results.

DESIGNATE: Phase-IIΔ Siplizumab in [Mostly] Honeymooners

This trial was started by the US government (National Institutes of Health, National Institute of Allergy and Infectious Diseases) and is being run by the Immune Tolerance Network.  It started in April-2023 and they are enrolling 120 people, which is a lot.

Initially adults were enrolled, but later the study started enrolling children as young as 8.  Everyone is within 18 months of diagnosis.  This is an open label trial, where each person will get one of four different doses of Siplizumab.  No placebo or control group.

The primary end points for this study look at changes in immune cells, so they are measuring how the treatment changes the immune system.  The secondary end points include adverse effects, C-peptides, and insulin use, and so cover both safety and effectiveness.

This study is in a pause right now.  They are evaluating the results from the first couple of people, and may change the study slightly based on what they learn.  When it moves forward, they will be recruiting at 20 locations all over the US, which will be listed in the links below:

Trial Web Page: https://www.designate-study.org/
Clinical trial registry: https://clinicaltrials.gov/study/NCT05574335

STRIDE: Phase-IIΔ Siplizumab in Honeymooners

This trial was started by a company (ITB-Med LLC), and is run by INNODIA.  It started in Jan-2023 and expects to finish in Jan-2025.  They are enrolling 96 adults. 

People must be within 100 days of diagnosis to be enrolled.  I think that there are four treatment groups, with one getting a placebo and the other three each getting a different dose of Siplizumab.  Nowhere are the doses listed.  Patients will get the treatment for 12 weeks; I think 1 dose per week, and will be followed for a year total. 

Although the description is a little vague, I think this study measures C-peptide as its primary end point, and adverse reactions as its secondary end point.  The first covers effectiveness and the second covers safety.

This study is still recruiting in Europe (Belgium, Germany, Italy, Poland, UK).  You can see the exact locations in the clinical trial registry: https://clinicaltrials.gov/study/NCT06025110

Eudract number: https://www.clinicaltrialsregister.eu/ctr-search/trial/2022-001713-39/SE

Discussion

These clinical trials have a lot in common.  The differences I do see are:

1. The STRIDE study recruits people within 100 days of diagnosis, while DESIGNATE recruits people within 18 months of diagnosis.
2. The STRIDE study recruits adults only, while DESIGNATE started out recruiting adults, but later added children.
3. STRIDE is single blind, DESIGNATE is open label. 
4. My understanding is that the two studies use different dosing techniques. 
   

An obvious question is: why did two different studies start at almost the same time, which are so similar?  I don't know the answer.  Both studies were motivated by the same company, the one producing the drug, and I'm always in favor of doing more studies, more quickly.  I'm happy they are moving forward along two separate paths at the same time.

One issue in the DESIGNATE trial is the recruitment cut off of 18 months after diagnosis.  I don't understand that.  The honeymoon time period is generally accepted to end after 12 months or a little earlier, so this means that DESIGNATE will include some honeymooners and some non-honeymooners.  I can only hope that they will analyze the data for both people recruited in their honeymoon phrase and separately for people who are not, just in case it works at one time but not at the other.

I'm not sure of the details of the history of Siplizumab, but I think it was originally developed by MedImmune,which was later bought by AstraZeneca.  However, the rights were later bought by ITB-Med, who are responsible for testing the drug on people with T1D. 

The Immune Tolerance Network, which is running DESIGNATE, is a collaborative network for clinical research, funded by the National Institute of Allergy and Infectious Diseases, part of the [United States] National Institutes of Health (especially through the special type 1 diabetes appropriation), and JDRF.

INNODIA, which is running STRIDE is a European non-profit network dedicated to preventing and curing type 1 diabetes.  It is funded by the European Commission’s Innovative Medicines Initiative (IMI-JU Joint Undertaking), The Hemsley Charitable Trust, JDRF, and EFPIA partners.

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 kid 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!

Why I Don't Blog On How To Vote For T1D (part 1)

This whole blog posting is very US (and US politics) focused, but the issues are similar in other countries.  Also, this blog is trying to describe the existing situation, not argue that the current situation is good, bad, or should be changed.  It is just describing how things are.

When I first starting blogging, over 15 years ago, I thought it might be a useful idea to have a "Voter's Guide To T1D" style blog.  I would discuss who to vote for if all you cared about was T1D, why one candidate was the best for T1D research, how your vote would impact T1D research, and so on.  I never quite got around to it.  Since then, I've changed my opinion and decided that such a blog could never be written because T1D should not effect how people voted.

As people affected by T1D, we should want two things from our government, which means two things to consider when we vote.  One is the best possible care for T1D and the other is the best possible research into finding a cure for T1D.  Some people are more care focused, and others more cure focused, but those are the biggest T1D issues out there.

The democratic way to view this is to ask the question:  "if T1D is important to me, who should I vote for to improve these things."  The idea is that your vote should be directed by what you care about.  My insight is that is not true, because your vote is actually about how to solve problems, not what problems are important.

For example, consider research.  In the US, more liberal people think that research can be encouraged by funding research.  More conservative people, by lowering regulation of research. Neither of these groups are for or against research, they differ on how to encourage it.

Obviously, the perfect person to vote for is someone who cares about T1D and who has the same political leanings as you, and obviously the worse person to vote for is someone who doesn't care about T1D and has the opposite political leanings as you. 

However, in choosing between someone who has your political leanings, but does not care about T1D and someone who does not have your political leanings, but cares a lot about T1D, who should you vote for?  The answer is: you should choose the person with your politics.  (And this is true even if you are a single issue voter, and that issue is T1D.)

Why?  Because the person who does not share your politics will do things that they think will help T1D, but you will not agree with, even for T1D.  For example, if you are a liberal who cares about T1D, but vote for a conservative because they care a lot about T1D, they are going to lower government regulations on big businesses.  And you are going to think, it just encourages businesses to fleece me and not do any more research. 

Therefore, you should vote your own politics and not vote for who "cares" more about T1D, unless it is a primary and both politicians have your politics in general, and it is just a question of more or less T1D.  If you have that luxury, definitely vote more T1D!  However, that assumes the choice is between to people who otherwise share your views.

In the past, there might have been politicians who were generally liberal, but conservative on T1D research, or the other way around, so you might want to vote for them because they matched your T1D views, even if they did not match your more general views.  But I think those politicians are rare to nonexistent today.  Today, politicians are either liberal about nearly everything or conservative about nearly everything.

So therefore, blogging about a particular politicians positions on T1D is a waste of time.  The important thing is their general politics, and others can blog on that much better than I can.

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 kid 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.