Normally, sometime in October, I write a summary of all the clinical trials funded by JDRF. However, this year I didn't, which was a mistake. But late is better than never, so in this post I summarize the clinical trails that Breakthrough T1D (formerly JDRF) is funding as of October 1st, 2024.
I hope to remind everyone of how important Breakthrough T1D is to the human trials of potential cures for T1D, which I track.
Let me give you the punch line up front: 65% of the treatments currently in human trials have been funded by Breakthrough T1D, and this goes to 77% for the Phase-III and Phase-II studies! This is a strong impact; one that any non-profit should be proud of. Below is a list of all the treatments, grouped by phase, and separated into trials that Breakthrough T1D has funded, and those Breakthrough T1D has never funded.
The List, Divided by Phases
Below is the list of all treatments, divided into six phases: FDA Approved, In Process of Approval, Phase-III, Phase-II, Phase-IIΔ, and Phase-I. Phase-II trials are "classic" phase-II trials, which are done after a Phase-I trial. What I call Phase-IIΔ trials test treatments which never went through phase-I trials on people with T1D. (I used to call those Phase-II? but I think using punctuation that way is confusing, so I'm using a delta instead: Phase-IIΔ.) They've been shown safe in other diseases, so have skipped phase-I trials on people with T1D. These Phase-IIΔ trials might be Phase-II from the point of view of size and safety, but they are Phase-I in terms of effectiveness, so I'm putting them in their own category.
For T1D research, phase-I studies are usually about 10 people and test for both safety and efficiency. In other diseases, phase-I trials are sometimes only done on healthy people, or only test for safety issues, but this is not the way T1D research is usually done. Over 90% of phase-I studies are done on people with T1D, and over 90% test for both safety and effectiveness.
Phase-II trials are about 100 people, and phase-III about 300. After two successful phase-III trials, the FDA considers approval for general use. These two studies can be run at the same time, and are often identical. Occasionally, only one phase-III trial is required for approval.
Approved or In Process of FDA Approval
In 2024, nothing was approved and nothing is in process of approval.Phase-III Human Trials
Summary: currently there are 3 treatments in phase-III clinical trials. 2 are funded by JDRF:
Summary: there are 19 trials in phase-II, and 15 of them have been funded by Breakthrough T1D, while 4 have not. Here are the treatments that have been funded by Breakthrough T1D:- Diamyd by Diamyd
- Oral Insulin (Preventative)
Not funded by JDRF:
- Ladarixin by Dompé
- ATG and GCSF by Haller at University of Florida (Established)
- Abatacept in honeymooners and as a prevention by Orban at Joslin Diabetes Center and Skyler at University of Miami (Prevention)
- Aldesleukin (Proleukin) at Addenbrooke’s Hospital, Cambridge, UK
- Diamyd in several combinations by Ludvigsson at Linköping University and Larsson at Lund University (Honeymoon and Prevention)
- Difluoromethylornithine (DFMO) by Panbela
- Gleevec by Gitelman at UCSF
- Gluten Free Diet: Three Studies (Preventative)
- Rituximab and Abatacept by TrialNet
- Stem Cell Educator by Zhao (Established)
- Tauroursodeoxycholic Acid (TUDCA)
- Tocilizumab by Greenbaum/Buckner at Benaroya Research Institute
- TOL-3021 by Bayhill Therapeutics (Honeymoon and Established)
- Umbilical Cord Blood Infusion by Haller at University of Florida
- Ustekinumab by University of British Columbia
- Verapamil by Shalev/Ovalle at University of Alabama at Birmingham
- ATG and autotransplant by several research groups: Burt, Snarski, and Li
- Dual Stem Cell by Tan at Fuzhou General Hospital
- Stem Cells of Arabia (Established)
- Vitamin D by Stephens at Nationwide Children's Hospital (Prevention)
Summary: there are 19 trials in phase-IIΔ, and 10 of them have been funded by Breakthrough T1D, while 9 have not. Here are the treatments that have been funded by Breakthrough T1D:
- Alpha Difluoromethylornithine (DFMO) by DiMeglio
- Baricitinib by St Vincent's Institute of Medical Research
- GABA by Diamyd
- Golimumab by Janssen (Honeymoon and Established)
- Hydroxychloroquine by Greenbaum (At Risk)
- Intranasal Insulin by Harrison at Melbourne Health (Prevention)
- Iscalimab (CFZ533) by Novartis
- Influenza Vaccination at Aarhus University Hospital
- Pleconaril and Ribavirin by Oslo University Hospital
- Siplizumab by NIH and ITB-Med LLC
- Abrocitinib or Ritlecitinib by NIH/Pfizer
- Azithromycin by Forsander
- BMF-219 by Biomea Fusion (Established)
- Fenofibrate at Warsaw Medical University
- Ixekizumab/Taltz by Vastra Gotaland Region
- Liraglutid (At Risk)
- NNC0114-0006 and Liraglutide by Novo-Norsk (Established)
- Rapamycin Vildagliptin Combo by IRCCS (Established)
- Visbiome by Medical College of Wisconsin
Summary: there are 21 trials in phase-I, and 13 of them are funded by Breakthrough T1D, while 8 are not. Here is the list funded by Breakthrough T1D:
- AG019 and Teplizumab by ActoGeniX
- DIMID1 (Faecal Microbiota Transplantation) at AMC Hospital
- Diamyd by Diamyd (At Risk)
- CGSF by Haller at University of Florida
- Golimumab (At Risk)
- MER3101 by Mercia (previously IBC-VS01 by Orban)
- Mozobil by University of Alberta (Established)
- PRV-101 (Coxsackie B Vaccine) by Provention Bio (Prevention)
- Semaglutide by Dandona at University of Buffalo
- TOPPLE T1D by Novo Nordisk (Established)
- VC-01 by Viacyte (Established)
- VCTX210A by Viacyte/CRISPR (Established)
- VX-264 by Vertex (Established)
- AVT001 by Avotres
- Baby Teeth Stem Cells by CAR-T Biotechnology
- Extracorporeal Photopheresis by ADSCC
- Gluten Free Diet by Carlsson at Lund University
- NN1845 (Glucose Sensitive Insulin) by Novo Nordisk
- OPT101 by Op-T (Established)
- PIpepTolDC at City of Hope Medical Center
- ProTrans by NextCell (Established)
62 in total
40 funded by JDRF
So 65% of the human trials currently underway are funded (either directly or indirectly) by JDRF. Everyone who donates to Breakthrough T1D should be proud of this huge impact; and everyone who works for Breakthrough T1D or volunteers for it, should be doubly proud.
Of these treatments 14 (23%) are being tested on people with established T1D, of those, 9 are funded by Breakthrough T1D. So 64% of the trials recruiting people with established T1D are funded by Breakthrough T1D.
Compared to Last Year
In 2023 there were 59 treatments in clinical trials, in 2024 there are 62 (growth of 5%).
In 2023 there were 59 treatments in clinical trials, in 2024 there are 62 (growth of 5%).
In 2023 there was 2 treatments in Phase-III trials, in 2024 there are 3 (growth of 50%).
In 2023 there were 17 treatments in Phase-II trials, in 2024 there are 19 (growth of 11%).
In 2023 there were 14 treatments in Phase-IIΔ trials, in 2024 there are 19 (growth of 36%).
In 2023 there were 25 treatments in Phase-I trials, in 2024 there are 21 (dropped by 16%).
In 2023 there were 17 treatments in Phase-II trials, in 2024 there are 19 (growth of 11%).
In 2023 there were 14 treatments in Phase-IIΔ trials, in 2024 there are 19 (growth of 36%).
In 2023 there were 25 treatments in Phase-I trials, in 2024 there are 21 (dropped by 16%).
I think that the drop in Phase-I trials was mostly caused by me doing a good housecleaning to remove old, moribund trials, when I had not done that in several previous years.
A Little Discussion
The money that we donate does many things:
- It finances more clinical trials (especially early clinical trials).
- It finances making clinical trials (especially early clinical trials) larger and better designed.
- It helps push possible cures to the next level of trial. It finances moving phase-I trials to phase-II, and phase-II to phase III.
I like to say that there are two reasons for donating money for research into T1D. People who like the research being done should donate money to move it forward, faster. People who don't like the research being done should donate money to start up different research which (presumably) they will like more. So no matter which group you are in, you should donate. 😀
Trial Populations
The list above uses the following marks to show the nature of the treatments, and if one treatment is being tested in different populations, then it will be listed more than once.
Honeymoon: Most trials are done on people within the first year of diagnosis. All the studies listed above which are not Established, At Risk, or Prevention are in this Honeymoon category.
Honeymoon: Most trials are done on people within the first year of diagnosis. All the studies listed above which are not Established, At Risk, or Prevention are in this Honeymoon category.
Established: One or more trials are open to people who have had type-1 diabetes for over a year.
At Risk: One or more trials are open to people who have 2 or more autoantibodies, but have not yet started showing symptoms of type-1 diabetes.
Prevention: This treatment is aimed at preventing type-1 diabetes, not curing it.
If a trial is not marked, then it is for people in the honeymoon (first year) of T1D.
I give an organization credit for funding a treatment if they funded it at any point in development; I don't limit it to the current trial.
At Risk: One or more trials are open to people who have 2 or more autoantibodies, but have not yet started showing symptoms of type-1 diabetes.
Prevention: This treatment is aimed at preventing type-1 diabetes, not curing it.
If a trial is not marked, then it is for people in the honeymoon (first year) of T1D.
I also give credit if JDRF funds research indirectly, through another organization. For example, JDRF funds nPOD, Immune Tolerance Network, and INNODA and so I give Breakthrough T1D credit for clinical trials based on their work.
How I Count Trials for This Comparison
- I don't count trials where the Breakthrough T1D funded some basic research, but not the research which lead to a specific clinical trial. I'm sure this under estimates JDRF's impact. For example OPT101 is an anti CD154 drug. JDRF has funded many studies on CD154, but not the particular research that is being tested here. Similarly with Ixekizumab, JDRF has funded related research on that drug, but not the clinical trial or the research immediately leading to the clinical trial here.
- I mark the start of a research trial when the researchers start recruiting patients (and if there is any uncertainty, when the first patient is dosed). Some researchers talk about starting a trial when they submit the paper work, which is usually months earlier.
- For trials which use combinations of two or more different treatments, I give funding credit, if the organization in the past funded any component of a combination treatment, or if they are funding the current combined treatment.
- I have made no attempt to find out how much funding different organizations gave to different research. This would be next to impossible for long research programs, anyway.
- Funding of research is not my primary interest, so I don't spend a lot of time tracking down details in this area. I might be wrong on details.
- I only include intervention studies here, because those are the only type of study that the FDA will accept for the eventual approval of a new treatment.
Research Not Listed Here
I sometimes get asked why some piece of research is not listed here, and so here are some of those answers:
- VX-880 is a transplant study which requires life long immunosuppression. Read here why I do not consider these to be cures.
- Levicure's Combination Therapy has only been tested in a retrospective study, not an intervention based clinical trial.
- Oral Insulin: This trial was a phase-III trial, meaning that it was large and designed to provide enough information so that, if successful, the treatment could be widely used. However, as it turned out, only part was successful, and that part was phase-II sized, so I don't think we will see widespread use based on this trial alone. You can think of this as a phase-III trial with phase-II results.
Please remember that my blog (and therefore this posting) covers research aimed at curing, delaying, or preventing type-1 diabetes that is currently being tested in humans. There is a lot more research going on than is counted here.
Please think of this posting as being my personal "thank you" note to all the Breakthrough T1D staff, volunteers, and everyone who donates money to research a cure for type-1 diabetes:Thank You!
Finally, if you see any mistakes or oversights in this posting, please tell me! There is a lot of information packed into this small posting, and I've made mistakes in the past.
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.
