In the US, we are in the "Walking Season" when JDRF (Juvenile Diabetes Research Foundation) asks us to walk
to raise money for a cure for type-1 diabetes. So I'd like to do my part, by reminding you
all of how important JDRF is to the human trials of potential cures for T1D, which I track.
In Processes To Submit For FDA Approval
Summary: currently there is 1 drug in process of being submitted to the US FDA for approval for sale, and it was funded by JDRF.
- Teplizumab by Provention Bio (At Risk)
Phase-III Human Trials
- Oral Insulin (Preventative)
- Teplizumab by Provention Bio
Note: Teplizumab is listed separately here, because it is being tested separately for people with honeymoon type 1 diabetes.
- AAT (Alpha-1 Antitrypsin) by Kamada
- ATG and GCSF by Haller at University of Florida (Established)
- Abatacept by Orban at Joslin Diabetes Center
- Abatacept by Skyler at University of Miami (Prevention)
- Aldesleukin (Proleukin) at Addenbrooke’s Hospital, Cambridge, UK
- Diamyd, Ibuprofen ("Advil"), and Vitamin D by Ludvigsson at Linköping University
- Diamyd, Etanercep, and Vitamin D by Ludvigsson at Linköping University
- Diamyd and Vitamin D by Larsson at Lund University (Prevention)
- Gleevec by Gitelman at UCSF
- Gluten Free Diet: Three Studies (Preventative)
- Stem Cell Educator by Zhao (Established)
- Tocilizumab by Greenbaum/Buckner at Benaroya Research Institute
- TOL-3021 by Bayhill Therapeutics
- TOL-3021 by Bayhill Therapeutics (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 14 trials in phase-II?, and 8 of them have been funded by JDRF, while 6 have not. Here are the treatments that have been funded by JDRF:
- Alpha Difluoromethylornithine (DFMO) by DiMeglio
- GABA by Diamyd
- Golimumab by Janssen
- Golimumab by Greenbaum (Established)
- Hydroxychloroquine by Greenbaum (At Risk)
- Intranasal Insulin by Harrison at Melbourne Health (Prevention)
- Iscalimab (CFZ533) by Novartis
- Rituximab by Pescovitz at Indiana University
- Azithromycin by Forsander
- Ladarixin by Emanuele Bosi of Dompé Farmaceutici
- Liraglutid (At Risk)
- NNC0114-0006 and Liraglutide by Novo-Norsk
- Rapamycin Vildagliptin Combo by IRCCS (Established)
- Visbiome by Medical College of Wisconsin
Summary: there are 18 trials in phase-I, and 12 of them are funded by JDRF, while 6 are not. Here is the list funded by JDRF:
- AG019 and Teplizumab by ActoGeniX
- Alefacept by TrialNet
- CGSF by Haller at University of Florida
- Golimumab by (At Risk)
- MER3101 by Mercia (previously IBC-VS01 by Orban)
- MonoPepT1De by Cardiff University
- Mozobil by University of Alberta (Established)
- MultiPepT1De (Multi Peptide Vaccine) by Powrie at King’s College London
- Nasal insulin by Harrison at Melbourne Health (Prevention)
- Tauroursodeoxycholic Acid (TUDCA) by Goland at Columbia University
- Pro insulin peptide by Dayan at Cardiff University
- VC-01 by Viacyte (Established)
- AVT001 by Avotres
- Baby Teeth Stem Cells by CAR-T Biotechnology
- Gluten Free Diet by Carlsson at Lund University
- Mesenchymal Stromal Cell by Carlsson at Uppsala University
- Microvesicles (MVs) and Exosomes by Nassar at Sahel Teaching Hospital
- ProTrans by NextCell (Established)
56 in total
40 funded by JDRF
So 71% of the human trials currently underway are funded (either directly or indirectly) by JDRF. Everyone who donates to JDRF should be proud of this huge impact; and everyone who works for JDRF or volunteers for it, should be doubly proud.
9 of these treatments (16%) are being tested on people with established T1D.
Of these, 6 are funded by JDRF.
So 66% of the trials recruiting people with established T1D are funded by JDRF.
Compared to Last Year
In 2019 there were 56 treatments in clinical trials, in 2020 there are 56 (no change).
In 2019 there was 1 treatment in process of approval to sell, in 2020 there is 1 (no change).
In 2019 there was 2 treatment in Phase-III trials, in 2020 there are 2 (no change).
In 2019 there were 21 treatments in Phase-II trials, in 2020 there are 21 (no change).
In 2019 there were 14 treatments in Phase-II? trials, in 2020 there are 14 (no change).
In 2019 there were 18 treatments in Phase-I trials, in 2020 there are 18 (no change).
A Little Discussion
The big break through from 2019 was that Provention Bio expected to submit Teplizumab for approval in 2020. Their most recent press release says they are still on that schedule. They expect to complete their application to the US FDA in the 4th quarter.
The money that we all donate is the thing that is going to move more Phase-II studies into Phase-III studies, the Phase-I studies to Phase-II, create more Phase-I studies, and so on. If you don't like where we are on research, donating money is the way to make it better. And if you do like where we are, then money is the way to push these things forward into the market. If you're worried about your money going to non-research, then you can do what I do: fill out the attached form or go to the following website and send it in with your donation: http://thejdca.org/good-giving-landing-page/ (Unfortunately I don't know how to do this for on-line donations.)
Notes on How Trials Are Grouped
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.
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.
Phase-II trials are "classic" phase-II trials; they are done after a successful Phase-I trial in type-1 diabetes. What I call Phase-II? trials are done on known safe treatments, so they don't need Phase-I trials, but have never been tested on type-1 diabetes before. These Phase-II? trials might be Phase-II from the point of view of size and safety, but they are Phase-I in terms of effectiveness, so I'm putting them in their own category.
- 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. Also, I list experiments separately if they use at least one different drug.
- The ITN (Immune Tolerance Network) has JDRF as a major funder, so I count ITN as indirect JDRF funding.
- I have made no attempt to find out how much funding different organizations gave to different research. This would be next to impossible for long research programs, anyway.
- Funding of research is not my primary interest, so I don't spend a lot of time tracking down details in this area. I might be wrong on details.
- I 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.
- The PreventT1D study (Vitamin D and Omega-3s) is a "field study" so not included.
- A Rotavirus Vaccine study which was published this year was a population based study, so also not included.
- I've removed Dr. Faustman's BCG research from my list of potential
cures, because it is no longer aimed at a cure. For more information
read this blog:
https://cureresearch4type1diabetes.blogspot.com/2018/09/every-year-in-september-or-october-i.html and for even more details
- 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.
- Serova's Cell Pouch and DRI's BioHub: These two clinical trials are both testing one piece of infrastructure which might be used later in a cure. They are testing a part of a potential cure. However, in both cases, the clinical trials being run now require immunosuppression for the rest of the patient's life, so I'm not counting them as testing a cure.
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.