In the US, we are in the "Walking Season" when JDRF asks us to walk to raise money for a cure. So I'd like to do my part, by reminding you all of how important JDRF is to the human trials of potential cures for type-1 diabetes, which I track.
Let me give you the punch line up front: 70% of the treatments currently in human trials have been funded by JDRF. (And the number is 71% for the later phase trials) This is a strong impact; one that any non-profit should be proud of. This summary does not include Artificial Pancreas research or stem cell growth trials, because there are so many of those that it would be hard to include them all. For a recent summary of some (not not all) AP research, please read this blog posting: http://cureresearch4type1diabetes.blogspot.com/2014/07/ada-2014-type-1-diabetes-cure-research.html
Below is a list of all the potential cures, grouped by phase of trial that they are currently in, and separated into potential cures that JDRF has funded, and potential cures that JDRF has never funded.
This list is a list of treatments, and many of these are being tested in more than one clinical trial. For example, the "ATG and autotransplant" treatment is actually running three trials, but since they are all testing the same treatment, it is only one item in the list. The list below uses the following marks to show the nature of the treatments:
(Established) One or more trials are open to people who have had type-1 diabetes for over a year.
(Prevention) This treatment is aimed at preventing type-1 diabetes, not curing it.
Also remember that I give an organization credit for funding a treatment if they funded it at any point in development; I don't limit it to the current trial. For example, JDRF is not funding the current trials for AAT, but they did fund earlier research into it, which helped it grow into human trials. I include indirect funding of various kinds. For example, the JDRF funds nPOD and helps to fund ITN and several other organizations, so I include research done by these other groups as well, as being indirectly JDRF funded.
Cures in Phase-III Human Trials
Summary: currently there are no treatments aimed at curing type-1 diabetes which are in phase-III trials (under the definition of cure that I use). This is the second year in a row there have been no phase-III trials underway, and it's not a good thing. Even worse, I don't see a phase-III study starting even next year. However, phase-III trials grow out of phase-II trials, and there was big growth in the number of phase-II trials this year. I'm very hopeful that in a few years, these will naturally result in a number of phase-III trials.
Cures in Phase-II Human Trials
Summary: there are 21 trials in phsae-II, and 15 of them have been funded by JDRF, while 6 have not. Here are the treatments that have been funded by JDRF:
- AAT (Alpha-1 Antitrypsin) by Grifols Therapeutics and also Kamada
- Abatacept by Orban at Joslin Diabetes Center
- Abatacept by Skyler at University of Miami (Prevention)
- Aldesleukin (Proleukin) at Addenbrooke’s Hospital, Cambridge, UK
- Diabecell by Living Cell Technologies (Established)
- Diamyd, Ibuprofen ("Advil") and Vitamin D by Ludvigsson at Linköping University
- Gleevec by Gitelman at UCSF
- Oral Insulin (Preventative)
- Rituximab by Pescovitz at Indiana
- Stem Cell Educator by Zhao (Established)
- Teplizumab (AbATE study team)
- Teplizumab by Herold/Skyler/Rafkin (Preventative)
- Umbilical Cord Blood Infusion by Haller at University of Florida
- Ustekinumab by University of British Columbia
- Xoma 52 by Xoma Corp (Established)
Not funded by JDRF:
- ATG and autotransplant by Burt, and also Snarski, and also Li
- Atorvastatin (Lipitor) by Willi at Children's Hospital of Philadelphia
- BCG by Faustman at MGH (Established)
- Brod at University of Texas-Health Science Center
- Secukinumab by Novartis Pharmaceuticals
- Vitamin D by Stephens at Nationwide Children's Hospital (Prevention)
Cures in Phase-I Human Trials
Summary: there are 19 trials in phase-I, and 13 of them are funded by JDRF, while 6 are not. Here is the list funded by JDRF:
- Alefacept by TrialNet
- ATG and GCSF by Haller at University of Florida (Established)
- ßAir bio-artificial pancreas by Beta-O2's at Uppsala University Hospital in Sweden (Established)
- TOL-3021 by Bayhill Theraputics (Established)
- CGSF by Haller at University of Florida
- Trucco at Children’s Hospital of Pitt / Dendritic Cells (DV-0100) by DiaVacs (Established)
- IBC-VS01 by Orban at Joslin Diabetes Center
- Leptin by Garg at University of Texas
- Lisofylline by DiaKine
- Nasal insulin by Harrison at Melbourne Health (Prevention)
- Polyclonal Tregs by both Trzonkowski and Gitelman
- Pro insulin peptide by Dayan at Cardiff University
- VC-01 by Viacyte (Established)
Not funded by JDRF:
- CGSF and autotransplant by Esmatjes at Hospital Clinic of Barcelona (Established)
- Encapsulated Islets at University clinical Hospital Saint-Luc (Established)
- Etanercept (ENBREL) by Quattrin at University at Buffalo School of Medicine
- Monolayer Cellular Device (Established)
- Rilonacept by White at University of Texas
- The Sydney Project, Encapsulated Stem Cells (Established)
Summary of all Trials
40 in total
28 funded by JDRF
So 70% of the human trials currently underway are funded (either directly or indirectly) by JDRF. Everyone who donates to JDRF should be proud of this huge impact; and everyone who works for JDRF or volunteers for it, should be doubly proud.
Just Looking at Trials on Established Type-1 Diabetics
13 of these treatments (33%) are being tested on established type-1 diabetics.
Of these, 8 are funded by JDRF
So 62% of the trials recruiting established type-1 diabetics are funded by JDRF.
Compared to Last Year
In 2013 there were 37 treatments in clinical trials, in 2014 there are 40 (growth of 8%)
In 2013 there were no treatments in Phase-III trials, in 2014 there are none (no change).
In 2013 there were 15 treatments in Phase-II trials, in 2014 there are 21 (growth of 40%).
In 2013 there were 22 treatments in Phase-I trials, in 2014 there are 19 (drop of 14%).
How I Count Trials for This Comparison
- I give an organization credit for funding a cure if it funded that cure at any point in it's development cycle.
- I mark the start of a research trial when the researchers start recruiting patients (and if there is any uncertainty, when the first patient is dosed). Some researchers talk about starting a trial when they submit the paper work, which is usually months earlier.
- If there are different clinical trials aimed at proving effectiveness as a cure and as a preventative, or effectiveness in honeymooners and established diabetics, then those are counted separately.
- For trials which use combinations of two or more different treatments, I give funding credit, if the organization in the past funded any component of a combination treatment, or if they are funding the current combined treatment. Also, I list experiments separately if they use at least one different drug.
- The ITN (Immune Tolerance Network) has JDRF as a major funder, so I count ITN as indirect JDRF funding.
- I have made no attempt to find out how much funding different organizations gave to different research. This would be next to impossible for long research programs, anyway.
- Funding of research is not my primary interest, so I don't spend a lot of time tracking down details in this area. I might be wrong on details.
- I use the term "US Gov" for all the different branches and organizations within the United States of America's federal government (so includes NIDDK, NIAID, NICHD, etc.)
- I don't work for the US Gov, JDRF, or any of the other organizations discussed here. I have a more complete non-conflict of interest statement on my web site.
Some Specific Notes:
- The FDA's clinical trials web site lists two studies being done on Cyclosporine and Lansoprazole ("Prevacid") as a combination treatment. These trials were listed over 18 months ago but not started recruiting patients. I have not included them in my list of clinical trials.
- LX4211: This drug is a SLGT2, and I don't think it is likely to be a cure. It might turn into a treatment that can be paired with insulin for better results, but not a cure.
- 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.
- INGAP: This treatment was in human trials twice, but long in the past. The current testing is being done by a high school student, and I'm not counting it as cure research until I see better results than were seen before (and which previously led nowhere).
This is an update and extension to blog postings that I've made for the previous five years:
Finally, please remember that my blog (and therefore this posting) covers research aimed at curing or preventing type-1 diabetes that is currently being tested in humans. There is a lot more research going on, not covered here.
Please think of this posting as being my personal "thank you" note to all the JDRF staff, volunteers, and everyone who donates money to research a cure for type-1 diabetes:
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.
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.