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.
Let me give you the punch line up front: 71% of the treatments currently in human trials have been funded by JDRF. (And the number is 83% for the later phase trials!) 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 groups that JDRF has funded, and those JDRF has never funded.
The list below 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. On the other hand, if it is in many clinical trials, all with established T1D, then it will be listed only once, no matter how many different trials are being run.
Established: One or more trials are open to people who have had type-1 diabetes for over a year.
Presymptomatics: 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. 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 also include indirect funding of various kinds. The JDRF funds nPOD, ITN, and several other organizations, so I include research done by these other groups as well.
The Difference Between Phase-II and Phase-II? Trials
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.
Waiting For FDA Approval
Summary: currently there is 1 drug in process of getting FDA approval for sale, and it is funded by JDRF.
Note: Provention Bio is preparing to submit Teplizumab for FDA approval for presymptomatics (people who have tested positive for two autoantibodies related to T1D, but who are not yet taking insulin) in 2020. In clinical trials in this population, Teplizumab delayed the onset of T1D and helped preserve some insulin production for two years. However it is unclear how long these effects will last.
Phase-III Human Trials
Summary: currently there are 2 treatments in a phase-III clinical trial. Both are funded by JDRF:
Phase-II Human Trials
Summary: there are 21 trials in phase-II, and 17 of them have been funded by JDRF, while 4 have not. Here are the treatments that have been funded by JDRF:
Not funded by JDRF:
Phase-II? Human Trials
Summary: there are 14 trials in phase-II, and 8 of them has been funded by JDRF, while 6 have not. Here are the treatments that have been funded by JDRF:
Not funded by JDRF:
Phase-I Human Trials
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:
Not funded by JDRF:
Summary of all Trials
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.
Just Looking at Trials on Established Type-1 Diabetics
12 of these treatments (21%) are being tested on established type-1 diabetics.
Of these, 8 are funded by JDRF.
So 75% of the trials recruiting established type-1 diabetics are funded by JDRF.
Compared to Last Year
In 2018 there were 59 treatments in clinical trials, in 2019 there are 56 (a drop of 5%).
In 2018 (and every previous year) there were no treatments waiting for approval to sell, in 2019 there is 1.
In 2018 there was 1 treatment in Phase-III trials, in 2019 there are 2 (growth of 100%).
In 2018 there were 22 treatments in Phase-II trials, in 2019 there are 21 (a drop of 5%).
In 2018 there were 12 treatments in Phase-II? trials, in 2019 there are 14 (growth of 17%).
In 2018 there were 24 treatments in Phase-I trials, in 2019 there are 18 (a drop of 25%).
A Little Discussion
The big break through this year is that Teplizumab has completed the clinical trials that Provention Bio thinks are required to get FDA approval. This is the first time any drug aimed at changing the course of T1D has ever gotten so far in the regulatory process.
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.)
How I Count Trials for This Comparison
Some Specific Notes:
This is an update and extension to blog postings that I've made for the previous seven years:
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 than is counted 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:
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. I'll be at the San Francisco (California) JDRF One Walk as part of "The Narwhals" team. Come by and say "hi", or strike up a conversation about research. I love to talk about research!
Joshua Levy
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 adult 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.