Monday, October 7, 2013

JDRF Funding for a Cure 2013

In the US, we are starting 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 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: 65% of the treatments currently in human trials have been funded by JDRF. (And the number is 73% 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.

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.

The 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: there are no treatments aimed at curing type-1 diabetes which are in phase-III trials   (under the definition of cure that I use).
In the past, I have listed DiaPep277 here, but their most recent results make it pretty clear that they are seeing a treatment result, not a cure result.  So I removed it from the list this year.  Also there are two studies being done on Cyclosporine and Lansoprazole ("Prevacid") as a combination treatment.  Those trials are filed with the FDA, but have not started recruiting patients, so they are not listed here, either.  Maybe next year.

Cures in Phase-II Human Trials
Summary: there are a total of 15: 11 of them have been funded by JDRF, and 4 have not. Here are the treatments that have been funded by JDRF:
  • Abatacept by Orban at Joslin Diabetes Center
  • 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
  • Oral Insulin (Preventative)  
  • Rituximab by Pescovitz at Indiana
  • Sitagliptin and Lansoprazole at Sanford Health
  • Stem Cell Educator by Zhao (Established)
  • Teplizumab (AbATE study team)
  • Umbilical Cord Blood Infusion by Haller at University of Florida
  • 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
  • Brod at University of Texas-Health Science Center
  • Vitamin D by Stephens at Nationwide Children's Hospital  (Prevention)
Cures in Phase-I Human Trials
Summary: there are a total of 22: 13 of them are funded by JDRF and 9 are not. Here is the list funded by JDRF:
  • Alefacept by TrialNet
  • AAT (Alpha-1 Antitrypsin) by OmniBio and also Kamada 
  • ATG and GCSF by Haller at University of Florida  (Established)
  • TOL-3021 by Bayhill Theraputics   (Established)
  • CGSF by Haller at University of Florida
  • Trucco at Children’s Hospital of Pittsburgh   (Established)
  • IBC-VS01 by Orban at Joslin Diabetes Center
  • Leptin by Garg at University of Texas
  • Nasal insulin by Harrison at Melbourne Health
  • Polyclonal Tregs by both Trzonkowski and Gitelman 
  • Pro insulin peptide by Dayan at Cardiff University
  • Proleukin and Rapamune by Greenbaum at Benaroya Research Institute   (Established)
  • Lisofylline by DiaKine
Not funded by JDRF:
  • BCG by Faustman at MGH  (Established)
  • 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
  • GABA by Lunsford at the University of Alabama at Birmingham.
  • Monolayer Cellular Device  (Established)
  • Rilonacept by White at University of Texas
  • The Sydney Project, Encapsulated Stem Cells  (Established) 
  • Pioglitazone by Wilson at Stony Brook 
Summary of all Trials
37 in total
24 funded by JDRF
So 65% 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
11 of these treatments (29%) are being tested on established type-1 diabetics.
Of these, 6 are funded by JDRF
So 55% of the trials recruiting established type-1 diabetics are funded by JDRF.

Compared to Last Year
In 2012 there were 38 treatments in clinical trials, in 2013 there are 37 (drop of 3%)
In 2012 there was 1 treatment in Phase-III trials, in 2013 there are none (drop of 100%).
In 2012 there were 14 treatments in Phase-II trials, in 2013 there are 15  (growth of 7%).
In 2012 there were 23 treatments in Phase-I trials, in 2013 there are 22 (drop of 4%).

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.
  • 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.
This is an update and extension to blog postings that I've made for the previous five years:
http://cureresearch4type1diabetes.blogspot.com/2012/09/jdrf-funding-for-cure-2012.html
http://cureresearch4type1diabetes.blogspot.com/2011/10/jdrf-funding-research-for-cure-2011.html
http://cureresearch4type1diabetes.blogspot.com/2010/09/jdrf-funding-research-for-cure-2010.html
http://cureresearch4type1diabetes.blogspot.com/2009/09/jdrf-funding-research-for-cure.html
http://cureresearch4type1diabetes.blogspot.com/2008/10/jdrf-funding-of-cure-research-phases-ii.html

Finally, please remember that my blog (and therefore this posting) covers research aimed a 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:
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 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.

22 comments:

Leo said...

Hi
Great to see this summary but I do not see my favorite for a cure in it... I do want you to take a third look (according to your blog you have two entries about them) at Sernova & Edmonton's lead scientist dr James Shapiro in case you have missed it .. They are in clinical trials like 6 months already and they recently announced preliminary positive results http://sernova.com/press/?ID=166
Keep up the good work informing us
Best Regards
Lazar Tomovski

Joshua Levy said...

Sernova's work is interesting. However, their current clinical trial is using full immunospression. (It is no different than many other transplant trials which I do not follow, because they require long term immunospression. I consider that trading one set of issues for another set, which is no better, and might be worse.

I know that they have plans to add sertoli cells to their Cell Pouch, and they hope they will not need immunospression. At that point, they really will be testing a cure, and I will list their trial then. (Obviously, I'm already following it.)

Some might think of this as "splitting hairs", but (in my opinion) the current trial is not aimed at curing type-1 diabetes. It is laying the foundation for a future trial, which will.

Joshua

Leo said...

Hi
I totally agree with you!
I must have skipped reading this "To protect the islets from immune cell attack, the patients are given an antibody and immunosuppressive medications."
I do hope sertoli cells do their job
Best Regards
Lazar

QUALITY SERVICES said...
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Suzy Q said...

Joshua you are the best! Thanks for doing all of the hard work of compiling and reporting on the research :-)

Suzanne
mom to Merrick, dx 7/10

GabLarConBriCar said...

JDRF's prominence in the T1 cure community is obviously undeniable. In this sense, I generally agree with your post.

However, as very well pointed out by JDCA and others, their deficiencies are notorious. Just to mention the three most notable ones:

- only a fraction of their resources goes to cure, while most of their funding campaigns tell donors their money is for cure.

- their "partnership" with the industry is at least questionable in its logic. In summary, charity funding goes to big pharmas to develop ways to sell more insulin (read artificial pancreas, smart-inteligent insulin, even basic science! and others).

- corporate governance standards are way deficient. Details in http://www.thejdca.org/wp-content/uploads/2013/08/Corporate-Governance-STAR-Review.pdf

Emily Riopelle said...

I agree fully with GabLarConBriCar that JDRF could be using a much bigger percentage of their funds toward research. However, you have Faustman's BCG trials listed under not funded by JDRF. I contacted JDRF and they told me that they have funded Dr. Faustman in the past, but they are not currently. Not sure why this is, I think her research seems very promising.

Leo said...

Hi
hmm I have tweeted Sernova this

@SernovaCorp Hi, I just want to ask if you are using, for the current clinical trial, full immunospression or you are using sertoli cells

and the answer was this

we are infact using our patented Sertolin™ cells. Please check out our website & feel free to ask us anything else!

I am puzzled !!

Joshua Levy said...

Leo: I don't think 140 character tweet is a good way to learn about what they are doing! :-) Their clinical trial paperwork does not mention Sertolin cells at all: http://www.clinicaltrials.gov/ct2/show/NCT01652911. You might want to ask them where it says they are using Sertolin cells or what press release they issued that says they are using Sertolin cells right now. I just don't see it. --Joshua

Joshua Levy said...

Emily: My understanding is that JDRF funded Dr. Faustman prior to her BCG work (over 10 years ago), but never funded her BCG work. My list is by cure, not by researcher, so Dr. Faustman's BCG work is non-JDRF funded, even though she was funded by them prior to the BCG work.

As far as the BCG work goes, she has published a phase-I study, which was unsuccessful. (Failed pre-set primary end point.) She has said that she has already raised enough money to run a phase-II trial, so I expect to see results from that as well. But as the first trial did not work out, I'm not expecting much from the second. To the best of my knowledge the second trial has not yet filed cliniical trial registry paperwork, or started recruiting. --Joshua

Joshua Levy said...

This is my reply to GabLarConBriCar (her quotes start with a dash "-"). Each of her key points gets a separate comment from me:

- only a fraction of their resources goes to cure, while most of their funding campaigns tell donors their money is for cure.

Well, it is certainly true that about 61% of JDRF's funding goes to cure based research. For comparison, about 30% goes to treatment, and about 8% to prevention. (Artificial Pancreas research is under treatment, and is about 12% of total). My guess, is that people who have had type-1 for a long time lobby for more money into treatments, while those newly diagnosed lobby for more into cure research. So I'm sure JDRF is "getting yelled at in each ear" when it comes to allocation.

But as for their funding campaigns, it depends where you look. Their logo, at the top of most (all?) of their web pages, says very specifically: "Improving Lives. Curing Type-1 Diabetes". (and Cure is in a different color.) That seems spot-on to me. On the other hand, near the bottom it says things like "Walk to Cure Diabetes" and "Ride to Cure Diabetes", which does over emphasize the cure part. If you click on "Research Progress", you see four sub areas, all of equal size: JDRF Research, Cure, Treat, and Prevent, so that seems pretty reasonable. I think if you look at the whole web page, you get a feel for all the things that JDRF funds. But if you only look at one slogan, yes, that one slogan might only talk about the cure.

Budget info: http://jdrf.org/about-jdrf/fact-sheets/research-funding-facts/

--Joshua

Joshua Levy said...

This is my reply to GabLarConBriCar (her quotes start with a dash "-"). Each of her key points gets a separate comment from me:

- their "partnership" with the industry is at least questionable in its logic. In summary, charity funding goes to big pharmas to develop ways to sell more insulin (read artificial pancreas, smart-inteligent insulin, even basic science! and others).

Certainly some people consider big pharma to be the evil enemy. If you are one of those people, then giving even one cent to big pharma is too much! My view is simple: anything to cure type-1 diabetes. If that means funding a university researcher, that's fine. If that means funding a small start up, that's fine. Same for a big company. I think it's very important that you fund companies differently than university researchers! You need a lot more controls, and contracts, and so on. But that is exactly what JDRF does.

I have previously blogged about JDRF partnering with "big pharma" specifically for AP research here:
http://cureresearch4type1diabetes.blogspot.com/2010/01/why-fund-j-and-j-animas-dexcom-close.html
In that blog posting, you can see that I'm nervous about it, but I do think it can be done well, and I do see the benefits of doing it (if done carefully).

Also, you seem to think that "smart insulin" is just another way of selling insulin. I disagree completely! I think smart insulin could easily be a functional cure (or within a hair's breath of one). Take one injection a day, or maybe one a week, and no carb counting, no blood checks, no long term complications, no middle of the night lows, etc. I'm sure you would claim that because you need to take an injection once a day, it's not a cure, but is it really all that different? As for AP some will consider that cure, and some will not. (Although if it leads to an implanted device, then more people will consider it a cure.) But even if not a cure, it will have a huge impact on long term complications. In a sense, that is how JDRF thinks of it, it is funded out of their "treatment" section, not the "cure" section. Finally, I don't think JDRF funds basic research at large pharma companies: were you thinking of something specific, when you wrote that?

--Joshua

Joshua Levy said...

This is my reply to GabLarConBriCar (her quotes start with a dash "-"). Each of her key points gets a separate comment from me:

- corporate governance standards are way deficient. Details in http://www.thejdca.org/wp-content/uploads/2013/08/Corporate-Governance-STAR-Review.pdf

Did you even read that JDCA report that you linked to? It compared four diabetes charities and found that JDRF was the best one for governance! Of course, it pointed out many ways that JDRF could get better. And I'm sure it can improve. But in the meantime, it did the best of the four compared.

JDRF also does well on other measures of governance. Charity Navigator has a specific "Accountability & Transparency" section, and JDRF got 4 stars there. (So did the other major charities: ADA, DRI, and Joslin).

--Joshua

GabLarConBriCar said...

Dear Joshua, thanks for your individual answer to each one of the three items I had commented upon. Here are my contributions to your points

1- on how much JDRF spends on cure, your numbers are way different than what JDCA reports. Of course I have no way to audit who is right. Please see page 4 of the following report, where the JDCA clearly indicates that out of the 205 mln spending, the JDRF spends 28% on cure research (25% idealized + 3% practical).

http://www.thejdca.org/wp-content/uploads/2013/05/How-Much-Practical-Cure-Research-Do-the-Non-Profits-Fund.pdf

2- No, I don't consider big pharma the evil enemy. I consider it immoral that funding raised by a charity for cure purposes is given to a profit oriented company for basic science or to develop products to sell more insulin.

3- Corporate Governance: yes, I did read the report. The JDRF rating is 2 out of 5 overall, which I do not consider brilliant at all. In the specific field of accountability, it got 1 out of 5.

Joshua Levy said...

GabLarConBriCar: About your point 2 above, maybe you should give an example of JDRF paying for basic research at a for-profit company. You've complained about it twice, but does it really happen at all? (I know that JDRF does fee-for-research contracts, but that is different. JDRF is not giving away money in those cases, it's paying for a specific service of some kind, and owns the results. It's like JDRF paying for PR services or payroll services, etc.)

As for selling more insulin, as I've said before, I think that "smart insulin" type products are a potential path to a cure, so proper for JDRF to fund. You many differ, of course.

Joshua

Joshua Levy said...
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Joshua Levy said...

One more thing: I'm not convinced that it is always immoral for a non-profit to give money to a for-profit. Non-profits have specific goals. If the best way for a non-profit to achieve their goal is to give money to a for-profit company, then why not?

Many non-profits find it is better to outsource some of their activities to for-profit companies. It makes sense to me to use capitalism for those things it is best for, while non-profits do those things that capitalism does not do well.

Joshua

jazs07 said...

@GabLarConBriCar Joshua is right it is not immoral for private own companies to donate or fund a clinical research, who cares if it will benefit the company well we can also benefit from such studies. Recruiting research volunteers is quite hard these days for some reasons, some take part if there is money involve.

GabLarConBriCar said...

Josh, thanks for this productive exchange. I don't think it is even debatable that JDRF's funding of basic research takes place, as the JDRF itself clearly states this.

Please see a specific example of charity money going to fund a 45 bln market cap company here:

http://ezinearticles.com/?Cure-for-Type-1-Diabetes-Is-One-Step-Closer&id=6821710

and a Facebook discussion about it here

https://www.facebook.com/permalink.php?story_fbid=310947548961980&id=161403643908447

I also refer to my comment on Dr. Gerry Fishbones generic defence of JDRF's partnership with the industry.

https://www.facebook.com/notes/dr-gerry-fishbones-blog/another-frequently-asked-question-of-jdrf/358419790861969

GabLarConBriCar said...

Dear jazs07

Please note I never said it is immoral "for private companies to donate". I said it is immoral for a CHARITY to donate its money to a for-profit company who is in the business of selling insulin. By the way, I find it naive from JDRF's standpoint and totally immoral from the Recipient's standpoint. How can Eli Lilly even accept JDRF money? That is what I am referring to.

GabLarConBriCar said...

Joshua, I do understand and in see your "one more thing" comment.

Of course, if the non-profit contribution to a for-profit results in a cure, I'd be very happy. So, from an abstract and highly theoretical standpoint, you are absolutely right. The thing is, in this case, we are talking about the for-profit who is actually in the very lucrative business of treating the disease "as is", meaning cronic and forever-dependant on it.

I would absolutely agree with JDRF funding a cure-oriented for-profit underfunded company (e.g. run by a brilliant scientist with great ideas but no money, helping him/her not to depend exclusively on private equity, debt and other hard to find alternatives), not a treatment-oriented rich company.

prerna said...
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