Thursday, August 02, 2007

Show Me The Money!

August 1 marked the start of JDRF's "Promise to Remember Me" campaign. A major objective of the campaign this year is to convince the Federal Government to renew the Special Statutory Funding Program for Type 1 Diabetes Research, which is set to expire in 2008. President George W. Bush signed the last extension of that program (in 2000 or 2001, I can't recall), so its possible he will also approve it this year if the legislation is sent to the White House for approval. What's at stake is more than one-third (35% to be exact) of all Federal funding allocated to type 1 diabetes research which comes from that "special" program. (The remainder comes from the annually appropriated funds to the NIH to support type 1 diabetes, and a tiny, highly targeted funding program also exists within the Department of Defense to fund the development of remote sensing technology, which is seen as having application not only for diabetes management, but also for the U.S. military.)

Some have estimated that JDRF funds as much as 25%-30% of all research conducted for type 1 diabetes (excluding pharmaceutical research, which has been minimal for type 1). Unlike type 2 diabetes which receives almost all of its research funding from the Federal Government (again, excluding pharmaceutical research, which is substantial), type 1 diabetes is unique in that such a large portion of the research done is actually financed by the people who live with the disease itself. That speaks volumes about the determination of the individuals and families who live with this condition and to bring their disease to an end, and it also enables them to have a much larger say in what the research priorities should be for their disease since they are raising the funds to pay for it. But the other side of that equation is that to some extent, it has also enabled the National Institutes of Health to shift focus more towards the more common form of diabetes (type 2), which is why type 1 diabetes is the only major disease to obtain Federal funding in this unique manner.

As I reviewed the e-mails I've received from JDRF over the past week or so, I began thinking about the organization's research priorities. Former CEO Peter Van Etten really pushed stem cell research, and his reasoning was that if scientists could culture an unending supply of beta cells in laboratories, more patients could receive transplants and be cured. He also reasoned that they could develop better ways of transplanting islets which would enable them to be done without immunosuppression. Some did not agree with his views, but no one could say he did not work to set priorities and strategies for JDRF.

However, I have struggled to put my finger on JDRF's new(er) CEO Arnold W. Donald and what exactly his contribution has been. I was initially unimpressed, and quite frankly, the guy rubbed me the wrong way. But after a more thorough examination, I do see some signs of progress. Notably, since he has taken the reigns, one of his primary objectives has been for JDRF's role to be helping to fill gaps in the drug and therapeutic development process, and in that regard, he has indeed shown significant progress and should be congratulated. The most visible example is the "artificial pancreas" concept, and while thats a visible manifestation, the article published in a February 2007 edition of The Wall Street Journal (see here for details, and here for the article itself) suggests that JDRF has pushed into less visible areas as well. This is an important new direction for the organization, and I think a good one.

For example, in the area of autoimmunity, JDRF has partnered with TolerRX in Massachussetts, and MacroGenics, a Maryland-based company to fund the first Phase III trial JDRF has ever sponsored. Phase III trials are the last stage before applying to the FDA for approval to market a product, and the idea is that by helping at this stage, some of these efforts may be commercialized which would otherwise never have made it that far due to a lack of funding. The trial is testing an anti-CD3 antibody in patients who have recently been diagnosed with type 1 diabetes. The research these Phase III trials are building on showed that this antibody could preserve beta cell function in newly diagnosed patients for up to 18 months after diagnosis. His reasoning is that if JDRF can prevent diabetes from spreading in the newly diagnosed, then JDRF could also gain insight, and perhaps the ability to reverse it. These are important steps, but frankly, don't do much for the million or so people who live with this condition and work to fill JDRF's coffers each year.

But JDRF has not forgotten that. They did report progress in filling the gaps in getting treatments for complications farther along the product development pipeline. For example, several drugs aimed at alleviating diabetic eye disease are now in clinical trials. JDRF is also collaborating with Genentech to test a drug called Lucentis in a Phase II trial for diabetic macular edema, and JDRF is also planning a Phase II trial testing this drug against diabetic retinopathy. Lucentis has already been approved by the FDA for age-related macular edema, so the journey to the market for diabetic complications could be accelerated.

But gaps in treatment for type 1 diabetes remain, and continue to widen when compared to type 2 diabetes. There have been only a handful of improvements in treatment since insulin was discovered. And not everyone is enthusiastic about a so-called artifical pancreas. I'd like to see Mr. Donald move JDRF to step in to fill a number of important therapeutic gaps. One notable example, a development stage biopharmaceutical company based in Sweden called Creative Peptides who is engaged in the discovery and development of biopharmaceutical drugs to treat long-term complications of type 1 diabetes, and their lead drug (synthetic C-Peptide made from rDNA origin) development project is currently in phase II clinical trials. The company has secured U.S. and European patents for their work (see here for the U.S. Patent information). Started by a number of researchers from the esteemed Karolinska Institute in Stockholm, like all startups, the cost of trials is an impediment to faster progress. Ultimately, Creative Peptides may well succeed, but commercialization of their products will require a partnership with a large pharmaceutical company for manufacturing and product marketing expertise. That usually guarantees the larger partner marketing exclusivity for some period. But patients would likely benefit by having JDRF working to fill a gap and ensure that all manufacturers offer it.

My initial reaction to Arnold W. Donald was not positive (see my posting on that here), but after 18 months on the job, I must admit that I have indeed warmed to his big push for JDRF to "fill in the gaps". But in order for him to make JDRF a more donor-centric organization, I want to see more quantifiable progress and details reported on this regularly. To borrow a famous line from the movie Jerry McGuire, "Show me the money!"

3 comments:

Jenny said...

99.9% of the "research" about Type 2 diabetes is crap. Much of it is bogus studies run to increase off-abel prescribing of the inadequate and often dangerous oral drugs that keep people with Type 2 running an average A1c of 10%.

A lot more of it is still based on the discredited idea that eating fat causes obesity and that obesity causes diabetes.

And yet more of it is so poorly designed and so without controls or true statistical significance as to leave anyone with engineering training shaking their heads.

It all depends on where you're standing, I guess. To a lot of Type 2s it looks like the research going on for Type 1 is much more likely to translate into significant cures than does the ongoing production of inadequate oral drugs that has become ALL that Type 2s are offered.

BetterCell said...

I am happy to know that Creative Peptides is making incremental progress to get C-peptide on the Market.
As far as Federal Funding for Type 1 Diabetes is concerned, I am skeptical about $$ being earmarked for T1DM Research and Clinical Availability since most Politicians(as you already know) have a bias for IRD(aka Type 2 Diabetes) established. This may have to do with the tendency for many of these same Politicans who themselves succumb to IRD due to negative Lifestyle attributes, such as obesity.

Scott said...

Jenny,

I certainly agree on the crap research for diabetes research overall, including both type 2 and type 1. There is so much replication of the same tired mantra about control that overshadows the handful legitimate research. Also, in that regard, I do think that some of the research being done by JDRF could be very beneficial to the type 1 community. As I noted, the fact that such a large portion of the research is self-funded enables the collective community to determine what type of research actually gets done. In that regard, I do feel its a shame that the type 2 universe is not more actively involved in fundraising for the condition. There are a variety of reasons for that which I cannot begin to address here.

One thing I'd like to see (but suspect its unlikely) is for the NIH/NIDDK to have a more independent review board in much the same way as JDRF does, as it does help ensure that what the community deems to be junk research is not rubber-stamp approved.

Barry,

I agree that there is a political bias towards T2DM research, but the numbers speak for themselves. The issue I have is that much of the response has been knee-jerk reactions and stupid comments instead of a closer look at the causes behind the epidemic. The Farm Bill is a disgrace, and actually encourages farmers to grow commodities that we already have too much of (like corn) over fresh produce grown locally in many cases. The agribusiness has had to look for creative ways to use these commodities, thus high-fructose corn syrup was born, and appears in the typical scapegoats (soda, junk food), but also in things people don't often suspect, like canned produce for example just to name a few. Instead of paying farmers to grow more fresh produce, we end up with mountains of useless commodities that have a harmful impact as a result of the industry's creativity. Now, they are talking about ethanol, which might be useful but remains decades away from widespread deployment.

To add to this problem, look at a more local example. Consider that many low-income neighborhoods have a choice between liquor stores and maybe a fast food joint if they're in Harlen (the South Bronx may have a few, too), but the nearest supermarkets are subway ride away, making shopping a hassle for these residents, and then we blame them for becoming obese.

The good news on that front is that Tesco (the successful U.K. supermarket chain) will enter that niche with its Fresh & Easy Neighborhood Markets to appear initially in the West (they've targeted low-income areas in Los Angeles initially) but if successful, they could emerge in NYC in the not-too-distant future. The real question is why has the city made it so hard for Gristedes, Pathmark and others to operate in these areas? Shouldn't they be providing tax incentives for that? Instead, they get hassled by the zoning boards, high priced rents, and a barrage of permit requirements from a variety of city agencies, including the Department of Health and Mental Hygiene, yet its popular to people for unhealthy lifestyles. There are many sides to this issue, but most of what has been done has been only marginal changes where they need to be more substantial.