In last week’s interview segment, Dr. Denise Faustman talked about the basics of her research. This week, in the conclusion of her interview, Dr. Faustman and Diabetes Self-Management‘s Quinn Phillips discuss the controversy surrounding her work and its funding.
Quinn Phillips: Are you surprised — or disappointed — that there is no funding of your research by the National Institutes of Health (NIH) or a similar entity?
Denise Faustman: Well, remember that most drugs in the United States are developed by industry. So what usually happens is people invent in academia, but then those inventions are taken to industry, and after that — only when there’s a finished, polished drug — they come back to academia to be studied in patients. So usually when doctors get recruited to enroll their patients in a drug trial, they don’t have to raise the money — they’re given the money by the drug company. This is a very different project, because this is not a drug owned by a pharmaceutical company; this is a drug that is manufactured by 40 different companies around the world, and it’s readily available. We were pretty naive at the beginning, going to the industry and saying, “Hey, you want to support this drug? What? Money? It reverses disease!” It became pretty obvious that you have to have an economic model for industry to partner with you — you have to have something that’s unique that they can make their money from. And if you don’t have that, then industry has no interest in moving it forward. It’s just the brutal truth, you know. A company can’t have a loss of a billion dollars and not have anything to sell.
NIH money, historically, isn’t available for Phase I trials such as ours, and often a lot of NIH money is paired with industry money so that they get a bigger bang for their buck. NIH does a lot of these industry trials. So we don’t like to think about good guys and bad guys, but the real good guys here are people who donate to this program directly to see it move forward. And we aren’t hampered by the typical things that hamper clinical trials, like someone saying, “You have to use this drug, but guess what — you can’t study these patients in this way or that.”
QP: Using NIH money — taxpayer money — to develop a drug from which a company will reap enormous profits seems like corporate welfare to a lot of people. Do you think there are valid reasons for this system?
DF: I think that when you can leverage studies — let’s say you’re a government employee and you have $20 million, and someone else comes in and says, “I’ll give you $40 million, and we’ll do it together” — it looks good. And that’s the typical way drugs are developed. So when somebody comes along with the exception and says, “Hey, I’ve got this cheap thing,” it’s kind of a new paradigm — we’re breaking new ground. And also, a lot of the government programs are set up for only new-onset diabetics. That’s where all the for-profit drugs are being developed. So half the money for clinical trials we can’t even apply for, because we’re actually trying to reverse diabetes in people who have it. We’re excluded.
QP: Do you see that changing?
DF: I think once we start getting real data, things will change. But nothing in the government moves fast. We actually hope that things change in a broader sense, that people should be taking on the bigger conceptual projects and not just trying to give a new-onset diabetic a small difference in pancreas function for a week or a month. They should really be tackling the tough scientific questions of what to do for people who have the disease, other than new glucose meters, etc. So if anything, we hope our work sets a new paradigm that people with the disease should not be left out of innovative therapy.
QP: Given that you rely on philanthropic support, were you surprised by the JDRF’s reaction to your work [circulating a very critical e-mail, funding replication of the mouse study by other scientists, and then denying funds for human trials]? [Read more about the JDRF and Dr. Faustman here.]
DF: Yeah. I mean, a lot of foundations — and the JDRF is not alone — sponsor for-profit clinical trials, and they take an equity position in them to help raise money in tight times. So we were shocked by that, but we got back on our feet and are raising money. So in that sense it was disappointing, but we’ve moved on from there, and I think the public recognizes the validity of trying to do these types of trials in people who have the disease. It’s not a new glucose pump, meter, etc., but actually trying to restore function long-term in a pancreas. So we’ve been very fortunate regarding the public’s support.
QP: Is it useful to fund duplication of experiments, as the JDRF did with yours?
DF: Sure, all science needs to be duplicated. What you would hope they would do, of course — instead of pouring $5 million into the duplication — would be to then fund the trials. That’s where they stopped, but we kept moving. Disappointing, but we’re moving on.
QP: Do you have anything to say about the drug Gleevec, which was mentioned in a recent press release of yours? [Gleevec has been used to treat diabetic mice in industry-funded studies — read more about it in the blog post “Cancer Drugs Reverse Type 1 Diabetes in Mice”.]
DF: The studies are still at a little bit earlier stage. What people do when they evaluate these studies — when mouse doctors evaluate these studies — we look at three things: Does it work in a prediabetic animal? Does it work in a recent-onset diabetic animal? And does it work in a severely diabetic animal? Those studies show that it works in prediabetic animals and animals with just one blood sugar reading of 250 mg/dl. The data is not out there yet on whether it works on fully diabetic or long-term diabetic animals.
QP: Do you worry that because of the profit motivation for Gleevec, their research will catch up with yours?
DF: Well, it will be sponsored by the drug company, so it will fit the very common paradigm of drug development in the U.S. — that the company that owns that drug is going to develop it and pour money into it. If we thought we might lose money from that company, then we’d be worried — but we’re not getting money from that company now, so it’s OK. And I’m also of the thought that you want every approach moving forward as quickly and aggressively as possible, because we’re not at the golden gates yet with everything working — barely anything is working! So you’d better hope a lot of different approaches are moving forward.
QP: When will your Phase I human trials be over?
DF: About  months from now. So that’s really fast — most Phase I trials take five years or so; we’re going to be about two-and-a-half years.
QP: Is there anything else you’d like to tell the readers of Diabetes Self-Management?
DF: We’re working to reverse disease in people who already have it; that’s kind of unique. And we’re working with a generic drug that has the most impeccable safety record of any vaccine in the history of the world. So we can move pretty fast. And the project is totally supported by the p
ublic — it’s philan
QP: Are you still soliciting donations?
DF: Oh yes, we’ve got to raise money for Phase II! And if people want to register on our Web site for an interest in participating in the trial — obviously we have a lot of people interested — we take all their information down.
For more information about Dr. Faustman’s research, visit www.faustmanlab.org