Allen Spiegel, M.D., is an internationally recognized endocrinologist whose research has helped define the genetic basis of several endocrine diseases. A former director of the National Institute of Diabetes and Digestive and Kidney Diseases, Spiegel currently is dean of the Albert Einstein College of Medicine of Yeshiva University.
In 2003, he shared his thoughts about the importance of investing in basic research and the need for greater collaboration – among universities, the government, private industry and the general public – to address the challenges of diabetes.
Lens: What are some of the major priorities of the Institute?
Spiegel: Because of its enormous public health significance, diabetes is clearly one of our major priorities. For example, we’re investing substantial resources in the Beta Cell Biology Consortium (BCBC), because insulin-secreting beta cells are at the heart of both types 1 and 2 diabetes. The BCBC is a recently formed group that typifies a newer approach to biology and medical research. We still rely on the creativity of individual investigators, but to tackle some of the large issues, it’s really vital to bring together a number of investigators. That’s exactly what we’re trying to do.
The goal of the consortium, coupled with other efforts, is nothing less than to understand everything there is to know about the beta cell, every gene that is expressed. It is possible to do this now, at every stage of development; to identify stem cells in the pancreas; to work with embryonic stem cells, according to the President’s guidelines; to determine what is necessary to create a sufficient quantity of beta cells and islets needed to replace beta cell function in animal models of diabetes and eventually in patients.
Within the funding levels provided, the NIDDK will continue to support a scientifically robust research portfolio that addresses all the diseases within its mission. This portfolio includes fundamental laboratory research; “discovery” research that brings new insights from the laboratory into clinical testing; clinical research of promising treatment and prevention strategies; and the translation of important results from clinical trials, not only in the scientific literature, but also through public outreach, information and education efforts.
Lens: What will be needed to achieve success? What are the barriers?
Spiegel: There is cultural change needed at the level of the university and academic medical center in order to foster multi-disciplinary investigations. It is increasingly important to bring in the mathematical and physical sciences, computational biology. The sheer volume of data we are inundated with from advances in genomics and proteomics, for example, mandates that biology take on more of a ‘math’ kind of tone.
Equally important is the need to break down some barriers between universities, and allow investigators to come together so they can apply their talents. The current system of promotion tends to discourage this. If you are part of a collaborative effort, it’s hard to identify the individual contributions upon which, historically, decisions of tenure have been made.
Lens: How important is NIH funding in diabetes research?
Spiegel: Extremely important; relative stability in funding is particularly important. In the early 1990s, the funding of the NIH hit a real low. Under those circumstances, sadly, the success rate of applicants for research grants began to plummet. It had a ripple effect, discouraging individuals from going into science.
In the past three years, NIH-wide funding of diabetes research has increased substantially, from $688 million in the 2000-2001 fiscal year, to an estimated $946 million in the coming fiscal year (2003-2004).
One of the powerful messages that needs to be disseminated is: You can’t go from feast to famine. Investment in medical research is something that pays off, not only in terms of improving quality of life and preventing mortality, but economically. The entire biotechnology industry was built on the fundamental investment of NIH resources.
Lens: How important is it for universities and the government to collaborate with industry? What are the barriers to successful collaboration?
Spiegel: This is a vital partnership. A prime example is recombinant DNA technology, which largely came out of curiosity-driven research about restriction enzymes. Through the private sector, it was converted into an enormously successful enterprise that is delivering new, innovative treatments.
But this partnership with industry has to be assiduously framed and monitored, not only to guarantee appropriateness and safety in clinical trials, but also to ensure the sharing of resources, data and research findings. In recent years, universities have promulgated some very clear guidelines regarding the need to publish and how to address real or perceived conflicts of interest, both as individuals and institutions.
Lens: What are some other challenges to translating research findings into benefits for patients?
Spiegel: One translational block is in the dissemination of information. It’s well established, for example, that certain drugs called ACE inhibitors are unequivocally effective in halting the progression of diabetic kidney disease. Why aren’t we applying this knowledge far and wide?
We just launched the National Kidney Disease Education Program, which in its pilot phase is targeting four cities – Atlanta, Ga., Baltimore, Md., Cleveland, Ohio, and Jackson, Miss. – which have a disproportionately high level of end-stage kidney disease, mostly among African Americans. If we can get the information out about the importance of measuring protein in the urine and using ACE inhibitors where indicated, for example, we can really make an impact.
The use of information technology also can be very important. One really needs to look at chronic diseases through a different management model, one that focuses on flagging reminders, making sure people are monitored. This increases cost in the short run, but it can make a huge difference in the long run in terms of reduced hospitalization.
Lens: What about prevention?
Preventing disease is a high priority, and much of our research is aimed at understanding how better to prevent chronic and costly diseases such as diabetes. If we don’t do this, we’re really in for trouble simply because the health care system is not going to be able to sustain itself, faced with a diabetes epidemic and its attendant complications – amputations, blindness, kidney failure and cardiovascular mortality.
Our Diabetes Prevention Program (DPP) showed clearly that type 2 diabetes can be prevented or at least delayed in the majority population as well as all ethnic and minority groups, including African Americans, Hispanic Americans, American Indians and Asian American/Pacific Islanders who are at highly disproportionate risk. We are now building on the DPP results to develop cost-effective ways to prevent type 2 diabetes in adults and in adolescents, who increasingly are developing the disease.
With regard to obesity, we need research that will address ways to interfere with inappropriate cues and signals that lead to overeating. Behavioral efforts have been successful. That is the powerful message of the DPP. We’re not talking about running a marathon here. We’re saying that, for a group of individuals we can identify who are at particularly high risk, a targeted effort aimed at early lifestyle changes would be effective.
It becomes a matter of public policy, informed by serious research, that tells us whether having soft drinks in vending machines and the absence of physical education in schools predisposes children to obesity. We are launching a type 2 diabetes school-based prevention effort with appropriate rigorous controls that will inform the public policy debate.
But the public needs to participate. The public must ask its leaders to act on informed data. That’s the only way we can advance.