Editor’s Note: As Breakthrough T1D recognizes its 50th anniversary, we are highlighting stories that showcase the Power of Us: the people, partners, and events that have propelled us forward.
Below, a story from the Winter 1998 Research Update issue of “Countdown,” Breakthrough T1D’s now-discontinued magazine. The story focuses on a 1997 initiative that would help pave the way for Breakthrough T1D’s future approach to identifying research priorities.
The original, written by Wayne L. Clark, is a historical account and reflective of the year in which it was published. At that time, Breakthrough T1D was known as JDRI. The story does not include information about or reflect Breakthrough T1D’s current research priorities.
Explore the full spectrum of Breakthrough T1D’s current research priorities on BreakthroughT1D.org.
For blog-publishing purposes, the original copy from the 1998 story has been shortened.
Diabetes is complicated. The search for a cure has led to many scientists pursuing many different paths. In a world of limited resources, how do you make certain that those paths produce results quickly?
A research task force at the Breakthrough T1D is addressing that question and, in the process, leading a fundamental change in the way diabetes research is funded.
Breakthrough T1D is in the position to influence how research into the disease progresses: The Foundation provides more money for diabetes research than any other private, non-profit agency in the world. And its research task force is turning “business as usual” into a new way of looking at the world of research and focusing on the search for a cure.
A New Strategy
Breakthrough T1D will no longer wait to receive applications for research grants. Instead, gaps in scientific knowledge will be identified, and Breakthrough T1D will actively encourage applications from researchers working in those fields.
The same process that determines the funding strategy will be used to evaluate results, make 
adjustments in the strategy and continue to ensure that the very best research in Breakthrough T1D’s areas of interest will be funded.
“This approach is unique,” says Robert A. Goldstein, M.D., Ph.D., M.B.A., vice president of research at Breakthrough T1D. “This is not the typical way science is driven in most funding agencies. It is an effort to direct research, to some extent—to be more goal oriented, while at the same time preserving the scientific process and the high quality of the research.
Dr. Goldstein is co-chair of the task force, which is charged with determining the direction of research funding. Its major tool is the “map,” a deceptively simple looking document that is the result of some of the brightest minds in the scientific community pooling their collective knowledge. It organizes what is known about diabetes and charts various paths to a cure.
The ”Mapping” Concept
The mapping process has its roots, as is typical for Breakthrough T1D, in the passion of its volunteers. The driving force behind this innovation is Emily Spitzer, chairman of research, a parent of a child with diabetes, and a longtime volunteer who has served on the Breakthrough T1D lay review committee for many years.
“I had long been troubled that there were two different forces at work,” Spitzer says. “We were funding the best research, but we never said what we were interested in. It was reactive; we were taking the best of what researchers were presenting to us. …I realized we had to drive the agenda more than we were, to direct research toward making a difference for people with diabetes.”
Spitzer compares the process to the goal-directed research of a pharmaceutical company. Define the goal, assemble the knowledge you have about the area and determine the fastest way to reach the goal.
The mapping process takes into account who else is spending money on the area, how promising one line of research is relative to another and what the likely time frame is for success. It’s a blending of scientific judgement with the mission that drives Breakthrough T1D to find a cure, and to find it as soon as possible.
What “Cure” Will Mean
“Cure” has three broad definitions for the task force: prevention of diabetes, restoration of normal blood glucose metabolism in those who have the disease, and treatment of complications. A series of maps have been developed for each general area, to organize and depict the research that has been done and needs to be done.
They are graphic depictions of everything that is known about or suspected about the question at hand.
The next level of mapping looks at approaches for a cure. In looking at the loss of normal glucose metabolism following beta cell destruction, for instance, the map shows three alternatives: replacing the beta cells (as with a transplant), replacing their function (as with insulin injection) or finding another way to regulate glucose metabolism. The task force will determine which paths within each alternative shows the most promise.
The process is an endless, ever-deeper and more specific series of questions and answers.
Each level of questioning draws a better map of where Breakthrough T1D can have the greatest impact. It helps answer the final and most fundamental question: Where should we invest our research funds?
Dr. Goldstein says, “…Let’s say there are half a dozen pathways for a particular problem. Using the map, we can make a scientific judgement that one might be a little more productive than another. Or that one will work, but it’s going to take ten years.”
“We’re also able to use this process to make a judgment about resource allocation. For example, on this particular path, how much do the National Institutes of Health spend on it, and how much does industry spend?”
Into “Meaningful Parts”
The challenge of assembling the maps fell to Sandra Puczynski, Ph.D., a clinical research educator at the Medical College of Ohio. A Breakthrough T1D volunteer and the mother of a child with diabetes, Dr. Puczynski took on the task of bringing about consensus on what research directions make the most sense.
“This is a complicated disease, and the search for a cure crosses many boundaries, overlapping areas such as genetics and immunology, transplantation and prevention,” she says. “We need to determine what has been done, what needs to be done, where the greatest impact can be made, how much it will cost and who will fund it. In developing the maps, I worked with the staff at the National Institutes of Health, interviewed most of the members of the Breakthrough T1D Scientific Advisory Board about their specific areas of expertise, met with more than one hundred members of our Medical Science Review Committee, talked to representatives from pharmaceutical and biotechnology firms and searched the literature.”
Once the maps were drafted, the Breakthrough T1D research task force reviewed them. The task force consists of experts both within and outside diabetes and drawn from industry, biotechnology, the National Institutes of Health and academia. Management consulting firm McKinsey & Company helped pull the views together.
“The task force’s role is to look at the maps, identify gaps, and locate new opportunities,” Spitzer says. “With the help of experts from the McKinsey group, we have been able to bring our divergent views together, agree on maps and decide how to go forward. We can now direct research in an intelligent way.”
“The McKinsey group brought to the process a business marketing approach to science,” Dr. Puczynski says. “They have some scientific expertise, and they helped us focus on end points and set goals, looking at their impact in terms of different parameters. It is a strategic planning approach, one that can be difficult for those of us who think in scientific terms and understand that no process can capture all the information.”
“Will this Help Our Children?”
The quality of science must remain at Breakthrough T1D’s traditionally high standards, however. “We’ve never wanted to do away with peer review,” Spitzer says. “We only want to fund great science. But in the universe of great science, we only want to fund those things that are going to lead to a type 1 diabetes cure.
A significant benefit of the mapping process is that it lends itself to evaluation. If Breakthrough T1D decides to fund a particular path of research, it can revisit the map later on and determine if it got where we wanted it to go: whether the money was spent wisely, the results worthwhile, a measurable success. If not, the focus can be changed or a different path chosen.
Dr. Goldstein says, “…The question when we look at a research strategy has to be: Is this going to help our children? Is it going to help them next week, next year, or next decade?”
Dr. Goldstein and Spitzer are encouraging groups to submit applications to pursue their work and streamlining the review process. The message is going out that Breakthrough T1D is interested in applications not just from universities, but from companies and others.
Spitzer remembers when her daughter asked her if the work she was doing for the Breakthrough T1D would make her life better. The poignancy of that question and the importance of the answer being “yes” continues to motivate Spitzer to push for answers.
“I don’t have an intellectual interest in research,” Spitzer says. “Over the years, though, I’ve developed an appreciation for an understanding of that intellectual drive. What I ask of researchers is that they develop an appreciation for my interest, which is to move results to the patient. Through this process, we’ve brought our differing to the common goal of finding a cure.”
Learn more about Breakthrough T1D’s current research priorities.
