The Juvenile Diabetes Research Foundation (JDRF) has never been an organization to shy away from controversy. At last Tuesday's Bay Area Annual Research Meeting, Senior VP of Research and Development Dr. Paul Burn explained in detail the change in direction that the JDRF has pursued in the last year. Once committed exclusively to funding research for a cure for type 1 diabetes, the organization has stepped up funding for continuous glucose sensing and the artificial pancreas project.
Dr. Burn described JDRF now as a “therapeutics development organization.” In discussing the rationale for the shift, he said that JDRF was not satisfied with the rate of progress on the search for a cure. While for many years basic scientists were receiving grants and doing compelling research, their research did not often translate into products that could help people with diabetes in their daily lives. Curative research is a “long, costly, and high-risk process,” and most of the projects funded by the JDRF are still in an early stage. In shifting emphasis to the artificial pancreas (AP), the JDRF aims to bring concrete results to parents and children sooner.
To accomplish this, the JDRF is moving quickly on several new fronts. The organization is collaborating with industry and spurring technological innovation with milestone-driven, contract-based programs. It is also using corporate dollar matching programs to maximize its financial effectiveness. This year the organization has launched programs based on a proactive pursuit of top scientists; invitations to apply for grants are issued to scientists based on reviews of their work in leading scientific journals.
Most encouragingly, JDRF’s advocacy and lobbying efforts are primarily targeting the regulatory and reimbursement obstacles that can be so challenging. In bringing the AP to more people sooner and encouraging companies to innovate in this field, reimbursement is and will continue to be a critical issue. Dr. Tim Goodnow of Abbott Diabetes Care, who presented at the research meeting, said that the FDA’s willingness to work with Abbott on the regulatory side has been excellent and the whole process has been very positively influenced by the JDRF. In this past year alone the JDRF Government Affairs Branch had more than 20 meetings with legislators in Washington, D.C. This year, we have been very impressed at how much the JDRF has done to further the availability AND the accessibility of continuous monitoring.
Engaging JDRF member support on this major strategy change will be critical. At Tuesday’s meeting Executive Director Vicki Weiland announced that the Bay Area Chapter had raised over $7 million this year; the chapter aims to raise $50 million by 2009 to contribute to the global campaign goal of $1 billion raised by 2009.
Dr. Bruce Buckingham of Stanford presented on the current state of closed loop systems. He showed data suggesting that closed loop systems at present—which are sometimes criticized for failing to eliminate post-prandial hyperglycemia—still achieve results superior to manual self-management. In an algorithm study at Yale of 10 adults for 28 hours, the closed loop nighttime glycemic control was stellar. The days were less perfect, but with the closed loop system only 17% of the values were greater than 180 mg/dL, versus approximately 34% with continuous glucose monitoring combined with self-administrated boluses. Dr. Goodnow also cited a 21-day study with the Navigator system that showed that patients testing nine times per day still spent one-third of their time—approximately eight hours—above 180 mg/dL.
The best combination available to date is the “hybrid closed loop with bolus.” Dr. Buckingham presented information from a study of a closed-loop system in which users administered a small bolus prior to eating. Patients using this system had the tightest glycemic control, with mean blood glucose 135 mg/dL. Nocturnal mean was 114 mg/dL and peak post-prandial was 191 mg/dL.
Dr. Buckingham also emphasized that the closed loop system, apart from its effects on glycemic control, will alleviate the extremely high burden of care that children and parents must assume in dealing with type 1 diabetes. He said that it is difficult to accurately count carbohydrates and children often miss boluses. One study showed that two missed boluses per week raised A1c by 0.5%.
While progress on the AP is exciting, there are still a number of barriers. We believe the keys to a very successful AP will be spot-on algorithms, a very rapid insulin, and the possible integration of other hormones, such as glucagon or amylin. That said, the studies cited by Dr. Buckingham suggest that more near-term versions of the AP can still be useful for people with diabetes as we make incremental progress toward an ideal AP.
Accelerating the progress on continuous glucose sensing and the new work on the AP is compelling—diversification of strategy can only be good. Now the JDRF’s proverbial eggs are no longer in a single basket, as it is funding both shorter- and longer-term solutions for people with diabetes. That said, we don’t think that curative research needs to be dismissed to justify investment in therapeutic device-driven solutions. Some of the comments made at the meeting in support of the AP projects were overly negative regarding the prospects of curative research. We don’t see work toward a cure and work toward an AP as at all incompatible. While an AP would vastly, vastly improve the lives of people with diabetes, it would clearly be second-best to a cure, partly because it would still entail wearing and paying for medical devices. We applaud the JDRF’s “Six Goal” strategy, which focuses efforts on islet transplantation, stem cell research, beta cell regeneration, prevention of type 1, and prevention and reversal of complications as well as the AP. This strategy certainly covers a lot of the necessary bases.
Founded only 36 years ago by a group of parents of children with type 1 diabetes, the JDRF has already made huge strides as a fundraising powerhouse and an advocacy group for people with type 1 diabetes. In recent years the political clout of the JDRF has grown considerably—and that’s certainly great news as far as we’re concerned. The results of JDRF-funded work on the AP have already manifested in some of the newer devices coming to market this year. We look forward to seeing the lab research on closed-loop algorithms translated to real world solutions. When that happens, patients will be able to worry less and feel better due to the reduced incidence of hypoglycemia and hyperglycemia.