is becoming the standard treatment. Transplanting a whole pancreas often involves major surgery—which is significant in terms of potential risks. But even with such treatment, there are limitations: the number of donors is most of the time outweighed by the demand. Another constraint lies in the fact that transplants require the immune system to be suppressed in order to prevent rejection of the “foreign” organ. The use of immune-suppressing drugs can cause side effects to the recipient, and it can also leave him/her vulnerable to potential infection.
Significant progress in the treatment of type 2 diabetes includes the implementation of prevention initiatives aimed at developing new classes of medications to lower blood glucose and complement existing therapies. The current treatment strategy for type 2 diabetes continues to include traditional drugs focusing on β-cell failure and insulin resistance. It aims, additionally, at studying carefully the impact of such therapies on related comorbidities such as hypertension, obesity, and hypercoagulability.
Government Steps in the Implementation of Clinical Trials: Perspective
Diabetes is a lifelong condition and the number of children being diagnosed with type 1 diabetes is constantly growing. Although the symptoms can today be controlled, there is no cure. For many people, diabetes signifies long-term health issues and daily injections of insulin. How might clinical trials help? How might stem cells benefit patients? The landscape of novel therapies to advance treatment for people with diabetes is evolving.
Judith Fradkin, director of the Division of Diabetes, Endocrinology, and Metabolic Diseases at the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), discussed, not long ago, the NIDDK’s research initiatives of setting up and implementing clinical trials for diabetes that would be sponsored by the government and that are “not currently pursued by drug companies.”
One of the successes of the National Institutes of Health (NIH)/NIDDK multicenter trials pointed to by Fradkin, is the success in recruiting ethnically heterogeneous populations. The government-sponsored initiative includes about 45 percent of minority groups. The study looked at the incidence rates of diabetes based on three factors: the use of metformin, the presence of a placebo, and lifestyle intervention. The benefits of metformin and lifestyle interventions in the reduction of the incidence of type 2 diabetes were clear across all of the ethnic groups under study. This fact alone proved to be key, as it indicates that type 2 diabetes affects minority populations in a disproportionate manner compared to other racial groups.
An additional positive outcome that resulted from this study has been that, in addition to the inclusion of diverse ethnic groups, trials sponsored by the government have succeeded in classifying diabetic patients by phenotype. Such an unexpected finding has had important impact on the development of type 1 diabetes trials. The study also succeeded in creating some phenotyping following pharmacogenomics analyses. The results revealed that even for people with higher genetic risk for type 2 diabetes, lifestyle interventions may succeed in reducing their level of risk. This was described by the community of scientists as “a powerful result” indicating that genetics is far from being synonymous with destiny when it comes to developing type 2 diabetes.
NIDDK: A New Model for Clinical Trial Collaboration
Given the prevalence of diabetes in older adults and its constant increase in children, the expanded time course of the disease, and the costs associated with it, Fradkin believes that “diabetes is a good candidate for collaboration between NIH and the Centers for Medicare and Medicaid Services (CMS). Very few of the practices Medicare pays for have been rigorously examined in [randomized clinical trials] RCTs. Given NIDDK’s track record in conducting paradigm-shifting diabetes trials in diverse populations, conducting clinical trials in the Medicare population could offer an opportunity for cost savings.” Such collaboration would work in that NIH/NIDDK and CMS would share the costs related to research and clinical care, opening a new era of collaboration that could potentially benefit diabetic patients.
TrialNet: A Network Strategy for Type 1 Diabetes Trials
Another new venture that could be beneficial for diabetic patients in the long term is the establishment of TrialNet. Funded jointly by the Juvenile Diabetes Research Foundation International (JDRF), the NIH, and the American Diabetes Association (ADA), and approved by Congress, TrialNet is an international network of researchers and scientists whose objective is to explore new ways to delay, prevent, and potentially reverse the progression of type 1 diabetes. TrialNet researchers come from 18 clinical centers in Canada, the United Kingdom, Finland, Italy, New Zealand, Germany, and the United States, and more than 150 medical centers and physician offices.
New Treatment Pushed Forward by the Pharmaceutical Industry
While government initiatives for the fight against diabetes are emerging and flourishing, America’s biopharmaceutical research companies also continue to explore various strategies to combat diabetes and its related conditions. Some potential new treatments from the 180 existing medications in development today include enhancing the regeneration of insulin-producing cells (islets) and stimulating the formation of cells capable of generating insulin, a potential state-of-the-art treatment for type 1 diabetes. The treatment would consist of a human peptide made up of the bioactive part of a gene responsible for regenerating pancreatic islets.
Leaps Forward on the Research Horizon
The potential development of an effective vaccine or the use of stem cells to grow beta cells capable of making insulin are part of an array of promising lines of research intended to advance diabetes treatment. A number of novel devices are currently under way to better control blood sugar levels. In terms of vaccines, British scientists reported the existence of a well-known virus that could trigger type 1 diabetes by attacking insulin-making beta cells and triggering an infection. The underlying reasoning is that, if such process proves to be effective, it is highly possible that the individual’s immune response may not be able, in the end, to produce insulin. Based on this assumption, a vaccine with the ability to block infection by a virus might also be able to prevent diabetes from developing. In the United States, some Harvard University researchers have suggested that a widely used tuberculosis vaccine could treat type 1 diabetes by acting on the person’s immune response to kill beta cells. In current studies, type 1 diabetic mice started to produce insulin following treatment with the vaccine.
As for glucagon, which is produced in the pancreas and released when blood sugar levels are too low, a recent study stated that “suppressing the action of glucagon made insulin unnecessary to control blood sugar levels.” These results could suggest that if the action of glucagon is blocked, normal glucose tolerance might be restored.
Morenike Trenou
Independent Scholar
See Also: Adult Stem Cells: Overview; Clinical Trials Outside the United States; Hematopoietic Transplantation: Gene Therapy.
Further Readings
Cooke, D. W. and L. Plotnick. “Type 1 Diabetes Mellitus in Pediatrics.” Pediatric Review, v.29/11 (2008).
Emerging Risk Factors Collaboration. “Diabetes Mellitus, Fasting Blood Glucose Concentration, and Risk of Vascular Disease: A Collaborative Meta-Analysis of 102 Prospective Studies.” Lancet, v.375/9733 (2010).
International Diabetes Federation. http://www.idf.org (Accessed April 2014).
Kitabchi,
