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The Impact of Islet Cell Transplantation on the Type 1 Diabetes Market September 29, 2005
Decision Resources, Inc., Sep 2005, Pages: 13


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Type 1 diabetes is a chronic disease that is often accompanied by long-term complications such as nephropathy, neuropathy, and cardiovascular disease that frequently result in morbidity and mortality. Insulin therapy, while controlling the symptoms of the disease, does not constitute a cure, and many patients develop secondary complications. Pancreatic islet cell transplantation offers the potential of terminating the autoimmune response that drives the destruction of insulin-producing beta-cells and eliminating the need for life-long insulin therapy.

This report outlines the mechanisms and current therapies for type 1 diabetes and discusses some of the novel therapy options that are emerging for treatment of the disease, particularly pancreatic islet cell transplantation. We investigate the promise and the challenges associated with pancreatic islet cell transplantation and forecast the effect it is likely to have on the type 1 diabetes market, as well as its impact on insulin and emerging therapies.

Business Implications
- Type 1 diabetes affects more than 2 million people worldwide, and, although insulin treatments are effective in managing the symptoms of the disease, this therapy and other emerging drug treatments do not offer a cure. Pancreatic islet cell transplantation offers the potential of terminating the autoimmune response that drives the destruction of insulin-producing ß-cells and eliminating the need for life-long insulin therapy. Several transplant centers have reported remarkable success in achieving insulin independence and glucose control in islet transplant recipients.
- Several challenges associated with islet cell transplantation must be addressed before the procedure can become a mainstream therapy.
Patients who undergo this procedure must commit to lifelong immunosuppressive therapy; therefore, there is a need to identify effective immunosuppressive therapies and eliminate some of the severe side effects associated with this type of therapy. Also, because there is a limited number of pancreata available for islet cells, a viable and sustainable surrogate source of these cells must be identified to make this procedure more widely available. In addition, the percentage of transplant recipients who will achieve insulin independence remains to be determined, as does the question of how long this independence will last.
- Several therapies are currently in development for the treatment of type 1 diabetes. Sanofi-Aventis recently filed for approval of Exubera, its inhaled formulation of insulin, and we expect this product to have a significant impact on the market. Amylin's pramlintide acetate (Symlin) recently received approval as an adjunct therapy in combination with insulin for patients with type 1 diabetes. These drugs, as well as others in development, are likely to reduce the number of patients who do not respond to insulin therapy and therefore may reduce the number of patients who might require islet cell transplantation.



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