2016 - IPITA - Stem Cell Derived Beta Cells Workshop


Clinical Targets

15.0 - Clinical targets for stem cell-derived beta cell replacement therapies

Presenter: Bernhard, Hering, Minneapolis/St. Paul, United States
Authors: Bernhard Hering


A central element of the design of any clinical trial, especially also of early-phase trials of stem cell-derived beta cell products, is the definition of the study population. The aim is to select a trial population with a favorable benefit-risk ratio, while also achieving the study’s scientific objectives.
Stem cell-derived beta cell replacement technologies are rapidly evolving and the specific characteristics of each investigational cell therapy product and related technologies applied for preventing rejection will determine the patient group in whom the islet beta cell product’s activity, either beneficial or adverse, can be best detected and for whom participation in an early-phase stem cell-derived beta cell trial is the best option. Since early-phase pilot trials are likely to be small, single-arm, and open-label in which outcomes are compared to baseline status, the metabolic and clinical effects should be expected to be robust enough for detection in the selected study population, if meaningful information is to be obtained from the study.
Medical treatments for diabetes are also rapidly evolving. The improved educational programs involving behavioral therapies, insulin analogs, continuous glucose monitoring systems, and other diabetes technologies such as sensor augmented pumps with predictive low glucose management technology will help more and more insulin-treated diabetic patients meet treatment goals. For those patients in whom diabetes is complicated by impaired awareness of hypoglycemia and recurrent severe hypoglycemia, extreme glycemic lability, and progressive microvascular lesions and in whom severe hypoglycemic episodes persist or glycemic goals cannot be maintained despite access to refined interventions under the close guidance of an expert diabetes care team, transplant interventions should be considered. Patients who present with these persistently unmet clinical needs may have type 1 diabetes, type 2 diabetes, or pancreatogenic diabetes resulting from total pancreatectomy. To warrant enrollment of these transplant candidates in pilot clinical trials of stem cell-derived beta cell products, lack of timely access to islet transplantation due to high islet dose requirements or other factors, or alternatively, a more favorable benefit-risk determination associated with the stem cell-derived islet than the alloislet, xenoislet or allopancreas transplant must be demonstrated. A more favorable benefit-risk determination could result from beta cell replacement technologies that will not require maintenance immunosuppression. It is also suggested that sponsors/investigators justify the rationale for a particular study population by reviewing the potential for benefit and the ability to detect the stem cell-derived beta cell product’s activity in the selected versus other possible cohorts of patients.
As the stem cell-derived beta cell products advance through research and development and the full potential of stem cell-derived beta cell transplantation is increasingly realized through continued innovation, the selection of appropriate recipients will need to be revisited.


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