After finishing the preparation of the light-activated compound for human use, we will perform proof-of-concept studies in adults, which is part of work package 4. In this work package, we aim to ensure safety and efficacy of the light-activated treatment with the photosensitizer-exendin construct in adults. This will be performed in three steps, including studies outside and inside the body to ensure the highest level of safety. We will focus on adult patients who will be scheduled for residual pancreas removal surgery due to an insulinoma. Insulinomas are benign beta cell derived tumors overproducing insulin, which are therefore the perfect model for the preparation of clinical trials in children with CHI, since both diseases are focusing on over-functioning of beta cells. First, we will test this light-activated treatment in the removed pancreas tissue (outside the body), coming from a partial pancreas removal surgery. If the treatment works in the pancreas outside the body, we will perform the same treatment inside the body of patients who are scheduled for pancreas removal surgery (step 2), so we can assess the effects on the pancreas outside of the body after surgery. If this is successful, the last step is to treat inside the body of patients who are not scheduled for surgical removal of the insulinoma.
Quick links to other Work Packages: 1 | 2 | 3 | 4 | 5 | 6 | 7 | 8 | 9