It is essential to reposition the perspective of therapeutic trials in a continuum between laboratory research, preclinical development, often carried out within industry, and above all the patient-caregiver relationship. Indeed, there is a whole range of issues that need to be addressed sequentially, in order to eventually be able to propose targeted therapeutic trials on limited populations capable of demonstrating the efficacy of these therapies, particularly in the context of rare diseases.
In the course of the lecture, a number of current and future trials were discussed, such as the development of therapeutic trials for Leber's optic neuropathy, now in phase III, carried out within the framework of work initiated and conducted at the Institut de la Vision and taken over by an industrial partner.
The production of therapeutic products, particularly gene therapy vectors, represents a major challenge for the development of clinical trials, and is currently the focus of a great deal of effort on the part of industry, but also of particular vigilance on the part of regulatory agencies, with the establishment in many countries of several public bodies to monitor clinical trials.
The crucial factor in the development of clinical trials is to adapt the trial to the stage of the disease. We have repeatedly stressed that, in the initial stages of a trial, it must be carried out on patients with very advanced stages of the disease, stages at which therapeutic hope is limited and the possibility of targeting study cells relatively low.
