Danuta Radzioch
McGill University, Quebec
Title: Importance of characterization of variation in the secondary endpoint measures prior to the trial: A key to a successful outcome of phase 1 trial and progression to a phase 2 stage of the study
Biography
Biography: Danuta Radzioch
Abstract
The presentation describes importance of characterization of variation in the secondary endpoint measures prior to the trial: a key to a successful outcome of phase I trial and progression to phase 2 stage of the study based on the example of the outcomes from the recently completed phase I trial study Fenretinide (4-HPR), a master regulator of lipid metabolism controlling inflammation in patients with cystic fibrosis. The presentation describes bench to clinical development of the project and back to bench study to fully explain the mechanism of action of the drug explaining its effect on secondary end points. Dr. Radzioch laboratory has been pursuing research on cystic fibrosis lung disease for more than 15 years. Cystic fibrosis disease involves multiple organ pathology causing patients to live with many symptoms such as malabsorption of nutrients, low body mass, and malfunction of the pancreas, diabetes, infertility and osteoporosis. Aberrant regulation arachidonic acid (AA, an omega-6 fatty acid) and docosahexaenoic acid (DHA, an omega-3 fatty acid) were already known in cystic fibrosis but until now there was no treatment which could correct this problem. Dr. Radzioch laboratory demonstrated that the severe aberrations in lipid metabolism also happen during severe infections and spinal cord injury. CF mice were treated with fenretinide (4-HPR); a drug previously used in cancer trials helped the CF mice to improve their ability to fight lung infection with Pseudomonas.