Programme led by
Royal College of Surgeons in Ireland, 123 St Stephens Green, Dublin 2, Ireland
Domain / Application Expertise
Daniela studies the molecular mechanisms involved in neurodevelopmental disorders with a particular interest in Schizophrenia and Rett Syndrome. Her expertise in growth factors and are widely recognized, and she is a regular invited and plenary speaker at relevant international meetings.
Daniela's first translational work on IGF1 and Rett syndrome stands out for its clinical relevance. Rett syndrome is a severe disorder that lacks a treatment: she was the first to show that IGF1, a growth factor used in children with growth impairments, is able to ameliorate the symptoms of the disease in a mouse model of Rett, and therefore has the potential of leading to a treatment for Rett and other neurodevelopmental disorders (Tropea et al., 2009). Nine clinical trials derived from this study, which showed no side-effects of IGF1 administration in Rett patients (Pini et al., 2012, Pini et al., 2014, 2016).
Daniela has access to exclusive EEG data and blood samples from patients involved in IGF1-related trials and collaborates with MIT, Neuren Pharmaceuticals, Tuscany Rett Center and Boston Children's Hospital to study the RNA expression in the blood of Rett patients before and after treatment with IGF1. Her team aims to identify molecular determinants of IGF1' action in the patients who responded to treatment. She plans to focus on the identification of measurable biomarkers and on the effects of IGF1 for the treatment of neurodevelopmental disorders. This research has application in the design and interpretation of current and future clinical trials for assessing the efficacy of IGF1 (current active trails in Rett Syndrome, FragileX, Phelan McDermid and autism).