PREVENTROP · New approach to treatment of the blinding disease Retinopathy of Prematurity (ROP)

The overall objective is to develop a novel preventative intervention for the blinding disease retinopathy of prematurity (ROP) and other complications of prematurity. The PREVENTROP consortium proposes to conduct preclinical studies (pharmacological, pharmacodynamics, pharmacokinetics and toxicological) in models and/or clinical studies (including phase III clinical trial) of an EU designated orphan medicinal product. This orphan medicinal product has been granted the EU orphan designation.We have completed both a Phase I study and the first section of the Phase II study administering the growth factor complex IGF-I/IGFBP-3 (Premiplex®) successfully to preterm infants in order to prevent ROP– a discovery that we have taken from bench to bedside.Due to improved neonatal care, the survival of preterm babies has increased dramatically during the last decades. The downside of this improvement in survival rates is higher morbidity affecting these vulnerable infants. It should be emphasised that in a preterm infant any lasting morbidity will have a negative impact on the quality of life for a whole life span for both the individual and their families.One of the most severe morbidities affecting these infants is retinopathy causing severe visual impairment and blindness.Our research findings have rendered several editorials pointing at the hope of prevention strategies which if successful will change the paradigm for ROP and other morbidities in preterm infants.There are three major impacts of the present proposal; (1) clinical availability of a new orphan designated drug product, Premiplex®, and (2) improved care of preterm infants and (3) a significant contribution towards the goal of the International Rare Disease Research Consortium (IRDiRC) by delivering one new therapy for a rare disease.