A new treatment for infant blindness

Retinopathy of prematurity (ROP) is a major cause of blindness in babies born prematurely and receiving intensive care. Global statistics show that nearly 40 % of perinatal blindness can be attributed to ROP. The abnormal growth of retinal blood vessels seems to be the aetiology of ROP and the sole treatment for the past 50 years is laser treatment or cryotherapy.

However, these methods destroy the peripheral part of the retina, often inducing loss of peripheral vision. As a result, new treatments or preventative measures are urgently required. Scientists under the umbrella of the EU-funded 'New approach to treatment of the blinding disease Retinopathy of Prematurity (ROP)' (PREVENTROP) initiative are working to address this issue.

The preventative strategy is based on the replacement of the in utero factors which are disrupted when the infant is born prematurely. One such molecule is insulin-like growth factor 1 (IGF-I) and preliminary work indicates that IGF-1 replacement to normal levels will prevent ROP.

The overall objective of the PREVENTROP study is to evaluate their approach in animal models and then proceed to clinical trials to evaluate the pharmacokinetics, pharmacodynamics and safety of IGF-I administration. So far, the consortium has set out to assess its efficacy by administering the growth factor until endogenous production reached normal levels.

Continuous assessment of various parameters has led to the determination of a safe dose of IGF-I in premature infants which will be implemented in a multi-centre European study. Partners have also developed a novel blood-based method for measuring IGF-I levels rapidly that requires only a tiny amount of blood.

The outcome of the PREVENTROP project is the development of an orphan drug designated to treat ROP in premature infants. Given the destructive nature of existing interventions and that visual handicap is a life-long issue, this novel drug seems superior at halting disease progression.