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Published on: April 2, 2021
IGF-1 in retinopathy of prematurity, a CNS neurovascular disease
Raffael Liegl1, Chatarina Löfqvist2, Ann Hellström2
1Department of Ophthalmology, Boston Children's Hospital, Harvard Medical School, Boston, MA, USA.
Insights
Insulin-like growth factor 1 (IGF-1) may help prevent blindness and brain damage in preterm infants. While a study showed IGF-1 improved intraventricular hemorrhage (IVH) severity, it did not impact retinopathy of prematurity (ROP).
Area of Science:
- Neonatal neuroscience
- Ophthalmology
- Endocrinology
Background:
- Very preterm birth can cause severe retinal and brain damage, leading to conditions like retinopathy of prematurity (ROP) and central nervous system (CNS) disorders.
- Low circulating Insulin-like Growth Factor 1 (IGF-1) levels are linked to ROP, while IGF-1 gene defects are associated with CNS impairments such as learning deficits and restricted brain growth.
Purpose of the Study:
- To review and compare the roles of IGF-1 in retinopathy of prematurity (ROP) and central nervous system (CNS) disorders.
- To evaluate the potential of recombinant IGF-1 treatment in preventing ROP and CNS complications in preterm infants.
Main Methods:
- Literature review comparing the function of IGF-1 in ROP and CNS development.
- Analysis of a recent phase 2 clinical study investigating IGF-1 effects in preterm infants.
Main Results:
- A phase 2 study indicated that IGF-1 treatment positively influenced the severity of intraventricular hemorrhage (IVH) in preterm infants.
- The same study found no significant effect of IGF-1 on retinopathy of prematurity (ROP).
Conclusions:
- IGF-1 shows potential therapeutic benefits for certain brain injuries like IVH in preterm infants.
- Further investigation, including a planned phase 3 trial, is warranted to fully understand IGF-1's efficacy in preventing ROP and CNS disorders.
Abstract:
The retina is part of the central nervous system and both the retina as well as the brain can suffer from severe damage after very preterm birth. Retinopathy of prematurity is one of the major causes of blindness in these children and brain neuronal impairments including cognitive defects, cerebral palsy and intraventricular hemorrhage (IVH) are also complications of very preterm birth. Insulin-like growth factor 1 (IGF-1) acts to promote proliferation, maturation, growth and survival of neural cells. Low levels of circulating IGF-1 are associated with ROP and defects in the IGF-1 gene are associated with CNS disorders including learning deficits and brain growth restriction. Treatment of preterm infants with recombinant IGF-1 may potentially prevent ROP and CNS disorders. This review compares the role of IGF-1 in ROP and CNS disorders. A recent phase 2 study showed a positive effect of IGF-1 on the severity of IVH but no effect on ROP. A phase 3 trial is planned.
