IGF-1 Pilot Study to Evaluate Safety and Efficacy for Core Autism Symptoms in Children

Autism Spectrum Disorder (ASD) is a complex neurodevelopmental condition characterized by persistent deficits in social communication and interaction, alongside restricted, repetitive patterns of behavior, interests, or activities. Current interventions primarily focus on behavioral and educational therapies, with limited pharmacological options directly targeting core ASD symptoms. Existing pharmacotherapies often address co-occurring conditions like irritability or anxiety, rather than the fundamental social and communication challenges. Insulin-like Growth Factor-1 (IGF-1) is a pleiotropic peptide hormone crucial for brain development, neuronal survival, and synaptic plasticity. Dysregulation of the IGF-1 pathway has been implicated in various neurodevelopmental disorders, including some forms of ASD, suggesting its potential as a therapeutic agent to modulate neural circuits underlying social and cognitive functions. This pilot study aims to explore this therapeutic potential.

This proposed pilot project will employ a double-blind, placebo-controlled crossover trial design to evaluate IGF-1's impact. Five children diagnosed with Autistic Disorder will participate. The study's primary objective is to assess the safety and feasibility of IGF-1 treatment in this population. Secondary outcomes will include evaluating improvements in autism-specific impairments, specifically in socialization, language delay, and repetitive behaviors. Additionally, the study will examine changes in functional outcomes related to global severity. The crossover design ensures that each participant receives both IGF-1 and placebo, allowing for within-subject comparisons and maximizing statistical power despite the small sample size. Specific dosing, route, and duration details are not provided in the abstract, but the design focuses on initial safety and preliminary efficacy signals.