Background: The purpose of treatment of children with autism is to help them to acquire functional skills in routine life and to control behaviors that are disruptive. Objectives: In this study, the safety and efficiency of cerebrolysin was determined by childhood autism rating scale (CARS) on behavioral, verbal and nonverbal developments of children with autism referred to children's medical hospital clinic of neurology. Methods: In a quasi-experimental study, 36 children with autism were enrolled and the effect of cerebrolysin on their behavioral, nonverbal and verbal development was determined by CARS questionnaire fulfilled before and one month after complete intervention. Results: All evaluated items except level and consistency of intellectual response had favorable reduction in scores of CARS questionnaire (P = 0.001). The total score decreased from 40.6 to 36.1 showing 11.1% improvement. Despite the efficiency of cerebrolysin observed in this study, its partial use may be limited by the large number of intramuscular injections, i.e. nine per months. Sustained release formulation may alleviate this limitation, if this therapy proves to be beneficial in future studies. Conclusions: According to the obtained results, the safety and feasibility of cerebrolysin administration could be considered and be effective on behavioral, nonverbal and verbal development in children with autism.