Treatments

Citalopram (Celexa), a medication commonly prescribed to children with autism spectrum disorders (ASD), was no more effective than a placebo at reducing repetitive behaviors, according to a multi-site clinical trial guided by lead author Bryan King, MD, director of child and adolescent psychiatry at Seattle Children's Hospital and professor and vice chair of psychiatry at the University of Washington School of Medicine.

Because citalopram is also prescribed for patients with obsessive compulsive disorders (OCD), these study results may challenge the widely held premise that repetitive behaviors in children with ASD are similar to repetitive behaviors often found in cases of OCD.

Rett Syndrome (RTT) is a severe form of X-linked mental retardation caused by mutations in the gene coding for methyl CpG-binding protein 2 (MECP2). Mice deficient in MeCP2 have a range of physiological and neurological abnormalities that mimic the human syndrome. Here we show that systemic treatment of MeCP2 mutant mice with an active peptide fragment of Insulin-like Growth Factor 1 (IGF-1) extends the life span of the mice, improves locomotor function, ameliorates breathing patterns, and reduces irregularity in heart rate. In addition, treatment with IGF-1 peptide increases brain weight of the mutant mice. Multiple measurements support the hypothesis that RTT results from a deficit in synaptic maturation in the brain: MeCP2 mutant mice have sparse dendritic spines and reduced PSD-95 in motor cortex pyramidal neurons, reduced synaptic amplitude in the same neurons, and protracted cortical plasticity in vivo. Treatment with IGF-1 peptide partially restores spine density and synaptic amplitude, increases PSD-95, and stabilizes cortical plasticity to wild-type levels. Our results thus strongly suggest IGF-1 as a candidate for pharmacological treatment of RTT and potentially of other CNS disorders caused by delayed synapse maturation.