IGF-1 and Rett syndrome

US researchers have used insulin-like growth factor 1 (IGF-1) to reverse some of the symptoms of the neurological disorder Rett syndrome in a mouse model.

Rett is an autistic-like disorder affecting girls, predominantly caused by a mutation in the gene coding for the MeCP2 protein.

MeCP2 is essential for neuronal maturation. A deficiency stops neurons from developing dendritic spines, which enable cell-cell communication.

The researchers, led by MIT scientists Mrignaka Sur and Rudolf Jaenisch, tested whether IGF-1 – a hormone which, amongst many other things, has a role in regulating cell growth and neuronal development – could restore proper neuronal cell maturation.

Administering IGF-1 to MeCP2 deficient mice ameliorated certain symptoms of Rett, including delayed or irregular locomotor function, breathing patterns and heart rate, the researchers report.

They also found the treatment extended the life span of the mice, increased brain weight and restored dendritic spinal density.

They suggest IGF-1 is a strong candidate for trials in patients with Rett, and potentially for other central nervous system disorders.

Their findings are published in the US journal Proceedings of the National Academy of Sciences.