IGF1 as a Potential Treatment for Rett Syndrome: Safety Assessment in Six Rett Patients
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AURT.2012.679801.pdf
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2.54 MB
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Author(s)
Pini, Giorgio
Scusa, Maria Flora
Congiu, Laura
Benincasa, Alberto
Morescalchi, Paolina
Bottiglioni, Ilaria
Di Marco, Pietro
Borelli, Paolo
Bonuccelli, Ubaldo
Della-Chiesa, Andrea
Date Issued
2012
Journal
Autism Research and Treatment
Publisher
Hindawi Publishing Corporation
Citation
Pini, Giorgio, Maria Flora Scusa, Laura Congiu, Alberto Benincasa, Paolina Morescalchi, Ilaria Bottiglioni, Pietro Di Marco, et al. “IGF1 as a Potential Treatment for Rett Syndrome: Safety Assessment in Six Rett Patients.” Autism Research and Treatment 2012 (2012): 1–14.
Version
Final published version
Abstract
Rett syndrome (RTT) is a devastating neurodevelopmental disorder that affects one in ten thousand girls and has no cure. The majority of RTT patients display mutations in the gene that codes for the methyl-CpG-binding protein 2 (MeCP2). Clinical observations and neurobiological analysis of mouse models suggest that defects in the expression of MeCP2 protein compromise the development of the central nervous system, especially synaptic and circuit maturation. Thus, agents that promote brain development and synaptic function, such as insulin-like growth factor 1 (IGF1), are good candidates for ameliorating the symptoms of RTT. IGF1 and its active peptide, (1–3) IGF1, cross the blood brain barrier, and (1–3) IGF1 ameliorates the symptoms of RTT in a mouse model of the disease; therefore they are ideal treatments for neurodevelopmental disorders, including RTT. We performed a pilot study to establish whether there are major risks associated with IGF1 administration in RTT patients. Six young girls with classic RTT received IGF1 subcutaneous injections twice a day for six months, and they were regularly monitored by their primary care physicians and by the unit for RTT in Versilia Hospital (Italy). This study shows that there are no risks associated with IGF1 administration.
MIT Department
Massachusetts Institute of Technology. Department of Brain and Cognitive Sciences
Picower Institute for Learning and Memory
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DOI of Published Version
https://doi.org/10.1155/2012/679801
