Rett Syndrome Association of Australia | Insulin-Like Growth Factor 1 (IGF-1) – A potential treatment in Rett syndrome?

Insulin-Like Growth Factor 1 (IGF-1) – A potential treatment in Rett syndrome?

Posted by Rett Syndrome Association of Australia in News 02 Sep 2012

Recent research conducted at the Massachusetts Institute of Technology saw Rett syndrome mice being treated with an active peptide fragment of Insulin-Like Growth Factor 1 (IGF-1). As a result, they were found to live longer, move better, and both their heart rates and breathing patterns improved. A study into the treatment of Rett syndrome individuals with IGF-1 is being conducted the Children’s Hospital, Boston.

Phase 1 of a trial with IGF-1 directly involving individuals with Rett syndrome, began at the Children’s Hospital, Boston (USA), in December 2010, and basically just looked at dosage and side effects.

An update on the trial was provided in September 2012 by Dr Steven Kaminsky, Chief Scientific Officer of the International Rett Syndrome Foundation. He advised that “The initial trial was a four week phase 1 study of safety and tolerability of IGF-1 in Rett syndrome. This initial phase 1 trial was followed by a 20 week open label (i.e., everyone received IGF-1) extension of the study. During this period, the patients were monitored for safety and potential long term side-effects of IGF-1. New technologies which may be useful to demonstrate efficacy of the drug during the phase 2 trial, were also tried.

After analyzing data from phase 1 and the open label extension, the Boston team decided to narrow the inclusion criteria for phase 2. The upper age limit was reduced to 10 years, while the lower age limit was increased to 5 years.  A recent press release from the International Rett Syndrome Foundation advised that “the phase 1 trial has deemed that IGF-1 is safe and well tolerated in girls diagnosed with Rett syndrome, and the data also suggests that certain breathing and behavioral symptoms associated with Rett syndrome were ameliorated after IGF-1 treatment.”

Phase 2 which recently reached its mid-point, is a double-blinded trial, meaning that neither the families nor the researchers know which participants are receiving IGF-1. The cross-over design of the trial means that each subject will receive 20 weeks of either a placebo or IGF-1 and then, after a 6-week washout period, participants will receive another 20 weeks of treatment but taking the drug they did not take on the first occasion. In other words, all girls enrolled in phase 2  receive 20 weeks of treatment with IGF-1. The most recent announcement about Phase 2 was made in May 2014 by the Boston Children’s Hospital, a copy of which can be viewed by clicking on the following file 

May 2014 – Announcement by Boston Children’s Hospital re Phase 2 of IGF-1 trial           

The above announcement has been sourced from the website of the International Rett Syndrome Foundation.



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