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Mecasermin (rh-IGF-1) treatment for Rett Syndrome is safe and well-tolerated
Date:3/10/2014

(Cincinnati, OH) The results from Boston Children's Hospital's Phase 1 human clinical trial in Rett syndrome came out today. A team of investigators successfully completed a Phase 1 clinical trial using mecasermin [recombinant human insulin-like growth factor 1 (IGF-1)], showing proof-of-principle that treatments like IGF-1 which are based on the neurobiology of Rett syndrome, are possible. The study 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. The completed Phase 1 trial and the now current Phase 2 study are funded in part by the International Rett Syndrome Foundation (IRSF) to Dr. Omar Khwaja (first author on the publication) who initially led the Phase 1 study and to Dr. Walter Kaufmann (corresponding author) who completed the Phase 1 study and is leading the Phase 2 study at Boston Children's Hospital. Kathryn Kissam, IRSF Board member said, "Our sincerest gratitude goes to Dr. Khwaja and Dr. Kaufmann for their true dedication to find treatments for our girls and women today."

The article, titled "Safety, pharmacokinetics, and preliminary assessment of efficacy of mecasermin (rhIGF-1) for the treatment of Rett syndrome"' in the scientific journal Proceedings of the National Academy of Sciences (PNAS) describes how the team assessed IGF-1 treatment in 12 girls with MECP2 mutations (9 with a clinical diagnosis of Rett syndrome). IGF-1 is approved by the Food and Drug Administration (FDA) for treatment of growth failure in children with severe primary IGF-1 deficiency. Because IGF-1 has not previously been tested on a pediatric population with Rett syndrome, this Phase 1 was designed first and foremost for safety.

Dr. Kaufmann now has an ongoing Phase 2 trial of IGF-1 treatment that intends to replicate the promising beneficial effects on behavior a
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Contact: Steve Kaminsky, Ph.D.
skaminsky@rettsyndrome.org
301-961-1559
International Rett Syndrome Foundation
Source:Eurekalert

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