Mecasermin treatment for Rett Syndrome – Phase 1 completed successfully
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 RTT, 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.'
