Claims for Patent: 9,212,204

Summary for Patent: 9,212,204

Title:	Treatment of rett syndrome using glycyl-L-2-methylprolyl-L-glutamic acid
Abstract:	This invention provides compounds, compositions and methods for treating Autism Spectrum Disorders (ASD) using glycyl-2-methylprolyl-glutamic acid (G-2-MePE) and analogs thereof. Autism Spectrum Disorders include Autism, Autistic Disorder, Asperger Syndrome, Childhood Disintegrative Disorder, Pervasive Developmental Disorder—Not Otherwise Specified (PDD-NOS), Fragile X Syndrome, and Rett Syndrome. Compositions containing compounds include water-soluble formulations, water-in-oil micro-emulsions, water-in-oil coarse emulsions, water-in-oil liquid crystals, nanocapsules, tablets, and orally administered gels. The compounds and compositions of this invention can be administered intravenously, intraventricularly, parenterally, or orally, and can be effective in treating neurodegeneration, promoting neurological function, treating seizure activity and other symptoms of ASD, and can prolong life in animals including human beings having Autism Spectrum Disorders.
Inventor(s):	Glass Lawrence Irwin, Bickerdike Michael John, Snape Michael Frederick
Assignee:	Neuren Pharmaceuticals Limited
Application Number:	US14605420
Patent Claims:	2. The method of claim 1 , said effective amount of G-2-MePE being in the range of about 1 mg/Kg to about 100 mg/Kg.3. The method of claim 1 , where said G-2-MePE is administered in a dose of about 60 mg/kg twice per day (“b.i.d.”).4. The method of claim 1 , where said G-2-MePE is administered to a mucosa of said human being.5. The method of claim 1 , further comprising administering to the human being a second therapeutic agent selected from a group consisting of: insulin-like growth factor-I (IGF-I) claim 1 , insulin-like growth factor-II (IGF-II) claim 1 , glycyl-prolyl-glutamate (GPE) claim 1 , transforming growth factor-β1 claim 1 , activin claim 1 , growth hormone claim 1 , nerve growth factor claim 1 , brain-derived neurotrophic factor (BDNF) claim 1 , growth hormone binding protein claim 1 , IGF-binding proteins (especially IGFBP-3) claim 1 , basic fibroblast growth factor claim 1 , acidic fibroblast growth factor claim 1 , the hst/Kfgk gene product claim 1 , FGF-3 claim 1 , FGF-4 claim 1 , FGF-6 claim 1 , keratinocyte growth factor claim 1 , androgen-induced growth factor claim 1 , int-2 claim 1 , fibroblast growth factor homologous factor-1 (FHF-1) claim 1 , FHF-2 claim 1 , FHF-3 claim 1 , FHF-4 claim 1 , keratinocyte growth factor 2 claim 1 , glial-activating factor claim 1 , FGF-10 and FGF-16 claim 1 , ciliary neurotrophic factor claim 1 , brain derived growth factor claim 1 , neurotrophin 3 claim 1 , neurotrophin 4 claim 1 , bone morphogenetic protein 2 (BMP-2) claim 1 , glial-cell line derived neurotrophic factor claim 1 , activity-dependant neurotrophic factor claim 1 , cytokine leukaemia inhibiting factor claim 1 , oncostatin M claim 1 , interleukin) claim 1 , α-interferon claim 1 , β-interferon claim 1 , γ- claim 1 , consensus interferon claim 1 , TNF-α claim 1 , clomethiazole; kynurenic acid claim 1 , Semax claim 1 , tacrolimus claim 1 , L-threo-1-phenyl-2-decanoylamino-3-morpholino-1-propanol claim 1 , andrenocorticotropin-(4-9) claim 1 , analog [ORG 2766] claim 1 , dizolcipine (MK-801) claim 1 , selegiline; mematine claim 1 , NPS1506 claim 1 , GV1505260 claim 1 , MK-801 claim 1 , GV150526; 2 claim 1 ,3-dihydroxy-6-nitro-7-sulfamoylbenzo(f)quinoxaline (NBQX) claim 1 , LY303070 claim 1 , LY300164; anti-MAdCAM-1mAb claim 1 , MECA-367 (ATCC accession no. HB-9478) claim 1 , fenobam claim 1 , fluoxetine claim 1 , and risperidone.6. The method of claim 1 , where said dose of said compound is 10 mg/kg three times per day or 30 mg/kg three times per day.7. The method of claim 1 , said treatment producing an improvement in said symptom as assessed by one or more behavioral test selected from the group consisting of The Rett Syndrome Natural History/Clinical Severity Scale claim 1 , Aberrant Behavior Checklist Community Edition (ABC) claim 1 , Vinelands claim 1 , Clinical Global Impression of Severity (CGI-S) and their carers completed the Caregiver Strain Questionnaire (CSQ) claim 1 , or one or more physiological test selected from the group consisting of electroencephalogram (EEG) spike frequency claim 1 , overall power in frequency bands of an EEG claim 1 , hand movement claim 1 , QTc and heart rate variability (HRV) claim 1 , and respiratory irregularities compared to control animals not suffering from said symptom.