Insulin-like Growth Factor-1 (IGF-1) Treatment for Phelan-McDermid Syndroms (PMS)

Description:

Summary

IGF-1 treatment for PMS has demonstrated significant improvement in both social impairment and restrictive behaviors.

 

Unmet need

PMS is a rare genetic syndrome, with a wide range of severity of symptoms, including intellectual disabilities, sleep disorders, and seizures, as well as motor and vocalization symptoms. PMS is caused by disruption of the SHANK3/ProSAP2 gene on the terminal of chromosome 22. Approximately 600 cases are known worldwide, but numbers are expected to increase as new genomic testing methods have made clinical testing more widely available.

 

Current Development Status

•       A pilot controlled trial of insulin-like growth factor-1 in children with Phelan-McDermid syndrome has been completed

•       Further clinical studies are ongoing

Applications

•       Phelan-McDermid Syndrome

•       Possible broader application to other Autism Spectrum Disorders

Advantages

•       IGF-1 is approved for short stature children and has a proven safe profile in pediatric  populations

•       IGF-1 and its N-terminal peptide {(1-3) IGF-1} both have potential to ameliorate certain symptoms associated with PMS and other related disorders

•       IGF-1/mescasermin (Increlex®) is administered intravenously, and the recombinant human DNA is synthesized in bacteria

•       Increlex® patent expired 2017

Publications

•       https://www.ncbi.nlm.nih.gov/pubmed/25685306

 

Patent Information:
Category(s):
Therapeutic
For Information, Contact:
Alison Fenney
Manager, Business Development and Licensing
Icahn School of Medicine at Mount Sinai
alison.fenney@mssm.edu
Inventors:
Joseph Buxbaum
Takeshi Sakurai
Keywords:
CNS
Orphan Diseases
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