New Treatment for AS – IGF-2 Receptor Ligand Reverses Multiple Deficits

Quick Overview

Dr. Cristina Alberini presented her research on the potential use of Insulin-like growth factor 2 (IGF-2) as a treatment for Angelman syndrome (AS) and other neurodevelopmental disorders at the 2018 FAST Science Summit. Her studies have shown that IGF-2 can enhance memory and reverse deficits in cognitive function, social interaction, and repetitive behavior in mouse models of AS. The effects of IGF-2 are mediated through the IGF-2 receptor and involve the clearance of excess proteins in cells. Dr. Alberini’s research also identified a novel ligand, L1, that targets the IGF-2 receptor and has similar effects. Further studies are ongoing to understand the mechanism of action and to explore the potential clinical translation of IGF-2 as a therapy for AS and other synaptic disorders.

Introduction

In a presentation at the 2018 FAST Science Summit, Dr. Cristina Alberini introduced her groundbreaking research on a potential new treatment for Angelman Syndrome (AS). Dr. Alberini, a world-renowned clinician and scientist at NYU in New York City, shared her findings on the use of Insulin-like growth factor 2 (IGF-2) as a memory enhancer and its potential therapeutic benefits for AS.

Background

Dr. Alberini’s research initially focused on understanding the biology of memory formation and storage in the brain. She studied the role of gene expression and synaptic plasticity in the formation of long-term memories. Through her studies, she identified IGF-2 as a growth factor that plays a crucial role in memory enhancement.

Memory Enhancement and Therapeutic Potential

Dr. Alberini’s research showed that IGF-2 can enhance memory formation in rats. When IGF-2 was injected into mice before or after a learning experience, it significantly improved memory performance across various types of memories, including fear memories and recognition memories. These findings led Dr. Alberini to explore the potential therapeutic benefits of IGF-2 in neurodevelopmental disorders.

IGF-2 and Angelman Syndrome

Dr. Alberini’s team tested the effects of IGF-2 in a mouse model of Angelman Syndrome, a neurodevelopmental disorder characterized by developmental delay, intellectual deficits, repetitive behavior, lack of speech, seizures, and motor problems. The results were remarkable – a single injection of IGF-2 reversed cognitive deficits, social interaction impairments, repetitive behavior, and motor deficits in the Angelman mice.

Mechanism of Action

Dr. Alberini’s research suggests that IGF-2 acts through the IGF-2 receptor and targets mechanisms of synapse and cell homeostasis. It promotes the clearance of excess proteins in the cells, a process known as macroautophagy. This mechanism is crucial in maintaining the health and functioning of neurons and preventing the accumulation of proteins associated with neurodegenerative diseases.

Future Directions

Dr. Alberini’s research on IGF-2 and its potential therapeutic benefits is ongoing. Her team is working to identify more specific ligands for the IGF-2 receptor and further understand the molecular mechanisms underlying its effects. They are also collaborating with other researchers to expand their studies to include rat models and explore the potential of IGF-2 in other neurodevelopmental disorders and neurodegenerative diseases.

Conclusion

Dr. Alberini’s research on IGF-2 as a memory enhancer and its potential therapeutic benefits for Angelman Syndrome is groundbreaking. Her findings offer hope for the development of new treatments for AS and other neurodevelopmental disorders. Further research is needed to fully understand the mechanisms of action and to translate these findings into clinical applications.