Project description
New pharmacological treatment for Down’s syndrome
Down’s syndrome (DS) is caused by an extra copy of all or part of the long arm of human chromosome 21. DS causes lifelong cognitive impairments (CIs) which have a significant negative impact on the individuals, their families and society. However, there are currently no approved pharmacological treatments for CIs in people with DS. The EU-funded ICOD project aims to fill this gap by providing a feasibility demonstration of a new therapy for CIs in DS. It will perform first-in-humans and efficacy clinical trials with a new molecular entity (AEF0217), which reverses CIs in animal models of DS. AEF0217 belongs to a new pharmacological class developed by the biotech Aelis Farma: the Signaling Specific Inhibitors of the Cannabinoid (CB1) receptor (CB1-SSi). ICOD will enable a safe and efficacious pharmacological treatment of CIs in people with DS.
Objective
Cognitive disabilities in individuals with Down syndrome (DS), also known as Trisomy 21, have no approved pharmacological treatment and represent a significant burden on DS individuals, their families and the healthcare system.
The ICOD project will deliver a clinical proof of concept for a new therapy of cognitive deficits in DS by performing first in humans (phase I) and clinical efficacy (Phase II) studies with AEF0217.
AEF0217 is a new chemical entity which offers a unique opportunity to treat cognitive deficits in DS. AEF0217 targets the CB1 receptor, a hyperactivity of which has been recently linked to cognitive deficit in DS. AEF0217 belongs to a new pharmacological class, named signalling specific inhibitors of the CB1 receptor (CB1-SSi), which allows to reverse cognitive deficit in animal models of DS. Because of its innovative MOA, AEF0217 is able to reduce selectively a hyperactivity of the CB1 without modifying the basal activity of this receptor and consequently without inducing behavioural side effects.
The overarching goal of the ICOD project is to make this innovative first-in-class drug available for DS individuals 7 years after clinical development initiation.
Keywords
Programme(s)
Call for proposal
(opens in new window) H2020-SC1-BHC-2018-2020
See other projects for this callSub call
H2020-SC1-2020-Two-Stage-RTD
Funding Scheme
RIA - Research and Innovation actionCoordinator
08003 Barcelona
Spain