The ICOD project aims to develop a pharmacological therapy of cognitive deficits in Down syndrome which is lacking today. A pharmacotherapy in this area will change clinical practice providing practitioner with a long-awaited therapeutic tool that will improve considerably the healthcare paradigm for treating cognitive deficits in individuals with Down syndrome.
The intellectual disability in Down syndrome and its social dependence, with the associated high health care costs, derive from a profound impairment in working memory and cognitive flexibility. This deficit has major consequences in the performance of simple cognitive tasks. Based on the preclinical proof of concept of AEF0217, it will be reasonable to expect that a patient, after several weeks or months of treatment with AEF0217 will improve enough cognitive flexibility and adaptive behaviours to significantly improve his/her daily skills, capacity of communication and social relationships and consequently his/her autonomy with a significant improvement in quality of life and reduction in healthcare costs.
Another significant advantage of AEF0217 is the excellent safety and tolerability which has been confirmed following the Phase 1 clinical program in healthy volunteers and in subjects with DS. This will allow a ready acceptance by families of individuals with DS facilitating development and adherence to the treatment. Thus, families are extremely protective of children with DS and consequently highly concerned by the adverse effects of new potential treatments, which can become an unsurmountable barrier to the adoption of a new treatment.
In conclusion, AEF0217 meets the needs of subjects with Down syndrome, their families and society.
