Advancing Down syndrome sustained therapies: therapeutic target reverses memory impairments in aged mouse model

A study led by MELIS-UPF, in collaboration with the Hospital del Mar Research Institute and IDIBAPS, validated a promising long-term pharmacological strategy to treat cognitive decline in Down syndrome. This research demonstrates that cannabinoid receptor 1 (CB1) is dysregulated in the brain of people with Down syndrome. Sustained pharmacological blockade of CB1 significantly improves memory deficits and neuroinflammation in a mouse model of the condition, showing cognitive benefits even when age-related neurodegeneration is not prevented. This study provides data needed to pave the way for long-term clinical trials in humans.

By directly linking receptor dysregulation to neurological outcomes, this effort provides the critical benchmarking data needed to pave the way for long-term clinical trials in humans.

Reference:
Vázquez-Oliver A, Pérez-García S, Romero-Pérez R, Pizarro N, Galarraga-Shinin D, Molina-Porcel L, de la Torre R, Maldonado R, Ozaita A (2025). Targeting dysregulated CB1 receptor in a Down syndrome mouse model improves neurological outcomes. Alzheimer’s & Dementia. DOI: 10.1002/alz.70874