Pharmacological interventions to improve cognition and adaptive functioning in Down syndrome: Strides to date

• Published in: American journal of medical genetics. Part A Vol. 173; no. 11; pp. 3029 - 3041
• Main Authors: Hart, Sarah J., Visootsak, Jeannie, Tamburri, Paul, Phuong, Patrick, Baumer, Nicole, Hernandez, Maria‐Clemencia, Skotko, Brian G., Ochoa‐Lubinoff, Cesar, Liogier D'Ardhuy, Xavier, Kishnani, Priya S., Spiridigliozzi, Gail A.
• Format: Journal Article
• Language: English
• Published: United States Wiley Subscription Services, Inc 01.11.2017
• Subjects: Animal models; Animals; Clinical trials; Cognition; Cognition & reasoning; Cognition - drug effects; Cognitive Dysfunction - drug therapy; Cognitive Dysfunction - genetics; Cognitive Dysfunction - physiopathology; Down syndrome; Down Syndrome - drug therapy; Down Syndrome - genetics; Down Syndrome - physiopathology; Down's syndrome; Drug discovery; Humans; interventions; Mice; Pharmaceutical industry; clinical trials; interventions; Down syndrome; cognition
• ISSN: 1552-4825; 1552-4833
• DOI: 10.1002/ajmg.a.38465

Although an increasing number of clinical trials have been developed for cognition in Down syndrome, there has been limited success to date in identifying effective interventions. This review describes the progression from pre‐clinical studies with mouse models to human clinical trials research using pharmacological interventions to improve cognition and adaptive functioning in Down syndrome. We also provide considerations for investigators when conducting human clinical trials and describe strategies for the pharmaceutical industry to advance the field in drug discovery for Down syndrome. Future research focusing on earlier pharmaceutical interventions, development of appropriate outcome measures, and greater collaboration between industry, academia, advocacy, and regulatory groups will be important for addressing limitations from prior studies and developing potential effective interventions for cognition in Down syndrome.