Pharmacological interventions to improve cognition and adaptive functioning in down syndrome: strides to date.
Abstract
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.
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Pharmaceutical interventions translating these findings back into humans have not been successful.
“However, translating these findings back into humans in the form of pharmaceutical interventions has yet to prove successful (Hart et al., 2017).”
From Neurodevelopment in Down syndrome: Concordance in humans and models (2022) · cites this paper as Hart et al., 2017
None of the clinical trials of GABA antagonists have resulted in an improvement in cognition in people with Down syndrome.
“none of these trials have resulted in an improvement.”
From Neurodevelopment in Down syndrome: Concordance in humans and models (2022) · cites this paper as Hart et al., 2017
Several clinical trials have tested the effect of GABA antagonists on cognition in people with Down syndrome.
“several different clinical trials have tested the effect of GABA antagonists on cognition in people with DS (Hart et al., 2017)”
From Neurodevelopment in Down syndrome: Concordance in humans and models (2022) · cites this paper as Hart et al., 2017
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“Based on data from mouse models, several different clinical trials have tested the effect of GABA antagonists on cognition in people with DS (Hart et al., 2017), however, none of these trials have resulted in an improvement.”
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Self-citation
- Authors
- Hart, S. J., Visootsak, J., Tamburri, P., Phuong, P., Baumer, N., Hernandez, M., Skotko, B. G., Ochoa‐Lubinoff, C., Liogier D'Ardhuy, X., Kishnani, P. S., & Spiridigliozzi, G. A.
- Affiliations
- Duke Medical Center (US)
- F. Hoffmann‐La Roche Roche Pharma Research and Early Development Neuroscience, Roche Innovation Center New York New York New York
- Boston Children's Hospital (US)
- F. Hoffmann-La Roche AG (CH)
- Harvard University (US)
- Rush University Medical Center (US)
- F. Hoffmann‐La Roche Roche Pharma Research and Early Development Neuroscience, Roche Innovation Center Basel Basel Switzerland
- Corresponding author
- Hart, Sarah J.
- ORCID
- Venue
- American Journal of Medical Genetics Part A
- Publisher
- Wiley
- Published
- 2017-09-08
- Volume
- 173
- Issue
- 11
- Pages
- 3029-3041
- ISSN
- 1552-4825 (ISSN-L)
- 1552-4833
- OpenAlex
- W2751709943
- Cited by
- 54
- References
- 111
- Citations by year
- 2026: 4 · 2025: 5 · 2024: 9 · 2023: 1 · 2022: 9 · 2021: 9 · 2020: 7 · 2019: 8 · 2018: 1 · 2017: 1
- FWCI
- 0.76
- Type
- journal article
- Language
- en
- Open access
- closed
- Locations
- American Journal of Medical Genetics Part A (publishedVersion)
- PubMed (publishedVersion)
- Topics
- Health Sciences › Medicine › Public Health, Environmental and Occupational Health › Down syndrome and intellectual disability research
- Health Sciences › Medicine › Physiology › Alzheimer's disease research and treatments
- Health Sciences › Medicine › Psychiatry and Mental health › Dementia and Cognitive Impairment Research
- Keywords
- Down syndrome, pharmacological interventions, cognitive impairment, adaptive behavior, mouse model, clinical trials, drug discovery, outcome measures, pharmaceutical industry collaboration
- MeSH terms
- Animals, Cognition, Down Syndrome, Humans, Mice, Cognitive Dysfunction
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