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Drug repositioning and repurposing for Alzheimer disease
Ballard C, Aarsland D, Cummings J, O'Brien J, Mills R, Molinuevo JL, Fladby T, Williams G et al.. Nature reviews. Neurology, 2020-09-16
Topic: Amyloid, tau and blood-test diagnosis
Drug repositioning and repurposing can enhance traditional drug development efforts and could accelerate the identification of new treatments for individuals with Alzheimer disease (AD) dementia and mild cognitive impairment. Transcriptional profiling offers a new and highly efficient approach to the identification of novel candidates for repositioning and repurposing. In the future, novel AD transcriptional signatures from cells isolated at early stages of disease, or from human neurons or microglia that carry mutations that increase the risk of AD, might be used as probes to identify additional candidate drugs.
Key findings
- Transcriptional profiling offers a new and highly efficient approach to the identification of novel candidates for repositioning and repurposing.
- In the future, novel AD transcriptional signatures from cells isolated at early stages of disease, or from human neurons or microglia that carry mutations that increase the risk of AD, might be used as probes to identify additional candidate drugs.
- Phase II trials assessing repurposed agents must consider the best target population for a specific candidate therapy as well as the mechanism of action of the treatment.
- In this Review, we highlight promising compounds to prioritize for clinical trials in individuals with AD, and discuss the value of Delphi consensus methodology and evidence-based reviews to inform this prioritization process.
Why it matters
Drug repositioning and repurposing could speed up the identification of new treatments for people with Alzheimer's disease dementia and mild cognitive impairment. Transcriptional profiling is described as a new and highly efficient way to find candidate drugs, though future work is still needed to use early-stage disease signatures as probes.
Study details
- Evidence strengthLow
How this study scored 48/100
Published in a peer-reviewed journal. Scored zero for study design, sample size, effect reported, studied in people and randomised / blinded because the record does not state it.
| Component | Points | Why |
|---|---|---|
| Journal tier | 7 / 10 | Tier 3 — peer-reviewed journal (Nature reviews. Neurology) |
| Journal's citation record | 3 / 4 | Nature Reviews Neurology: journal h-index 269 (OpenAlex, 2026-10-29) |
| Study design | 0 / 12 | No study design recorded — scores zero, not guessed (not stated in the record — scored 0) |
| Strength of evidence | 1 / 5 | Rated “low” in our summary |
| Peer-review validation | 10 / 10 | Peer-reviewed. Published as an article in a named peer-reviewed journal. |
| How recent | 2 / 5 | 2020 — within 10 year(s) of the newest study here |
| Sample size | 0 / 4 | Participant count not stated — scores zero (not stated in the record — scored 0) |
| Effect reported | 0 / 3 | No effect size reported — scores zero (not stated in the record — scored 0) |
| Studied in people | 0 / 3 | Not stated whether this was studied in people — scores zero (not stated in the record — scored 0) |
| Randomised / blinded | 0 / 2 | No randomisation or blinding stated — scores zero (not stated in the record — scored 0) |
| Times cited | 5 / 7 | Cited 200 times (OpenAlex, 2026-10-10) |
| Citations per year | 4 / 5 | 25.0 citations a year over 8 year(s) since 2020 |
| Impact for its field and year | 3 / 6 | Cited 4.3× the average for papers of its field and year (OpenAlex FWCI) |
| Cited by studies here | 2 / 6 | Cited by 4 other studies in this library |
| Still being cited | 3 / 4 | Cited 100 times in 2024–2026 (2026 still in progress; OpenAlex) |
| Senior author's track record | 2 / 4 | Last-listed author Janet Sultana: h-index 26 (OpenAlex, whole career) |
| Institution's track record | 2 / 3 | University of Cambridge: institution h-index 1,973 (OpenAlex) |
| Multi-centre collaboration | 2 / 3 | OpenAlex lists 9 institution(s) in 5 countries |
| Indexed in PubMed | 2 / 2 | Indexed in PubMed (PMID 32939050) |
| Free to read | 0 / 2 | Behind a paywall — scores zero |
| Total | 48 / 100 | — |
What each of these 20 components measures, and what this score is not →
Read the original
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Cite this study APA, Vancouver, BibTeX · download .bib or .ris
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APA 7th
Ballard, C., Aarsland, D., Cummings, J., O'Brien, J., Mills, R., Molinuevo, J. L., et al. (2020). Drug repositioning and repurposing for Alzheimer disease. Nature reviews. Neurology. https://doi.org/10.1038/s41582-020-0397-4
Vancouver
Ballard C, Aarsland D, Cummings J, O'Brien J, Mills R, Molinuevo JL, et al. Drug repositioning and repurposing for Alzheimer disease. Nature reviews. Neurology. 2020. doi:10.1038/s41582-020-0397-4. PMID: 32939050.
BibTeX
@article{ballard2020-32939050,
author = {Ballard, C. and Aarsland, D. and Cummings, J. and O'Brien, J. and Mills, R. and Molinuevo, J. L. and Fladby, T. and Williams, G. and others},
title = {{Drug repositioning and repurposing for Alzheimer disease}},
journal = {Nature reviews. Neurology},
year = {2020},
doi = {10.1038/s41582-020-0397-4},
pmid = {32939050},
url = {https://doi.org/10.1038/s41582-020-0397-4}
}
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