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Side Effects of Minocycline Treatment in Patients With Fragile X Syndrome and Exploration of Outcome Measures
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Minocycline can rescue the dendritic spine and synaptic structural abnormalities in the fragile X knock-out mouse. This is a review and preliminary survey to document side effects and potential outcome measures for minocycline use in the treatment of individuals with fragile X syndrome. We surveyed 50 patients with fragile X syndrome who received minocycline for at least 2 weeks and found that the most common reported side effect is gastrointestinal difficulty, including loss of appetite. The families reported an improvement in language and behavioral areas. Outcome measures in the design of future randomized clinical trials should include both behavioral and language measures. As with any other treatments, we emphasize that randomized clinical trials are needed to determine the efficacy of minocycline in fragile X syndrome.
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“This work stimulated a survey study of families whose children were treated with minocycline, and 70% noted an improvement in behavior or language (126).”
From Fragile X syndrome: causes, diagnosis, mechanisms, and therapeutics (2012) · cites this paper as [126]
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Puścian, A., Winiarski, M., Borowska, J., Łęski, S., Górkiewicz, T., Chaturvedi, M., Nowicka, K., Wołyniak, M., Chmielewska, J. J., Nikolaev, T., et al. (2022) · Molecular Psychiatry · doi:10.1038/s41380-022-01527-5 · Cited by 11
“In the case of the FX patients application of minocycline, a drug repeatedly shown to normalize both, MMP-9 levels and behavioral performance in mice [15, 23, 24], turned only partially effective in improving cognitive function and only mildly helped in alleviating anxiety-related symptoms [25, 27, 93, 94].”
BackgroundClinical significance of matrix metalloproteinase-9 in Fragile X Syndrome
Laroui, A., Galarneau, L., Abolghasemi, A., Benachenhou, S., Plantefève, R., Bouchouirab, F. Z., Lepage, J., Corbin, F., & Çaku, A. (2022) · Scientific Reports · doi:10.1038/s41598-022-19476-y · Cited by 10
Fragile X Syndrome and Targeted Treatments
Tassanakijpanich, N., Cabal-Herrera, A. M., Salcedo‐Arellano, M. J., & Hagerman, R. J. (2020) · Journal of Biomedicine and Translational Research · doi:10.14710/jbtr.v6i1.7321 · Cited by 10
“In a survey of clinical response to minocycline in 50 patients with FXS performed by Utari et al. (2010), the most common side effect reported was gastrointestinal issues such as loss of appetite, gastrointestinal discomfort and diarrhea.”
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Repurposing of Minocycline, a Tetracycline Antibiotic, for Neurodegenerative Disorders
Bhandari, R., Sood, P., Thakur, D., & Kuhad, A. (2023) · Springer eBooks · doi:10.1007/978-981-19-5399-6_27 · Cited by 7
Potential Use of Modulators of Oxidative Stress as Add-on Therapy in Patients with Anxiety Disorders
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“Minocycline is a second generation tetracycline derivative, introduced as an antimicrobial agent in 1967 [158, 159], which can also be considered belonging to”
BackgroundEmerging autism and Fragile X syndrome treatments
Parkhill, M., Salmaso, N., D’Angiulli, A., Lee, V., & Aguilar‐Valles, A. (2025) · Trends in Pharmacological Sciences · doi:10.1016/j.tips.2025.02.004 · Cited by 7
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“Unfortunately, minocycline may cause gastrointestinal effects, such as loss of appetite [14] and, in the long term, hyperpigmentation of various tissues, autoimmune disorders (systemic lupus erythematosus or autoimmune hepatitis), and serious hypersensitivity reactions (hypersensitivity syndrome reaction, pneumonitis…”
BackgroundDetermination of various risk factors associated with acne vulgaris infection in Quetta, Pakistan
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Overview of Targeted Double-Blind, Placebo-Controlled Clinical Trials in Fragile X Syndrome
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Self-citationPharmacological Treatments for Fragile X Syndrome Based on Synaptic Dysfunction
Telias, M. (2019) · Current Pharmaceutical Design · doi:10.2174/1381612825666191102165206 · Cited by 4
Neurobiology of Autism Spectrum Disorders
Martins, G. J. (2017) · Springer eBooks · doi:10.1007/978-3-319-42713-3_2 · Cited by 4
How dendritic spines shape is determined by MMP-9 activity in FXS
Dziembowska, M. (2023) · International review of neurobiology · doi:10.1016/bs.irn.2023.10.001 · Cited by 2
Therapeutic approaches for the future treatment of Fragile X
Sastre, A., Campillo, N. E., Gil, C., & Martı́nez, A. (2015) · Current Opinion in Behavioral Sciences · doi:10.1016/j.cobeha.2015.01.003 · Cited by 2
Hare, E. B., Lozano, R., Partington, L. C., & Hagerman, R. J. (2016) · Springer eBooks · doi:10.1007/978-3-319-18096-0_64 · Cited by 2
Self-citationMatrix Metalloproteinases in Fragile X Syndrome
Ethell, D. W., & Sidhu, H. (2017) · Elsevier eBooks · doi:10.1016/b978-0-12-804461-2.00015-9 · Cited by 2
mRNA Translation and Proteasomal Degradation in Health and Neurological Disease
Isaac, A. R., Cozachenco, D., Wagner, B. D. A., Chauvet, M. G., & Lourenco, M. V. (2026) · Physiology · doi:10.1152/physiol.00023.2025 · Cited by 1
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Fragile X Clinical Features and Neurobiology
Leigh, M. J., & Hagerman, R. J. (2013) · Elsevier eBooks · doi:10.1016/b978-0-12-397267-5.00044-3 · Cited by 1
Self-citationPhase II and III drugs for the treatment of fragile X syndrome
Politte, L. C., & McDougle, C. J. (2012) · Expert Opinion on Orphan Drugs · doi:10.1080/21678707.2013.750240 · Cited by 1
Fragile X clinical features and neurobiology
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Self-citationFragile X Syndrome: From Pathophysiology to New Therapeutic Perspectives
D’Antoni, S., Spatuzza, M., Carmela, M., Aloisi, E., Musumeci, S. A., & Vincenza, M. (2012) · InTech eBooks · doi:10.5772/31387
“Clinical trials have been started for patients with FXS and an open-label trial has been recently completed to study the effects of minocycline in patients with FXS (Utari et al., 2010).”
BackgroundGrieco, J. C. (2015) · Digital Commons - University of South Florida (University of South Florida)
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Fragile X Syndrome: Steps towards Therapy
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Social dysfunction and mental retardation
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Fragile X clinical features and neurobiology
Leigh, M. J., & Hagerman, R. J. (2020) · Elsevier eBooks · doi:10.1016/b978-0-12-813955-4.00022-2
Self-citation
Metadata
- Authors
- Utari, A., Chonchaiya, W., Rivera, S. M., Schneider, A., Hagerman, R. J., Faradz, S. M. H., Ethell, I. M., & Nguyen, D. V.
- Affiliations
- University of California Davis Medical Center (US)
- Diponegoro University (ID)
- University of California, Riverside (US)
- University of California, Davis (US)
- ORCID
- Venue
- American Journal on Intellectual and Developmental Disabilities
- Publisher
- Informa UK Limited
- Published
- 2010-09-01
- Volume
- 115
- Issue
- 5
- Pages
- 433-443
- ISSN
- 1944-7558 (ISSN-L)
- 1944-7515
- PMC
- PMC4031088
- OpenAlex
- W2134986933
- Cited by
- 105
- References
- 71
- Citations by year
- 2026: 3 · 2025: 1 · 2024: 3 · 2023: 3 · 2022: 3 · 2021: 3 · 2020: 6 · 2019: 9 · 2018: 2 · 2017: 8 · 2016: 3 · 2015: 5 · 2014: 11 · 2013: 17 · 2012: 14
- FWCI
- 8.10
- Type
- journal article
- Language
- en
- Open access
- green
- europepmc.org
- Locations
- American Journal on Intellectual and Developmental Disabilities (publishedVersion)
- PubMed (publishedVersion)
- Europe PMC (PubMed Central) (acceptedVersion)
- PubMed Central (acceptedVersion)
- Funders
- Centers for Disease Control and Prevention (90DD05969)
- National Center for Research Resources (UL1RR024146)
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (HD036071, HD 02274, P30HD002274, R01HD036071)
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Genetics and Neurodevelopmental Disorders
- Life Sciences › Neuroscience › Cognitive Neuroscience › Autism Spectrum Disorder Research
- Health Sciences › Medicine › Public Health, Environmental and Occupational Health › Down syndrome and intellectual disability research
- Keywords
- minocycline, fragile X syndrome, adverse drug reactions, gastrointestinal adverse effects, appetite loss, language outcomes, behavioral effects, outcome measures, randomized controlled trials, tetracycline antibiotics, synaptic abnormalities
- MeSH terms
- Adolescent, Anti-Bacterial Agents, Child, Child Behavior Disorders, Child, Preschool, Female, Follow-Up Studies, Fragile X Syndrome, Humans, Infant, Language Development Disorders, Male, Minocycline, Randomized Controlled Trials as Topic, Neuroprotective Agents, Young Adult
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