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Updated: Feb 5, 2026
Comprehensive Autopsy Program for Individuals with Multiple Sclerosis
Published on: July 19, 2019
Promising effect of rapamycin on multiple sclerosis
Bahram Bagherpour1, Mansoor Salehi2, Rasool Jafari3
1Acquired Immunodeficiency Research Center, Isfahan University of Medical Sciences, Isfahan, Iran; Gastrointestinal and Hepatobiliary Diseases Research Center, Poursina Hakim Research Institute for Health Care Development, Isfahan, Iran.
Abstract:
The routine therapies for relapsing-remitting multiple sclerosis (RRMS) are common disease-modifying medications, yet are not effective in all patients. The aim of the present clinical trial was to evaluate the therapeutic effects of rapamycin on the clinical and radiological aspects, regulatory T cells proliferation and FOXP3 and GARP gene expression in the patients with RRMS. In this study, eight patients with RRMS were chosen and included in the trial. Patients received rapamycin (Rapacan, Biocon, India) for six months. Magnetic resonance imaging (MRI) of the patients' brain was taken before and after the therapy. Patients' expanded disability status scale (EDSS), and FoxP3 and GARP gene expression, and Treg cell proliferation were also been evaluated. All the patients had some degrees of significant reduction in mean plaque area size (P = 0.012, Z = -2.520), and minimum and maximum size of the plaques (P = 0.012, Z = -2.521). EDSS of 50% of patients was decreased after the treatment, yet it was not significant (P = 0.059, Z = -1.89). The expression rate of FOXP3 (P = 0.003) and GARP genes in Tregs increased after the therapy. We found a promising response to rapamycin among our cases with minor side effects and it may be considered as a therapeutic option of this disease.
Insights
Rapamycin shows promise for treating relapsing-remitting multiple sclerosis (RRMS) by reducing brain lesions and increasing regulatory T cell gene expression. This therapy may offer a new option for RRMS patients unresponsive to standard treatments.
Area of Science:
- Neuroimmunology
- Clinical Therapeutics
- Molecular Medicine
Background:
- Standard disease-modifying medications for relapsing-remitting multiple sclerosis (RRMS) are not universally effective.
- There is a need for novel therapeutic strategies to manage RRMS.
- Regulatory T cells (Tregs) play a crucial role in immune regulation and have been implicated in MS pathogenesis.
Purpose of the Study:
- To evaluate the efficacy of rapamycin in patients with RRMS.
- To assess the impact of rapamycin on clinical and radiological outcomes.
- To investigate the effects of rapamycin on Treg proliferation and FOXP3/GARP gene expression.
Main Methods:
- A clinical trial involving eight RRMS patients treated with rapamycin for six months.
- Brain MRI scans were performed before and after treatment to assess lesion size.
- Expanded Disability Status Scale (EDSS), Treg cell proliferation, and FOXP3/GARP gene expression were evaluated.
Main Results:
- Significant reduction in mean, minimum, and maximum plaque area size observed post-rapamycin therapy (P=0.012).
- A decrease in EDSS was noted in 50% of patients, though not statistically significant (P=0.059).
- Increased expression of FOXP3 (P=0.003) and GARP genes in Tregs was observed after treatment.
Conclusions:
- Rapamycin demonstrated a promising therapeutic response in RRMS patients.
- The drug led to significant radiological improvements and modulated key immune markers.
- Rapamycin may represent a viable therapeutic option for RRMS, with minor side effects observed.
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