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sirolimus + hydroxychloroquine (Brigham and Women's Hospital)
- Development stage
- Unknown
- Lead developer
- Brigham and Women's Hospital
- Modality
- Small Molecules
- Administration
- Oral
01
Overview
Sirolimus + hydroxychloroquine is an investigational combination therapy being evaluated for the treatment of lymphangioleiomyomatosis (LAM) and tuberous sclerosis complex (TSC). Sirolimus, a well-established mTOR inhibitor, is used to suppress the abnormal proliferation of smooth muscle-like cells in LAM. However, mTOR inhibition can induce autophagy, a cellular survival mechanism that may limit the efficacy of sirolimus monotherapy. Hydroxychloroquine is included in this regimen as an autophagy inhibitor to block this survival pathway and potentially enhance the destruction of LAM cells. This combination was primarily investigated in the Phase I SAIL (Sirolimus and Autophagy Inhibition in LAM) trial conducted by Brigham and Women's Hospital.
- Other names
- sirolimus and hydroxychloroquinerapamycin and hydroxychloroquine
02
Targets
mTOR (Mammalian target of rapamycin kinase)
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