Article
Immunology
Paramarajan Piranavan, Andras Perl
Summary: The study showed that sirolimus is well tolerated and effective in controlling renal and non-renal manifestations of SLE over the long term, reducing anti-DNA antibody levels and steroid use while increasing complement levels. Adverse effects were minimal, with only a small number of patients experiencing mouth sores, headaches, and gastrointestinal discomfort, leading to discontinuation in only two cases.
CLINICAL IMMUNOLOGY
(2021)
Article
Dermatology
A. J. Duran-Romero, J. C. Hernandez-Rodriguez, J. Ortiz-Alvarez, J. J. Dominguez-Cruz, M. T. Monserrat-Garcia, J. Conejo-Mir Sanchez, J. Bernabeu-Wittel
Summary: Oral sirolimus shows good efficacy and safety in treating high-flow vascular malformations, with most patients experiencing partial relief and tolerating the treatment well. It can be a useful option for long-term stabilization of patients with high-flow vascular malformations.
CLINICAL AND EXPERIMENTAL DERMATOLOGY
(2022)
Article
Oncology
Andrew J. Wagner, Vinod Ravi, Richard F. Riedel, Kristen Ganjoo, Brian A. Van Tine, Rashmi Chugh, Lee Cranmer, Erlinda M. Gordon, Jason L. Hornick, Heng Du, Berta Grigorian, Anita N. Schmid, Shihe Hou, Katherine Harris, David J. Kwiatkowski, Neil P. Desai, Mark A. Dickson
Summary: The study evaluated the efficacy and safety of nab-sirolimus in patients with malignant PEComa, showing an overall response rate of 39% with rapid and durable responses. Patients with TSC2 mutations were more likely to achieve a positive response. nab-sirolimus was considered an important new treatment option for this disease based on response rate, disease control, and safety profile.
JOURNAL OF CLINICAL ONCOLOGY
(2021)
Article
Radiology, Nuclear Medicine & Medical Imaging
Rachelle Durand, Janet R. Reid, Jean B. Belasco, Sphoorti Shellikeri, Juan S. Calle-Toro, Anne Marie Cahill, Abhay Srinivasan
Summary: This study retrospectively characterized changes seen on MRI of children with extensive LMs treated with sirolimus. Significant reductions in volume and signal on T2-weighted MRI were observed with sirolimus treatment, with greater decreases in volume seen in younger children and craniocervical lesions.
AMERICAN JOURNAL OF ROENTGENOLOGY
(2021)
Review
Immunology
Martyna Tomaszewicz, Anna Ronowska, Maciej Zielinski, Agnieszka Jankowska-Kulawy, Piotr Trzonkowski
Summary: CD4(+)CD25(high)FoxP3(+) regulatory T cells (Tregs) are a subset of lymphocytes that can suppress the immune system through direct interactions with other cells and the secretion of suppressive compounds. Modulating the metabolic program of Tregs may improve or weaken their stability and function, making it a crucial tool in managing infections and autoimmunity.
FRONTIERS IN IMMUNOLOGY
(2023)
Article
Clinical Neurology
Marc Pawlitzki, Christopher Nelke, Melanie Korsen, Sven G. Meuth, Tobias Ruck
Summary: The study provides evidence for the use of sirolimus in treating patients with IBM, showing significant and prolonged clinical improvement without adverse effects. While no meaningful changes were observed in CD8(+) T-cell subsets, further research should explore the potential myoprotective effects of sirolimus.
EUROPEAN JOURNAL OF NEUROLOGY
(2022)
Review
Gastroenterology & Hepatology
Xiangyu Yan, Songhan Huang, Yang Yang, Ziwen Lu, Feiyu Li, Liyong Jiang, Yong Jiang, Jun Liu
Summary: This systematic review and meta-analysis found that the use of mammalian target of rapamycin (mTOR) inhibitors, sirolimus (SRL) or everolimus (EVL), as immunosuppressants in liver transplantation (LT) recipients with hepatocellular carcinoma (HCC) improved overall survival (OS) and recurrence-free survival (RFS) while reducing the risk of renal toxicity.
LIVER TRANSPLANTATION
(2022)
Article
Oncology
Jonathan A. Chacon-Barahona, Jeffrey P. MacKeigan, Nathan J. Lanning
Summary: In this study, we examined the viability of cancer cells under different metabolic conditions. We found that the pentose phosphate pathway is essential for survival under glycolytic conditions, while mTOR signaling is essential for survival under oxidative phosphorylation conditions. Additionally, we identified similarities and differences in the gene expression profiles of glycolytic and non-glycolytic tumors, supporting the classification of tumors based on metabolic phenotype.
Article
Medicine, Research & Experimental
Kevin M. Motz, Ioan A. Lina, Idris Samad, Michael K. Murphy, Madhavi Duvvuri, Ruth J. Davis, Alexander Gelbard, Liam Chung, Yee Chan-Li, Samuel Collins, Jonathan D. Powell, Jennifer H. Elisseeff, Maureen R. Horton, Alexander T. Hillel
Summary: Laryngotracheal stenosis (LTS) is characterized by fibrotic narrowing of the larynx and trachea and the involvement of CD4+ T cells. This study investigates the role of mTOR signaling in CD4+ T cells in promoting LTS fibrosis and suggests that targeting mTOR with sirolimus can effectively treat LTS by inhibiting profibrotic Th17 cells. The findings also suggest the possibility of local delivery of sirolimus with a drug-eluting stent for LTS treatment.
Article
Oncology
Elise F. F. Nassif, Cissimol P. P. Joseph, Rossana Lazcano, Jocelyn T. T. Joseph, Prapassorn Thirasastr, Alexander J. J. Lazar, Neeta Somaiah
Summary: TSC-mutated sarcomas are rare types of sarcoma that are sensitive to mTOR inhibitors. Nab-sirolimus, an albumin-bound mTOR inhibitor, is currently the only FDA-approved treatment for tumors with TSC mutation. We report two cases where patients with TSC-mutated sarcomas responded well to the combination of gemcitabine and sirolimus after prior treatment with gemcitabine-based chemotherapy and nab-sirolimus. This combination could be a viable therapeutic option for patients who have failed nab-sirolimus treatment.
FRONTIERS IN ONCOLOGY
(2023)
Article
Clinical Neurology
Daniel Ebrahimi-Fakhari, Gabrielle Stires, Eunice Hahn, Darcy Krueger, David Neal Franz
Summary: Maternal sirolimus appears to be a safe and effective treatment option for prenatally detected rhabdomyomas due to tuberous sclerosis complex. Different dosing regimens and trough levels were used, with gradual shrinkage of cardiac rhabdomyomas observed in all patients. Weekly follow-up visits with fetal ultrasound, echocardiography, and laboratory work are recommended during the treatment period.
PEDIATRIC NEUROLOGY
(2021)
Article
Multidisciplinary Sciences
Ping-Min Chen, Eri Katsuyama, Abhigyan Satyam, Hao Li, Jose Rubio, Sungwook Jung, Sylvia Andrzejewski, J. David Becherer, Maria G. Tsokos, Reza Abdi, George C. Tsokos
Summary: CD38 up-regulation in CD8(+) T cells of SLE patients negatively affects T cell function by inhibiting mitochondrial fitness and mitophagy. Targeted administration of a CD38 inhibitor improves T cell function and viral clearance, suggesting CD38 as a potential therapeutic target to mitigate infection rates in people with SLE.
Article
Endocrinology & Metabolism
G. Lanzolla, M. N. Maglionico, S. Comi, F. Menconi, P. Piaggi, C. Posarelli, M. Figus, C. Marcocci, M. Marino
Summary: This study aimed to evaluate the efficacy of sirolimus as a second-line treatment for Graves' orbitopathy. The results showed that sirolimus had a better overall outcome at 24 weeks compared to methylprednisolone, improving quality of life, reducing proptosis and clinical activity score, with no serious adverse events observed.
JOURNAL OF ENDOCRINOLOGICAL INVESTIGATION
(2022)
Review
Immunology
Christian Geier, Andras Perl
Summary: The mTOR pathway plays a critical role in cell lineage specification, especially in adaptive immune responses. Its inhibition can reverse aberrant cell differentiation, potentially benefiting the treatment of autoimmune diseases and viral infections.
AUTOIMMUNITY REVIEWS
(2021)
Article
Medicine, Research & Experimental
Hong Zhang, Xiaoyang Su, Stephen K. Burley, X. F. Steven Zheng
Summary: This study reveals the molecular mechanism by which mTOR promotes the "Warburg Effect" in HCC, which is important for the metabolism and development of HCC, as well as the anticancer response of mTOR-targeted therapy.