Sirolimus for angiomyolipoma in tuberous sclerosis complex or lymphangioleiomyomatosis.

By J. Bissler, F. McCormack, L. Young, J.M. Elwing, G. Chuck, J. Leonard, V. Schmithorst, T. Laor, A. Brody, J. Bean and 2 more. New England Journal of Medicine, 2008

1,215 citations recorded by the source corpus.

Subfields: Molecular Biology, Neurology, Physiology

View Paper: https://www.semanticscholar.org/paper/bd13fda685871f365ef33ebafd8bcbc368c21115

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Citations in the corpus

The TSC1-TSC2 complex: a molecular switchboard controlling cell growth. (2008, result): “Although it is still early days with these trials, the results thus far demonstrate that mTORC1 inhibitors reproducibly decrease the size of tumours with TSC gene mutations [54–56].”
LKB1 and AMPK control of mTOR signalling and growth (2009, background): “…Cloughesy et al. 2008) Furthermore, in a pair of phase II clinical trials involving tuberous sclerosis (TSC) and lymphangioleiomyomatosis (LAM) patients, partial responses to the rapamycin analogues were observed, including regression of angiomyoliomas (Bissler et al. 2008, Davies et al. 2008).”
Disentangling the heterogeneity of autism spectrum disorder through genetic findings (2014, neutral): “33,34 Now, with safety profiles established, several international studies are investigating the use of mTOR inhibitors for neurocogni tive deficits in children with TSC. 35 The main challenges to successful treatment with mTOR inhibitors lie in the adverse effects of immunosuppressants and…”
PTEN loss in the continuum of common cancers, rare syndromes and mouse models (2011, neutral): “For example, sirolimus was tested in a Phase II trial of patients with tuberous sclerosis, which, like Cowden syndrome, is a highly morbid familial syndrome in which the loss of a tumour suppressor gene leads to mTOR activatio”
Multifaceted role of mTOR (mammalian target of rapamycin) signaling pathway in human health and disease (2023, background): “gov number, NCT00457808, and NCT00414648).(241,242) Nab-sirolimus (albuminbound nanoparticles-based sirolimus formulation) displayed a strong antitumor effect in cases with perivascular epithelioid cell tumors (PEComa).”
Multi-compartment microscopic diffusion imaging (2016, background): “Our findings have clear translational significance as SMT may help oversee treatment success in promising clinical trials (Franz et al., 2006; Bissler et al., 2008; Krueger et al., 2010; Tillema et al., 2012) where patients receive mTOR inhibitors such as everolimus and sirolimus.”
The PI3K pathway in human disease (2017, neutral): “Commonly observed brain lesions are cerebral hamartomas and subepen-dymal giant cell tumors (SEGAs) (Franz et al., 2014; Jo´ (cid:1) zwiak et al., 2016).”
Efficacy and Safety of Sirolimus in the Treatment of Complicated Vascular Anomalies (2016, neutral): “CR 0 0 PR 47 (83) [70–95] 45 (85) [73–97] PD 7 (12) [2–23] 8 (15) [3–27] SD 3 (5) [0–13] 0”
Small molecule inhibition of the autophagy kinase ULK1 and identification of ULK1 substrates (2015, background): “In hamartoma syndromes, such as tuberous sclerosis complex and Peutz-Jeghers syndrome, inactivating mutations in the tumor suppressors TSC1, TSC2, PTEN, and LKB1/STK11 result in mTOR hyperactivation, and these mutations are targetable by mTOR inhibitors (Bissler et al., 2008).”
Control of PD-L1 Expression by Oncogenic Activation of the AKT-mTOR Pathway in Non-Small Cell Lung Cancer. (2016, background): “Rapamycin has a black box warning from the FDA stemming from a study of renal transplant patients who were also taking cyclosporine and corticosteroids (43), but multiple trials of single-agent rapamycin or rapamycin analogues in cancer patients have shown no evidence of increased incidence of immun”