Recent Advances and Challenges of mTOR Inhibitors Use in the Treatment of Patients with Tuberous Sclerosis Complex
Joint Authors
Palavra, Filipe
Robalo, Conceição
Reis, Flávio
Source
Oxidative Medicine and Cellular Longevity
Issue
Vol. 2017, Issue 2017 (31 Dec. 2017), pp.1-11, 11 p.
Publisher
Hindawi Publishing Corporation
Publication Date
2017-03-12
Country of Publication
Egypt
No. of Pages
11
Main Subjects
Abstract EN
Tuberous sclerosis complex (TSC) is a genetic condition characterized by the presence of benign, noninvasive, and tumor-like lesions called hamartomas that can affect multiple organ systems and are responsible for the clinical features of the disease.
In the majority of cases, TSC results from mutations in the TSC1 and TSC2 genes, leading to the overactivation of the mammalian target of rapamycin (mTOR) signalling pathway, which controls several cell functions, including cell growth, proliferation, and survival.
The establishment of a connection between TSC and mTOR led to the clinical use of drugs known as mTOR inhibitors (like rapamycin, also known as sirolimus and everolimus), which are becoming an increasingly interesting tool in the management of TSC-associated features, such as subependymal giant cell astrocytomas, renal angiomyolipomas, and also epilepsy.
However, the intrinsic characteristics of these drugs and their systemic effects in such a heterogeneous condition pose many challenges in clinical practice, so that some questions remain unanswered.
This article provides an overview of the pharmacological aspects of mTOR inhibitors about the clinical trials leading to their approval in TSC-related conditions and exposes current challenges and future directions associated with this promising therapeutic line.
American Psychological Association (APA)
Palavra, Filipe& Robalo, Conceição& Reis, Flávio. 2017. Recent Advances and Challenges of mTOR Inhibitors Use in the Treatment of Patients with Tuberous Sclerosis Complex. Oxidative Medicine and Cellular Longevity،Vol. 2017, no. 2017, pp.1-11.
https://search.emarefa.net/detail/BIM-1196740
Modern Language Association (MLA)
Palavra, Filipe…[et al.]. Recent Advances and Challenges of mTOR Inhibitors Use in the Treatment of Patients with Tuberous Sclerosis Complex. Oxidative Medicine and Cellular Longevity No. 2017 (2017), pp.1-11.
https://search.emarefa.net/detail/BIM-1196740
American Medical Association (AMA)
Palavra, Filipe& Robalo, Conceição& Reis, Flávio. Recent Advances and Challenges of mTOR Inhibitors Use in the Treatment of Patients with Tuberous Sclerosis Complex. Oxidative Medicine and Cellular Longevity. 2017. Vol. 2017, no. 2017, pp.1-11.
https://search.emarefa.net/detail/BIM-1196740
Data Type
Journal Articles
Language
English
Notes
Includes bibliographical references
Record ID
BIM-1196740