PMID- 27815691
OWN - NLM
STAT- MEDLINE
DCOM- 20170807
LR  - 20211204
IS  - 1534-6293 (Electronic)
IS  - 1528-4042 (Linking)
VI  - 16
IP  - 12
DP  - 2016 Dec
TI  - mTOR Inhibitors in Children: Current Indications and Future Directions in 
      Neurology.
PG  - 102
AB  - The mammalian/mechanistic target of rapamycin (mTOR) pathway is a key signaling 
      pathway that has been implicated in genetic epilepsy syndromes, neurodegenerative 
      diseases, and conditions associated with autism spectrum disorder and cognitive 
      impairment. The mTOR pathway has become an exciting treatment target for these 
      various disorders, with mTOR inhibitors such as rapamycin being studied for their 
      potential therapeutic applications. In particular, tuberous sclerosis complex 
      (TSC) is a genetic disorder resulting from overactivation of the mTOR pathway, 
      and pharmacologic therapy with mTOR inhibitors has emerged as a viable treatment 
      option for the systemic manifestations of the disease. In this review, we discuss 
      the approved indications for mTOR inhibitors in TSC, the potential future 
      applications of mTOR inhibitors in TSC and other neurological conditions, and the 
      safety considerations applicable to mTOR therapy in the pediatric population.
FAU - Jeong, Anna
AU  - Jeong A
AD  - Department of Neurology and the Hope Center for Neurological Disorders, 
      Washington University School of Medicine, 660 South Euclid Avenue, Box 8111, St. 
      Louis, MO, 63110, USA.
FAU - Wong, Michael
AU  - Wong M
AD  - Department of Neurology and the Hope Center for Neurological Disorders, 
      Washington University School of Medicine, 660 South Euclid Avenue, Box 8111, St. 
      Louis, MO, 63110, USA. wong_m@wustl.edu.
LA  - eng
PT  - Journal Article
PT  - Review
PL  - United States
TA  - Curr Neurol Neurosci Rep
JT  - Current neurology and neuroscience reports
JID - 100931790
RN  - 0 (Enzyme Inhibitors)
RN  - EC 2.7.1.1 (MTOR protein, human)
RN  - EC 2.7.11.1 (TOR Serine-Threonine Kinases)
RN  - W36ZG6FT64 (Sirolimus)
SB  - IM
MH  - Autism Spectrum Disorder/drug therapy/metabolism
MH  - Child
MH  - Enzyme Inhibitors/pharmacology/*therapeutic use
MH  - Epilepsy/*drug therapy
MH  - Humans
MH  - Signal Transduction/drug effects
MH  - Sirolimus/pharmacology/*therapeutic use
MH  - TOR Serine-Threonine Kinases/*antagonists & inhibitors/metabolism
MH  - Tuberous Sclerosis/*drug therapy/metabolism
OTO - NOTNLM
OT  - Everolimus
OT  - Rapamycin
OT  - Sirolimus
OT  - Tuberous sclerosis
OT  - mTOR
EDAT- 2016/11/07 06:00
MHDA- 2017/08/08 06:00
CRDT- 2016/11/06 06:00
PHST- 2016/11/06 06:00 [entrez]
PHST- 2016/11/07 06:00 [pubmed]
PHST- 2017/08/08 06:00 [medline]
AID - 10.1007/s11910-016-0708-8 [pii]
AID - 10.1007/s11910-016-0708-8 [doi]
PST - ppublish
SO  - Curr Neurol Neurosci Rep. 2016 Dec;16(12):102. doi: 10.1007/s11910-016-0708-8.