PMID- 32409220
OWN - NLM
STAT- MEDLINE
DCOM- 20201105
LR  - 20211204
IS  - 1532-2653 (Electronic)
IS  - 0967-5868 (Linking)
VI  - 77
DP  - 2020 Jul
TI  - The efficacy of cannabidiol on renal angiomyolipoma and subependymal giant cell 
      tumor volume in tuberous sclerosis complex.
PG  - 85-88
LID - S0967-5868(20)30565-8 [pii]
LID - 10.1016/j.jocn.2020.05.030 [doi]
AB  - In patients with tuberous sclerosis complex (TSC) the upregulation of the 
      mechanistic target of rapamycin (mTOR) pathway leads to the development and 
      growth of subependymal giant cell tumors (SGCTs) and renal angiomyolipomas 
      (AMLs). Drugs that inhibit the mTOR pathway, such as sirolimus, can reduce the 
      size of both SGCTs and AMLs. Recent preclinical studies have suggested 
      cannabidiol (CBD) may mediate the mTOR pathway, however, its exact effects are 
      unclear. This study examines the volumes of SGCTs and renal AMLs in patients with 
      TSC during treatment with purified CBD for refractory epilepsy. We 
      retrospectively reviewed the medical records of patients with TSC with 
      radiological evidence of AMLs and SGCTs who were being treated with plant-derived 
      highly purified CBD in oral solution (Epidiolex(R), GW Research Ltd) for refractory 
      epilepsy at Massachusetts General Hospital. Patients who had surgical 
      intervention for AMLs or SGCTS, and patients who had been treated with mTOR 
      inhibitors were excluded. The volumes of SGCTs and dominant renal AML were 
      measured before and after CBD initiation using abdominal and brain scans and 
      compared. Patient demographics and CBD doses were collected from medical records. 
      Six out of the seven dominant renal AMLs and three out of the three SGCTs 
      increased in volume during CBD treatment. One AML had a decrease in volume after 
      CBD initiation which was not considered significant. The results suggest that 
      unlike mTOR inhibitors, CBD treatment does not decrease the volume of SGCTs or 
      AMLs in TSC patients.
CI  - Copyright (c) 2020 Elsevier Ltd. All rights reserved.
FAU - Barnett, James R
AU  - Barnett JR
AD  - Massachusetts General Hospital, Boston, MA, USA.
FAU - Grinspoon, Reid A
AU  - Grinspoon RA
AD  - Massachusetts General Hospital, Boston, MA, USA.
FAU - Harisinghani, Mukesh
AU  - Harisinghani M
AD  - Massachusetts General Hospital, Boston, MA, USA.
FAU - Caruso, Paul A
AU  - Caruso PA
AD  - Massachusetts General Hospital, Boston, MA, USA.
FAU - Thiele, Elizabeth A
AU  - Thiele EA
AD  - Massachusetts General Hospital, Boston, MA, USA. Electronic address: 
      ETHIELE@mgh.harvard.edu.
LA  - eng
PT  - Journal Article
DEP - 20200511
PL  - Scotland
TA  - J Clin Neurosci
JT  - Journal of clinical neuroscience : official journal of the Neurosurgical Society 
      of Australasia
JID - 9433352
RN  - 19GBJ60SN5 (Cannabidiol)
RN  - EC 2.7.1.1 (MTOR protein, human)
RN  - EC 2.7.11.1 (TOR Serine-Threonine Kinases)
SB  - IM
MH  - Adult
MH  - Angiomyolipoma/*drug therapy/pathology
MH  - Cannabidiol/pharmacology/*therapeutic use
MH  - Female
MH  - Giant Cell Tumors/*drug therapy
MH  - Humans
MH  - Kidney Neoplasms/*drug therapy
MH  - Male
MH  - Retrospective Studies
MH  - TOR Serine-Threonine Kinases/drug effects/metabolism
MH  - Treatment Outcome
MH  - Tuberous Sclerosis/*drug therapy/pathology
MH  - Tumor Burden/drug effects
OTO - NOTNLM
OT  - Cannabidiol
OT  - Epilepsy
OT  - Renal angiomyolipoma
OT  - Subependymal giant cell tumor
OT  - Tuberous sclerosis complex
EDAT- 2020/05/16 06:00
MHDA- 2020/11/06 06:00
CRDT- 2020/05/16 06:00
PHST- 2020/03/10 00:00 [received]
PHST- 2020/05/03 00:00 [accepted]
PHST- 2020/05/16 06:00 [pubmed]
PHST- 2020/11/06 06:00 [medline]
PHST- 2020/05/16 06:00 [entrez]
AID - S0967-5868(20)30565-8 [pii]
AID - 10.1016/j.jocn.2020.05.030 [doi]
PST - ppublish
SO  - J Clin Neurosci. 2020 Jul;77:85-88. doi: 10.1016/j.jocn.2020.05.030. Epub 2020 
      May 11.