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Experimental drugs for the treatment of idiopathic intracranial hypertension (IIH): shedding light on phase I and II trials.

Khatkar, Pavan
; 
Hubbard, Jess C
; 
Hill, Lisa
; 
Sinclair, Alexandra J
; 
Mollan, Susan P
Abstract
Introduction: Idiopathic intracranial hypertension is a neurological condition characterized by a raised intracranial pressure and papilledema that causes debilitating headaches. While the extent of the pathophysiology is being discovered, the condition is emerging as a systemic metabolic disease distinct to people living with obesity alone. Idiopathic intracranial hypertension is becoming more common and therefore establishing licensed therapeutics is a key priority. Area covered: The translation of preclinical work in idiopathic intracranial hypertension is evident by the two early phase trials evaluating 11-β-hydroxysteroid dehydrogenase inhibitor, AZD4017, and a glucagon like peptide-1 receptor agonist, Exenatide. This review summarizes these two early phase trials evaluating targeted medicines for the treatment of intracranial pressure. The modulation of these two distinct mechanisms have potential for therapeutic intervention in people living with idiopathic intracranial hypertension. Expert opinion: The clinical trial landscape in idiopathic intracranial hypertension is a challenge due to the rarity of the disease and the lack of agreed meaningful trial outcomes. Further preclinical work to fully understand the pathogenesis is required to enable personalized targeted drug treatment. Keywords: 11-β-hydroxysteroid dehydrogenase; Cerebrospinal fluid; glucagon-like peptide 1; headache; intracranial pressure; obesity; outcome measures; papilledema.
MIDER Authors
Hubbard, Jess, C
Mollan, Susan, P
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Date
2023-11-25
Type
Article
Subject
Ophthalmology, Neurology, Pharmacology
Collections
UHB Neurology
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Citation
Khatkar P, Hubbard JC, Hill L, Sinclair AJ, Mollan SP. Experimental drugs for the treatment of idiopathic intracranial hypertension (IIH): shedding light on phase I and II trials. Expert Opin Investig Drugs. 2023 Nov 25:1-9. doi: 10.1080/13543784.2023.2288073. Epub ahead of print. PMID: 38006580.
Journal / Source Title
Expert Opinion on Investigational Drugs
DOI
10.1080/13543784.2023.2288073
URI
http://hdl.handle.net/20.500.14200/3183
PMID
38006580
Publisher
Taylor and Francis Group
Publisher’s URL
https://www.tandfonline.com/doi/full/10.1080/13543784.2023.2288073?src=
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