Systemic Therapy Development in Von Hippel–Lindau Disease: An Outsized Contribution from an Orphan Disease

Vivek Narayan; Eric Jonasch

doi:10.3390/cancers14215313

Systemic Therapy Development in Von Hippel–Lindau Disease: An Outsized Contribution from an Orphan Disease

Vivek Narayan, Eric Jonasch

Genitourinary Medical Oncology

Research output: Contribution to journal › Review article › peer-review

2 Scopus citations

Abstract

Over the last several decades, an improved understanding of von Hippel–Lindau disease and its underlying biology has informed the successful development of numerous anti-cancer agents, particularly for the treatment of advanced renal cell carcinoma. Most recently, this has culminated in the first regulatory approval for a systemic therapy for VHL disease-associated neoplasms. This review will trace the clinical development of systemic therapies for VHL disease and additionally highlight anticipated challenges and opportunities for future VHL systemic therapy.

Original language	English (US)
Article number	5313
Journal	Cancers
Volume	14
Issue number	21
DOIs	https://doi.org/10.3390/cancers14215313
State	Published - Nov 2022

Keywords

hemangioblastoma
HIF2 inhibitor
renal cell carcinoma
systemic therapy
von Hippel–Lindau

ASJC Scopus subject areas

Oncology
Cancer Research

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10.3390/cancers14215313

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title = "Systemic Therapy Development in Von Hippel–Lindau Disease: An Outsized Contribution from an Orphan Disease",

abstract = "Over the last several decades, an improved understanding of von Hippel–Lindau disease and its underlying biology has informed the successful development of numerous anti-cancer agents, particularly for the treatment of advanced renal cell carcinoma. Most recently, this has culminated in the first regulatory approval for a systemic therapy for VHL disease-associated neoplasms. This review will trace the clinical development of systemic therapies for VHL disease and additionally highlight anticipated challenges and opportunities for future VHL systemic therapy.",

keywords = "hemangioblastoma, HIF2 inhibitor, renal cell carcinoma, systemic therapy, von Hippel–Lindau",

author = "Vivek Narayan and Eric Jonasch",

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AU - Jonasch, Eric

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AB - Over the last several decades, an improved understanding of von Hippel–Lindau disease and its underlying biology has informed the successful development of numerous anti-cancer agents, particularly for the treatment of advanced renal cell carcinoma. Most recently, this has culminated in the first regulatory approval for a systemic therapy for VHL disease-associated neoplasms. This review will trace the clinical development of systemic therapies for VHL disease and additionally highlight anticipated challenges and opportunities for future VHL systemic therapy.

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KW - HIF2 inhibitor

KW - renal cell carcinoma

KW - systemic therapy

KW - von Hippel–Lindau

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Systemic Therapy Development in Von Hippel–Lindau Disease: An Outsized Contribution from an Orphan Disease

Abstract

Keywords

ASJC Scopus subject areas

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