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Published in: Journal of Translational Medicine 1/2010

Open Access 01-12-2010 | Research

A pilot clinical trial testing mutant von Hippel-Lindau peptide as a novel immune therapy in metastatic Renal Cell Carcinoma

Authors: Osama E Rahma, Ed Ashtar, Ramy Ibrahim, Antoun Toubaji, Barry Gause, Vincent E Herrin, W Marston Linehan, Seth M Steinberg, Frank Grollman, George Grimes, Sarah A Bernstein, Jay A Berzofsky, Samir N Khleif

Published in: Journal of Translational Medicine | Issue 1/2010

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Abstract

Background

Due to the lack of specific tumor antigens, the majority of tested cancer vaccines for renal cell carcinoma (RCC) are based on tumor cell lysate. The identification of the von Hippel-Lindau (VHL) gene mutations in RCC patients provided the potential for developing a novel targeted vaccine for RCC. In this pilot study, we tested the feasibility of vaccinating advanced RCC patients with the corresponding mutant VHL peptides.

Methods

Six patients with advanced RCC and mutated VHL genes were vaccinated with the relevant VHL peptides. Patients were injected with the peptide mixed with Montanide subcutaneously (SQ) every 4 weeks until disease progression or until the utilization of all available peptide stock.

Results

Four out of five evaluable patients (80%) generated specific immune responses against the corresponding mutant VHL peptides. The vaccine was well tolerated. No grade III or IV toxicities occurred. The median overall survival (OS) and median progression-free survival (PFS) were 30.5 and 6.5 months, respectively.

Conclusions

The vaccine demonstrated safety and proved efficacy in generating specific immune response to the mutant VHL peptide. Despite the fact that the preparation of these custom-made vaccines is time consuming, the utilization of VHL as a vaccine target presents a promising approach because of the lack of other specific targets for RCC. Accordingly, developing mutant VHL peptides as vaccines for RCC warrants further investigation in larger trials. Trial registration: 98C0139
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Metadata
Title
A pilot clinical trial testing mutant von Hippel-Lindau peptide as a novel immune therapy in metastatic Renal Cell Carcinoma
Authors
Osama E Rahma
Ed Ashtar
Ramy Ibrahim
Antoun Toubaji
Barry Gause
Vincent E Herrin
W Marston Linehan
Seth M Steinberg
Frank Grollman
George Grimes
Sarah A Bernstein
Jay A Berzofsky
Samir N Khleif
Publication date
01-12-2010
Publisher
BioMed Central
Published in
Journal of Translational Medicine / Issue 1/2010
Electronic ISSN: 1479-5876
DOI
https://doi.org/10.1186/1479-5876-8-8

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