Gene therapy for peripheral arterial disease using sendai virus vector

From preclinical studies to the phase I/IIa clinical trial

Yoshikazu Yonemitsu, Takuya Matsumoto, Yoshihiko Maehara

Research output: Chapter in Book/Report/Conference proceedingChapter

3 Citations (Scopus)

Abstract

Great potential for limb salvaging with basic fibroblast growth factor (FGF-2) expressed from a Sendai virus vector with the full-length viral genome (SeV/FGF-2) in the target muscle was demonstrated in animal models of severe limb ischemia. The exogenously expressed FGF-2 induced other endogenous angiogenic factors, including vascular endothelial growth factor and hepatocyte growth factor in a highly concerted fashion, and this was shown to underlie the remarkable limb salvaging. These results led to the initiation of a phase I/IIa clinical trial for peripheral arterial disease (PAD) of humans. To be better prepared for unforeseen adverse events, this clinical trial used the F gene-deleted, nontransmissible, and hence safer (than SeV/FGF-2) version that carried the human FGF-2 gene (ΔFSeV/FGF-2; product code, DVC1-0101). Overall, DVC1-0101 appeared to be safe, giving rise to no serious adverse events in various criteria. Moreover, it exerted a significant therapeutic effect from a number of clinical aspects. DVC1-0101 represents the first case of the potential diverse medical applications of SeV vector. It is now hoped to move on to an advanced phase of clinical trial with a larger number of patients and placebo group to firmly establish the safety and efficacy of DVC1-0101.

Original languageEnglish
Title of host publicationSendai Virus Vector: Advantages and Applications
PublisherSpringer Japan
Pages185-199
Number of pages15
ISBN (Electronic)9784431545569
ISBN (Print)4431545557, 9784431545552
DOIs
Publication statusPublished - Sep 1 2013

Fingerprint

Sendai virus
Clinical Trials, Phase I
Peripheral Arterial Disease
Fibroblast Growth Factor 2
Genetic Therapy
Extremities
Clinical Trials
Hepatocyte Growth Factor
Angiogenesis Inducing Agents
Viral Genome
Therapeutic Uses
Vascular Endothelial Growth Factor A
Genes
Ischemia
Animal Models
Placebos
Safety
Muscles

All Science Journal Classification (ASJC) codes

  • Medicine(all)
  • Immunology and Microbiology(all)

Cite this

Yonemitsu, Y., Matsumoto, T., & Maehara, Y. (2013). Gene therapy for peripheral arterial disease using sendai virus vector: From preclinical studies to the phase I/IIa clinical trial. In Sendai Virus Vector: Advantages and Applications (pp. 185-199). Springer Japan. https://doi.org/10.1007/978-4-431-54556-9_8

Gene therapy for peripheral arterial disease using sendai virus vector : From preclinical studies to the phase I/IIa clinical trial. / Yonemitsu, Yoshikazu; Matsumoto, Takuya; Maehara, Yoshihiko.

Sendai Virus Vector: Advantages and Applications. Springer Japan, 2013. p. 185-199.

Research output: Chapter in Book/Report/Conference proceedingChapter

Yonemitsu, Yoshikazu ; Matsumoto, Takuya ; Maehara, Yoshihiko. / Gene therapy for peripheral arterial disease using sendai virus vector : From preclinical studies to the phase I/IIa clinical trial. Sendai Virus Vector: Advantages and Applications. Springer Japan, 2013. pp. 185-199
@inbook{4354c0ff46e24b4fb13f315acce63a16,
title = "Gene therapy for peripheral arterial disease using sendai virus vector: From preclinical studies to the phase I/IIa clinical trial",
abstract = "Great potential for limb salvaging with basic fibroblast growth factor (FGF-2) expressed from a Sendai virus vector with the full-length viral genome (SeV/FGF-2) in the target muscle was demonstrated in animal models of severe limb ischemia. The exogenously expressed FGF-2 induced other endogenous angiogenic factors, including vascular endothelial growth factor and hepatocyte growth factor in a highly concerted fashion, and this was shown to underlie the remarkable limb salvaging. These results led to the initiation of a phase I/IIa clinical trial for peripheral arterial disease (PAD) of humans. To be better prepared for unforeseen adverse events, this clinical trial used the F gene-deleted, nontransmissible, and hence safer (than SeV/FGF-2) version that carried the human FGF-2 gene (ΔFSeV/FGF-2; product code, DVC1-0101). Overall, DVC1-0101 appeared to be safe, giving rise to no serious adverse events in various criteria. Moreover, it exerted a significant therapeutic effect from a number of clinical aspects. DVC1-0101 represents the first case of the potential diverse medical applications of SeV vector. It is now hoped to move on to an advanced phase of clinical trial with a larger number of patients and placebo group to firmly establish the safety and efficacy of DVC1-0101.",
author = "Yoshikazu Yonemitsu and Takuya Matsumoto and Yoshihiko Maehara",
year = "2013",
month = "9",
day = "1",
doi = "10.1007/978-4-431-54556-9_8",
language = "English",
isbn = "4431545557",
pages = "185--199",
booktitle = "Sendai Virus Vector: Advantages and Applications",
publisher = "Springer Japan",
address = "Japan",

}

TY - CHAP

T1 - Gene therapy for peripheral arterial disease using sendai virus vector

T2 - From preclinical studies to the phase I/IIa clinical trial

AU - Yonemitsu, Yoshikazu

AU - Matsumoto, Takuya

AU - Maehara, Yoshihiko

PY - 2013/9/1

Y1 - 2013/9/1

N2 - Great potential for limb salvaging with basic fibroblast growth factor (FGF-2) expressed from a Sendai virus vector with the full-length viral genome (SeV/FGF-2) in the target muscle was demonstrated in animal models of severe limb ischemia. The exogenously expressed FGF-2 induced other endogenous angiogenic factors, including vascular endothelial growth factor and hepatocyte growth factor in a highly concerted fashion, and this was shown to underlie the remarkable limb salvaging. These results led to the initiation of a phase I/IIa clinical trial for peripheral arterial disease (PAD) of humans. To be better prepared for unforeseen adverse events, this clinical trial used the F gene-deleted, nontransmissible, and hence safer (than SeV/FGF-2) version that carried the human FGF-2 gene (ΔFSeV/FGF-2; product code, DVC1-0101). Overall, DVC1-0101 appeared to be safe, giving rise to no serious adverse events in various criteria. Moreover, it exerted a significant therapeutic effect from a number of clinical aspects. DVC1-0101 represents the first case of the potential diverse medical applications of SeV vector. It is now hoped to move on to an advanced phase of clinical trial with a larger number of patients and placebo group to firmly establish the safety and efficacy of DVC1-0101.

AB - Great potential for limb salvaging with basic fibroblast growth factor (FGF-2) expressed from a Sendai virus vector with the full-length viral genome (SeV/FGF-2) in the target muscle was demonstrated in animal models of severe limb ischemia. The exogenously expressed FGF-2 induced other endogenous angiogenic factors, including vascular endothelial growth factor and hepatocyte growth factor in a highly concerted fashion, and this was shown to underlie the remarkable limb salvaging. These results led to the initiation of a phase I/IIa clinical trial for peripheral arterial disease (PAD) of humans. To be better prepared for unforeseen adverse events, this clinical trial used the F gene-deleted, nontransmissible, and hence safer (than SeV/FGF-2) version that carried the human FGF-2 gene (ΔFSeV/FGF-2; product code, DVC1-0101). Overall, DVC1-0101 appeared to be safe, giving rise to no serious adverse events in various criteria. Moreover, it exerted a significant therapeutic effect from a number of clinical aspects. DVC1-0101 represents the first case of the potential diverse medical applications of SeV vector. It is now hoped to move on to an advanced phase of clinical trial with a larger number of patients and placebo group to firmly establish the safety and efficacy of DVC1-0101.

UR - http://www.scopus.com/inward/record.url?scp=84930734502&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=84930734502&partnerID=8YFLogxK

U2 - 10.1007/978-4-431-54556-9_8

DO - 10.1007/978-4-431-54556-9_8

M3 - Chapter

SN - 4431545557

SN - 9784431545552

SP - 185

EP - 199

BT - Sendai Virus Vector: Advantages and Applications

PB - Springer Japan

ER -