Cited by 143Ocular Oncology and TreatmentsVirus-based gene therapy researchRetinal Development and Disorders

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Response of Retinoblastoma With Vitreous Tumor Seeding to Adenovirus-Mediated Delivery of Thymidine Kinase Followed by Ganciclovir

Chévez-Barrios, Patricia, Chintagumpala, Murali, Mieler, William, Paysse, Evelyn, Boniuk, Milton, Kozinetz, Claudia, Hurwitz, Mary Y., Hurwitz, Richard L.·2005·Journal of Clinical Oncology

DOI 10.1200/jco.2004.00.1883

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Abstract

Purpose: To evaluate the feasibility and safety of adenovirus-mediated gene therapy as a treatment for tumor seeds in the vitreous of children with retinoblastoma. Patients and Methods: An Institutional Biosafety Committee–, Institutional Review Board–, Recombinant DNA Advisory Committee–, and US Food and Drug Administration–approved phase I study used intrapatient dose escalation of adenoviral vector containing a herpes simplex thymidine kinase gene (AdV-TK) followed by systemic administration of ganciclovir to treat bilateral retinoblastoma with vitreous tumor seeding refractory to standard therapies. Vitreous tumor seeds were treated by intravitreous injection of AdV-TK adjacent to disease sites. Each injection was followed by ganciclovir delivered intravenously every 12 hours for 7 days. Results: Eight patients with vitreous tumor seeds were enrolled. One patient who was treated with 10 8 viral particles (vp) had resolution of the tumor seeds around the injection site. The seven patients who were treated with doses ≥ 10 10 vp had resolution of their vitreous tumor seeds documented by fundoscopy. Toxicity included mild inflammation at 10 10 vp and moderate inflammation, corneal edema, and increased intraocular pressure at 10 11 vp. One patient was free of active vitreous tumor seeds 38 months after therapy. There has been no evidence of extraocular spread of tumor along the needle tract in any patient. Conclusion: AdV-TK followed by ganciclovir can be administered safely to children with retinoblastoma. Suicide gene therapy may contribute to the treatment of children with retinoblastoma tumor seeds in the vitreous, a resistant complication of retinoblastoma.

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Method

Authors: Chévez-Barrios, P., Chintagumpala, M., Mieler, W., Paysse, E., Boniuk, M., Kozinetz, C., Hurwitz, M. Y., & Hurwitz, R. L.
Affiliations: From the Departments of Pathology, Ophthalmology, and Pediatrics, Texas Children's Cancer Center; the Center for Cell and Gene Therapy, Baylor College of Medicine, Texas Children's Hospital; and the Methodist Hospital, Houston, TX
Houston Methodist (US)
ORCID: Chintagumpala, Murali
Mieler, William
Paysse, Evelyn
Kozinetz, Claudia
Hurwitz, Mary Y.
Venue: Journal of Clinical Oncology
Publisher: American Society of Clinical Oncology (ASCO)
Published: 2005-11-01
Volume: 23
Issue: 31
Pages: 7927-7935
ISSN: 0732-183X (ISSN-L)
1527-7755
DOI: 10.1200/jco.2004.00.1883
OpenAlex: W2166510225
Cited by: 143
References: 32
Citations by year: 2026: 1 · 2025: 2 · 2024: 1 · 2023: 4 · 2022: 7 · 2021: 5 · 2020: 4 · 2019: 3 · 2018: 1 · 2017: 9 · 2016: 4 · 2015: 10 · 2014: 7 · 2013: 5 · 2012: 10
FWCI: 8.23
Type: journal article
Language: en
Open access: closed
Locations: Journal of Clinical Oncology (publishedVersion)
PubMed (publishedVersion)
Funders: National Cancer Institute (R21CA097762, CA97762)
Topics: Health Sciences › Medicine › Ophthalmology › Ocular Oncology and Treatments
Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
Health Sciences › Medicine › Ophthalmology › Retinal Development and Disorders
Keywords: retinoblastoma, adenoviral vector, herpes simplex thymidine kinase, ganciclovir, suicide gene therapy, gene therapy toxicity, pediatric retinoblastoma, intravitreal injection, dose escalation
MeSH terms: Adenoviruses, Human, Antiviral Agents, Child, Combined Modality Therapy, Feasibility Studies, Genetic Vectors, Humans, Neoplasm Recurrence, Local, Neoplasm Seeding, Retinoblastoma, Thymidine Kinase, Vitreous Body, Genetic Therapy, Ganciclovir, Gene Transfer Techniques, Retinal Neoplasms

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Response of Retinoblastoma With Vitreous Tumor Seeding to Adenovirus-Mediated Delivery of Thymidine Kinase Followed by Ganciclovir

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