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

Abstract

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.

Diagnosis and current management of retinoblastoma

“…Molecular therapy for retinoblastoma, taking advantage of the specific genetic defect in retinoblastoma, is currently under study (Huang et al, 1988;Xu, 2003;Chevez-Barrios et al, 2005).…”

Phase I and biodistribution study of recombinant adenovirus vector-mediated herpes simplex virus thymidine kinase gene and ganciclovir administration in patients with head and neck cancer and other malignant tumors

“…[8][9][10][11][12][13][14] There have been no reports evaluating this agent in NPC or head and neck cancer, both of which are common among Chinese people, often relapse locally and are often resistant to radiotherapy and chemotherapy. In this study, the majority of subjects had been diagnosed with NPC or head and neck cancer.…”

“…Phase I/II trials have subsequently been conducted using AdV/TK/GCV in a number of malignant tumors, including ovarian carcinoma, brain tumors, prostate cancer, hepatocellular carcinoma and so on. [9][10][11][12][13][14] All of these trials have shown that the approach is relatively safe, but efficacy has been localized to the injection site, which has limited its use. However, this treatment may be particularly suitable for some tumors that have the tendency to relapse locally, such as head and neck cancer or nasopharyngeal carcinoma (NPC).…”

Progress and Prospects: Gene Therapy Clinical Trials (Part 1)

“…A phase I clinical trial has been conducted to evaluate the feasibility and safety of adenovirus-mediated 'suicide' gene therapy for vitreous tumour seeds in one eye of children with bilateral retinoblastoma refractory to conventional treatment. 1 This trial evaluated the effects of repeated intravitreal injections of an adenoviral vector (group C serotype 5) expressing a herpes simplex thymidine kinase gene followed by systemic administration of ganciclovir. The vitreous tumour seeds appeared to respond to the intervention in all eight children.…”