Suppression of TP53 function is nearly ubiquitous in human
cancers, and a significant fraction of
cancers have mutations in the TP53 gene itself. Therefore, the wild-type TP53 gene has become an important target gene for transformation research of cancer gene
therapy. In 2003, the first anti-
tumor gene therapy drug rAd-p53 (recombinant human p53 adenovirus), trade name Gendicine™, was approved by the China Food and Drug Administration (CFDA) for treatment of
head and neck squamous cell carcinoma (
HNSCC) in combination with
radiotherapy. The recombinant human TP53 gene is delivered into
cancer cells by an adenovirus vector constructed to express the functional p53
protein. Although the only currently approved used of Gendicine is in combination with
radiotherapy for treatment of
HNSCC, clinical studies have been carried out for more than 20 other applications of Gendicine in treating
cancer, including treatment of advanced
lung cancer, advanced
liver cancer, malignant gynecological
tumors, and
soft tissue sarcomas. Currently more than 30,000 patients have been treated with Gendicine. This review provides an overview of the clinical applications of Gendicine in China. We summarize a total of 48 studies with 2,561 patients with solid
tumors, including 34 controlled clinical studies and 14 open clinical studies, i.e., clinical studies without a control group. There are 11 studies for
head and neck cancer, 10 for
liver cancer, 6 for malignant gynecological
tumors, 4 for
non-small cell lung cancer, 4 for
soft tissue sarcoma, 4 for malignant effusion, 2 for gastrointestinal
tumors, and 7 for other types of
cancer. In all the reported clinical studies, the most common side effect was self-limited
fever. Intratumoral injection and
intra-arterial infusion were the most common routes of administration. Overall, Gendicine combined with
chemotherapy,
radiotherapy, or other conventional treatment regimens demonstrated significantly higher response rates compared to standard
therapies alone. Some of the published studies also showed that Gendicine combination regimens demonstrated longer progression-free survival times than conventional treatments alone. To date, Gendicine has been clinically used in China for treatment of
cancers other than
HNSCC for more than ten years, mainly for patients with advanced or unresectable malignant
tumors. However, the establishment of standard treatment regimens using TP53 gene
therapy is still needed in order to advance its use in clinical practice.