Retinoblastoma, an eye cancer affecting the retina, most predominantly occurs in the children. A cancer-killing, virus-based therapy showed promising effects against cancer in a pilot clinical trial and mouse models.
Although further work is needed, the therapy lays the groundwork for new treatment options for the cancer, which is currently treated with disfiguring surgery.
Researchers estimate that retinoblastoma causes 8,000 cases each year, a figure that represents 11% of all cancers in children under the age of one. Most cases result from inactivation of the gene RB1, which normally plays a critical role as a tumor suppressor. Chemotherapy is the standard-of-care for retinoblastoma, but intensive rounds of such drugs can damage the retina and cause long-term vision problems. In some cases, surgery is needed to remove the eye entirely - an invasive procedure called enucleation that results in loss of vision.
Importantly, the authors administered VCN-01 to two pediatric patients with retinoblastoma and observed the virus successfully replicated in tumor cells and did not cause systemic inflammation. Taken together, the findings warrant further development of VCN-01 as a potential treatment for patients with retinoblastoma and RB1 inactivation, Pascual-Pasto et al. say.