ONC-001

Evaluating the Safety of REQORSA® Immunogene Therapy as a Monotherapy (Completed)

A Phase 1 dose escalation trial was conducted at The University of Texas MD Anderson Cancer Center evaluating the systemic, intravenous delivery of REQORSA® (TUSC2/FUS1) as a monotherapy in stage IV recurrent, metastatic lung cancer patients. The primary objective of this Phase 1 trial was to assess the toxicity of REQORSA administered systemically and intravenously, to determine the maximum tolerated dose, or MTD, and to determine a recommended Phase 2 dose of REQORSA alone.

The First Systemically Delivered Gene Therapy Used for Cancer in Humans

The study showed for the first time that a tumor suppressor gene can be delivered systemically, intravenously and selectively to a patient’s cancer cells using a systemic nanoparticle vector. Although this trial was not designed to show changes in outcomes, a halt in cancer growth was observed in a number of patients. REQORSA was well tolerated with tumor responses noted in lung primary and metastatic cancers in the liver, pancreas, and lymph nodes. In addition, pre- and post-treatment patient biopsies demonstrated that intravenous REQORSA selectively and preferentially targeted patients’ cancer cells.

Metabolic Tumor Response in a Metastatic Lung Cancer Subject

This subject survived after subsequent therapy more than seven years after the final treatment with REQORSA, to our knowledge, without evidence of cancer progression in the responding sites.

To learn more about scientific evidence and studies supporting REQORSA and the TUSC2 gene, please refer to our TUSC2 Bibliography page.