Clinical Trials


Evaluating the Safety of GPX-001 as a Monotherapy

A phase I dose escalation trial was conducted at The University of Texas MD Anderson Cancer Center evaluating intravenous GPX-001 (TUSC2/FUS1) monotherapy in stage IV recurrent, metastatic lung cancer patients. The primary objective of this Phase I trial was to assess the toxicity of GPX-001 administered intravenously and to determine the maximum tolerated dose, or MTD, and recommended Phase II dose of GPX-001 alone.

The study showed for the first time that a tumor suppressor gene can be delivered intravenously and selectively to a patient’s cancer cells using a systemic nanovesicle vector. Although this trial was not designed to show changes in outcomes, a halt in cancer growth was observed in a number of patients. GPX-001 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 GPX-001 selectively and preferentially targeted patients’ cancer cells.

To learn more about scientific evidence and studies supporting GPX-001 and the TUSC2 gene, please refer to our TUSC2 Bibliography page.