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First clinical experience using a 'pathotropic' injectable retroviral vector (Rexin-G) as intervention for stage IV
Erlinda M Gordon1, Gerardo H Cornelio, Conrado C Lorenzo
1Epeius Biotechnologies Corporation, Glendale, CA 91203, USA. emgordon@epeiusbiotech.com
Abstract:
Metastatic or non-resectable (stage IV) pancreatic cancer has a rapidly fatal outcome (median survival: 3-6 months), thus making gene therapy a viable therapeutic option. The objectives of the clinical studies are to evaluate the safety/toxicity and potential anti-tumor response/efficacy of intravenous (i.v.) infusions of a 'pathotropic' retroviral vector bearing a cytocidal gene construct (Rexin-G) as a gene transfer intervention for stage IV pancreatic cancer. An intra-patient dose escalation regimen was used wherein increasing doses of Rexin-G were given i.v. daily for 8-10 days. Completion of this regimen was followed by a one-week evaluation period for toxicity, after which, the maximum tolerated dose of Rexin-G was administered for another 8-10 days. In a second protocol, i.v. Rexin-G was administered frontline for 6 days followed by 8 doses of weekly gemcitabine. The NIH Common Toxicity Criteria Vs.2 was used to assess toxicity, and the NCI-RECIST criteria and tumor volume measurements were used to evaluate potential anti-tumor responses. We report the results of the first 3 patients that participated in the studies. Rexin-G arrested tumor growth in 3 of 3 patients without experiencing dose-limiting toxicity. No bone marrow suppression, significant alterations in liver and kidney function, nausea and vomiting, mucositis or hair loss were observed. Two patients are alive with stable disease approximately 5 and 14 months from diagnosis, and 1 patient is alive with progressive disease 20 months from diagnosis. The encouraging results of this first clinical experience will guide the design and planning of phase I/II clinical trials to establish the safety and efficacy of Rexin-G as the first targeted injectable gene therapy vector for stage IV pancreatic cancer.
Insights
Gene therapy using Rexin-G shows promise for stage IV pancreatic cancer. This novel treatment arrested tumor growth in all initial patients without significant side effects, offering a new therapeutic avenue.
Area of Science:
- Oncology
- Gene Therapy
- Cancer Research
Background:
- Stage IV pancreatic cancer has a poor prognosis with limited survival.
- Gene therapy presents a potential therapeutic option for advanced pancreatic cancer.
- Existing treatments often have significant side effects and limited efficacy.
Purpose of the Study:
- To evaluate the safety and efficacy of Rexin-G, an injectable gene therapy vector, for stage IV pancreatic cancer.
- To assess the anti-tumor response and toxicity of intravenous Rexin-G infusions.
- To establish preliminary data for future Phase I/II clinical trials.
Main Methods:
- Utilized an intra-patient dose escalation regimen of intravenous Rexin-G.
- Administered Rexin-G daily for 8-10 days, followed by toxicity evaluation.
- Assessed toxicity using NIH Common Toxicity Criteria and anti-tumor response using NCI-RECIST criteria.
Main Results:
- Rexin-G demonstrated tumor growth arrest in all three initial patients.
- No dose-limiting toxicities or significant side effects (e.g., bone marrow suppression, organ dysfunction, nausea, hair loss) were observed.
- Two patients achieved stable disease for approximately 5 and 14 months, with one patient alive with progressive disease at 20 months.
Conclusions:
- Intravenous Rexin-G gene therapy is a potentially safe and effective treatment for stage IV pancreatic cancer.
- Rexin-G shows promising anti-tumor activity and an acceptable safety profile in early clinical studies.
- These findings support further investigation of Rexin-G in Phase I/II clinical trials for pancreatic cancer.
