Gene and Virus Therapy Shared Resource

  • Diagram of the gene and virus therapy development process

    Preclinical research (left) evaluates the efficacy of a viral vector in vitro and in animal models, providing data that justify testing the concept in human clinical trials.

    Translation from the lab to clinical trials (center) requires capabilities to characterize the product, manufacture a clinical-grade product using good manufacturing practices and integrate these services with the clinical trial protocol. Clinical trials (right) then get under way.

    All procedures and results are reviewed by the FDA, the National Institutes of Health Recombinant DNA Advisory Committee and Mayo Clinic committees, such as the Institutional Review Board and Institutional Biosafety Committee.

Development process.

The Gene and Virus Therapy Shared Resource supports Mayo Clinic Cancer Center investigators in translating virus-based therapies from the research laboratory into preclinical and clinical trials.

Before a promising virus-based cancer therapy can be evaluated in a clinical trial, the gene or viral product must be converted into medical-grade material — high enough quality for use in humans — and produced in sufficient quantities.

These tasks cannot take place in research laboratories. They must be done using federally mandated procedures and practices, including good manufacturing practices, which require specific personnel training and expertise, unique facilities and equipment, and robust quality assurance programs — all of which are available in the Gene and Virus Therapy Shared Resource.

The Gene and Virus Therapy Shared Resource meets the rigorous requirements set forth by the Food and Drug Administration (FDA) for phase I and II clinical trials, including the ability to provide enough clinical-grade material to determine the viral product's efficacy and toxicological and pharmacological characteristics. It also has expertise with the FDA's investigational new drug regulations.

Services

The Gene and Virus Therapy Shared Resource has three components that provide services to investigators. Staff members from these units work in collaboration with each investigator to determine what services are needed for each project, perform the services, and document the protocols and results.

Projects

The Gene and Virus Therapy Shared Resource supports researchers who are studying a wide range of cancers. Project examples include these phase I clinical trials:

  • Ovarian cancer. Intraperitoneal administration of an attenuated strain (Edmonston strain) of measles virus, genetically engineered to produce carcinoembryonic antigen, in patients with recurrent ovarian cancer.
  • Multiple myeloma. Systemic administration of Edmonston strain of measles virus, genetically engineered to express the sodium-iodide symporter with or without cyclophosphamide, in patients with recurrent or refractory multiple myeloma.
  • Brain cancer. Intratumoral administration of MV-CEA in patients with recurrent glioma.
  • Prostate cancer. In situ gene therapy for locally recurrent prostate cancer following radiation therapy failure using sodium-iodide symporter and radioiodine.
  • Mesothelioma. Oncolytic measles virotherapy in mesothelioma.
  • Liver cancer. Intratumoral injection of vesicular stomatitis virus expressing interferon beta in patients with hepatocellular carcinoma.

Contact

Mark J. Federspiel, Ph.D.
Director
Gene and Virus Therapy Shared Resource
Email: federspiel.mark@mayo.edu

Watch a video about the Gene and Virus Therapy Shared Resource.