New resources to assist with gene transfer clinical studies

November 07, 2001

Bethesda, MD-The National Center for Research Resources (NCRR), a part of the National Institutes of Health (NIH), announced today the award of cooperative agreements in the amount of $2.65 million to establish National Gene Vector Laboratories (NGVLs) at five locations:

• Baylor College of Medicine (Houston, TX)

• City of Hope National Medical Center and Beckman Research Institute (Duarte, CA)

• Indiana University (Indianapolis, IN)

• Southern Research Institute (Birmingham, AL)

• University of Florida (Gainesville, FL)

In gene transfer clinical studies, a vector serves as the vehicle to deliver a healthy gene to a part of the patient's body. This allows the correction of a genetic disorder or alteration of the properties of the cell. In this manner, gene transfer protocols, using several viral and non-viral vectors, have addressed such diseases as cystic fibrosis, severe combined immune deficiencies, hemophilia, cancer, heart disease, and AIDS.

"While the potential for gene transfer is great, the technical requirements and the expense of vector development, production, and safety testing often limits the capacity of investigators to proceed with their research," said Dr. Judith Vaitukaitis, NCRR Director. "A cooperative national effort will help move this promising research forward."

Frequently, physician scientists have had neither the ability to generate clinical grade vector in sufficient quantity nor the financial resources or time required to obtain the needed vector from commercial manufacturers. The limited availability of clinical quality vector constituted a barrier to progress in the field of gene transfer.

NCRR established a NGVL network in 1995 to help clinical researchers obtain adequate quantities of clinical-grade vectors. With today's announcement, the NGVLs will have not only the capability to produce and distribute a variety of human gene vectors, but also the capacity to provide toxicology studies at no cost to investigators for Phase I and Phase II human gene transfer clinical trials.

Toxicology data are frequently required by the Food and Drug Administration to minimize the likelihood of adverse events occurring during clinical research. This aspect of the NIH support is critical since many investigators' project grants do not provide funds for such studies. Since toxicology data are frequently considered proprietary and, therefore, not shared, toxicology studies are often repeated at great cost to the research community and funding entities. These new awards will help to eliminate these problems.

The responsibilities of the NGVLs:

• Indiana University will serve as the NGVL Coordinating Center to receive investigators' requests for NGVL services and to provide oversight of compliance with NGVL policies, procedures, and administrative matters. The Indiana NGVL will produce lentiviral and retroviral vectors; maintain a generally accessible master file that contains toxicology data on adeno-associated virus (AAV), a commonly used vector; and establish a repository to distribute an AAV reference standard to investigators;

• City of Hope National Medical Center and Beckman Research Institute will produce non-viral vectors;

• Baylor College of Medicine will produce adenoviral vectors; and

• Southern Research Institute and University of Florida will perform toxicology studies on the gene vector preparations.
Seven other NIH components will participate in the NGVL cooperative agreements, in addition to NCRR. They are: the National Cancer Institute; National Institute of Diabetes and Digestive and Kidney Diseases; National Institute of Neurological Disorders and Stroke; National Institute of Arthritis and Musculoskeletal and Skin Diseases; National Institute of Dental and Craniofacial Research; National Institute of Allergy and Infectious Disease; and the National Institute of Child Health and Human Development.

NCRR is the nation's leading Federal sponsor of resources that enable advances in many areas of biomedical research. NCRR support provides the scientific research community with access to a diverse array of biomedical research technologies, instrumentation, specialized basic and clinical research facilities, mammalian and non-mammalian animal models, genetic stocks, and biomaterials such as cell lines, tissues, and organs.

NIH/National Center for Advancing Translational Sciences (NCATS)

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