GMP Facility Will Enable Genetic Treatments, Immune Therapies, Other Advances

Michael C. Purdy

Dec. 15, 2003 – Early next month, scientists working at a new research facility at Washington University School of Medicine in St. Louis will begin spending their days in a high-tech haven of cleanliness and environmental control comparable to the orbiting International Space Station. While the rest of the world spends the day in an invisible sea of a million or more tiny contaminant particles per cubic foot of air, state-of-the-art air filters and dedicated systems for controlling air flow and pressure will keep researchers at the Good Manufacturing Practice (GMP) facility, working in head-to-toe protective suits, in a much thinner soup of only about 10,000 particles per cubic foot of air.

The cells and other materials researchers work on in the GMP facility also will be kept inside sophisticated isolation units and stored in refrigerators and freezers extensively tested for their ability to maintain temperature within strict limits.

The extraordinary dedication to control and isolation in the GMP facility doesn't primarily stem from a need to keep dangerous materials from getting out of the laboratory. Instead, these measures will keep dangerous contaminants from getting into what the GMP facility makes: cells modified or manipulated for use in the latest innovative techniques for the treatment of cancer and other diseases. Like a tray of surgical tools, the cells are destined for use inside patients and must be produced to exact standards of consistency and purity. The GMP facility takes its name from the Food and Drug Administration (FDA) term for these standards.

Faculty and staff members from the School of Medicine and the Siteman Cancer Center celebrated the opening of the GMP facility at a Dec. 12 open house. After the opening, the facility will be permanently transformed into an ultra-clean area accessible only to personnel outfitted in special suits and protective gear.

The 2,615-square-foot facility will make it easier for researchers to engineer and manipulate cells for use in a variety of new treatments, including approaches that harness the power of the immune system to attack cancer and genetic modifications that enhance existing vulnerabilities in tumor cells or open up new lines of attack against such cells.

"The GMP facility is focused on translational research – taking something developed in the lab and bringing it to the patient's bedside," says Gerhard Bauer, laboratory director. "It's going to be a state-of-the-art, absolutely modern facility, one of the best available in the country."

The facility includes six manufacturing rooms capable of working independently on different projects.

"One of the unique things about this laboratory is that we had pre-facility design meetings with the FDA and completed the design and construction in consultation with them," says Bauer. "We're not going to be doing anything that would be a danger to the public, but we have to maintain strict safeguards against contamination because of the medical context in which these tissues will be used."

The GMP facility also adheres to the standards of the Good Tissue Practices Act, a new law going into effect in early 2004 that will require even minimal tissue manipulations to be performed in a facility that meets strict criteria for contamination prevention.

The first clinical trial using products produced in the GMP facility is set to begin early next year.John DiPersio, MD, PhD, the Lewis T. and Rosalind B. Apple Professor of Medicine and deputy director of the Siteman Cancer Center, plans to modify immune system cells known as T cells for use in the treatment of leukemia patients.

Physicians plan to inject patients with the T cells to destroy any recurrent tumors after bone marrow transplants. However, T cells also are involved in graft-versus-host disease, a primary cause of illness and death in such patients. To avoid the development of this disease, researchers will modify the cells to self-destruct in the presence of an anti-viral drug.

According to Bauer, researchers working on 30 different projects at Washington University already have made arrangements to have materials produced at the GMP facility. The University also plans to make the facility's services available to other universities and private companies.