IU researcher plays role in recent FDA approval of Neupogen drug to treat people exposed to radiation

Jul 15 2015

An Indiana University Melvin and Bren Simon Cancer Center researcher played a role in the recent Food and Drug Administration approval of a drug to treat people exposed to potentially lethal doses of radiation.

Christie M. Orschell, Ph.D., a senior research professor at the Indiana University School of Medicine and a researcher at the cancer center, and colleagues performed preclinical work that contributed to the approval of Neupogen (filgrastim) to treat adult and pediatric patients exposed to myelosuppressive doses of radiation. Such exposure may happen in a radiation nuclear event.

Radiation destroys the bone marrow, resulting in loss of blood cells and increasing the risk of infection and uncontrolled bleeding, according to Dr. Orschell. Neupogen can help patients by facilitating recovery of bone marrow cells that develop into neutrophils, white blood cells that help fight off infections.

"The approval of Neupogen is an important step in advancing medical countermeasures for radiation," Dr. Orschell said. "Still, we're continually investigating new drugs that are easier to administer and perhaps only require a single injection."

Neupogen is the first radiation countermeasure approved under the FDA's Animal Rule, which was drafted to guide the development of drugs when human efficacy studies cannot ethically be performed. In March 2015, the FDA approved Neupogen for use following an acute exposure to a radiation dose capable of causing severe loss of bone marrow cells.

Dr. Orschell and her lab of nine researchers developed a mouse model to test medical countermeasures against radiation as part of a consortium of investigators working together to find drugs to treat irradiated people. Data from the Orschell lab contributed to the understanding of how Neupogen may work in humans.

Dr. Orschell explained that a mouse model is used to mimic a disease in humans. "Our mouse model of acute radiation syndrome has become one of the standard models to test medical countermeasures under the Animal Rule," Dr. Orschell said.

In 2005, the National Institute of Allergy and Infectious Diseases awarded a federal contract to the University of Maryland School of Medicine, which established the consortium of institutions to facilitate the development of medical countermeasures that could be used in an emergency mass casualty situation involving radiation injuries. IU was an integral part of that consortium.

Dr. Orschell's ongoing work recently earned her $750,000 in funding from the Department of Defense to study drugs for civilians or first responders who report to a site following radiation exposure. "In the case of first responders, you would have an opportunity to administer the drug to them before they are exposed at the site," she said.

Source: Indiana University