Under the Project BioShield (PBS) Act (2004) (PDF - 191 KB) and the Pandemic and All-Hazards Preparedness Act (2006) (PDF - 248 KB), BARDA was charged with developing medical countermeasures (MCMs) to address radiological and nuclear threats to the United States determined by the Department of Homeland Security as Material Threats. Improvised Nuclear Devices (IND) and Radiological Dispersal Devices (RDD) have been the primary threats with INDs (nuclear denotation) having the highest priority. Radiation exposure injuries are complex and can be exacerbated by nuclear blasts causing other injuries like trauma and thermal burns. BARDA’s Radiological/Nuclear MCM Program supports advanced development of therapeutic candidates for all aspects, illnesses and injuries that may result from a radiological/nuclear event, thus increasing preparedness to treat patients in both field and definitive care (hospital) environments.
The BARDA Radiological/Nuclear MCM Program has supported development of over 35 candidates to treat the complex array of injuries, diseases and syndromes that can occur in patients after radiological and/or nuclear events. By collaborating with other key federal agencies that support basic research and early development (like NIH and DoD), BARDA has transitioned more than 20 MCM candidates into advanced development. Major accomplishments under Project BioShield include the late-stage development and procurement of recombinant cytokines (G-CSF and GM-CSF) in 2013, which are cell-signaling proteins used in treatment of neutropenia caused by exposure to ionizing radiation. The cytokine Neupogen® (G-CSF) received FDA approval in March, 2015 for the indication to increase survival in patients exposed to acute doses of radiation high enough to suppress bone marrow and immune system function. The approval was also granted to Neulasta®, a pegylated formulation of Neupogen® in the same year. In 2016, additional G-CSF and GM-CSF products were procured to increase our preparedness posture. The procurement of these anti-neutropenia cytokines exemplifies BARDA’s strategic goal of repurposing existing products as MCMs to leverage commercial development, distribution infrastructure and taxpayer investments in order to increase preparedness for radiological and nuclear threats.
BARDA’s Radiological/Nuclear MCM development pipeline consists of ten (10) MCM candidates targeting traumatic injuries and acute radiation syndrome (including hematopoietic subsyndromes affecting blood-cell production and subsyndromes of the gastrointestinal tract, lungs and skin) that may result from IND denotation. The Program also supports advanced development of a pediatric formulation for a licensed decorporation agent that can reduce internal contamination of radionuclides in children. The Radiological/Nuclear MCM Program also leverages the investments of BARDA’s new National MCM Response Infrastructure by collaborating extensively with the Nonclinical Studies Network on natural history studies, animal model development, evaluation of potential MCM candidates and efficacy assessments. For example, studies are conducted in appropriate scenarios and animal models to optimize the use of currently-available FDA-approved drugs, treatments and supportive care methods to treat patients with radiation subsyndromes. This Program also works with BARDA’s diagnostics and medical devices programs to support advanced development of biodosimeters that can determine whether or not an individual’s level of absorbed ionizing radiation requires definitive care.
Through investments in MCM candidates that can help medical professionals triage patients and more effectively treat radiation syndromes and related injuries, BARDA will increase the Nation’s preparedness to respond to individuals in most need of critical care following a radiological/nuclear event. Future investments may include advanced development and regulatory approval of cytokines similar to G-CSF and GM-CSF from other manufacturers to diversify the market and increase competition. BARDA’s investments will continue support a robust armamentarium of countermeasures, optimized clinical management plans and well-trained communities of medical professionals and institutions capable of responding to radiological/nuclear threats.