The U.S. Food and Drug Administration (FDA) has approved a new drug that has its origins at the Oklahoma Medical Research Foundation as the first biologic treatment for patients with a rare genetic defect that can cause a potentially life-threatening clotting disorder.
The drug is a treatment for patients with complications related to severe congenital Protein C deficiency, a genetic defect found in one to two newborns for every million births.
The drug has roots in discoveries made almost two decades ago by a pair of OMRF scientists: Charles Esmon, Ph.D., and Naomi Esmon, Ph.D. OMRF filed for patents on those discoveries in 1988, and the patents issued in 1993 and 1994.
Patients with insufficient Protein C levels experience abnormally high numbers of blood clots, which can occur in the blood vessels of the skin, eyes, brain, kidneys and elsewhere. The new drug is intended to treat those patients when they are faced with a life-threatening situation from blood clots in the veins or from a skin and systemic clotting disorder known as purpura fulminans (PF). In clinical trials, it was found to be effective in 94 percent of patients suffering from PF and other acute thrombotic episodes.
“This drug offers much-needed treatment for patients with this severe deficiency, which could lead to blindness, severe brain damage, multi-organ failure and even death,” said Charles Esmon. “It is clear that for this group of patients, this therapy offers new hope for a quality life.”
The drug is made from the plasma of healthy human blood donors and is a concentrated form of Protein C, a substance normally manufactured in the liver that circulates in the plasma in very small amounts.
This is the second drug with roots in Charles Esmon’s lab at OMRF to receive FDA approval. Esmon’s work also led to the development of a treatment for severe sepsis, a blood infection that is the leading cause of death in America’s intensive care units. That drug received FDA approval in 2001.
Chartered in 1946, OMRF (www.omrf.org) is an independent, nonprofit biomedical research institute dedicated to understanding and developing more effective treatments for human disease. Its scientists focus on such critical research areas as Alzheimer’s disease, cancer, lupus and cardiovascular disease. OMRF is home to Oklahoma’s only member of the National Academy of Sciences.