Okla. Medical Research Found.-Based Drug Found Highly Effective in Treating Rare Blood Disorder
Posted on: Wednesday, 1 February 2006, 21:00 CST
By Journal Record Staff
A drug based on discoveries made at the Oklahoma Medical Research Foundation has been shown to be highly effective in treating a rare, often debilitating blood disorder.
The drug's manufacturer, Connecticut-based Alexion Pharmaceuticals, announced that the drug, eculizumab, sharply reduced the need for blood transfusions in clinical trials involving patients with the blood disorder paroxysmal nocturnal hemoglobinuria. The company said that it planned to file for approval from the Food and Drug Administration by end of the year, following the completion of another trial examining the drug's safety.
The drug is based on discoveries made at OMRF by Peter Sims and Therese Wiedmer in collaboration with researchers at Yale University. The two institutions filed for patents on the research in 1989 and first licensed the discoveries to Alexion in 1992.
Eculizumab would be the first drug specifically to treat paroxysmal nocturnal hemoglobinuria, an acquired genetic disorder in which a person's own immune system destroys the body's red blood cells. This causes hemoglobin to spill into the bloodstream, which can lead to life-threatening blood clots, as well as anemia, severe abdominal pain, chronic fatigue and pulmonary hypertension.
An estimated 8,000 to 10,000 people in North America and Western Europe suffer from paroxysmal nocturnal hemoglobinuria. Patients must typically undergo frequent blood transfusions, and many are unable to live normal lives as a result of the disease.
This is an illness that decimates patients' lives and can be fatal, said J. Donald Capra, president of OMRF. If the work our scientists did can help offer them a new treatment avenue, it will be a major step forward for those patients and an extremely gratifying moment for OMRF.
In the clinical trial, approximately half the patients given the drug had stable hemoglobin levels over six months without needing a transfusion. None of those receiving a placebo showed such results.
It has been a very long time - 17 years - since OMRF first filed for the patents on this discovery, Capra said. But if this drug proves safe and effective, it will have been worth the wait.
If the drug clears remaining regulatory hurdles, it will be the third OMRF-based drug to reach the market. The others are Xigris, the only FDA-approved treatment for severe sepsis, a deadly blood infection that kills 200,000 Americans each year, and Ceprotin, used to treat children suffering from a life-threatening protein deficiency in their blood.
Source: Journal Record - Oklahoma City
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