Originally Released: January 1, 1996
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Results of a large multi-center study, reported in the February 1st issue of the New England Journal of Medicine, show that a new drug offers significant promise for patients with an incurable lung disease called primary pulmonary hypertension (PPH). The drug, Prostacyclin, received FDA approval in September. It is the first drug ever approved to treat that disease. FDA approval was based on data submitted from this study and another randomized, multi-center trial. Both studies were led by researchers at the University of Maryland Medical Center.
Primary pulmonary hypertension is a disorder of the blood vessels inside the lungs. It makes it hard for blood to flow into the lungs, causing lung pressure to rise. Eventually, the condition can cause the right side of the heart to fail. Patients experience shortness of breath, chest pain, and fainting. PPH mainly affects young women in their 20's or 30's, often around or right after pregnancy. Each year, 400 new cases of the disease are diagnosed in the United States.
About 50 percent of people with primary pulmonary hypertension can be treated effectively with medications that dilate the vessels. The others may go on to become very ill and, until the approval of this new drug, their only other option was a lung transplant.
"This is a real breakthrough in the treatment of this disease," says Lewis J. Rubin, M.D., professor of medicine and head of the division of Pulmonary and Critical Care Medicine at the University of Maryland Medical Center and the lead investigator in both studies. "While PPH is not a curable disease, it often can be treated quite effectively. This drug offers long term treatment for some patients or it can help stabilize other patients while they wait for a transplant."
The larger of the two studies to investigate Prostacyclin involved 81 patients at 15 medical centers. Forty-one patients received Prostacyclin and 40 others received conventional therapy, which included oxygen, diuretics, anti-coagulants and vasodilators like calcium channel blocker medications. Of the patients taking Prostacyclin, 75 percent improved considerably, meaning blood flow in the lungs improved. Survival, exercise tolerance and quality of life also got better. The other 25 percent of the patients taking Prostacyclin remained the same or got worse with the drug. None of the patients on conventional therapy improved. They either remained the same or got worse. Eight patients in the group who received conventional therapy died.
Prostacyclin is a natural substance made by the lining of the blood vessels. The substance can open up constricted vessels in the lungs without causing major side effects. Prostacyclin is administered in pre-set doses with a portable infusion pump. The pump is connected to a catheter that leads to the right side of the heart. Patients receiving the drug must stay on it indefinitely and they need increasing amounts of the drug over time.
Prostacyclin is manufactured by Glaxo Wellcome, which provided funding for both studies, along with the National Institutes of Health.
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