New medication offers new hope for survival 'This is my lifeline,' says patient with rare disease

February 01, 1996|By Jonathan Bor | Jonathan Bor,SUN STAFF

A new drug that must be continually infused by a portable pump offers hope to people suffering from a life-threatening disease marked by high blood pressure in the lungs, doctors said yesterday.

The drug, Prostacyclin, is a synthetic version of a natural substance that is produced by the linings of blood vessels. Yesterday, doctors declared that it brought significant improvement to three-quarters of the patients who tried it in a three-month study.

"For the first time in this disease, a drug has been shown to improve not only exercise tolerance and quality of life, but also survival," said Dr. Lewis J. Rubin, head of pulmonary medicine at the University of Maryland Medical Center and one of the study's chief investigators.

The disease, primary pulmonary hypertension, is marked by an abnormally high resistance to blood flow in the arteries supplying the lungs. This makes it hard for the heart to pump blood into the adjacent organ, causing the heart to enlarge and weaken.

The patient becomes short of breath, has difficulty walking and -- in most cases -- dies of heart failure. Roughly a quarter of all patients respond to conventional drugs that dilate the vessels. Some can benefit from lung transplants, but patients often die waiting for organs.

Janice Rosenzweig, 45, a Baltimore attorney who enrolled in the study, said Prostacyclin immediately ended the shortness of breath that had made it difficult for her to walk a block. "This drug is my lifeline," she said.

Taking the drug is no small matter. Inside her purse, she carries a lightweight pump that continually delivers the drug through a catheter and into an artery that feeds the heart. The drug costs $88,000 a year, most of it covered by insurance.

Primary pulmonary hypertension is extremely rare, afflicting about 1,500 people in the United States. But U.S. and European doctors have observed a recent upsurge, particularly among patients who have taken diet pills.

Dr. Rubin said the diet pills fall into two categories -- stimulants that act much like amphetamines and chemicals that suppress appetite by raising levels of a brain chemical known as seratonin.

Most patients taking the pills do not develop pulmonary hypertension, he said. But some, for reasons that are not understood, have a susceptibility that is triggered by the diet drugs, he said.

It is not clear how Prostacyclin works. Doctors first thought it reduced blood pressure by relaxing the blood vessels, Dr. Rubin said. More recently, they have leaned toward another explanation: that it reduces platelets' stickiness.

An article reporting the results of the study appears in today's New England Journal of Medicine.

The experiment, conducted at 15 medical centers, was one of two studies that formed the basis for the Food and Drug Administration's decision in September to approve the drug for commercial sale. Eighty-one patients who had failed conventional therapy took part. Half were given Prostacyclin, while half continued their therapy.

Three-quarters of those taking the new drug improved considerably, while all the patients in the other group remained the same or got worse. Eight patients died; all were on conventional drugs.

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