Tracleer and PPH News
Bill would fund research for PHT treatment
By RYAN HOLEYWELL
WASHINGTON, June 24 (UPI) -- Bipartisan legislation introduced this week in the House would authorize $250 million over the next five years for a new research effort at the National Institutes of Health aimed at finding treatments for pulmonary hypertension, a little-known disorder that affects at least 100,000 Americans.
The Pulmonary Hypertension Research Act, co-sponsored by Reps. Kevin Brady, R-Texas, and Tom Lantos, D-Calif., may give hope to people like John Kostur, a 42-year-old Maryland resident who suffers from PHT and must wear a device the size of a cassette-tape player that continuously pumps a drug called Flolan into his bloodstream.
"I hope that they'll find a cure," Kostur told United Press International. "It's very sad to see this pump on a child."
Four years ago Kostur began experiencing severe shortness of breath and was diagnosed with a sinus problem associated with his deviated septum, but after passing out at work one day and being rushed to a hospital, doctors at Johns Hopkins University were the first to diagnose Kostur with his condition, which he thinks he has had his whole life.
PHT is a blood-vessel disorder that occurs when pressure in the pulmonary artery, which leads from the heart to the lungs, rises above normal levels.
In PHT patients, the right side of the heart is forced to pump harder to transport blood to the lungs, causing the heart to enlarge and ultimately fail. The disease can be fatal and is most common in women age 21 to 40, although it can occur in men and women of all ages. Symptoms can include fatigue, chest pain, shortness of breath, dizziness and fainting.
Patients develop the disease as a result of other cardiac problems, but a smaller group, suffering from what is called primary pulmonary hypertension, develops the disease without any known underlying cause. There is a familiar form of the disease, but more often than not it is an isolated finding within a family.
Historically, those diagnosed with the disease have a poor survival rate, but today some can survive for 15 or 20 years.
At a news conference announcing the legislation, Brady said that a few years ago there was only one drug to treat the disease, but today four drugs can be used, and six more are being tested. He also said the survival rate has improved slightly over the last few years.
The gene responsible for the disease was identified in 2000, opening up new opportunities for studying the disease.
Dr. Stuart Berger, medical director of the Herma Heart Center at the Children's Hospital of Wisconsin in Milwaukee, said researchers are looking at possible molecular pathways that cause the disease. They also are trying to understand what triggers the affected blood vessels to relax and contract, he said.
"Once the mechanisms can be understood, we can get a better handle on how to attack these mechanisms and reverse ... or some day prevent (PHT)," Berger told UPI.
He cautioned, however, that a cure for the disease is "a long way away," because there are so many different theories about it.
"Until we understand that completely, I think we're not quite there in terms of an underlying cure," Berger said.
At the news conference, Lantos said he thinks the legislation will pass, because the Senate majority and minority leaders, as well as both the House majority and minority whips, have pledged their support.
Lantos said PHT is "one of the most undiagnosed, misdiagnosed" diseases.
Brady said patients report an average of three physicians who misdiagnose their affliction before a fourth one finally identifies it. He added that the key to improving patients' odds is early diagnosis.
Carl Hicks, a board member of the Pulmonary Hypertension Association, said that 10 years ago only 195 patients had been diagnosed with the disease, a number that has grown exponentially because PHT has become more widely recognized.
Hicks, whose daughter was diagnosed 11 years ago, spoke at the news conference in front of an audience that included a group of PHT patients. As he finished his remarks, tears streamed down his face and he hugged Lantos.
"We will always be deeply indebted to you," Hicks told the congressman.
Charity Sunshine Tillemann-Dick, Lantos's 21-year-old granddaughter, suffers from PHT. An opera singer, Charity performed at the Kennedy Center last week with Secretary of State Condoleezza Rice to help raise awareness about the issue.
"I hope my medications will be able to work, I hope I'll be able to sing, I hope I'll be able to get up in the morning," she told reporters, "but the truth is hope is empty without action."
Earlier this month the Food and Drug Administration approved the use of sildenafil -- the key ingredient in the anti-impotence pill Viagra -- as a PHT treatment. An FDA study showed sildenafil improved patients' ability to exercise and could become a useful treatment.
Ryan Holeywell is an intern for UPI Science News. E-mail: sciencemail@upi.com
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