Primary Pulmonary Hypertension News - Menu Bill would fund research for PHT treatment By RYAN HOLEYWELL 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 Pulmonary Hypertension Research Act of 2001 H.R. 1297 Pulmonary hypertension is characterized by sustained, abnormally high blood pressure in the arteries of the lungs. There are two kinds of pulmonary hypertension: primary and secondary. Primary, or unexplained, pulmonary hypertension (PPH) is a rare lung disorder that occurs for no apparent reason. There is no cure for PPH, and it has a low survival rate. Secondary pulmonary hypertension (SPH) means the cause of SPH is known. Common causes of SPH are emphysema, cystic fibrosis, and congenital heart disease. Estimates of the incidence of PPH range from one to two people per million in the general public. In the United States, it has been estimated that 300 new cases of PPH are diagnosed each year; the greatest number are reported in women between the ages of 21 and 40. Based on data from the National Heart, Lung, and Blood Institute (NHLBI)-supported National Patient Registry for PPH, which recruited patients between 1981 and 1985, it was estimated that approximately 6 percent of the cases of PPH are due to an inherited defect. This may be an underestimate; however, since it appears that not everybody who inherits the defective gene develops the disease, the familial component is not always discernable. Interest in pulmonary hypertension has escalated due to a rise in the number of cases associated with the appetite-suppressant drugs fenfluramine-phentermine (fen-phen) and dexfenfluramine (Redux). The treatment for PPH remains limited. The most successful treatment currently in use is continuous intravenous prostacyclin, a potent vasodilator. Despite its favorable outcome in some patients, it is very costly, complicated to administer, and associated with a number of systemic side effects. Lung transplantation is an option for some patients when all other forms of treatment have been unsuccessful. There are now several new drugs being evaluated in clinical trials that hold promise for a more effective treatment for this devastating disease. H.R. 1297, the Pulmonary Hypertension Research Act of 2001, would have amended the Public Health Service Act to provide for pulmonary hypertension research at NHLBI. As cited in the "Findings," the bill seeks to further the Federal investment in pulmonary hypertension to take advantage of the potential for finding a cure or an effective treatment. Provisions of the Legislation/Impact on NIH H.R. 1297 would have required the Director of NHLBI to expand, intensify, and coordinate the Institute's activities with respect to pulmonary hypertension, including coordination with similar activities of other Institutes and Centers of the National Institutes of Health. In addition, the Director of NHLBI would have been required to make grants to, or enter into contracts with, public or nonprofit private organizations for the development and operation of three Centers of Excellence to conduct relevant basic and clinical research, provide research training for scientists and health professionals, provide information and continuing education for health professionals, and disseminate information to the public. The Director of NHLBI also would have been required to coordinate information exchange between Centers, including the periodic preparation of reports. Support for each Center would not have exceeded a period of 5 years, although an extension for up to an additional 5 years could have been made upon appropriate technical and scientific peer review. The bill authorized up to $25 million for fiscal years 2002 through 2006. H.R. 1297 was introduced on March 29, 2001, by Representative Kevin P. Brady (R-TX) and was referred to the House Energy and Commerce Committee. There was no further action on this legislation during the 107th Congress. 107th Congress H.R. 1297 and H. Con. Res. 381 Pulmonary hypertension is characterized by sustained, abnormally high blood pressure in the arteries of the lungs. There are two kinds of pulmonary hypertension: primary and secondary. Primary, or unexplained, pulmonary hypertension (PPH) is a rare lung disorder that occurs for no apparent reason. There is no cure for PPH, and it has a poor survival rate. Secondary pulmonary hypertension (SPH) means the cause is known. Common causes of SPH are emphysema, cystic fibrosis, and congenital heart disease. Estimates of the incidence of PPH range from 1 to 2 people per million in the general public. In the United States, an estimated 300 new cases of PPH are diagnosed each year; the greatest number of cases are reported in women between the ages of 21 and 40. Based on data from the National Heart, Lung, and Blood Institute- (NHLBI-) supported National Patient Registry for PPH, which recruited patients between 1981 and 1985, approximately 6 percent of the cases of PPH are due to an inherited defect. This may be an underestimate, however, since it appears that not everybody who inherits the defective gene develops the disease, and thus, the familial component is not always discernable. Interest in pulmonary hypertension has escalated because of an increase in the number of cases associated with the appetite-suppressant drugs fenfluramine-phentermine (fen-phen) and dexfenfluramine (Redux). The treatment for PPH remains limited. The most successful treatment currently in use is continuous intravenous prostacyclin, a potent vasodilator. Despite its favorable outcome in some patients, the drug is very costly, complicated to administer, and associated with a number of systemic side effects. Lung transplantation is an option for some patients when all other forms of treatment have been unsuccessful. Several new drugs that hold promise for a more effective treatment for this devastating disease are being evaluated in clinical trials. H.R. 1297, the Pulmonary Hypertension Research Act of 2000, would amend the Public Health Service Act to provide for pulmonary hypertension research at NHLBI. As cited in the "Findings," the bill seeks to further the Federal investment in pulmonary hypertension research to take advantage of the potential for finding a cure or an effective treatment. H. Con. Res. 381 is a concurrent resolution aimed at increasing awareness of pulmonary hypertension. Concurrent resolutions deal with the prerogatives or internal affairs of Congress as a whole and do not have the force of law. H.R. 1297 would require the Director of NHLBI to expand, intensify, and coordinate the Institute's activities with respect to pulmonary hypertension, including coordination with similar activities of other Institutes and Centers of the National Institutes of Health. In addition, the Director of NHLBI would be required to make grants to, or enter into contracts with, public or nonprofit private organizations for the development and operation of three Centers of Excellence to conduct relevant basic and clinical research, research training for scientists and health professionals, information and continuing education for health professionals, and information dissemination to the public. The Director of NHLBI also would be required to coordinate an information exchange between the Centers, including the periodic preparation of reports. Support for each Center would not exceed a period of 5 years, although an extension for up to an additional 5 years could be made upon appropriate technical and scientific peer review. The bill authorizes up to $25 million for fiscal years 2002 through 2006. H. Con. Res. 381 expresses the Sense of the Congress that 1) the role played by national and community organizations and health care providers in promoting awareness of pulmonary hypertension should be recognized and applauded, 2) the Federal Government has a responsibility to raise awareness of pulmonary hypertension, increase research funding, and consider ways to improve access to health care, 3) the Director of NHLBI should continue to take a leadership role through expansion of research, 4) the Director of the Centers for Disease Control and Prevention should give priority consideration to pulmonary hypertension awareness programs, 5) a National Pulmonary Hypertension Awareness Month should be established, and 6) the President should issue a proclamation recognizing a National Pulmonary Hypertension Awareness Month. H.R. 1297 was introduced on March 29, 2001, by Representative Kevin P. Brady (R-TX) and was referred to the House Committee on Energy and Commerce. No further action has occurred on H.R. 1297 since its introduction. H. Con. Res. 381 was introduced on April 18, 2002, by Representative Robert A. Brady (D-PA) and was referred to the House Committee on Energy and Commerce. 1st Session H. R. 3005 To amend the Public Health Service Act to provide for the expansion, intensification, and coordination of the activities of the National Heart, Lung, and Blood Institute with respect to research on pulmonary hypertension. IN THE HOUSE OF REPRESENTATIVES June 21, 2005 Mr. BRADY of Texas (for himself, Mr. LANTOS, Mr. BLUNT, and Mr. HOYER) introduced the following bill; which was referred to the Committee on Energy and Commerce A BILL To amend the Public Health Service Act to provide for the expansion, intensification, and coordination of the activities of the National Heart, Lung, and Blood Institute with respect to research on pulmonary hypertension. Be it enacted by the Senate and House of Representatives of the United States of America in Congress assembled, SECTION 1. SHORT TITLE. This Act may be cited as the `Pulmonary Hypertension Research Act of 2005'. SEC. 2. FINDINGS. The Congress finds as follows: (1) In order to take full advantage of the tremendous potential for finding a cure or effective treatment, the Federal investment in pulmonary hypertension must be expanded, and coordination among the national research institutes of the National Institutes of Health must be strengthened. (2) Pulmonary hypertension is a serious and often fatal condition where the blood pressure in the lungs rises to dangerously high levels. In PH patients, the walls of the arteries that take blood from the right side of the heart to the lungs thicken and constrict. As a result, the right side of the heart has to pump harder to move blood into the lungs, causing it to enlarge and ultimately fail. (3) In the United States it has been estimated that 300 new cases of PPH are diagnosed each year, or about two persons per million population per year; the greatest number are reported in women between the ages of 21 and 40. While at one time the disease was thought to occur among young women almost exclusively; we now know, however, that men and women in all age ranges, from very young children to elderly people, can develop PPH. It also affects people of all racial and ethnic origins with African Americans suffering from a mortality rate twice as high as that affecting Caucasians. (4) The low prevalence of PPH makes learning more about the disease extremely difficult. Studies of PPH also have been difficult because a good animal model of the disease has not been available. (5) In about 6 to 10 percent of cases, PPH is familial. The familial PPH gene is located on chromosome 2 and was discovered in July 2000. This discovery provided new insights for determining the molecular basis of PPH and opened new avenues of study for understanding the fundamental nature of the disease. (6) In the more advanced stages of PPH, the patient is able to perform only minimal activity and has symptoms even when resting. The disease may worsen to the point where the patient is completely bedridden. (7) PPH remains a diagnosis of exclusion and is rarely picked up in a routine medical examination. Even in its later stages, the signs of the disease can be confused with other conditions affecting the heart and lungs. The use of new diagnostic standards has been positively related to the rates of diagnosis. (8) In 1981, the National Heart, Lung, and Blood Institute established the first PPH-patient registry in the world. The registry followed 194 people with PPH over a period of at least 1 year and, in some cases, for as long as 7.5 years. Much of what we know about the illness today stems from this study. (9) As research progresses, so do treatments for PH. Currently, there are four FDA-approved medications for PH and three more in trials. However, all medications not are affective on all patients. Lung transplantation is often considered a treatment of last resort for PH. (10) Because we still do not understand the cause or have a cure for PPH, basic research studies are focusing on the possible involvement of immunologic and genetic factors in the cause and progression of PPH, looking at agents that cause narrowing of the pulmonary blood vessels, and identifying factors that cause growth of smooth muscle and formation of scar tissue in the vessel walls. (11) Secondary pulmonary hypertension (`SPH') means the cause is known. Common causes of SPH are the breathing disorders emphysema and bronchitis. Other less frequent causes are the inflammatory or collagen vascular diseases such as scleroderma, CREST syndrome or systemic lupus erythematosus (`SLE'). Other causes include congenital heart diseases that cause shunting of extra blood through the lungs like ventricular and atrial septal defects, chronic pulmonary thromboembolism, HIV infection, and liver disease. Sickle cell anemia is also linked to SPH with preliminary studies suggesting that approximately one third of sickle cell patients develop SPH. SEC. 3. EXPANSION, INTENSIFICATION, AND COORDINATION OF ACTIVITIES OF NATIONAL HEART, LUNG, AND BLOOD INSTITUTE WITH RESPECT TO RESEARCH ON PULMONARY HYPERTENSION. Subpart 2 of part C of title IV of the Public Health Service Act (42 U.S.C. 285b et seq.) is amended by inserting after section 424B the following section: `PULMONARY HYPERTENSION `SEC. 424C. (a) In General- `(1) EXPANSION OF ACTIVITIES- The Director of the Institute shall expand, intensify, and coordinate the activities of the Institute with respect to research on pulmonary hypertension. `(2) COORDINATION WITH OTHER INSTITUTES- The Director of the Institute shall coordinate the activities of the Director under paragraph (1) with similar activities conducted by other national research institutes and agencies of the National Institutes of Health to the extent that such Institutes and agencies have responsibilities that are related to pulmonary hypertension. `(b) Centers of Excellence- `(1) IN GENERAL- In carrying out subsection (a), the Director of the Institute shall make grants to, or enter into contracts with, public or nonprofit private entities for the development and operation of centers to conduct research on pulmonary hypertension. `(2) RESEARCH, TRAINING, AND INFORMATION AND EDUCATION- `(A) IN GENERAL- With respect to pulmonary hypertension, each center assisted under paragraph (1) shall-- `(i) conduct basic and clinical research into the cause, diagnosis, early detection, prevention, control, and treatment of such disease; `(ii) conduct training programs for scientists and health professionals; `(iii) conduct programs to provide information and continuing education to health professionals; and `(iv) conduct programs for the dissemination of information to the public. `(B) STIPENDS FOR TRAINING OF HEALTH PROFESSIONALS- A center under paragraph (1) may use funds under such paragraph to provide stipends for scientists and health professionals enrolled in programs described in subparagraph (A)(ii). `(3) COORDINATION OF CENTERS; REPORTS- The Director shall, as appropriate, provide for the coordination of information among centers under paragraph (1) and ensure regular communication between such centers, and may require the periodic preparation of reports on the activities of the centers and the submission of the reports to the Director. `(4) ORGANIZATION OF CENTERS- Each center under paragraph (1) shall use the facilities of a single institution, or be formed from a consortium of cooperating institutions, meeting such requirements as may be prescribed by the Director. `(5) NUMBER OF CENTERS; DURATION OF SUPPORT- The Director shall, subject to the extent of amounts made available in appropriations Acts, provide for the establishment of not less than three centers under paragraph (1). Support of such a center may be for a period not exceeding 5 years. Such period may be extended for one or more additional periods not exceeding 5 years if the operations of such center have been reviewed by an appropriate technical and scientific peer review group established by the Director and if such group has recommended to the Director that such period should be extended. `(c) Data System; Clearinghouse- `(1) DATA SYSTEM- The Director of the Institute shall establish a data system for the collection, storage, analysis, retrieval, and dissemination of data derived from patient populations with pulmonary hypertension, including where possible, data involving general populations for the purpose of identifying individuals at risk of developing such condition. `(2) CLEARINGHOUSE- The Director of the Institute shall establish an information clearinghouse to facilitate and enhance, through the effective dissemination of information, knowledge and understanding of pulmonary hypertension by health professionals, patients, industry, and the public. `(d) Public Input- In carrying out subsection (a), the Director of the Institute shall provide for means through which the public can obtain information on the existing and planned programs and activities of the National Institutes of Health with respect to primary hypertension and through which the Director can receive comments from the public regarding such programs and activities. `(e) Reports- The Director of the Institute shall prepare biennial reports on the activities conducted and supported under this section, and shall include such reports in the biennial reports prepared by the Director under section 407. `(f) Authorization of Appropriations- For the purpose of carrying out this section, there is authorized to be appropriated $50,000,000 for each of the fiscal years 2006 through 2010.'. |