I can assure you that there are new leads of great promise that were not in our hands 1 year ago. Investigators in Pennsylvania have evidence today that the pathological process within a cystic fibrosis gland may be reversible. A drug administered intravenously to cystic fibrosis children has temporarily restored abnormal salivary gland secretions toward normal. This is the first time to our knowledge that a drug has altered the abnormal processes of a cystic fibrosis gland this way. This drug is a valuable tool in the manipulation of secretions of a diseased gland and will allow basic studies of the nature of the altered glandular disturbance. A second exciting and promising finding from Duke University indicates that there may be a blood factor associated with cystic fibrosis, which we just alluded to. A biological assay has been developed which has permitted the Duke workers to identify a unique property in the serum protein fraction of cystic fibrosis patients. Of equal importance is that parents of cystic fibrosis patients also have this factor but to a lesser degree. When normal populations were tested for presence of this material, one out of 20 so-called normal individuals possessed this property in their blood serum protein. This coincides with the expected incidence of carriers of the cystic fibrosis gene in normal populations. Duke investigators have consistently demonstrated that this may be a cystic fibrosis genetic marker. They are now trying to isolate this material to identify and study it. They want to find out what role it plays in cause and effect in cystic fibrosis. They believe that this could allow them to discover the origin and control mechanisms operating in this material. This is our first clue that there might be a substance circulating in the blood which could play a role in the disease process. There is also an effort to develop a simple test for cystic fibrosis gene carriers from this new findings. Basic studies in cystic fibrosis have been hampered because of the severe restriction and limitation that must be placed on doing research on very ill children. Now that there is evidence that parents also have the defect but to a lesser degree, more meanful research may be possible using cystic fibrosis parents as volunteers. Because there are an estimated 10 to 12 million Americans who carry the cystic fibrosis gene, the finding that there may be a partial metabolic defect in carriers is exceedingly important. Almost everyone agres that the finding of a cystic fibrosis counterpart in an animal could enhance research on this disease. This time last year there was no known way of producing symptoms in animals that could be directly linked to cystic fibrosis processes. Today, gentlemen, we are happy to say, less than 1 year since you last reviewed this phenomenon of medicine we can tell you that there is reason to believe that an animal system can be developed which could revolutionize cystic fibrosis research. One group of investigators in Wisconsin has shown that cystic fibrosis saliva and sweat contain some substance which causes a dramatic change in the rat salivary gland. When cystic fibrosis sweat or saliva is injected into the rat salivary gland, that gland loses its ability to handle salt normally. This property appears unique to cystic fibrosis sweat and saliva since normal sweat or saliva have no measurable effect on salt in this animal test system. The potential for this animal system is immense. Enzymes, hormones, drugs, and various agents can be tested for their ability to modify this salt loss effect. This may be our first screening system for more and new agents to treat cystic fibrosis. This could be important in many other areas such as kidney disease and intestinal problems. Already Wisconsin workers are trying to correlate findings about sweat and saliva with those of the Duke investigators. Both of them may be looking at the same substance. Workers at the University of Miami, collaborating with investigators of the Arthritis and Metabolic Diseases Institute, have uncovered evidence of a unique substance in cystic fibrosis tissue using sensitive techniques. They are trying to determine the relationship of their tissue factor to that in the blood of cystic fibrosis patients at Duke and in sweat and saliva at Wisconsin. Mr. FLOOD. Cystic fibrosis tissue? Dr. PATTERSON. A tissue factor that may be localized in the cells and not just circulating in the blood, you see. These are some of the exciting clues that have been developed from research during the past year. The research has been going on for some time with the support of the NIH and the National Cystic Fibrosis Research Foundation, but this past year, in terms of promising leads, appears to have been a bonus year. Mr. CASEY. Mr. Chairman, you recall when the NIH was here they were telling us that the reason that their request was not for a greater amount was that they were moving slowly because they had not found any breakthrough to tie into, to really move forward. It sounds to me like they are getting right to the point where they could-they are getting some leads now that should be followed up. Dr. PATTERSON. Yes; this is true. In the past 6 months, even more so than the previous 6 months. Certainly we can control to some degree the nutritional problems of the cystic fibrosis child. We can keep the lungs clear and prevent lung damage in children who are diagnosed and treated from an early age. But we have not found an agent that will liquefy the viscid secretions that plug the ducts of glands and organs. We have found an effective means to lower the incidence of death from the lung involvement in cystic fibrosis, but sadly will lose part of this victory to eventual liver involvement, or to involvement of some other vital organ that yields to the progressive accumulation of viscid mucus. We must not guess about these things. We must do research and find the answers. We must pursue, with all our resources, the new clues and leads. There is a great surge of interest in research in cystic fibrosis since the developments I have described have come to light. In fact, there are many more requests for research support from the National Cystic Fibrosis Research Foundation than we can meet. We cannot afford to lose these men's ideas, nor their interest, nor their talent. So, we turn to you in the Congress to provide the means in the Arthritis and Metabolic Diseases Institute to take up this slack. We recommend for fiscal year 1968 an increase in the funds for the Institute of $950,000, for research related to cystic fibrosis and for the training of doctors in that field; $700,000 for research and $250,000 for training and we recommend further that the funds be identified in the budget of the Institute in such a way that they will go to the purposes for which we recommend them. I can assure you gentlemen and Mr. Chairman that these funds will be well spent and will make a noteworthy contribution to the health of our Nation's children. Thank you for your kind attention. Mr. FLOOD. You have a real killer, do you not? PULMONARY CENTERS STATEMENT OF DR. GIULIO J. BARBERO Mr. FLOOD. We will now hear Dr. Giulio J. Barbero on the question of pulmonary centers, and with him will be Dr. LeRoy W. Matthews. Dr. Barbero is the chairman of the department of pediatrics, Hahnemann Medical College, Philadelphia, Pa. (Biographical sketch of Dr. Barbero follows:) Name: Giulio J. Barbero. CURRICULUM VITAE Home address: 502 West Front Street, Media, Pennsylvania. Place of birth: Mount Vernon, New York. Education: Bangor High School, Bangor, Maine, 1940. College: University of Maine, Orono, Maine, 1943 B.S. Medical: University of Pennsylvania, Philadelphia, Pa., 1947 M.D. Internship: Hospital of University of Pennsylvania-Rotating, 1947-1948. Residency: The Children's Hospital of Philadelphia-Pediatrics, 1949-1950, 1952-1953 Post-Graduate: Hosp. of Univ. of Pa.-Pediatric-Gastroenterology and Nutritional Research, 1948-1949. Licensure: Pennsylvania and Maine, 1948. Certified by American Board of Pediatrics, 1954. Academic Positions: Ass't Professor in Pediatrics, Univ. of Pa., 1957-1965. Associate Professor in Pediatrics, Univ. of Pa., 1965. Ass't Instructor in Pediatrics, Graduate School of Medicine, 1955. Senior Physician and Director, Division of Gastroenterology, Children's Hospital of Phila., 1959. Consultant, Atlantic City Seashore House, 1964. Consultant, U.S. Naval Hospital, Phila., Pa., 1964. Memberships: Philadelphia Physiological Society, 1956. Philadelphia Pediatric Society, 1956. American Society for Pediatric Research, 1957. American Gastroenterological Association, 1960. New York Academy of Sciences, 1957. American Academy of Pediatrics, 1959. American Psychosomatic Society, 1960. The John Morgan Society, 1961. National Cystic Fibrosis Research Foundation: Chairman, Professional Education Committee, 1962-1964. Chairman, Research Evaluation & Development Committee, 1964. Dr. BARBERO. Thank you. Mr. Flood, members of the committee, I welcome the opportunity to present this consideration of the needs in cystic fibrosis. We are here to recommend to this committee and the Congress a program of pediatric pulmonary disease centers for the treatment of cystic fibrosis and other children's diseases, to be established in the chronic respiratory control program of the public health Service Bureau of Disease Prevention and Environmental Control. This recommendation is being made to the Congress because of the large number of children with crippling and often fatal pulmonary diseases who are not being properly treated today because of the lack of adequate treatment facilities and the lack of physician specialists competent to diagnose their illnesses and prescribe the proper controls and therapeutic measures for them. The program would include bronchitis, asthma, bronchiectasis, cystic fibrosis and other respiratory diseases with the exception of tuberculosis. And it would embrace coordinated programs of teaching clinical research and training. The benefits of a program such as this have already been demonstrated in pilot efforts financed by the National Cystic Fibrosis Research Foundation. Until recently, cystic fibrosis was considered a hopeless disease. The vital statistics of the United States show that in 1962, 85 percent of deaths due to cystic fibrosis occurred before 10 years of age. In spite of this we have found in one pilot center providing comprehensive care for over 400 children and young adults with cystic fibrosis, the mortality rate has been reduced to less than 2 percent per year and the crippling effects of pulmonary involvement markedly decreased over the past 10 years. Mr. CASEY. Where is this? Dr. BARBERO. At Western Reserve. The gentleman to my left will make such comments and fill us in. Even more important is the demonstration that application of an intensive, comprehensive and prophylactic treatment program prevents the development of serious lung disease in patients diagnosed early. The establishment of pediatric pulmonary disease centers is the most practical and economical method of increasing the availability and improving the quality of care in this field. Total care of the patient with chronic pulmonary disease requires the specialized knowledge and services of a large number of medical specialists and new diagnostic and therapeutic methods are complex and also require specialized personnel and facilities. Very few physicians in general or in pediatric practice have the training, time, or facilities to provide a complete program of therapy. Very few general or local hospitals can afford the specialized facilities. Children are not little adults. The personnel who treat children with chronic respiratory disease must be specially trained and must use equipment and facilities tailored to the size, limited ability to cooperate, and psychologic needs of children. Since many of the new and more effective therapeutic measures are prophylactic in nature it is essential that both the patient and the parents are thoroughly instructed in the nature of the disease, and how the therapy affects the course of disease. Neither patients nor parents will carry out timeconsuming prophylactic treatment if they do not understand why they are doing it. Such instruction requires the team approach. General instruction should be provided by the physician but specific instruction is most effectively and economically provided by trained nurses, physical therapists, inhalation therapists and social workers. Mr. FLOOD. Inhalation therapists, that means the administration of oxygen? Dr. BARBERO. No, it is much more extensive than that. Mr. FLOOD. There are institutes and technical training areas that certificate physicians as inhalational experts, are there not? Dr., BARBERO. Yes. Followup instruction provided in the home by the personnel of community health services is also often essential. Such personnel should be utilized to evaluate the effectiveness of therapy, to determine the effect of the treatment program both emotionally and financially on the patient and the family and to observe the patient for early psychologic problems. A close relationship between the Center and the community health services should exist. Ideally, a child with severe neonatal or chronic respiratory disease should be referred to a Pediatric Pulmonary Disease Center for accurate diagnosis and initiation of a comprehensive treatment program tailored to his particular needs. Mr. FLOOD. Are you speaking of inpatient care? Dr. BARBERO. Totally, both inpatient and outpatient. I am talking about the total personnel which are listed later on here, of a group which will have all varied facilities available to them to tailor them to the individual's need. If possible, he should be worked up and treated as an outpatient but hospital facilities should also be available. The child with severe involvement should be admitted and the trained personnel and special facilities of the Center utilized to establish the diagnosis and to provide the intensive treatment necessary to bring the disease under control. In the outpatient clinic or in the hospital both the patient and the parents should be thoroughly instructed in the disease and the treatment procedures to be utilized in subsequent home therapy. The child should then return home on treatment to be followed routinely by his local physician and at frequent intervals by the Center. This would obviously require close liaison between the Center and the local physician and a significant exchange of information. General problems could then be handled locally and the child referred back to the Center whenever its specially trained personnel and facilities are required. In the few areas where this is now possible |