.* SERVIC eg « 2 4 Public Health Service . = 2 s = % % %, >: € DEPARTMENT OF HEALTH & HUMAN SERVICES March 24, 1987 National Institutes of Health National Heart, Lung, and Blood Institute Bethesda, Maryland 20892 Dr. David A. Hamburg President “Carnegie Corporation of N.Y. 437 Madison Avenue New York, NY 10022 Dear Dr. Hamburg: In reference to your letter of March 18, I have written to Dr. Benedict (copy attached). It is estimated there are 20 to 40,000 al-antitrypsin deficient individuals in the United States with probably equal (or more) in Europe. Needless to say, since we developed this approach to therapy, we have been deluged with requests to admit patients into the study. As I outlined in the letter to Dr. Benedict, the major obstacle to having her husband participate in such a study is the fact that the Food and Drug Administration has limited the use of the material to the United States. For the first 18 months of our evaluation, they had limited the use of the material only to being administered at our Insti- tution; they have now allowed us to give it out to physicians around the United States. This may be insurmountable for Dr. Benedict’s husband. However, we would be pleased to evaluate him if they could come to the United States. Then the various aspects of participation in such studies could be discussed in detail. If they could arrange to have the clinical information from their physi- cian sent to me, we could make arrangements within a few weeks to have Dr. Dr. Benedict’s husband evaluated. I am enclosing some articles that I sent to Dr. Benedict; I thought these might be of interest. I am also enclosing an article relating to the therapy itself; the latter is scheduled to be published in the New England Journal of Medicine later next month. I do not know whether we can be helpful to Dr. Benedict’s husband but we will do everything possible. Sincerely yours, (Re, le Ronald G. Crystal, M.D. Chief, Pulmonary Branch National Heart, Lung, and Blood Institute cc: Joshua Lederberg RGC: Las