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Stephen L. DeFelice, MD

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Translational Science - How Doctornauts Can Help

Senator Tom Harkin (D-IA) has been one of the leaders in Congress on Translational Science.

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Orphan Drugs

Edited by Fred E. Karch, Marcel Decker, Inc. New York, 1982
Go directly to the publisher's page about this book at:

Contains "The Carnitine Story," a chapter by Stephen L. DeFelice, M.D. which can be read here on-line.


About the book...

Orphan Drugs examines, in detail, a complex, often frustrating question-why is it that many useful drugs are not available for the patients who need them? The developers of these drugs are unable to attract enough financial support from pharmaceutical manufacturers or federal agencies to fully test their discoveries. This innovative book illustrates the factors that lead to orphan status and presents model programs for their adoption.

With case histories of orphan drugs, written by individuals intimately involved in their development, the book meets the vital needs of pharmacologists and pharmacists, executives in the pharmaceutical industry, legislators and government regulatory officals, as well as consumer advocates and physicians. Orphan Drugs is certain to help these professionals provide patients with the best possible health care.


About the editor...

FRED E. KARCH is Clinical Assistant Professor of Pharmacology and Toxicology and Medicine, University of Rochester School of Medicine and Dentistry, Rochester, New York. Dr. Karch's research interests focus on the evaluation of adverse drug reactions and physician prescribing behavior, and he is the author of numerous papers in these and related areas. In recognition of his research contributions, he was awarded a National Research Service Award in Clinical Pharmacology by the National Institute of General Medical Sciences. He received his M.D. with distinction (1972) from the University of Rochester. Dr. Karch is a member of the American Society for Clinical Pharmacology and Therapeutics and a Fellow of the American College of Clinical Pharmacology.

Printed in the United States of America ISBN: O-8247-1681-7



What are "Orphan Drugs"? This is the name for a group of drugs which could save lives and alleviate suffering if they were made available. These drugs have already been discovered and could assist in the potential treatment of a wide range of illnesses such as: congestive heart failure, kidney infections, shock, gallstones, and epilepsy.

The question is: "Why aren't these drugs available?" There are many different reasons for this: money, insufficient market, commercial viability, patent problems, federal funding, etc.

This book presents case histories of six acknowledged orphan drugs, which illustrate the scope and complexity of the problem. These drugs cover: cardiovascular disease, disabling neurologic disorders, inherited metabolic diseases, chronic pulmonary disorders, and the treatment of chronic pain. By examining the individual cases, we can identify many of the factors that have kept these drugs from being developed.

In addition to discussing the orphan drugs themselves, we have also examined some of the efforts that have been made by federal agencies and the pharmaceutical industry to help adopt the orphan drugs.

Although much has been done, many valuable therapies remain undeveloped and unavailable. The last chapter discusses what can be done to help develop these drugs. Too many patients suffer needlessly while waiting for their orphan drugs.

Fred E. Karch







1 Why Orphan Drugs? - Fred E. Karch


2 L-5-Hydroxytryptophan - Melvin H. Van Woert

3 The Carnitine Story - Stephen L. De Felice

4 Triethylene Tetramine Dihydrochloride: A New Chelating Agent for Copper - J. M. Walshe

5 Alkylating Local Anesthetics - James F. Stubbins

6 Benzolamide: A Renal Carbonic Anhydrase Inhibitor - Thomas H. Maren

7 Development of Dopamine: An Example of Academic and Industrial Collaboration - Leon I. Goldberg and John F. Zaroslinski


8 The Drug Development Program of the National Cancer Institute: Its History, Results, and Impact on Marketing - C. Gordon Zubrod

9 The Antiepileptic Drug Development Program - Ronald L. Krall

10 Drugs for Parasitic Diseases - Sandra L. Ford

11 Contributions of the Pharmaceutical Industry - Thomas H. Althuis


12 How Can Society Facilitate the Adoption of Orphan Drugs? - Louis Lasagna