Orphan Drugs: Hope Where There Is Little or No Hope

NEW YORK, N.Y., February 18, 2004 – On a visit to his doctor, Gary Jacob received distressing news – not about himself, but a friend of the doctor’s. While playing with one of his children, the doctor’s friend fell and broke a rib. That was bad enough, but during the examination at the hospital, the father was hit with a startling and totally unexpected diagnosis – he had a disease known as multiple myeloma, a bone marrow blood cancer. The diagnosis was nothing less than a death sentence. Jacob knew of the anguish of multiple myeloma patients. The disease is incurable and nearly always fatal, one of the rare diseases that have few, if any, available treatments. They are known …