Young, just starting as a physician-scientist at the National Institutes of Health, found the cure. It was the first in a prolific chain of discoveries for which he has been nominated for a Samuel J. Heyman Service to America award.
Young, a finalist for the Science and Environment medal, is one of 33 federal employees nominated in nine categories who will be honored for outstanding service to advance the nation’s health and safety on Thursday, when winners will be announced.
Now the chief of the hematology branch at NIH’s National Heart, Lung, and Blood Institute, he is regarded as a world expert on bone-marrow-failure diseases. His Bethesda clinic draws young and old from around the world who cannot make the blood cells they need to survive. He is also a director of NIH’s Center for Human Immunology, Autoimmunity and Inflammation, and trains hematologists around the world.
Young recalled that the fear surrounding aplastic anemia when he was a medical resident in the 1970s was overwhelming.
“There was a terrifying sense of drama,” he said. “Perfectly healthy people started bleeding, becoming anemic and succumbing to awful infections.”
That’s because their bone marrow had stopped making platelet cells that help the blood clot, as well as red blood cells that carry oxygen and white blood cells that fight infection. The anemia is rare, striking from 600 to 900 people in the United States and a few thousand around the world each year.
The scariest part in those days, Young added, was looking at the samples of his anemic patients’ bone marrow. “It would be totally empty of blood cells — it was just yellow fat.”
Nobody knew how one acquired aplastic anemia, or who was at risk. Some doctors thought it could be contracted by coming into contact with a bad chemical or drug. Others speculated that it was an autoimmune disease; the body attacking its own bone marrow like it would a bacteria or a virus.
But that idea had never been proved, and in the early 1980s Young conducted the first nationwide clinical trial to test if a treatment that suppressed the immune system could cure the anemia and allow the bone marrow to regenerate.
“I had just begun my career — when I look back now, I can’t believe I had the gumption to try and organize such a huge study,” he said.
The trial included roughly 150 aplastic anemia patients at 12 clinical sites around the country. More than half of the patients who received immunosuppressive treatment improved.