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Saving the lives of patients with rare blood diseases

When Princeton University football star Jordan Culbreath was diagnosed in 2009 with the rare and potentially deadly blood disorder known as aplastic anemia, his prospects looked bleak. The life- threatening disease was wiping out the cells in his bone marrow, including red blood cells that carry oxygen, white blood cells that fight infection and platelets that help clot the blood.

Culbreath was fortunate to have found his way to the National Institutes of Health and a team of expert physicians led by Dr. Neal S. Young, where he received immune suppressant therapy and made a full recovery. He returned to the gridiron for the 2010 season and later graduated from college.

“I’m very lucky, I know that,” Culbreadth said last February when he received an award from an organization that seeks to raise awareness about rare diseases.

Culbreath’s recovery, however, was more than luck. It was the result of years of painstaking research and clinical testing led by Young and his colleagues, who are widely credited with pioneering the development of the treatments for patients with aplastic anemia and related syndromes.

The therapies tested by Young, the chief of the Hematology Branch of the National Heart, Lung and Blood Institutes at NIH, have resulted in a dramatic increase in survival rates for those suffering from aplastic anemia. When Young graduated from medical school in 1971, for example, almost all patients who developed severe aplastic anemia died within just a few months. Today, the survival rate is more than 70 percent.

(Ernie Branson/NIH)

Because of Young’s efforts, his clinic at NIH is considered one of the world’s major referral centers for bone marrow failure syndromes, including aplastic anemia. This disease strikes about 600 to 900 people a year in the United States.

“Neal Young is a great scientist and he has done an enormous amount of good in terms of aplastic anemia,’ said Dr. Arthur Nienhaus, a prominent physician at St. Jude Children’s Research Hospital in Tennessee and Young’s former boss at NIH.

“He is truly one of the great investigators of our generation in terms of hematology,” said Nienhaus.

At NIH, Young combines direct patient care and clinical trial research with basic science laboratory work in cell biology, molecular biology, virology, immunology and population-based epidemiologic studies.

Young said the NIH gives him great “intellectual freedom” to follow the science, undertake a wide range of investigations and engage in “transformative work” that provides benefits to the medical community and society. At the same time, Young said, he is able to have direct contact with patients, “take care of them in a very special way” and fulfill his role as a physician.

“I get great satisfaction seeing patients doing so much better,” said Young

In addition to researching and making breakthroughs on the immunologic and genetic bases of aplastic anemia, Young’s laboratory has studied and identified the B19 parvovirus, which can infect the bone marrow cells, and has developed a vaccine that is now in clinical trials.

Young also has been involved clinical and basic research in the areas of bone marrow failure, gene therapy for blood diseases and stem cell transplantation. He has published nearly 300 research articles and more than 100 reviews and book chapters; written or edited ten monographs, including a novel textbook of hematology; and mentored dozens of post-doctoral fellows.

Nienhus said Young has “a brilliant, incisive mind, assimilates knowledge very rapidly, comes back with original thoughts and is dedicated to understanding disease.” At the same time, he said, Young maintains an engaging personality and demonstrates extraordinary leadership.

This article was jointly prepared by the Partnership for Public Service, a group seeking to enhance the performance of the federal government, and washingtonpost.com. Go to www.servicetoamericamedals.org/nominate to nominate a federal employee for a Service to America Medal and http://www.washingtonpost.com/politics/fed-player to read about other federal workers who are making a difference.

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