Federal Success — NIH scientist fights back against bone marrow failures

Bone marrow — most of us don’t fully understand what it is, let alone why it fails. But Dr. Neal Young does.

He is the world’s leading expert in the field of bone marrow failure. His groundbreaking research at the National Institute for Health has led to life-saving treatments for a rare and deadly blood disorder known as aplastic anemia.

His work has earned him a spot as a finalist for the Service to America medals — the SAMMIES.

Let’s start at the beginning. Young says bones marrow is the inside of the bone that actually produces blood cells, basically the blood cell factory.

Back in the 1980s:“When I was in medical school 80-90% of those diagnosed with aplastic anemia died within a year. It’s a disease that effects people that were previously healthy. They developed symptoms quickly and died. Back then the focus from professors was on the literature, they wanted to discover where the disease came from. But that was tedious and ultimately didn’t really matter to the patients,” said Young.

Breakthrough:“A French physician who was doing bone marrow transplants in those early days noted that a suppressed immune system was beneficial. That was a game changer,” said Young.

Discoveries at NIH: Young conceived, designed and headed the first multicenter clinical trial in the United States for immunosuppressive therapy for aplastic anemia. The regimen he developed for this blood disease has become standard therapy for patients all over the world. This disease strikes about 600 to 900 people a year in the United States, and thousands across the globe.

Where are we now? Today people facing bone marrow disease face a 80-90% survival rate.

Why Government?

“I don’t want this to come off as political in any way, but without NIH I don’t think it would have been done,” said Young, “Basic research is the responsibility of the government. Simply because it’s hard to get the private sector on board. It makes sense because research is not necessarily fiscally responsible.”

  1. It would be almost impossible for private scientists to get a grant approved to bring together patients from across the country with varying levels of insurance to one place.
  2. The ability for scientist at NIH to go back and forth between the clinic and the lab.
  3. Research can go in a new directions without having to re-do the grant process.

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