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The uphill climb from discovery to drugstore

The chances are one in a million.

For the one, that can be good or bad. If you’re an inventor trying to patent a brand-new idea, being one in a million is probably good; if you’re a health care patient, it’s almost certainly bad. For patients with acquired hemophilia A, the odds were not in their favor.

Acquired hemophilia A is extremely rare and potentially fatal. The body’s immune system suddenly begins to attack the clotting factor in the blood, inactivating it and causing uncontrolled bleeding into the muscles, skin, and soft tissue. Although the condition is most commonly seen in elderly patients after surgery or trauma, or in postpartum women, doctors can pinpoint a cause in only about half of the cases.

In the early 1990s, Emory hematologist Pete Lollar and his research team were studying coagulation when they happened to discover a new, modified version of the very thing patients with acquired hemophilia A are missing — factor VIII, a blood-clotting protein. Lollar pursued the unexpected finding until it eventually led to a new treatment for the rare disease.

The drug Obizur was approved by the Food and Drug Administration (FDA) in 2014 with “orphan drug designation,” a special status established by the FDA to facilitate the development of medications for rare diseases. Still, it reached hemophiliac patients more than twenty years after Lollar — now the Hemophilia of Georgia Professor of Pediatrics in the Aflac Cancer and Blood Disorders Center at the School of Medicine and Children’s Healthcare of Atlanta — made the original discovery and filed an invention disclosure.

“The story is one of perseverance on the part of the inventors, the companies, and Emory itself,” says Todd Sherer, executive director of Emory’s Office of Technology Transfer (OTT) and associate vice president for research. “Its long and twisting road from discovery to market is a prime example of how complex it can be to bring a drug to market, but also how great an impact research can have on patients’ lives.”

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