Testing process for drugs is worth the wait

Two Oklahoma Medical Research Foundation scientists — Drs. Peter Sims and Therese Wiedmer — made the initial discoveries that led to the creation of Soliris, a drug that treats paroxysmal nocturnal hemoglobinuria, in 1989. So why did it take 18 years to turn that eureka moment into a drug?
By Stephen Prescott, M.D. Published: August 2, 2014
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Technically, Dr. Gregory Watkins entered this world on Aug. 10, 1949. But if you ask him his birthday, he might give you a different answer.

“I tell people that April 20 is my new birthday of my new life,” he says. That’s the day in 2007 that doctors began treating his rare blood disorder with Soliris, a new drug that had just received approval from the Food and Drug Administration.

For Watkins, a dentist at the Oklahoma City VA Medical Center, the wait had been a long one. Paroxysmal nocturnal hemoglobinuria (or PNH), a rare blood disease, had first struck him in 1995. For the next dozen years, he struggled with the disease, which is marked by painful esophageal spasms, anemia and potentially life-threatening blood clots.

“In the previous 12 years, every day I’d wake up and wonder, ‘How will I deal with the fatigue and pain of PNH? How will I make it through the day?’” But with Soliris, he says, “Those are no longer questions I ask myself.”

As long as the wait was for Watkins, it was even longer for the Oklahoma Medical Research Foundation. Of course, OMRF wasn’t a patient in need of treatment. But two OMRF scientists — Drs. Peter Sims and Therese Wiedmer — made the initial discoveries that led to the creation of Soliris. That work occurred in OMRF’s labs way back in 1989.

So why did it take 18 years to turn that eureka moment into a drug to treat patients like Dr. Watkins?

At OMRF, working with laboratory mice, Sims and Wiedmer developed a method to treat PNH using a highly specialized antibody, which is a sort of defense protein. When OMRF received a patent for this discovery in 1992, it licensed the patent to Alexion Pharmaceuticals.

The company’s first task was to “humanize” the antibody, which means replacing some parts of the mouse antibody proteins with human components. Once Alexion had accomplished this task, it tested the compound in preclinical models for safety and effectiveness.

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