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.
The compound, now referred to as an investigational new drug, then moved to human clinical testing, a rigorous process that typically consumes a dozen years. It also typically costs hundreds of millions of dollars.
In the first stage of clinical trials, doctors administer the drug to volunteers to ensure it doesn’t hit unintended targets — in other words, that it’s not toxic and causes minimal side effects. If the drug passes the first phase, then it progresses to a second, larger study to see if it hits the desired target (i.e., if it helps the body fight the disease).
In the third phase, physicians administer the drug to many more patients over a period of years at multiple clinical sites. For a drug to gain FDA approval, its phase III results should show clinical benefit with at least 95 percent certainty.
In the end, four out of five drugs tested in humans fail. Although the FDA approval process is time-consuming and excruciating to patients waiting for new drugs, that statistic alone tells us why it is necessary. Would you want to live in a world where new drugs emerged from labs much more quickly — but you never knew if they would work? Or, even worse, if they might cause calamitous side effects?
No, the current system is not perfect. We need to continue to bring new drugs to market more quickly. But we must do so in a way that protects patients. Because safe and effective drugs are worth the wait. Just ask Dr. Gregory Watkins.
Prescott, a physician and medical researcher, is president of the Oklahoma Medical Research Foundation.