At any cost
Oklahoma research helps save Oklahoma dentist, but at a price.
Faced with a rare and life-threatening blood disease, Greg Watkins dealt with severe fatigue and pain for more than 20 years. In 2007, a miracle cure was developed, but it came at a cost. It was the most expensive drug in world.
A McAlester dentist, Watkins was an avid runner and in top physical condition for most of his life. That changed in 1995 when he was diagnosed with Paroxysmal Nocturnal Hemoglobinuria, or PNH. It is an extremely rare condition with a low survival rate.
Now, more than 20 years later, he’s back at the gym with reclaimed vitality thanks to a drug that was born in the laboratories of the Oklahoma Medical Research Foundation.
The catch? The drug is over $400,000 a year.
‘IT WAS SCARY’
Dr. Martin Brodsky, a hematologist at Johns Hopkins University who specializes in PNH, had been treating Watkins for almost a decade when Watkins flew to Baltimore for a regular appointment in 2007.
“I remember him saying, ‘Well, we now have a cure,’ “Watkins said. “I never thought I would hear those words out of his mouth. Technically, it’s not a cure, but as long as I take the drug, I won’t have to worry about the disease.
“Then he told me how much it was going to cost — $400,000 a year. Actually it was more than that.”
Watkins took his first dose of the drug, Soliris, in April 2007, just two months after it was approved by the U.S. Food and Drug Administration. Each infusion was about $15,000 for nursing services and the drug.
“My insurance paid $790,000 for one year,” he said.
Between one and two people in 1 million have PNH. Because the disease is so rare, the drug that was developed through the research of OMRF scientists Drs. Peter Sims and Therese Wiedmer, was, until recently, the most expensive drug in the world. It was surpassed in 2015 by Glybera, which is $1.21 million per year.
“It was scary,” Watkins said. “The first fear was the cost. Would I lose my job? The second fear is, OK how is this going to work? After my first treatment, my LDH, which is a liver enzyme, was normal for the first time since 1991. I knew immediately this was going to work. I haven’t had any more PNH attacks.”
Watkins also was able to keep his job with the state Corrections Department in McAlester, which provided him adequate insurance to continue taking the treatment.
STILL A MYSTERY
PNH has been studied for years but remains a mystery. It is a blood disorder in which PNH patients produce abnormal red blood cells that may be destroyed by their own immune systems. The result is allover physical decline with extreme fatigue, severe abdominal pain, decreased organ function, blood clots and weakness.
The path to a treatment for PNH had begun long before Watkins was diagnosed. OMRF scientists Wiedmer and Sims had spent years researching the complement system, a part of the immune system that encourages antibodies to clear microbes and damaged cells from an organism. With PNH, the complement system attacks the body’s red blood cells.
In the late 1980s, Wiedmer and Sims developed an antibody that interrupts the part of the complement system that attacks red blood cells. That research was used by Connecticut-based Alexion Pharmaceuticals to produce Soliris, but no one at OMRF knew that that would be the outcome.
“It wasn’t that (Sims and Wiedmer) were after this disease. They were after a fundamental mechanism,” said Dr. Stephen Prescott, a physician and president of the OMRF. “This is not the only disease caused by abnormal complement. In fact, when (Alexion) started testing the antibody they made, they thought one of the most likely places this antibody would help would be bypass surgery.”
Luckily for PNH sufferers, it became what Watkins believes is a miracle drug that has saved hundreds of lives.
“It’s the single most important thing that we aspire to,” Prescott said. “We don’t always know how things are going to turn out, but we really celebrate … when we get to the finish line.”
Now with the Veterans Affairs Department in Oklahoma City, Watkins has lunch almost daily in the OMRF cafeteria, which is next door. He said he sees it as a “fullcircle” experience.
“I feel great. It gave me back my quality of life. I work four 10-hour days at the VA dental clinic — hard long days doing oral surgery,” he said. “I’m going to be 68 in August. I wouldn’t have been expected to live much past 50. Luckily for me, I lived 12 years after diagnosis, and there was a FDAapproved drug to treat it. I did not dream in my lifetime there would be a drug to treat my rare blood disease. In the end, I see God’s plan works miraculously. I’m blessed.”