For many years, physicians treated PNH patients like Sandy Roark by treating their symptoms. Doctors would tamp down patients’ immune system with steroids and other drugs, and they’d replace red blood cells with transfusions. The one potential fix for the disease—bone marrow transplant—came with a two-year survival rate of only slightly better than 50 percent. And even when patients lived, the surgery was not always successful.
So Sims and Wiedmer decided to attack the problem from a different angle.
“We figured that if we could interrupt the complement system, it would protect the red blood cells from the proteins that were destroying them,” Sims says. Working with red blood cells, they isolated the inhibitory molecule—the armor that, in everyone but PNH patients, protects red blood cells from the piercing attacks of the complement system. With that key piece of knowledge in hand, the pair developed a method to shut down the complement system by using a highly specialized antibody (a sort of defense protein) to disable a key protein in the complement system.
OMRF filed for a patent on the discovery in 1989. When the patent was issued in 1992, OMRF licensed the discovery to a biotechnology company that Sims had helped found. Over the next 15 years, that company, now known as Alexion Pharmaceuticals, built the discovery into a drug that could be administered to humans. The drug then underwent human clinical testing, a rigorous process that typically consumes a dozen years and hundreds of millions of dollars.
For most experimental drugs, this is where the story ends, as only 1 in 5,000 reaches the market. But this drug—which came to be known as Soliris—bucked the odds, building a remarkable track record of safety and efficacy throughout the three stages of testing in hospitals and clinics. As a result, in 2007 the U.S. Food and Drug Administration approved Soliris for the treatment of PNH. It was one of only 19 new drugs approved in all of 2007 and the first specifically approved for the treatment of PNH.
The moment when a discovery is transformed into a treatment for human disease, says Wiedmer, is one that every medical researcher dreams of. “We were so joyful.”
In May 2007, after seven grueling years at the mercy of PNH, Roark began treatment with Soliris. Before the drug, she was receiving blood transfusions every few weeks. Immediately after her first treatment, though, she saw a change. “I went three full months without a transfusion. It was wonderful.”
Just as importantly, the drug helped restore Roark’s quality of life. “What Soliris has done is it stopped all those symptoms,” she says. Before, when her hemoglobin count would drop, her heart would throb just from the effort of beating, and esophageal spasms would make every breath a labor. Worst of all, abdominal cramps would trigger her greatest fear: a clot. “Now,” she says, “I can keep going. I can work in the yard. I can do what I need to do. It’s wonderful.”