By Stephen Prescott, M.D.
When Novartis announced earlier this month that the U.S. Food and Drug Administration had approved the company’s new drug to treat sickle cell anemia, the press release didn’t mention Oklahoma. But it should have.
That drug, known as Adakveo, is as Oklahoman as redbuds and scissor-tailed flycatchers. Indeed, but for work done in our state over several decades, it wouldn’t exist.
The story begins with Dr. Rodger McEver.
McEver came to Oklahoma in 1988, establishing a lab at the Oklahoma Medical Research Foundation. His research focused on white blood cells. Specifically, he studied a protein known as P-selectin, which mobilizes white blood cells to hunt down and stop invaders like bacteria in the body.
In the lab, McEver created an antibody that blocks P-selectin’s functions. Research indicated that, in laboratory mice, P-selectin exacerbated the symptoms of sickle cell disease.
In sickle cell, red blood cells change from their customary round or oval shapes to the form of a crescent or “sickle.” They stack up inside vessels, which eventually results in inflammation and excruciating pain. In some cases, long-term lack of oxygen can lead to organ damage, stroke or even death.
The disease can affect people of all races, but it’s most prevalent in those with African-American heritage. Health authorities estimate that 100,000 people in the U.S. suffer from sickle cell disease, which has no known cure.
As a physician, McEver had treated these patients.
“Their suffering,” he said, “is extreme.”
To explore clinical applications in humans, McEver helped create a biotechnology company, Selexys. The company, which was based in Oklahoma City and received seed funding from i2E and a group of Oklahoma “angel” investors, fine-tuned the antibody, making an experimental drug that bound to human P-selectin and blocked its function.
An initial round of clinical testing found the drug to be safe and well tolerated by patients. Then, in a larger, multi-center trial in the U.S., Jamaica and Brazil, the medication showed a marked reduction in pain crises suffered by sickle cell patients.
Based on those results, Novartis purchased Selexys and its experimental drug. That medication, now known as Adakveo, is the first to offer patients with sickle cell targeted relief from pain crises. Current treatments, which include blood transfusions and pain medications, only alleviate symptoms as they arise.
“We know this drug can decrease the frequency of sickle cell pain crises in a significant and clinically meaningful way,” said the physician who led the drug’s most recent clinical trial.
The drug’s approval, he said, “is an important advancement for people living with this very difficult condition.”
One of those people has worked at OMRF for more than three decades. Her name is Mary Long, and she’s a member of the staff that cleans OMRF’s offices and labs.
Since her childhood in rural Oklahoma, she’s lived with periodic bouts of excruciating pain. She can’t take pain-relieving medications like ibuprofen or acetaminophen because of stomach issues. Bed rest and a heating pad can dull her symptoms, but they’re far from an adequate remedy.
While she realizes that sickle cell may be inflicting long-term damage on her body, she is more concerned with the children she knows who have the disease. “It just hurts me to know what they go through with this,” she said.
With the approval of the new drug, Long sees the potential to ease suffering and transform lives.
“Dr. McEver has used his knowledge, and it might make life better for many who suffer with sickle cell disease,” she said. “I really hope it does.”
For McEver, this would represent the ideal culmination of research that began more than three decades ago.
“It’s the dream of every physician, and certainly every scientist, to do something that can make a difference for patients,” he said.
A physician and medical researcher, Prescott is president of the Oklahoma Medical Research Foundation and can be reached at firstname.lastname@example.org.