By Scott Meacham
There is very big news this month for individuals and families affected by sickle cell disease (SCD). The U.S. Food and Drug Administration (FDA) has approved the first targeted treatment to reduce the frequency of pain crises in individuals living with SCD.
This new drug — Adakveo® from Novartis — decreases the frequency of the debilitating and unpredictable pain episodes caused by SCD. It is a life-changing medical breakthrough that started in the mind of my across-the-street neighbor, Rodger McEver, M.D., more than 35 years ago.
Rod’s career has been as an academic scientist whose clinical specialty is blood disease. Today, he is vice president of research at OMRF. Back in the eighties, he was at OMRF with a secondary appointment at the University of Oklahoma. That’s when the story of this groundbreaking therapy for sickle cell disease first began.
“I was not at all working on sickle cell disease,” Rod told me. “I was working on blood platelets. We discovered a protein that only appeared on the surface of the platelets when they were activated, usually in response to infection or injury.”
Rod’s research team wrote grants, and the National Institutes of Health (NIH) funded basic research to clone the gene that produced the protein (named P-selectin). It took years more research to understand what the protein did.
“We knew that sickle cell is a mutation in one of the proteins in red blood cells that carries oxygen,” Rod said. “That mutation causes the red blood cells to form chains that become insoluble. The rigid cells occlude blood vessels, blocking oxygen delivery. But we began to understand that there was more to sickle cell than the plugging of red blood cells.”
Invoked by the signals of inflammation where the vessels were plugged, the P-selectin protein brought in white blood cells and platelets, which released other mediators and amplified the response, causing the excruciating pain those with sickle cell must endure.
“That’s how our work intersected with sickle cell,” Rod said. “It’s a whole community discovering things, eventually you see the connections.”
After more research and filing patents, Rod and his co-founders founded Selexys Pharmaceuticals in Oklahoma City and fine-tuned a drug that blocks the function of P-selectin. Novartis gained the technology and experimental drug when it acquired Selexys in 2016. The FDA approval came after a 52-week study that showed a 45 percent decrease in acute pain episodes.
“As a research scientist, our rewards are primarily from the process of discovery,” Rod said. “It is wonderful to be in a profession where you can see something for the first time. That’s why people do research because they want to be the first to understand. I was very lucky to discover something and develop a drug in our own lab to be adapted for use in people.”
This new drug for sickle cell is a case study in why investment in basic science is so very important to all of us. Scientists can’t predict when and where, but the applications eventually occur. Basic research can’t be forced to fit into applications too soon—but by adding to the body of scientific knowledge, basic, academic research will eventually lead to solutions that change people’s lives.
We need to have the patience and the perseverance to stay the course with these researchers.
Scott Meacham is president and CEO of i2E Inc., a nonprofit corporation that mentors many of the state’s technology-based startup companies. i2E receives state appropriations from the Oklahoma Center for the Advancement of Science and Technology. Contact Meacham at [email protected]