A new era for treating sickle cell disease could spark a health-care revolution
Inside Jimi, normally pliable, disc-shaped red blood cells deformed into rigid crescents. Those microscopic sickle-shaped cells clumped together, unleashing a cascade of damage. Pain was a constant, but about once a month it erupted into pure agony — like glass had shattered inside his veins and shards were sawing back and forth.
How would monthly trips to the emergency room to manage his pain work with a newborn baby? Could he keep up with a toddler when everyday pain could keep him stuck in bed all day? Would he even live long enough to try?
“I knew sickle cell would win that battle as well,” Jimi said. “It won everything — with my career, with education, with everything I wanted to do.”
Then, midway through Amanda’s pregnancy, the couple read an article about Victoria Gray, a woman whose genes had been experimentally edited to treat her sickle cell disease. It was still too soon to know exactly how well it worked, but Jimi wanted in.
After decades of neglect, stigma and underfunding, sickle cell is getting the equivalent of the red carpet treatment in science. It’s the target of a competitive biotech race, with scientists and companies using a crop of cutting-edge tools to try to cure the debilitating illness.
The first gene therapies for sickle cell, including one based on the buzzy, Nobel Prize-winning technique called CRISPR, will be reviewed by regulators this year, and companies are preparing to launch the medicines if they get the green light. That puts the country at the cusp of two frontiers: a new era in treating a tragically overlooked disease, and the beginning of what could be a CRISPR revolution in medicine.
