Patient-Led Research
PATIENT PIONEER
Victoria Gray
By Rachel Reiff Ellis
Sickle cell disease is an inherited red blood cell disorder that affects the oxygen-carrying proteins (hemoglobin) in red blood cells, contorting them into a crescent moon shape, making movement through the body and oxygen delivery to tissues difficult. About 100,000 Americans have it, but it's most prevalent in individuals with African ancestry, occurring in 1 out of every 365 births.
Victoria Gray was one of those births. The first 33 years of Victoria's life had been riddled with restrictions, painful sickle cell crises, and heart troubles requiring weeklong stays in the hospital.
"I thought I would die from it, honestly," she says.
Then in 2019, Gray met Haydar Frangoul, MD, MS, a hematologist/oncologist and leading sickle cell researcher.
As Gray lay in her hospital bed recovering from her latest crisis, Frangoul offered her the chance to be the first to take part in a clinical trial for a revolutionary gene-editing therapy using a tool called CRISPR.
CRISPR (clustered regularly interspaced short palindromic repeats) is a tool that makes precise cuts in DNA at a specific location. In this case, the cuts increase the production of fetal hemoglobin.
Even though Gray would be the first person in the U.S. to try this treatment for a genetic disorder, she didn't hesitate. She was simply existing, not living. A woman of faith, she prayed for peace about her decision.
"I understood that I was stepping into something unknown," she says. "I also knew that if it worked, it would help not only me but others with the disease."
Frangoul and a team at Sarah Cannon Research Institute and HCA Healthcare's The Children's Hospital At TriStar Centennial in Nashville collected Gray's stem cells, a lab reengineered them, and then he transplanted them back into Gray's bloodstream.
Six months later, Gray noticed a difference.
"I would pinch my face and thigh, thinking my body was numb, or something was wrong, because I wasn't in pain," she says. "But then I realized, no — it's just working."
Four years have passed since her treatment, and Gray remains symptom-free. She sees Frangoul every 6 months to monitor her hemoglobin and check in. Her husband, Earl, marvels that medicine has made "the impossible possible" for Gray.
(L to R): Earl and Victoria Gray
"I'm grateful God gave my wife a chance to experience life without medical limitations," he says.
Gray has become a public face of CRISPR therapy, speaking to patient and researcher audiences around the world.
CRISPR has been tested in at least 75 people since Gray's treatment and could be approved in the United States this year.
"As a physician, I have seen firsthand the devastating toll that sickle cell disease can take on a person's life," Frangoul says. "I consistently hear from patients who are looking for better options to treat their disease, and Victoria has helped pave the way for this therapy. I am forever thankful to her — and all patients and their families who participate in clinical trials — for their courage and contribution."
Gray's next dream is to one day to start a nonprofit to help cover the cost of gene therapy for sickle cell patients.
"Throughout the years when I would research, the only thing I would see was death," she says. "Now we have a cure in the making. It's amazing. I hope to become a lifelong advocate for patients with not only just sickle cell disease but all life-threatening genetic disorders and diseases who could benefit from CRISPR gene therapy."
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