'Was once thought to be science fiction': Changing DNA to treat sickle cell disease

NASHVILLE, Tenn. (WTVF) — A life-saving gene therapy to treat sickle cell disease has been FDA approved. The first patient to receive the treatment came to Nashville to be part of a clinical trial years ago.

For Victoria Gray, sickle cell disease has been debilitating.

“It’s the type of pain that’s hard to describe, it’s like lightning shooting across my chest, all of my limbs and my bones ache,” Gray said.

It impacted her family, as she was home-bound and hospitalized several times.

"My oldest son… was acting out at school,” Gray said, “It’s because he thought I was going to die, so I knew I had to fight to be here with my children.”

She decided to get a bone marrow transplant from her brother, but he wasn’t a perfect match. That's when Dr. Haydar Frangoul offered an alternative option— a clinical trial. Dr. Frangoul was orchestrating the genome editing trial through the Sarah Cannon Research Institute and The Children’s Hospital at TriStar Centennial Medical Center.

“We collect their stem cells, we gene edit them, and the patients undergo some chemotherapy to kill the bone marrow that is killing sickle cell, and we infuse those newly genetically modified cells,” Frangoul said.

A more detailed look at the process of the trial inside the human body is available in the video above.

Gray was the first patient to enroll in that trial.

"I didn’t worry about the title of being the first, I went home, I prayed to God, and I said if this is what you have designed for me, for my healing, move off all fears all hesitation and give me peace," Gray said. "He gave me that peace, and I was able to start this journey without any fears at all."

Years later, she no longer suffers from symptoms related to the disease.

“I no longer get woken up from sleep at 2 o’clock in the morning and have to rush to the emergency room because I’m in pain," Gray said.

A medical miracle that's paving the way to help others.

“What was once thought to be science fiction, is now factual, and is hope for not only the sickle cell community, but everyone who’s suffering from rare diseases,” Gray said.

The treatment, called Casgevy, has been approved by the FDA. It was developed by Vertex Pharmaceuticals and CRISPR Therapeutics.

“When I got that news, I was at work, I had to leave, and I was in my car and I was crying but this time it was tears of joy,” Gray said.

Sickle cell disease impacts roughly 100,000 people in the United States, and the most of them are minorities. Currently, the treatments cost millions per patient— a major downside.

“This is the beginning, not the end," Gray said.

Advocates are fighting to get treatment costs down.

"I am really hopeful that insurance companies, and both public and private, will look at this as huge cost-saving not only for individuals with sickle cell diseases but for their families and the community as a whole," Frangoul said.

So in coming months, Dr. Frangoul plans to treat more sickle cell patients with the life-saving therapy.

In addition, patients like Gray will be followed for years to record their progress.