First Patient To Receive Gene Editing Treatment For Sickle Cell Doing Well In 2020

The first patient to receive a special gene editing treatment for sickle cell disease is surviving ― and even thriving ― a year later.

Victoria Gray, 34, whose story has been chronicled by NPR for the past year, was the first person with a genetic disorder in the U.S. to receive the cutting-edge treatment known as CRISPR, which stands for Clustered Regularly Interspaced Short Palindromic Repeats.

CRISPR allows scientists to directly alter a cell’s genome, and a 2019 Wired guide to the technology called it “the genetic equivalent of Microsoft Word,” enabling scientists to “edit DNA almost as easily as software engineers modify code” ― potentially leading to cures for diseases such as cancer. 

For the estimated 100,000 Americans affected by sickle cell disease, which predominantly emerges in people with ancestry from sub-Saharan Africa, CRISPR holds the possibility of a new lease on life. The disease is caused by a mutation in hemoglobin, the protein in red blood cells that transports oxygen throughout the body and expels carbon dioxide. A sickle cell patient’s red blood cells deform into a “sickle” shape, limiting their ability to carry oxygen and leading to issues such as anemia and an increased risk of stroke. It can normally only be cured by a bone marrow transfusion, which requires a donor.

CRISPR technology, however, can theoretically edit a patient’s cells to produce normal hemoglobin, and Gray, who has suffered from sickle cell disease since infancy, underwent treatment last July. Doctors removed bone marrow from her body, used CRISPR to modify the marrow’s blood cells, and then transferred the cells back into Gray. 

Nearly a year later, Gray, who lives in Forest, Mississippi, is responding well to the treatment, according to NPR’s latest update, and hasn’t suffered major pain or needed emergency room treatment or blood transfusions. Relapses may still be possible, and it is too early to tell if CRISPR can serve as a definitive treatment for sickle cell disease. Nevertheless, 2020 is a noticeable improvement for Gray compared to the previous two years, when she visited the hospital seven times on average and frequently took pain medication.

Gray told NPR that “it hasn’t been easy” caring for three children during a year shaken by COVID-19 as well as racial injustice protests throughout the United States, but she added that without CRISPR, she would have likely been hospitalized amid the pandemic and been unable to provide for her family.

“Since my treatment, I’ve been able to do everything for myself, everything for my kids. And so it’s been joy not only for me but for the people around me that’s in my life,” she said.

“High school graduations, college graduations, weddings, grandkids ― I thought I wouldn’t see none of that…. Now I’ll be there to help my daughters pick out their wedding dresses and we’ll be able to take family vacations, and they’ll have their mom every step of the way.”