Phillip Mullin, 15, of Alexandria, Louisiana, has been hospitalized more than 100 times because of sickle cell disease. On Friday he rang a bell at Manning Family Children’s Hospital in New Orleans, and his doctors called him the first child in the state to be functionally cured with gene therapy.
Sickle cell disease is overwhelmingly a Black disease. The CDC says more than 90% of the roughly 100,000 Americans who live with it are Black, and it occurs in about 1 in 365 Black births, against about 1 in 16,300 Hispanic births. The agency notes the true count is unknown.
Phillip received Lyfgenia, a one-time treatment approved by the FDA in December 2023 for patients 12 and older who have had pain crises. Doctors collect a patient’s own blood stem cells, alter them in a lab and infuse them back after chemotherapy, so the body makes healthy red blood cells. Phillip’s course took months: stem cell collection, blood exchange procedures, chemotherapy and a hard recovery. “Functional cure” is the hospital’s term for his result.
At the ceremony, Phillip spoke about what it cost him. “Sacrifices are necessary,” he said. “You can’t gain anything without first giving something in return.” He wants to become a doctor “that can relate to somebody… relate to a sickle cell patient.” He is a student at Holy Savior Menard High School and loves Legos, and the hospital built a Lego display for the day.
His mother, Shataka Figgins, said she expects to spend time “unpacking every fear, every negative emotion,” and remembered “every sleepless night and every moment I wondered how much more his body could take.” Archie Manning, the Saints great whose name is on the hospital, called Phillip a “wonderful young man.”
The treatment is out of reach for most families without insurance coverage. Its maker, Bluebird Bio, set the US list price at $3.1 million, and the label carries a boxed warning for blood cancer, with monitoring every six months for at least 15 years. At launch the company said it was in talks with Medicaid agencies representing 80% of people with sickle cell in the US. Phillip’s case shows what the therapy can do. Whether the next child in Louisiana gets it depends on those payers.
Phillip will be monitored for years under that warning’s schedule.
Have your say