But she has other concerns, especially about her 7- and 12-year-old daughters. Longer hospitalizations and months of recovery for gene therapy would be difficult for them, he said.
“They’re not children, but they need me,” Ms. Polanco said.
Patients ask when gene therapy is needed during the course of the disease. If they wait too long, the disease can lead to paralysis or permanent damage to organs and bones. But if the disease is mild and manageable, is it worth waiting? Houston resident Andre Marcel Harris, 33, decided the best option was to wait.
“I didn’t get sick like a lot of people,” he said. “At this point, gene therapy is not part of the conversation.”
Shamar Lewis, 20, of Orlando, Fla., has many concerns — the long-term side effects of gene therapy, the costs and months of treatment and recovery.
Still, sickle cell remains a burden. He spent several years of his youth unable to attend school due to ill health – even his school sent teachers to his home. He is now eager to finish high school and join the military, but knows he won’t be accepted by sickle cell. With gene therapy, “I can truly live my life,” he told his mother, Carla Lewis.
But what would that life look like?
Without the disease, Teona Woolford, 31, a sickle cell patient and advocate for Owings Mills, Md., said patients “don’t know who they are.” Many worry, “about trading a familiar problem — sickle cell — for something else and fear of the overall unknown.”