FDA-Approved Sickle Cell Therapy Gives Teen a New Life, Free from Pain

Nineteen candles lit up Caden Major’s birthday cake this year, but the true celebration was something he once thought impossible: life without sickle cell pain.

For as long as he could remember, the disease had stolen ordinary moments, keeping him out of class, until he had to finish school at home. Friendships were difficult to maintain, especially when he was younger and classmates didn’t understand why he was often absent and home in bed. Even everyday choices, like when to shower, carried risk, knowing a simple routine could trigger days and sometimes weeks of agony.

Eventually, the pain grew so relentless that it landed him in the hospital again and again, culminating in a two-month stay.

But a new FDA-approved gene therapy offered him a chance at a cure. This year, Caden became the first pediatric patient in South Florida to receive the treatment at Holtz Children’s Hospital and UHealth – University of Miami Health System.

“I was kind of nervous. I didn’t really know what could happen,” he said. “It was something completely new. But after spending two months in the hospital, I thought I had to give this a try. I was having pain every day, and it wasn’t getting better.”

His mother, Francita Tador, remembers that hospital stay as one of the darkest points in their journey.

“He was tired of the pain. He was tired of the doctors, the needles, the hospital admissions,” she said. “He had almost no life.”

The gene therapy treatment works by taking a patient’s own cells, genetically modifying them to add a gene that helps the body make healthy blood cells that don’t sickle, and then transplanting the modified cells back.

Once the cells were ready, Caden underwent days of chemotherapy to wipe out his existing bone marrow, a necessary step that often comes with tough side effects. Finally, the modified cells were infused back into his body to grow healthy new bone marrow.

Caden received his transplant on May 6, 2025, and, in total, spent about six weeks in the hospital. Like other transplant patients, his immune system was weakened during and after the procedure, requiring him to take extra precautions and limit contact with people to avoid infection.

“It was grueling, both emotionally and physically,” Caden said. “My hair fell out, I had mouth sores, I felt nauseous and tired, I was throwing up. Mentally, it was hard being in the hospital for so long and seeing my appearance changing — even my skin was peeling. I was shedding, like a lizard

Despite the challenges, soon after his cell transplant in May, the pain was gone.

“He had a remarkable response to the treatment,” said David Crawford, MD, director of pediatric stem cell transplantation at Holtz Children’s and UHealth. “Most kids with sickle cell have pain in the weeks following the transplant, but he was ok as soon as the new cells started growing. He’s essentially cured.”

A New Life

Now, with his immune system rebounding and his pain finally gone, Caden celebrated his 19th birthday with a new sense of hope for his future.

“I want to learn to drive, get my GED, and finish school. I want to go to college,” he said. “I have no pain, and it’s pretty great — it’s how normal people feel.”

Dr. Crawford is excited about the potential of the new treatment, which could benefit more people with sickle cell and other genetic conditions.

“It’s an amazing feeling to be part of this and to see firsthand how treatment like this can truly change someone’s life,” he said.

For Caden’s mother and the rest of the family, it’s a relief to see him moving toward a more normal life, building a future beyond the walls of his bedroom where he spent so much of his time.

“As a parent, it feels like something has been lifted off of you,” Francita said. “It was so heavy for so long. His quality of life was so poor, going steadily downward. I was scared as a mom. Now, he has a whole new life.”