21-Year-Old Becomes First in South Florida to Receive Cutting-Edge Gene Therapy for Sickle Cell Disease at Holtz Children’s Hospital

MIAMI, FL – Doctors at Holtz Children’s Hospital and UHealth – University of Miami Health System become first in South Florida to treat a patient with a new gene therapy for sickle cell disease. This treatment, currently available at only a few medical centers worldwide, offers hope to patients suffering from the chronic, painful, and often debilitating condition.

Holtz Children’s and UHealth are the first in South Florida to administer this therapy, known as Beam-101.

Javarian Weatherspoon, 21, of Orlando, started the new therapy as part of the clinical trial in December 2024, with the help of the multidisciplinary care teams at Holtz Children’s and UHealth, including the apheresis team, the cell therapy lab, pathology laboratory, and the pediatric stem cell transplantation teams.

Beam-101 is a new type of gene therapy from Beam Therapeutics that genetically modifies a patient’s own blood cells through a process called base editing. Unlike other types of gene therapies that cut or break a DNA strand, base editing is more precise and can correct or make edits to genes. For patients with sickle cell, their genes can be altered so that they produce a fetal form of hemoglobin in red blood cells, which do not sickle or change shape, lessening or eradicating symptoms of the disease.

“It means a lot for us as clinicians to now have another option that we can offer children living with sickle cell disease,” said David Crawford, MD, director of pediatric stem cell transplantation at UHealth and Holtz Children’s. “This is especially meaningful in cases where a patient does not have any donors or there are not any matching donors on the registry. Now, we can offer them an alternative to a transplant. It allows patients and families to have options.”

Suffering from Sickle Cell
Sickle cell disease is an inherited blood disorder that changes the shape of red blood cells, which carry oxygen throughout the body. When the red blood cells sickle or change shape, they can get stuck in the bloodstream, preventing oxygen flow and causing severe pain and crisis episodes that can lead to hospitalization.

Affecting more than 100,000 Americans, the disease can also lead to anemia, fatigue, and damage to organs, including the kidneys, which can result in the need for dialysis or a kidney transplant. Another complication is acute chest syndrome due to blocked blood vessels in the lungs that make it hard to breathe.

Often, the condition gets worse as patients near age 20. It can become so severe that people struggle to work or go to school.

This was the case for Javarian, whose symptoms began to worsen around age 17. He was regularly hospitalized due to debilitating pain, which caused him to miss school and made it difficult to work. Due to damage from the disease, he did not qualify for a bone marrow transplant.

Base Editing Gene Therapy
With Beam-101 base editing gene therapy, patients first have their stem cells harvested in the hospital. Once collected, the stem cells are sent to a specialized lab where they undergo base editing, which takes approximately three months.

Once ready, the cells are returned to the hospital. Before receiving the new cells, patients must undergo a round of chemotherapy to create space for the newly edited cells. After the cells are infused back into the patient, they need to stay in the hospital for about four weeks for monitoring.

“This offers new hope to patients who previously had little chance of recovery or relief from the chronic, debilitating symptoms of sickle cell disease,” Dr. Crawford said. “We have been able to cure or significantly improve patients’ conditions with transplants for many years; however, finding a donor is often challenging. This represents a new option that provides life-saving treatment.”

A New Chapter
Today, after the yearlong treatment process, Javarian is back home, and is focused on healing and regaining his strength. Since leaving the hospital, he hasn’t needed pain medication.
“I feel no pain at all. Some mornings it’s a little tough to get up, but once I get up, stretch, and take a little walk, I feel good,” he said. “It was worth it.”