Loma Linda University Health first in West to treat sickle cell patient with FDA-approved gene therapy

LOMA LINDA, Calif. — A patient at Loma Linda University Health recently became the first on the West Coast to receive a groundbreaking gene therapy for sickle cell disease, marking a major milestone in the use of FDA-approved cellular treatments.

The new therapy, lovoti eglogene autotemcel — also known as lovo-cel — reprograms a patient’s own stem cells to restore healthy hemoglobin production. Approved by the U.S. Food and Drug Administration in December 2023, the treatment offers a potential cure for the inherited blood disorder that disproportionately affects Black Americans and causes lifelong complications.

“This is a revolutionary treatment that produces correction of the patient’s own cells at the genetic level,” said Dr. Hisham Abdel-Azim, chief of the Division of Transplant and Cellular Therapy at Loma Linda University Cancer Center. “Each patient is their own donor, and we’re effectively treating people of this disease.”

Sickle cell disease is caused by a genetic mutation that distorts the shape of red blood cells, impairing their ability to carry oxygen and leading to severe pain, organ damage, and a higher risk of stroke. About 100,000 Americans live with the condition, according to the Centers for Disease Control and Prevention.

Traditional treatments include blood transfusions, bone marrow transplants, and pain management. But donor matches can be difficult to find — and in some cases, relatives who might be good candidates also carry the gene.

Lovo-cel offers a different path. Doctors extract a patient’s blood-forming stem cells, correct the defective gene in a lab, and re-infuse the modified cells to generate normal hemoglobin — effectively curing the disease.

At LLU Health, a multidisciplinary team coordinated the complex treatment. “It takes a village of experts, including physicians, nurses, and specialized clinical and administrative teams to provide this therapy,” Abdel-Azim said.

Dr. Mark Reeves, director of the Loma Linda University Cancer Center, said the institution is committed to offering novel therapies for hard-to-treat blood conditions. “We strive to provide specialized, cutting-edge therapies for patients with complex hematological diseases in our community and the broader California region,” Reeves said.

The cancer center, which is FACT-accredited and designated as a Center of Excellence by the California Department of Health, offers several FDA-approved and experimental gene and CAR T-cell therapies for both children and adults.

While gene therapy has been used to treat some cancers, including leukemia and lymphoma, its application to non-malignant diseases like sickle cell represents a major leap forward in personalized medicine.

“It’s not a malignant disease, but in many ways it’s worse because of the pain and hospital visits,” Abdel-Azim said. “This therapy brings real hope.”

Loma Linda University Health serves a large sickle cell population in the Inland Empire and Desert region and treats patients from across the country seeking access to this new therapy.

This article was written from a press release from Loma Linda University Hospital.