Loma Linda University Health recently treated a patient of sickle cell disease using a pioneering FDA-approved gene therapy that reconfigures the patient’s own cells to reseed healthy cells throughout the body in order to cure sickle cell disease.
LLUH was the first facility on the West Coast to use the newly approved treatment, called lovotibeglogene autotemcel, or or lovo-cel.
The treatment involves collecting patient’s own blood stem cells to be used as seed cells after genetic correction. Those are then gene-modified in a lab to insert non-defective DNA sequencing that enable cells to produce proper hemoglobin, which was lacking. After infusion, the patient’s body can produce normal hemoglobin, thus curing sickle cell disease.
“This is a revolutionary treatment that produces correction of the patient’s own cells at the genetic level,” said Hisham Abdel-Azim, MD, chief of the Division of Transplant and Cellular Therapy/Hematological Malignancies at Loma Linda University Cancer Center. “It’s great because each patient is their own donor, and we’re effectively treating people of this disease.”
Gene therapy is a personalized treatment that involves engineering a patient's own blood stem cells to correct the disease at the gene level. Similar technology is used to treat cancers such as leukemia, lymphoma, and myeloma using patients’ own immune cells. This technology is being used and researched to treat other forms of blood diseases and cancers. These technologies are offered at the Cancer Center for various diseases for children and adults where engineered receptors enable T cells to recognize and bind to specific proteins on the surface of cancer or diseased cells.
“At Loma Linda University Cancer Center (LLUCC), we strive to provide specialized novel cutting-edge therapies for these patients with complex hematological disease in our community and to the broader California community,” said Mark Reeves, MD, PhD, director of the Loma Linda University Cancer Center.
Sickle cell disease causes frequent intense periods of pain. The disease occurs when a genetic mutation alters the hemoglobin protein within red blood cells. This causes the cells to become crescent-shaped, like a sickle. These sickle cells can bunch together in the vessels and cause blockages. This can lead to sharp pain throughout the body, limit oxygen to the brain and cause strokes, and cause damage to vital organs such as the kidneys and liver.
“It’s not a malignant disease, but in many ways it’s worse than a malignant disease because of the pain and hospital visits,” Abdel-Azim said.
At LLUH, complex conditions are approached with a multidisciplinary disease teams’ approach to deliver treatment. “It takes a village of experts, including physicians, nurses, and specialized clinical and administrative teams to provide this complex therapy. It’s a great team effort” Abdel-Azim said.
Sickle cell disease is largely a disease in minority populations, specifically African-Americans. It affects approximately 100,000 Americans, according to the CDC.
The disease can be somewhat managed by keeping oxygen levels up and managing stress, but not by every patient. Standard treatments include pain management, blood transfusions, and bone marrow transplants. Finding donors can be difficult, and potential donors within families often mean the relative also has the disease.
Loma Linda University Health serves a large number of pediatric and adult patients suffering from sickle cell disease in the Inland Empire and Desert region, as well as other patients from across the nation to deliver this cutting-edge therapy.
Loma Linda University Cancer Center is the only center in the Inland Empire and Desert region that provides advanced cancer treatments. The institution provides various cutting-edge FDA approved and experimental gene and CAR T therapies for patients with non-malignant and malignant blood disease as well as solid tumors. The Cancer Center is FACT accredited and is designated a center of excellence by the California Department of Health for Cell Therapy with various modalities and indications for children and adults.
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