European Journal of Biomedical and Pharmaceutical Sciences

A CURRENT TREATMENT AND PROSPECTIVE GENE AND STEM CELL THERAPIES TO MANAGE SICKLE CELL DISEASE

Ghazala Nathu*, Noor Hammam, Muhammad Ashir and Adila Nathu

ABSTRACT

Sickle Cell Disease (SCD) management has significantly evolved, improving patient survival and quality of life. Traditional therapies focus on symptom management, such as blood transfusions to alleviate anemia and prevent complications like strokes. Hydroxyurea, a disease-modifying drug, increases fetal hemoglobin levels, reducing red blood cell sickling and vaso-occlusive crises (VOC). Similarly, L-glutamine decreases oxidative stress and hemolysis, minimizing pain episodes. Newer therapies like voxelotor, which stabilizes hemoglobin to prevent sickling, and crizanlizumab, a monoclonal antibody targeting P-selectin to reduce VOC, offer additional relief from severe complications. These advancements have substantially improved life expectancy and reduced the burden of SCD symptoms. For patients seeking a potential cure, hematopoietic stem cell transplantation (HSCT) from matched sibling donors remains the standard. Advances in cord blood and haploidentical donor transplantation with modified conditioning regimens have expanded the donor pool, making this option more accessible. Gene therapy is an emerging curative approach currently in clinical trials, offering promising results. It involves modifying the patient’s stem cells to correct the underlying genetic mutation. This multidisciplinary approach to SCD treatment reflects significant progress in addressing both the symptoms and root cause of the disease.

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