Expert opinion on emerging drugs · 2026
Exploring the latest emerging drugs for the treatment of sickle cell disease.
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Cannas Giovanna
Аннотация
INTRODUCTION: Sickle cell disease (SCD) is an inherited autosomal recessive hemoglobinopathy that causes significant morbidity and mortality in children and adults. The availability of novel therapeutic agents that are safe, effective, and affordable and new cell therapy techniques remains highly desirable for the treatment of SCD.
AREAS COVERED: After a brief reminder of the main SCD complications, this review summarizes emerging disease-modifying agents and promising cell therapy strategies for the treatment of patients with SCD. A comprehensive search to look for the efficacy and safety of new agents was made on PubMed.ncbi.nlm.nih.gov for published studies and ClinicalTrials.gov for registered trials.
EXPERT OPINION: The landscape of therapy in patients with SCD has recently moved toward more personalized therapeutic approaches. Promising preliminary data were obtained with some disease-modifying agents and gene therapies became available and promising for curing patients. However, the high cost remains a major limiting factor for these new therapies, which are far from being used in all patients, even in the more developed countries. Hydroxyurea is still regarded as the best disease-modifying treatment for SCD and allogeneic hematopoietic stem cell transplantation as the standard 'curative' therapy. Continued research is still warranted to sustain therapeutic advancements and development in SCD.
AREAS COVERED: After a brief reminder of the main SCD complications, this review summarizes emerging disease-modifying agents and promising cell therapy strategies for the treatment of patients with SCD. A comprehensive search to look for the efficacy and safety of new agents was made on PubMed.ncbi.nlm.nih.gov for published studies and ClinicalTrials.gov for registered trials.
EXPERT OPINION: The landscape of therapy in patients with SCD has recently moved toward more personalized therapeutic approaches. Promising preliminary data were obtained with some disease-modifying agents and gene therapies became available and promising for curing patients. However, the high cost remains a major limiting factor for these new therapies, which are far from being used in all patients, even in the more developed countries. Hydroxyurea is still regarded as the best disease-modifying treatment for SCD and allogeneic hematopoietic stem cell transplantation as the standard 'curative' therapy. Continued research is still warranted to sustain therapeutic advancements and development in SCD.