Current and Future Therapeutics for Treating Patients with Sickle Cell Disease

Sickle cell disease (SCD) is the most common genetic blood disorder in the United States, with over 100,000 people suffering from this debilitating disease. SCD is caused by abnormal hemoglobin (Hb) variants that interfere with normal red blood cell (RBC) function. Research on SCD has led to the dev...

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Bibliographic Details
Published in:Cells (Basel, Switzerland) Switzerland), 2024-05, Vol.13 (10), p.848
Main Authors: Barak, Mariam, Hu, Christopher, Matthews, Alicia, Fortenberry, Yolanda M
Format: Article
Language:English
Subjects:
Anemia, Sickle Cell - genetics
Anemia, Sickle Cell - therapy
Blood
Blood diseases
Blood vessels
Bone marrow transplantation
Clinical trials
Clinical Trials as Topic
Drug approval
Erythrocytes
Gene therapy
Genetic Therapy
Glycosylated hemoglobin
Health aspects
Hemoglobin
Humans
hydroxyurea
Ischemia
Mortality
Patients
Physiological aspects
Sickle cell anemia
Sickle cell disease
Stroke
therapeutics
vaso-occlusion
Veins & arteries
voxelotor
Young adults
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Summary:Sickle cell disease (SCD) is the most common genetic blood disorder in the United States, with over 100,000 people suffering from this debilitating disease. SCD is caused by abnormal hemoglobin (Hb) variants that interfere with normal red blood cell (RBC) function. Research on SCD has led to the development and approval of several new SCD therapies in recent years. The recent FDA-approved novel gene therapies are potentially curative, giving patients an additional option besides a hematopoietic bone marrow transplant. Despite the promise of existing therapies, questions remain regarding their long-term pharmacological effects on adults and children. These questions, along with the exorbitant cost of the new gene therapies, justify additional research into more effective therapeutic options. Continual research in this field focuses on not only developing cheaper, more effective cures/treatments but also investigating the physiological effects of the current therapies on SCD patients, particularly on the brain and kidneys. In this article, we undertake a comprehensive review of ongoing clinical trials with completion dates in 2024 or later. Our exploration provides insights into the landscape of current therapeutics and emerging novel therapies designed to combat and potentially eradicate SCD, including the latest FDA-approved gene therapies.
ISSN:2073-4409
2073-4409
DOI:10.3390/cells13100848