Advancements in genomics research have opened up new avenues of treatments for sickle cell disease. This article highlights some of the most promising innovative approaches.
Sickle cell disease is a group of inherited blood disorders, the most commonly known of which is sickle cell anemia. The disease is caused by an amino acid substitution at position 6 of the β-globin chain of hemoglobin. In the last five decades, immense efforts have been dedicated to understanding the pathophysiological complexities of sickle cell disease. Sadly, effective treatments have been elusive and the process of treatment development has been slow. However, sickle cell disease treatment is changing rapidly, courtesy of ongoing genetics and genomics research.
Some of the latest developments in sickle disease treatment include:
1- Modifying a patient’s genotype via hemopoietic stem cell transplantation (HSCT). This approach was used nearly thirty years ago for the first time. Today, HSCTs have become an important feature in treating patients suffering from sickle cell disease. Gene addition and gene editing also have key roles in sickle cell disease treatment.
2- Certain approaches have been used that target hemoglobin S (HbS) polymerization. This is beneficial since it deals with the disease process prior to a sickling event rather than repairing the sequelae from sickling.
3- Targeting vaso-occlusion and inflammation have also been adopted for sickle cell disease treatment. Therapies pertaining to this phenomenon got the FDA’s approval in July 2017.
Final Thoughts
A combination of therapies, if perfected and implemented correctly, may prove to be instrumental in improving the quality of life for sickle disease patients and help make up for a century of neglect by the medical research community.
Reference
Salinas Cisneros, G., & Thein, S. L. (2020). Recent Advances in the Treatment of Sickle Cell Disease. Frontiers in Physiology, 11, 435. https://doi.org/10.3389/fphys.2020.00435
