THE LATEST ADVANCEMENTS IN SICKLE CELL DISEASE

   - Gene Addition Therapy: This approach involves introducing a functional copy of the hemoglobin gene into the patient's cells to produce healthy hemoglobin. Clinical trials using lentiviral vectors (delivery system for gene addition therapy) have shown promising results in restoring normal hemoglobin production and reducing SCD-related complications.

   - Gene Editing: Researchers are exploring gene editing techniques, such as CRISPR-Cas9, to directly modify the genetic mutation responsible for SCD. CRISPR-Cas9 is a revolutionary gene editing technology that allows scientists to make changes to the DNA of living organisms.This approach aims to correct the faulty gene and restore normal hemoglobin production. Although still in the early stages, gene editing holds a promise for potential cures.

   - Voxelotor (Oxbryta): Voxelotor is an oral medication used to treat sickle cell disease (SCD). It belongs to a class of drugs called hemoglobin modifiers and works by increasing the ability of abnormal hemoglobin to bind with oxygen. By doing so, Voxelotor helps prevent the formation of sickle-shaped red blood cells, reduces painful episodes caused by blocked blood vessels and improves oxygen levels in individuals with SCD. It is prescribed for individuals aged 12 years and older. This drug has demonstrated effectiveness in reducing the incidence of vaso-occlusive crises (painful episodes) and improving hemoglobin levels in clinical trials.

   - Crizanlizumab (Adakveo): Crizanlizumab is an antibody medication that is used to manage sickle cell disease (SCD). It targets a molecule called P-selectin, which is involved in the adhesion of sickle red blood cells to blood vessel walls, leading to painful vaso-occlusive crises. By binding to P-selectin, Crizanlizumab helps prevent the interaction between sickle cells and blood vessel walls, reducing the frequency and severity of these crises. It is administered through monthly intravenous infusions and is prescribed for individuals aged 16 years and older with a history of vaso-occlusive crises in SCD.

   - L-Glutamine (Endari): L-Glutamine is an oral medication that helps reduce oxidative stress and improve red blood cell functions. By providing an alternative energy source for red blood cells, L-glutamine improves their function and decreases sickling. Clinical trials have shown its efficacy in reducing the frequency of pain crises in SCD patients.

   - Hydroxyurea: Although not a new treatment, Hydroxyurea remains a standard therapy for SCD. It is a medication used in sickle cell disease (SCD) to increase fetal hemoglobin production, reducing vaso-occlusive crises, pain episodes, and complications. It improves the quality of life for individuals with moderate to severe SCD and requires regular monitoring and dosage adjustments.

Stem cell transplantation, also known as Hematopoietic stem cell transplantation (HSCT), is a potentially curative treatment for sickle cell disease (SCD). It involves replacing the patient's diseased bone marrow, which produces abnormal red blood cells, with healthy hematopoietic stem cells. These stem cells can be obtained from a compatible donor (allogeneic transplantation) or from the patient's own bone marrow (autologous transplantation). The transplanted stem cells have the potential to generate healthy red blood cells, effectively reversing the underlying cause of SCD.

However, stem cell transplantation is a complex procedure with potential risks and complications, a strict criteria for patient selection and donor matching need to be considered. It is typically reserved for individuals with severe SCD or those who have not responded to other treatments. Advancements in stem cell transplantation techniques have made the procedure more accessible and successful for sickle cell disease (SCD) patients. Reduced-intensity conditioning regimens and the use of haploidentical donors have expanded the availability and improved the success rates of transplantation.

There has been a focus on improving supportive care for SCD patients. This includes better pain management strategies, enhanced education and counseling, and comprehensive care coordination. Multidisciplinary SCD clinics are being established to provide specialized care and address the diverse needs of patients living and born with this genetic disorder.

A growing awareness and advocacy for SCD has led to increased funding for research, improved access to care and better education for healthcare providers. This has resulted in a more comprehensive and holistic approach to managing SCD and improving the quality of life for patients. However, there are myths and misconceptions revolving around sickle cell disease and more so in the developing countries necessitating for more awareness raising campaigns among the masses.