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Africa: As we remember, sickle cell disease is still not completely curable despite a century-long trial




Sickle cell disease was first discovered in the United States around 1910. The first documented patient was a young African-African medical student from Grenada, named Walter Clement Noel.

It was 40 years later that it was determined that sickle cell disease (SCD) was due to a genetic phenomenon affecting the hemoglobin chain of red blood cells.

Since then, there are few cures. The only pharmacological treatment for SCD was hydroxyurea until the 20th century.

Still, its use began in 1995, but as a diverted drug.

Treatment options

It was a coincidence that SCD was first cured through a bone marrow transplant in 1984 because the patient was being treated for leukemia.

It commends the neglect of this disease in all aspects of research, etiology, diagnosis and treatment options.

Nonetheless, SCD diagnostics have greatly benefited from technological advances, from basic microscopy to genomic sequencing.

The designation of SCD orphan disease status by regulatory agencies such as the US Food and Drug Administration has increased interest in developing therapeutic drug products in the treatment of disease.

In considering future research, it is important to recognize that we are dealing with diseases that are considered to affect a small percentage of the population.

The prevalence of SCD in African countries is about 1-2% of the population, with carrier status exceeding 20%.

For example, in the Lake Victoria basin, the prevalence of SCD carriers is approximately 25%. Mortality rates in most African countries are still high, primarily due to lack of established programs for the management of SCDs.

There is renewed interest in understanding the magnitude of SCD disease in sub-Saharan Africa in order to improve the survival and quality of life of SCD patients.

Such an understanding enhances the development of strategies to reduce the burden of disease in the next generation and the development of appropriate policies for the management of SCD in different regions.

To this end, an African-wide network of research groups involved in the development of databases across different countries that creates a large cohort of people who co-exist with SCD to support the development of new interventions in the treatment of SCD. there is.

Bone marrow transplantation

There are several clinical trials of existing drugs that have been diverted for the management of SCDs and new molecules and have shown promising results in their treatment.

Although research into whether gene editing or bone marrow transplantation can replace (the only known underlying cure for sickle cell disease) is in its infancy, it also provides the probes needed to understand SCD. Therefore, the target of new drug is identified.

However, because SCD is a multisystem disease, it needs to be combined with many therapies, like chronic diseases, so collaboration is needed.

Broken health system

As with other globally dispersed diseases, SCD morbidity and mortality have a dichotomous disparity that reflects the state of the country’s healthcare system.

Thus, pharmaceutical companies, academia, philanthropists, and other consortiums of illnesses, in collaboration with researchers in SCD-endemic countries, will mine existing large chemical and technical databases to identify promising interventions. is needed.

Utilization of this framework requires a well-characterized cohort of people living with SCD, and what interventions may improve quality of life and offer treatments. Need to understand.