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Africa must participate in finding a gene therapy cure for sickle-cell disease

Nature Medicine volume 28, pages 2451–2452 (2022)Cite this article

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Sickle-cell disease (SCD) is the most common serious inherited disease, with 300,000 affected individuals born yearly worldwide, with three-quarters of them in Africa.

At present, there are both disease-modifying and curative interventions for the management of SCD1. Among the former, exchange blood transfusion and hydroxyurea have the most robust research evidence. Hydroxyurea is a disease-modifying medicine that reduces mortality and improves the quality of life, with a clinically significant response in more than 50% of patients. The efficacy of hydroxyurea in Africa has been validated in a recent clinical trial, which showed that treatment reduced the incidence of infections, exchange transfusions and death2. Crizanlizumab, voxelotor and l-glutamine were recently approved for use in SCD as disease-modifying medicines. Crizanlizumab and voxelotor each cost more than US$100,000 per year per patient and are out of reach of patients with SCD in Africa. Hydroxyurea is not expensive at around $1 per day, but because it needs to be taken for life it becomes prohibitive for most patients. In practice, less than 20% of SCD patients in Africa consistently receive hydroxyurea. Implementation of widespread hydroxyurea use could be achieved if African countries and development partners, as well as entities such as the Global Fund, were to collaboratively support this effort, similar to what is being done successfully for HIV, tuberculosis and malaria3.

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Acknowledgements

The authors thank L. Luzzatto for his contribution in the preparation of this correspondence.

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Authors and Affiliations

  1. Department of Haematology and Blood Transfusion, Muhimbili University of Health and Allied Sciences (MUHAS), Dar-es-Salaam, Tanzania

    Grace Moshi & Julie Makani

  2. Department of Pathology and Laboratory Medicine, KK Women’s and Children’s Hospital Singapore, Duke-NUS Medical School, Singapore, Singapore

    Grace Moshi

  3. Department of Pediatrics, Division of Pediatric Hematology/Oncology, Aflac Cancer Center and Blood Disorders Service of Children’s Healthcare of Atlanta, Emory University School of Medicine, Atlanta, GA, USA

    Vivien A. Sheehan

  4. Sickle Cell Programme, Department of Haematology and Blood Transfusion, School of Medicine, MUHAS, Dar-es-Salaam, Tanzania

    Julie Makani

  5. Sickle Pan-African Consortium (SPARCO) Clinical Coordinating Centre, SickleInAfrica, Muhimbili University of Health and Allied Sciences (MUHAS), Dar-es-Salaam, Tanzania

    Julie Makani

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  1. Grace Moshi
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  2. Vivien A. Sheehan
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  3. Julie Makani
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Correspondence to Julie Makani.

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Moshi, G., Sheehan, V.A. & Makani, J. Africa must participate in finding a gene therapy cure for sickle-cell disease. Nat Med 28, 2451–2452 (2022). https://doi.org/10.1038/s41591-022-02033-5

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