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Equitable access to cell and gene therapies in South Africa: opportunities and hurdles

Gene Therapy volume 30, pages 180–186 (2023)Cite this article

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At present, more than 1200 cell and gene therapy (CGT) clinical trials are ongoing [1], and up to 50 such treatments are expected to be clinically available by 2030 [2]. A major contribution comes from the entry of chimeric antigen receptor (CAR) T-cell therapies into the market. These cells are genetically engineered to express a synthetic receptor that binds to antigens expressed on the surface of tumour or virally infected cells, resulting in T-cell activation and a re-directed immune response against the targeted cells [3]. Anti-CD19 and anti-B-cell maturation antigen CAR T-cells have been shown to be effective against relapsed/refractory B-cell malignancies [3, 4] and multiple myeloma [5], respectively, which has led to recent approvals by regulators (Table 1) [6]. Other similar products, collectively referred to as ex vivo gene-modified cell therapies, have also been approved for the treatment and cure of rare monogenic disorders, including severe combined immunodeficiency due to adenosine deaminase deficiency [7], β-thalassaemia [8] and metachromatic leukodystrophy [9]. In addition, gene therapies—where therapeutic/corrective genes are delivered directly to patients—have also been approved for an inherited form of blindness and spinal muscular atrophy (Table 1). In South Africa, over and above being able to address these conditions, CGT approaches hold promise for potentially being able to cure human immunodeficiency virus (HIV), a condition that has severe health and socioeconomic consequences [10, 11]. Although not a feature of the currently approved treatments, gene-editing tools—and in particular clustered regularly interspaced palindromic repeat (CRISPR)—are significantly advancing the development of CGT, allowing for an unprecedented repertoire of clinical applications [12, 13]. Compared to small molecules and protein-based treatments (such as monoclonal antibodies), which require repeated administration to maintain a therapeutic effect, CGT treatments offer a solution in which a single dose could provide a durable therapeutic effect.

Table 1 List of approved cell and gene therapies.
Full size table

South Africa, rich in its cultural and ethnic diversity, mineral resources, and biodiversity, is characterised by a heavy burden of disease. Its history, centred on a legacy of institutionalised oppression and discrimination, has left in its wake the continuing socioeconomic challenge of inequality. According to the World Bank, ‘South Africa remains a dual economy with one of the highest inequality rates in the world’ [14] and there is no clearer measure of this than a Gini index of 63 in 2021 [15]. Based on this metric, it is the most unequal country in the world. Inequality in wealth is even higher: the richest 10% of the population held around 71% of net wealth in 2015, while the bottom 60% held 7%. Unemployment at 32.5% at the end of 2020 remains a key challenge. This has been exacerbated by the COVID-19 pandemic which saw the economy contract by 7% in 2020 [14]. The healthcare sector has not been spared from this inequality. The private health care sector, which serves 16% of the population [16, 17], is able to afford world-class therapies to insured patients, while the public sector, accessed by 84% of the population [16, 17], due to resource constraints, has to make decisions which ensure that the most beneficial treatments are prioritised for the largest number of patients. In addition, South Africa suffers from poor returns on human resource investments with up to 30% of locally trained doctors having emigrated [18].

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Funding

This work was funded by the South African Medical Research Council (Extramural Unit for Stem Cell Research and Therapy) and the University of Pretoria through the Institute for Cellular and Molecular Medicine.

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Authors and Affiliations

  1. Institute for Cellular and Molecular Medicine, Department of Immunology, and South African Medical Research Council Extramural Unit for Stem Cell Research and Therapy, Faculty of Health Sciences, University of Pretoria, Pretoria, South Africa

    Candice Laverne Hendricks & Michael Sean Pepper

  2. Antion Biosciences SA, Geneva, Switzerland

    Marco Alessandrini

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  2. Marco Alessandrini
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CLH, MA and MSP all contributed equally to the concept, preparation and editing of the paper.

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Correspondence to Michael Sean Pepper.

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Hendricks, C.L., Alessandrini, M. & Pepper, M.S. Equitable access to cell and gene therapies in South Africa: opportunities and hurdles. Gene Ther 30, 180–186 (2023). https://doi.org/10.1038/s41434-021-00309-y

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