South African Medical Research Council Extramural Unit for Stem Cell Research and Therapy; and Institute for Cellular and Molecular Medicine, Department of Immunology, School of Medicine, Faculty of Health Sciences, University of Pretoria, South Africa.
S Afr Med J. 2018 Dec 13;109(1):20-22. doi: 10.7196/SAMJ.2018.v109i1.13425.
The fields of cell and gene therapy are moving rapidly towards providing innovative cures for incurable diseases. A current and highly topical example is immunotherapies involving T-cells that express chimeric antigen receptors (CAR T-cells), which have shown promise in the treatment of leukaemia and lymphoma. These new medicines are indicative of the changes we can anticipate in the practice of medicine in the near future. Despite their promise, they pose challenges for introduction into the healthcare sector in South Africa (SA), including: (i) that they are technologically demanding and their manufacture is resource intensive; (ii) that the regulatory system is underdeveloped and likely to be challenged by ethical, legal and social requirements that accompany these new therapies; and (iii) that costs are likely to be prohibitive, at least initially, and before economies of scale take effect. Investment should be made into finding novel and innovative ways to introduce these therapies into SA sooner rather than later to ensure that SA patients are not excluded from these exciting new opportunities.
细胞和基因治疗领域正在迅速为无法治愈的疾病提供创新疗法。当前一个非常热门的例子是涉及嵌合抗原受体 (CAR T 细胞) 的 T 细胞免疫疗法，该疗法在白血病和淋巴瘤的治疗中显示出了前景。这些新药物表明我们可以预期在不久的将来医学实践会发生变化。尽管它们很有前景，但它们在引入南非 (SA) 的医疗保健部门方面带来了挑战，包括：(i) 技术要求高，制造资源密集；(ii) 监管系统不发达，并且可能受到伴随这些新疗法的伦理、法律和社会要求的挑战；(iii) 成本可能很高，至少在最初是这样，而且在规模经济产生之前是这样。应该投资寻找新颖和创新的方法，尽快将这些疗法引入南非，以确保南非患者不会被排除在这些令人兴奋的新机会之外。
