Work

Cellular therapies for sickle cell disease: Should we be calling them a cure?

Year: 2025
Type: article
Abstract: Recent advances in gene therapy and expanded access for haematopoietic stem cell transplantation (HSCT) have made conversations about a cure more pertinent for individuals living with sickle cell disease (SCD).These advances, including the approval of exa-cel gene therapy in the United Kingdom, 1 have amplified the language of cure within policy, media, and clinical discussions.However, as this language becomes more visible, questions emerge about what cure means for individuals living with SCD, for healthcare systems, and for communities historically underserved and marginalized within research and clinical care.The term cure, derived from the Latin "curare," meaning "to care for" or "to heal," suggests the complete elimination of symptoms caused by disease and a restoration to a previous state of health, 2 but in the context of a lifelong disease like SCD, the meaning is more complex.In this article, we begin with the personal reflections of one author (F.D.) living with SCD as she grapples with the possibility and uncertainty of cellular therapies (Box 1).Next, we survey how the language of cure is used in public discussions about SCD cellular therapies.We share the account of one author (Z.G.-S.) whose experience befits the word cure, before exploring complexities that challenge the use of curative language.Finally, we highlight important considerations for health professionals and health systems relating to cellular therapies for SCD.
Source: HemaSphere
Language: English
Subfield: Genetics
Field: Medicine
Open Access status: gold
Estimated APC paid: $2,480
OpenAlex ID: W4417231059
FWCI: 1.054
Cites: 11
Cited by: 2
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