Dozens of people living with sickle cell disease are reportedly symptom-free after undergoing experimental gene-editing treatment.1,2 The treatment is expected to be the first gene-editing treatment to receive FDA approval. “The results are very promising, but it might take some time to complete the appropriate clinical trials to ensure generalizability to all participants, safety, and durability of the response,” cautions Charis Eng, MD, PhD, FACP, chair of the Genomic Medicine Institute at the Cleveland Clinic. Some other ethical concerns:
• Accessibility and equity. This is a concern because many sickle cell patients have limited medical and financial resources and might not be able to access the treatment, according to Eng.
• The treatment is not appropriate for all sickle cell patients. “Sickle cell disease carries broad clinical phenotype and expressivity,” Eng explains.
Some patients are living with severe disease, so the benefits of the gene-editing treatment far outweigh any risk. For those with milder disease, the benefits of the treatment might not be worth the risk.
• Since the treatment was widely publicized, there is the possibility of false hope. “Like any other treatment, it is important that patients are aware of limitations, but without crushing their hopes that a cure might indeed be feasible,” Eng says.
Eng recommends clinicians use the term “remission” instead of “cure” when talking with patients. For every medical discovery, there always is a risk of side effects and that the response may not be permanent. Thus, it is possible the effect on sickle cell patients will prove to be only temporary. “But for patients living with chronic conditions, even a moderate improvement in symptoms and quality of life are vital,” offers Eng, adding that this is especially true for debilitating conditions such as sickle cell disease.
Clinicians should be prepared to properly direct patients to appropriate research trials. “Specialist clinicians are typically aware of relevant clinical trials that their patients may be eligible for,” Eng says.
Some patients may search for this information proactively. “It is important that clinicians and patients work together to identify the right research trials for the right patients,” Eng stresses. “Joint decision-making is so important and important for the patient’s feel-good factor.”
REFERENCES
1. Zarghamian P, Klermund J, Cathomen T. Clinical genome editing to treat sickle cell disease-A brief update. Front Med (Lausanne) 2023;9:1065377.
2. Stein R. Sickle cell patient’s success with gene editing raises hopes and questions. NPR. March 16, 2023.
Like any other treatment, it is important patients are aware of limitations, but without crushing their hopes that a cure might be feasible.
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