Print version ISSN 0256-9574
SAMJ, S. Afr. med. j. vol.102 n.6 Cape Town Jun. 2012
Stem cell therapy and amyotrophic lateral sclerosis
To the Editor: We have been made aware of several patients with neurological diseases attending stem cell therapy centres, and in particular individuals with amyotrophic lateral sclerosis (ALS). The opening of stem cell clinics in South Africa has led to an increased number of patients pursuing this therapy at great financial cost and with high expectations. Regarding this technology, we would like to emphasise the following:
- There is a complete absence of sound clinical evidence for the efficacy of stem cell therapy for ALS and neurodegenerative diseases in general. No randomised controlled studies or even open-label studies with long-term follow-up exist. Furthermore, a prospective case series in the Netherlands showed no benefit from treatment with olfactory ensheathing cells in patients with ALS.1
- Long-term safety data on the use of stem cell therapy in neurological disorders are lacking.
- Currently, the main utility for stem cell technology in neurological disorders is the ability to offer human in vitro models for understanding disease mechanisms and facilitating drug discovery. Although the potential for cell-based therapy exists, current claims of efficacy in ALS and other neurodegenerative disorders are premature and unsubstantiated.
We have strong scientific, ethical and economic objections to clinics offering stem cell therapy on a commercial basis, as well as medical practitioners recommending (or not advising against) this modality for neurological disorders. Patients with incurable diseases such as ALS are desperate and emotionally vulnerable to the claims of institutions allegedly being able to heal a number of diseases that modern medicine is unable to. Although we respect their autonomy and right to self-determination, patients are seldom equipped to assess the evidence for or against different treatment modalities, and are therefore reliant on medical professionals in decision-making. Moreover, regulations guiding stem cell research and therapy are sorely lacking in South Africa.2 It is therefore the healthcare professional's moral duty to present patients and their families with relevant information in an understandable manner. In the case of stem cell therapy, this involves a thorough discussion about the absence of scientific evidence and the likelihood that substantial sums will be paid by patients for no discernible benefit. Failing to do so, in our view, is unethical and not in the best interests of the patient.
1. Piepers S, van den Berg LH. No benefits from experimental treatment with olfactory ensheathing cells in patients with ALS. Amyotroph Lateral Scler 2010;11(3):328-330 [PMID: 20433414]. [ Links ]
2. Pepper M. The stem cell regulatory environment in South Africa: cause for concern. S Afr Med J 2009;99(7):505-507 [PMID: 20460017]. [ Links ]
Division of Neurology
Tygerberg Academic Hospital/Faculty of Health Sciences