Hematopoietic stem cell transplantation and hematopoietic stem cell gene therapy in X-linked adrenoleukodystrophy.

[adrenomyeloneuropathy]

Allogeneic hematopoietic stem cell transplantation (HSCT ) is the only therapeutic approach that can arrest cerebral demyelination of X-linked adrenoleukodystrophy (ALD ) in boys and results in long -term in a good quality of life , provided the procedure is performed at an early stage of disease . Similar benefits of allogeneic HSCT have been demonstrated in adults with cerebral ALD . However , it is not yet known whether allogeneic HSCT can prevent or rescue adrenomyeloneuropathy . Allogeneic HSCT remains associated with significant morbidity and mortality risks , particularly in adults , and not all ALD patients have donors despite the availability of cord blood . The absence of biological markers that can predict the evolutivity of cerebral disease is a major limitation to propose in due time allogeneic HSCT to ALD patients . Recently , HSC gene therapy using lentiviral vector was shown to have comparable efficacy than allogeneic HSCT in two boys with cerebral ALD who had no Human-leukocyte-antigen (HLA )-matched donor . If these results are confirmed in an extended series of patients , HSC gene therapy may become the first therapeutic option for all ALD male patients who develop cerebral demyelination .