| Definition | Elivaldogene autotemcel is a gene therapy consisting of genetically modified autologous cells. It is used to provide functional copies of human adrenoleukodystrophy protein (ALDP) in patients with adrenoleukodystrophy,[L39491] an X-linked genetic disorder characterized by missing or non-functional _ABCD1_ gene that codes for ALDP.[A243691] ALDP is a key protein that normally breaks down fatty substances in the body called very long-chain fatty acids (VLCFAs). Without sufficient levels of functional ALDP, VLCFAs accumulate in the body leading to inflammation and destruction of myelin, which is an insulating layer and essential component of nerves.[L40298]
Cerebral adrenoleukodystrophy is an inflammatory cerebral phenotype that more commonly affects young men: it is associated with progressive brain function loss and poor survival rates without treatment. Allogeneic hematopoietic stem-cell transplantation has been the primary treatment option; however, it is associated with a risk of graft failure and graft-versus-host disease (GVHD), calling for alternative treatment options. Gene therapy with autologous hematopoietic stem cells was introduced and investigated as a possible treatment for patients with adrenoleukodystrophy.[A243691] Elivaldogene autotemcel works by delivering genes into the patient's body to produce functional ALDPs. It was first approved by the European Commission in July 2021 under the market name Skysona.[L40298] |
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