What this covers
Metachromatic leukodystrophy (MLD) is a rare, often devastating inherited disorder of the nervous system, one of a wider group of conditions called leukodystrophies. Using MLD as an example, this review outlines the therapeutic approaches that have been tested in animal models and clinical trials, including enzyme-replacement therapy, bone marrow and umbilical cord blood transplants, transplantation of genetically modified haematopoietic stem cells, and gene therapy.
Key takeaways
- At the time of writing there were no disease-specific treatments for the leukodystrophies, making the search for effective therapies for MLD especially pressing.
- The authors argue that a successful therapy must deliver persistent, high-level production of the missing enzyme, arylsulfatase A, within the central nervous system.
- Gene therapy using adeno-associated viruses is presented as the most promising route for clinical development, offering the best balance of potential effectiveness and reduced safety risk.
Why it matters
Mapping out which approaches show the most promise helps guide research towards treatments that could one day change the course of MLD and related leukodystrophies.
