What this covers
Metachromatic leukodystrophy (MLD) is a rare, progressive disorder of the nervous system, and several treatments have been tried over the years. This systematic review gathered the available evidence on the benefits and risks of two approaches — donor stem cell transplantation (allogeneic HSCT) and the gene therapy atidarsagene autotemcel (arsa-cel) — comparing each with the natural course of the untreated disease. From nearly 7,000 records, 35 papers covering 12 studies were included.
Key takeaways
- Donor stem cell transplantation showed no benefit for survival, movement or thinking in late-infantile patients, whose disease progressed much as it would untreated.
- Transplantation was linked to serious complications, including treatment-related death, graft-versus-host disease and the need for re-transplantation.
- Most late-infantile and early-juvenile patients treated with the gene therapy showed normal development or slower decline, and late-infantile patients also gained a survival benefit compared with the untreated course and with transplantation.
Why it matters
By weighing these treatments side by side, the review suggests gene therapy offers meaningful benefits for certain forms of MLD, while underlining that more research is needed because the findings come from studies without randomised controls.
