Based on data from 309 patients listed in the SMA France Registry and suffering from type I proximal spinal muscular atrophy (SMA), French clinicians conducted an initial comparative study of nusinersen (Spinraza) and gene therapy (Zolgensma) as first-line treatments.
- Data on the motor, respiratory and nutritional functions of 24 children, 12 treated with gene therapy and 12 with nusinersen, were rigorously matched and compared.
- The median age of the children at the start of treatment was 6.1 months.
- Two years after the start of treatment, three deaths were reported, one in the gene therapy group and two in the nusinersen group, with no proven direct link to these treatments.
- Motor function improved similarly with both treatments.
- One in 11 patients (9%) receiving gene therapy required nutritional support, compared with 5 in 10 (50%) receiving nusinersen.
- Night-time ventilation was required in 5 of the 11 patients (45%) receiving gene therapy, compared with 8 of the 10 patients (80%) receiving nusinersen.
- The incidence of unsatisfactory clinical response was lower with gene therapy (25%) than with nusinersen (67%).
These results support gene therapy as the first-line treatment for children with symptomatic SMA type I, but will need to be confirmed by clinical trials comparing the two treatments.
