A U.S. center reports delivering tofersen to 11 people with SOD1 ALS before approval
Original source
Optimizing Research Operations and Resource Utilization in ALS Care: Insights From the Tofersen Antisense Oligonucleotide Expanded Access Protocol. (opens in a new tab)Compass summarised this from the study's abstract.
Study details
- Studied in
- Human
Related topics
Massachusetts General Hospital provided monthly intrathecal tofersen to 11 adults with symptomatic superoxide dismutase 1 (SOD1) ALS through expanded access protocols from July 2021 to July 2023. The center administered 120 doses, with no treatment-related serious adverse events reported. The program cost $336,620 and used donated drug, insurance and philanthropic support.
Why this matters
The report shows that a specialist academic center was able to provide tofersen to people who were not eligible for traditional clinical trials before U.S. approval. It describes the practical resources and funding needed for this type of access, not evidence that the program improved ALS outcomes or changed treatment for everyone with ALS.
Limitations and context
This was a single-center observational report involving 11 people with SOD1 ALS. It describes treatment delivery, safety and operating costs rather than testing efficacy, and the findings may not generalize to other centers or patients. The source is a journal article, but no comparison group or treatment-effect assessment is reported.