What the trial was testing
The VALOR enrolled 139 patients with als. The study was sponsored by Biogen and tracked outcomes across the full group of patients who matched the trial's eligibility profile.
It was a large trial designed to confirm whether the treatment works well enough for wider use. Trials at this stage are designed to produce evidence regulators and physicians can act on — not just observations to follow up later.
What the results showed
Tofersen lowered harmful SOD1 protein levels in spinal fluid but didn't improve physical function in the first 28 weeks.
The New England journal of medicine · 2022 · NCT03070119
These findings — that people who started tofersen right away had slightly better muscle function after one year than those who waited six months — were published in the The New England journal of medicine and represent the headline result of the study.
Researchers tracked outcomes across 139 patients enrolled in the trial. The result was consistent enough across the group that the team felt confident reporting it.
What this means for patients
For patients with als, this result changes the calculus on what to ask their care team about. Whether it changes day-to-day care depends on factors like disease subtype, prior treatments, and where the patient is in their care journey.
What you can do now
Tofersen was FDA-approved in 2023 for adults with ALS linked to SOD1 mutations, though it works differently than traditional treatments. The drug requires spinal injections and can cause side effects including headaches and falls. If you have SOD1 ALS, talk with your doctor about whether tofersen is right for you and what to expect from treatment.
Eligibility for the treatments mentioned above depends on specific test results and clinical history. Bring this summary, the trial name, and your most recent labs or pathology report to your next visit.
Open als trials
A Study to Evaluate the Tolerability, Safety and Efficacy of VGN-R13 in Patients with ALS
The purpose of this trial is to evaluate safety and efficacy of intrathecal delivery of VGN-R13 as a treatment of Amyotrophic Lateral Sclerosis (ALS).
Needs of ALS Patients With C9orf72 Mutation and Their Caregivers
Individuals with Amyotrophic Lateral Sclerosis (ALS) carrying the C9orf72 HRE mutation (C9Pos) often exhibit different phenotypic traits compared to other patients (C9Neg), presenting a more aggressive form of the disease, with also a higher frequency of comorbidity with frontotemporal dementia (ALS-FTD) and a greater prevalence of family history of ALS and/or other neurodegenerative diseases. Considering these characteristics (comorbidity with FTD and family history of ALS and/or other neurodegenerative pathologies) as factors that have their importance from an assistance point of view, both C9orf72 patients and their caregivers may have particular needs in several respects, and in this sense this investigation is configured. This survey aims to describe the various aspects related to the care and quality of life of C9orf72 ALS patients and their respective caregivers, with the goal of identifying actionable steps to improve the quality of life for both.