promising results from clinical trial: low-dose interleukin-2 may extend survival in ALS patients

A groundbreaking clinical trial has demonstrated that the addition of low-dose interleukin-2 (IL2LD) to standard treatment protocols for amyotrophic lateral sclerosis (ALS) could slow disease progression and increase patient survival. The MIROCALS randomized controlled trial, conducted across multiple European countries including the UK, France, Italy, Sweden, and Ireland, involved 220 recently diagnosed ALS patients who initially received riluzole, the current standard therapy. Participants were subsequently randomized to receive either IL2LD or a placebo over 18 months.

ALS, also known as motor neuron disease, is a progressive neurodegenerative disorder affecting around 45,000 people in Europe. It damages the motor nerves responsible for muscle control, leading to muscle weakness, paralysis, and eventually death. The disease exhibits variable progression, with some individuals succumbing within a year of onset while others live a decade or longer.

IL2LD, a molecule known for regulating the immune system, has been shown to boost regulatory T cells (Tregs), which play a role in reducing neuroinflammation. The study aimed to evaluate the safety of IL2LD as an adjunct therapy and its potential to slow neurodegeneration.

Results revealed that IL2LD is safe and well-tolerated among ALS patients. Significantly, approximately 80% of participants with lower levels of a cerebrospinal fluid biomarker indicating motor neuron damage experienced a survival benefit. Specifically, those with reduced levels of phosphorylated neurofilament heavy chain protein (pNFH) saw their risk of death decrease by over 40%.

These findings highlight the potential of immune system modulation as a therapeutic approach in ALS. While IL2LD is not yet approved for clinical use in this context, the positive outcomes suggest it could be further developed as a disease-modifying treatment alongside existing therapies like riluzole.

Professor Andrea Malaspina of Queen Mary University of London emphasized that these results mark a significant milestone in ALS research, proposing immunomodulation as a promising strategy to alter disease progression and improve patient outcomes. The study underscores the importance of biomarker-guided therapies in understanding and treating neurodegenerative diseases.

For more details, see the published article in The Lancet: link. The research was conducted with support from leading neurology experts and research institutions across Europe, highlighting a collaborative effort in the fight against ALS.