To understand more about gene therapies, you can explore the resources below:
Mechanisms for therapies targeting genetic forms of ALS
Alliance ALS/MND Genetics: What can we do about genetic ALS/MND?
The ALS Association Therapies Targeting ALS-Linked Genetic Mutations
The ALS Association Antisense Therapy for ALS
Clinical trials
There are ongoing studies to test experimental treatments that are aimed at individuals with specific ALS genetic variants. Please find an up-to-date list below.
For more information on clinical trials and for a list of all current ALS clinical trials in Canada, refer to our Clinical Trials page.
SOD1
| Treatment | Company | Type of treatment | Phase of clinical trial | Recruitment status | Canadian sites? | More Details |
|---|---|---|---|---|---|---|
| AMT-162 | uniQure | Intrathecal AAV-microRNA | Phase 1 (not yet started) | n/a | n/a | [1]SOD1 Gene Therapy Study for ALS |
| ALN-SOD | Regeneron/Alnylam | RNA interference (RNAi) | Phase 1 (not yet started) | n/a | n/a | |
| AP-101 | AL-S Pharma | Intravenous antibody | Phase 2 | Recruiting | Edmonton, London, Toronto, Montreal (Neuro) | [1]AP-101 ALS Trial Details |
| Tofersen | Biogen | Intrathecal antisense oligonucleotide | Phase 3 ATLAS | Recruiting | Calgary, Toronto, Montreal (Neuro) | [1] Tofersen ALS Pre-Symptomatic Study [2] ATLAS Study Website |
FUS
| Treatment | Company | Type of treatment | Phase of clinical trial | Recruitment status | Canadian sites? | More Details |
|---|---|---|---|---|---|---|
| Ulefnersen | Ionis | Intrathecal ASO | Phase 3 | Recruiting | Montreal (Neuro) | [1]FUSION: FUS-ALS Study Details |
C9orf72
Nothing currently – more coming soon.
TARDBP
Nothing currently – more coming soon.
Observational studies
Learning from individuals who carry specific genetic variants that increase their risk of ALS is critical for researchers’ ability to understand and develop treatments targeting those genes, but also for all people living with ALS. Contributing to research by enrolling in observational studies as a genetic carrier who is not experiencing ALS symptoms can help us understand the biological processes that might occur at the earliest stages of the disease. An ongoing, landmark clinical trial called ATLAS, is examining the ability of a treatment called tofersen to potentially slow down or stop the disease in people who carry SOD1 genetic variants before any symptoms arise, and it is a direct result of learning from SOD1 gene carriers in observational studies.
Please find some observational studies for ALS below:
ALS Families Project - Eleanor and Lou Gehrig ALS Center
ALS Signal – Explore ALS Clinical Research, Trials and Studies.
*Select Genetic ALS Observational Studies to see all current studies.
ALS Trial Navigator: a comprehensive resource to empower your ALS clinical trial search.
The DIALS (Dominant Inherited ALS) Network
PREVENT ALS
Pre-fALS (Pre-symptomatic Familial ALS)
Next section: Genetically Focused ALS Organizations & Events