Table 1. Summary of therapeutic approaches that have entered clinical trials for the treatment of amyotrophic lateral sclerosis.

Therapeutic	Therapeutic approach	Therapeutic target	Class of drug	Eligible ALS population	Trial phase	ClinicalTrials.gov Identifier
Tofersen (BIIB067)	Gene therapy: antisense oligonucleotide	SOD1 transcript	Antisense oligonucleotide	SOD1 ALS	III	NCT02623699
BIIB078	Gene therapy: antisense oligonucleotide	C9orf72 repeat expansion	Antisense oligonucleotide	C9orf72 ALS	I	NCT03626012
Masitinib	Modulating neuroinflammation	Tyrosine kinase inhibitor	Small molecule	Familial or sporadic ALS	III	NCT03127267
Ibudilast (MN-166)	Modulating neuroinflammation	Phosphodiesterase inhibitor	Small molecule	Familial or sporadic ALS	II / III	NCT04057898
Fasudil	Modulating neuroinflammation	Rho kinase inhibitor	Small molecule	Not specified	II	NCT03792490
Ravulizumab	Modulating neuroinflammation	Complement component 5 inhibitor	Monoclonal antibody	Familial or sporadic ALS	III	NCT04248465
Zilucoplan	Modulating neuroinflammation	Complement component 5 inhibitor	Small molecule	Familial or sporadic ALS	II / III	NCT04297683
Anakinra	Modulating neuroinflammation	IL-1 receptor antagonist	Monoclonal antibody	Familial or sporadic ALS	II	NCT01277315
Tocilizumab	Modulating neuroinflammation	IL-1 receptor antagonist	Monoclonal antibody	Not specified	II	NCT02469896
Arimoclomol	Clearance of protein aggregates	Upregulates heat shock proteins	Small molecule	Not specified	III	NCT03491462
Colchicine	Clearance of protein aggregates	Enhances expression of HSPB8	Small molecule	Sporadic ALS	II	NCT03693781
Rapamycin	Clearance of protein aggregates	Stimulate autophagy	Small molecule	Familial or sporadic ALS	II	NCT03359538
BIIB100/KPT- 350	Targeting nucleocytoplasmic transport	XPO1/CRM1 inhibitor	Small molecule	Not specified	I	NCT03945279
HEALEY platform trial	Various	Various	Various	Familial or sporadic ALS	II / III	NCT04297683

ALS, amyotrophic lateral sclerosis; IL-1, interleukin 1; SOD1, superoxide dismutase; XPO1, exportin 1.