THERAPEUTICS

Brands

• Rilutek

Generic?

• No

Class

• Neuromuscular drug

Commonly Prescribed for

(FDA approved in bold)

• Amyotrophic lateral sclerosis (ALS)

• Cerebellar ataxia

• Depression or anxiety disorders

How the Drug Works

• The mode of action is unknown and probably involves multiple mechanisms. It strongly suppresses the persistent Na+ current in a wide variety of neurons, potentiates calcium-dependent K+ current, reduces presynaptic transmitter release, inhibits voltage-gated calcium channels, and enhances neuronal survival through production of neurotrophic factors (glial cell-derived neurotrophic factor [GDNF], brain-derived neurotrophic factor [BDNF]) and activation of mitogen-activated protein kinase [MAPK] pathway

How Long Until It Works

• Steady state is reached in 5 days, but it can take months to assess any clinical effect from the drug

If It Works

• ALS is a degenerative disease and deterioration is the general rule. Riluzole can increase survival or time to tracheostomy but is not a cure

If It Doesn't Work

• It is difficult to determine if the treatment is effective, especially because ALS progression varies greatly from patient to patient. Supportive care is the mainstay of current ALS treatment. This may include monitoring and treatment of gait, swallowing, and respiratory difficulties

Best Augmenting Combos for Partial Response or Treatment-Resistance

• No other medication is indicated for the treatment of ALS progression

Tests

• Measure serum transaminases, including ALT levels, at baseline and monthly for 3 months. Then evaluate every 3 months for the first year and periodically after that. Once ALT exceeds 5 times normal, begin checking weekly, and discontinue if ALT exceeds 10 times normal or clinical symptoms, such as jaundice, occur

ADVERSE EFFECTS (AEs)

How the Drug Causes AEs

• Unknown

Notable AEs

• Nausea, weakness, dizziness, diarrhea, abdominal pain, pneumonia, tremor, anorexia, somnolence, and paresthesias. Elevation of hepatic transaminases

Life-Threatening or Dangerous AEs

• Neutropenia and hepatic effects. Neutropenia is uncommon (less than 1/1000 in clinical trials). Hepatic transaminase elevation is common (about 50% of patients will experience 1 elevated level) but usually clinically insignificant