Amyotrophic lateral sclerosis (ALS) is a severe neurodegenerative disease characterized by the progressive loss of motor neurons, nerve cells that control the voluntary muscles. This leads to symptoms that include overall muscle weakness, causing difficulties with moving, breathing, eating, and speaking.
There is no cure for ALS yet, but there are treatments to help manage the condition. There are also medications available to help ease specific disease symptoms and a number of experimental treatments in the pipeline.
The U.S. Food and Drug Administration (FDA) has approved four treatments specifically aimed at slowing the progression of ALS: Rilutek (riluzole tablet), Tiglutik (riluzole suspension), Exservan (riluzole oral film), and Radicava (edaravone).
None of these treatments reverse the damage already caused by ALS, but researchers think they might increase patients’ life expectancy. Doctors often prescribe them in combination with symptomatic treatments to improve patients’ quality of life.
The treatment is thought to prevent the release of glutamate, a neurotransmitter, or cell-signaling molecule, that rises to toxic levels in the brain and spinal cord of ALS patients, damaging nearby nerve cells. By reducing the buildup of glutamate, scientists think Rilutek may prevent or slow the glutamate-induced deterioration of motor neurons.
The FDA approved Rilutek in 1995 as the first systemic treatment to slow ALS progression and prolong survival. It remained the sole systemic ALS therapy in the U.S. for more than 20 years, until Radicava became available.
Other countries also have approved the therapy to treat ALS, including the European Medicines Agency (EMA), which approved Rilutek in 1996.
Tiglutik (riluzole oral suspension)
Tiglutik is an oral suspension that doctors can administer using a syringe. Many ALS patients develop dysphagia (trouble swallowing) as their disease progresses, and this can lead to difficulty taking pills such as Rilutek.
Tiglutik has a similar mechanism of action to Rilutek because both contain riluzole.
Exservan (riluzole oral film)
Exservan is a riluzole oral film that Aquestive Therapeutics developed. The treatment uses Aquestive’s PharmFilm technology. It is an orange film containing 50 mg of riluzole, which patients take twice a day by placing it on the tongue, where it dissolves automatically.
Researchers have not performed independent clinical trials of Exservan; the FDA approved it based on the results of trials testing Rilutek, the oral tablet form of riluzole.
The most common side effects of Exservan are numbness in the mouth, lack of energy, nausea, high blood pressure, and abdominal pain.
Mitsubishi Tanabe Pharma America developed Radicava, which is administered as an injection into a vein. Researchers think the therapy works through a different process than riluzole, although they don’t fully understand its exact mechanism of action. It is thought it acts as an antioxidant or a so-called free radical scavenger.
Free radicals are a byproduct of energy production by the cell. Normally, the body quickly removes free radicals; if not, they can cause damage and lead to cell death through a process called oxidative stress — an imbalance between free radical production and the antioxidants that work to neutralize them.
Researchers think damage from free radicals is one of the mechanisms involved in the progressive nerve cell death that marks ALS. By helping to remove free radicals quickly, Radicava may be able to reduce damage to the nervous system and slow disease progression.
Radicava is not available in Europe. Mitsubishi withdrew its application to the EMA for marketing authorization based on the committee’s evaluation of the preliminary data. Radicava is not currently available in Australia either, and no application has been made to the country’s Therapeutic Goods Administration for its approval.
Last updated: May 6, 2020
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