From the Guidelines
Several promising new treatments and clinical trials are exploring options for ALS, with a focus on improving morbidity, mortality, and quality of life. Recently approved medications include Relyvrio (sodium phenylbutyrate and taurursodiol), which slows disease progression by protecting neurons from stress, and Qalsody (tofersen), specifically for SOD1 gene mutation ALS 1. Radicava (edaravone) remains an established treatment that reduces oxidative stress. Ongoing clinical trials are investigating stem cell therapies to replace damaged neurons, gene therapies targeting specific mutations like C9orf72, and immunotherapies to modulate the immune response in ALS.
Researchers are also exploring repurposed drugs like AMX0035 (a combination of sodium phenylbutyrate and taurursodiol) and masitinib (a tyrosine kinase inhibitor). Additionally, trials are examining the potential of antisense oligonucleotides to silence harmful gene expression and neuroprotective compounds that might preserve motor neuron function. These emerging treatments aim to address different aspects of ALS pathology, from protein misfolding to neuroinflammation, offering hope for more effective management of this complex neurodegenerative disease.
Some key considerations for ALS management include:
- Dietary counseling to adapt the texture of solid and liquids to facilitate swallowing and avoid aspiration 1
- Postural maneuvers, such as chin-tuck posture, to protect the airway during swallowing 1
- Management of sialorrhea, which can be distressing and increase the risk of aspiration and pneumonia, using anticholinergic agents, botulinum toxin therapy, or radiation therapy 1
- The use of thicker liquids, semisolid foods with a high water content, such as jellified water, as better alternatives to thinner liquids to help alleviate aspiration 1
It is essential to prioritize the most recent and highest-quality studies when making definitive recommendations for ALS treatment, focusing on improving morbidity, mortality, and quality of life. The most recent study 1 provides valuable insights into the respiratory management of patients with neuromuscular weakness, including ALS, and highlights the importance of addressing sialorrhea and other respiratory complications.
From the FDA Drug Label
The FDA drug label does not answer the question.
From the Research
Current Treatments for ALS
- There are currently three disease-modifying treatments approved by the FDA to slow the progression of amyotrophic lateral sclerosis (ALS): edaravone, riluzole, and sodium phenylbutyrate and taurursodiol (PB/TURSO) 2.
- Edaravone, a novel neuroprotective agent, has been shown to slow down the loss of physical function in ALS patients by 33% compared to placebo 3.
- Riluzole, a glutamatergic neurotransmission inhibitor, is the only drug approved by the USA Food and Drug Administration for ALS treatment with modest benefits on survival 4, 5.
Clinical Trials and Emerging Agents
- Emerging agents offer some hope for patients with ALS, including an oral tyrosine kinase inhibitor, RIPK1 inhibition, the use of mesenchymal stem cells, antisense oligonucleotides, and modification of the patient's own mesenchymal stem cells 2.
- A fourth therapy has been recently approved under accelerated approval and is contingent upon verification of clinical benefit in confirmatory trials 2.
- Clinical trials are ongoing to investigate the efficacy of these emerging agents in treating ALS.
Pharmacokinetics and Drug Development
- The clinical pharmacokinetics of riluzole and edaravone have been summarized, with edaravone achieving the desired systemic concentrations to possibly drive the local brain concentrations for its efficacy in ALS patients with lesser variability than riluzole 6.
- There is an opportunity for the development of an oral dosage form of edaravone, which may potentially benefit the therapy option for ALS patients by avoiding hospitalization costs 6.
- Combination strategies of edaravone and/or riluzole with suitable P-glycoprotein efflux-blocking drugs may be considered to gain more favorable outcomes in ALS patients 6.