New drugs for Amyotrophic Lateral Sclerosis

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We have presented on several occasions a summary of drugs that can be used in the fight against the progression of Amyotrophic Lateral Sclerosis. Here is a list of drugs that are currently (mid-2020) undergoing phase III clinical trials. When reading this list you should be aware of the following limitations:

  • None of these drugs can stop the progression of the disease, let alone reverse its course.
  • Most of these drugs have a dose-related effect. For example, a beneficial effect at low doses can become harmful at high doses. Of course, the concept of dose is very vague, it depends on the weight but also on other factors such as insulin resistance, which are rarely taken into account.
  • Our pharmacological imagination is shaped by our childhood illnesses where the administration of oral pills allows healing within a few days. However, the oral route is one of the least effective methods of administration, particularly for diseases of the central nervous system.
  • An adult has little experience of illness lasting longer than a few days. The drugs presented here do not have visible effects, they just slow the progression of the disease. Psychologically it is difficult to take medication and to continue to observe a deterioration in his condition.
  • A drug can completely have a beneficial action on a tissue and have a harmful action on one or more other tissues ("side" effects). The very term "medication" is therefore misleading, it is the result of a temporary compromise that is specific to the patient.
  • There are surprisingly several drugs known to bring a 30% improvement in life expectancy after diagnosis of ALS, that is to say several months, but it is at the cost of adverse effects which make the prospect unattractive.
  • Although the disease is referred to as ALS, there are multiple subcategories of ALS that only have in common the fact they are motor neuron diseases. For example below BIIB067 is a gene therapy that only targets patients with a mutation in the SOD1 gene (~ 2% of cases). The only point common to most cases of ALS is malformed TDP-43 protein granules located in the cytoplasm instead of the nucleus. But these granules are also observed in other neurodegenerative diseases. it is not known whether it is a protective mechanism like stress granules, or a cause of the disease.

  • BIIB067

  • Tauroursodeoxycholic Acid
  • Ravulizumab
  • Levosimendan
  • Arimoclomol
  • Deferiprone
  • Ibudilast
  • Memantine Hydrochloride
  • Cu(II)ATSM

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This book retraces the main achievements of ALS research over the last 30 years, presents the drugs under clinical trial, as well as ongoing research on future treatments likely to be able stop the disease in a few years and to provide a complete cure in a decade or two.

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