AMX0035 is a drug combination of sodium phenylbutyrate (PB) and tauroursodeoxycholic acid (TUDCA). It works by regulating the mitochondrial (cell power supply) and endoplasmic reticulum (quality control centers). Put simply, both processes should work in unison, but when they don’t, cells in the body die, and internal, neurological inflammation develops. AMX0035 regulates cells in the body to prevent the stress that kills those cells and prevents neurological inflammation from developing. This might sound familiar to you with any neurological disease or trauma.
Why this drug is important to everyone with neurological disease and trauma? You might be thinking that you don’t have ALS, so how could this discovery be important to you. In the study of neurological disease and trauma, what works in one area of study often is translated into other areas. Some clinical trials are considering AMX0035 in the treatment of Alzheimer’s Disease and Parkinson’s Disease. Phase II of the study of AMX0035 and ALS has produced some excellent results. Because ALS has no known cure, the drug is going to be released for people to use now. This is sort of a loophole in drug testing where humanitarian issues take precedent due to the severity and negative outcomes of some diseases.
University of Colorado researchers demonstrated that phenylbutyrate stops the progression of Parkinson's disease in mice by turning on a gene called DJ-1 that can protect dopaminergic neurons in the midbrain from dying. Sodium phenylbutyrate is used to treat urea cycle disorders, because its metabolites offer an alternative pathway to the urea cycle to allow excretion of excess nitrogen. It is an orphan drug, it's not clear if sodium phenylebutyrate can be substituted with Butyrate x TUCDA.
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