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Researchers at the Autonomous University of Barcelona Institute of Biotechnology and Biomedicine (IBB-UAB) in collaboration with biopharmaceutical company SOM Biotech have carried out a drug repositioning study that has discovered that Tolcapone could improve treatment of transthyretin-related hereditary amyloidosis (ATTR), a minority disease. This drug is currently used to treat Parkinson and SOM Biotech has discovered and patented its use with ATTR.

The results of the study, published in Nature Communications, show that Tolcapone is useful for all variants of the disease and can be up to four times more effective than the only treatment currently available to treat the polyneuropathic variety of ATTR. The alternative to curb progression of the disease is a liver or heart transplant.

The biophysical trials that have been conducted (in vitro in cell cultures and ex vivo in human plasma and model mice with the disease) have shown that Tolcapone imitates the process that binds transthyretin to the thyroid hormone. It binds the protein by joining to the protein subunits and stabilizing its structure, thus preventing it from breaking down into subunits. The compound, called SOM0226, works both to inhibit the beginning of the process of aggregating transthyretin amyloid and to help curb the progression of the disease.

Transthyretin-related hereditary amyloidosis is caused by a mutation in the transthyretin protein that cause toxic aggregates of amyloid fibers that build up in various organs, including the brain, kidney, nerves, eyes and myocardia. The variants studied are polyneuropathy and hereditary amyloid cardiomyopathy (which affects the peripheral nerves and the myocardia) and senile systemic amyloidosis (which also affects the myocardia). According to the study, SOM0226 is effective in treating these variants and, moreover, can also cross the blood-brain barrier. This means it could also be used to treat the variants that affect the central nervous system, which currently have no treatment.

The researchers believe that the drug could be available on the market for this new application within five years. In fact, a clinical trial has already been conducted with patients with the neuropathic variant, led by Dr. Josep Gámez, head of the VHIR Peripheral Nervous System group. According to the results of the trial, the compound stabilized 100% of the transthyretin protein present in the plasma of patients with the condition.


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