Soliris (eculizumab) is a treatment developed and commercialized by Alexion Pharmaceuticals and approved by the U.S. Food and Drug Administration for the treatment of paroxysmal nocturnal hemoglobinuria, hemolytic uremic syndrome, and myasthenia gravis.

Research suggests that Soliris may also be potentially beneficial in patients with Gaucher disease.

How Soliris works

Gaucher disease is caused by mutations in the GBA gene providing instructions for making the beta-glucocerebrosidase enzyme. As a result, toxic levels of the substrate of this enzyme, glucocerebroside, accumulate in cells, especially immune cells such as monocytes and macrophages. These cells then accumulate in several organs such as the spleen, liver, lungs, and bone marrow causing damage.

In a recent report published in Nature, the authors demonstrated that the accumulation of glucocerebroside is regulated by abnormal activation of the complement protein C5.

The active ingredient in Soliris is a monoclonal antibody against C5, which is cleaved into C5a and C5b when activated. C5a binds to its receptor found on the surface of many cells and controls the production of an enzyme, UDP-glucose ceramide glucosyltransferase, which synthesizes glucocerebroside, therefore triggering the accumulation of glucocerebroside. C5b forms a complex with complement factor 9, and the C5-9 complex is responsible for triggering inflammation, destruction of red blood cells, and blood clot formation.

Soliris prevents the cleavage of C5 into C5a and C5b, giving it the potential to prevent both glucocerebroside accumulation in cells and chronic inflammation and related pathology in Gaucher disease.

Soliris in clinical trials 

The common underlying theme in all three diseases for which Soliris is currently approved is the abnormal activation of C5. As shown in a number of clinical trials, several of the symptoms of these diseases such as chronic inflammation and hemolysis are alleviated by Soliris treatment.

Although the effect of Soliris in Gaucher disease has not yet been tested in clinical trials, the study published in Nature showed that in a mouse model of Gaucher disease, the genetic or pharmacological inhibition of C5 cleavage prevented the accumulation of glucocerebroside in the cells, reduced the symptoms associated with the disease, and extended the lifespan of the animals.

The results of this study imply that Soliris could be a good therapeutic candidate for Gaucher disease, but its potential benefits need to be further studied before being tested in clinical trials.


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