Researchers See Potential in Treatment for Gaucher Disease in Emerging Therapeutic Approach

Researchers See Potential in Treatment for Gaucher Disease in Emerging Therapeutic Approach

Increased interest in the developing field of pharmacological chaperones as a therapy in certain lysosomal conditions such as Gaucher disease prompted a study which, scientists say, confirms the potential of the prodrug concept in pharmacological chaperone therapeutics.

An international group of multidisciplinary researchers conducted a study that assessed the chaperone activity when combined with an innovative iminosugar prodrug (a medication that becomes active once ingested) and found the iminosugar had no negative impact on the therapeutic effect of the pharmacological chaperone.

The study, “Investigation of original multivalent iminosugars as pharmacological chaperones for the treatment of Gaucher disease,” was published in the current issue of Carbohydrate Research

Pharmacological chaperones are an emerging approach to treat the cause of misfolded proteins that are the hallmark of lysosomal storage diseases such as Gaucher disease. The therapeutic mechanism of action that makes this approach so enticing to researchers is their ability to stabilize the entire protein structure by glue-like interactions of small molecules that are able to hold part of the protein structure together.

To more thoroughly understand the concept of pharmacological chaperones, you can watch this video provided by Amicus pharmaceuticals.

Lab-based and clinical studies have shown that pharmacological chaperones may have advantages over current drug treatments, such as enzyme replacement therapy alone, for their effectiveness and ease of oral administration.

Researchers in this study aimed to further explore the research that had already been completed in the field of pharmacological chaperones and assess whether particular iminosugars — sugar rings commonly found in plants and are considered a primary component of their medicinal properties — had an impact on the therapeutic effect of these chaperones.

The authors used experimental methods such as immunofluorescence staining used to evaluate cellular protein concentrations,  cytotoxic assays used to detect cell viability, and cell culture to conduct the study.

After analysis of the experimental results, researchers concluded that the use of the iminosugar prodrug does have potential for use in pharmacological chaperone therapeutics.

“In conclusion, we have synthesized and evaluated new multivalent iminosugars, some of them conjugated with a morpholine moiety and/or designed as prodrugs for the treatment of Gaucher disease,” the authors wrote. “Our study confirms the potentiality of the prodrug concept but also shows that the efficiency of this approach depends mainly on the nature of the multivalent scaffold used.”

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