Researchers Hope to Repurpose Bladder Drug for Mucopolysaccharidosis

According to an article from R&D, researchers at the New York City-based Icahn School of Medicine at Mount Sinai have entered an agreement with two pharmaceutical companies to aid in repurposing the drug pentosan polysulfate sodium for use in patients with mucopolysaccharidosis.

About Mucopolysaccharidosis

Mucopolysaccharidosis (or MPS) is a group of inherited conditions that describe when the body fails to properly break down mucopolysaccharides (also known as glycosaminoglycans) – long, unbranched sugar molecules that are found throughout the body but especially in mucous and synovial fluids.

The conditions are highly rare, affecting only about four in every 100,000 births.

When the body fails to adequately break down these lengthy sugar molecules, they build up in cells, blood, and connective tissue. Due to the cumulative nature of MPS, the condition often isn’t diagnosed until much later in life. Most with MPS are born apparently healthy and enjoy relatively normal development until mucopolysaccharide levels build throughout the body. When that happens, physical or sometimes mental declines can occur. It can lead to changes in appearance, as well as in organ function and cognitive ability.

Mucopolysaccharidosis has some 11 recognized forms, differentiated by their underlying genetic cause and respective treatment methods.

Drug Repurposing

Drug repurposing is a hot topic in the medical world. Simply put, drug repositioning is a strategy where a drug approved for one condition is adapted for use in another.

Drug synthesis is a long and arduous process – costing millions of dollars and up to 17 years of research and development to yield even a single new pharmaceutical. Drug repurposing is comparatively cheap, and can result in new approvals in as little as 3 to 12 years. Those interested in a more in-depth look at the technique can read about it here.

Pentosan polysulfate sodium is the drug scientists at the Icahn School are teaming up to repurpose. It’s normally administered as an anti-inflammatory for bladder pain – but the researchers determined in an animal study that the drug was also capable of improving the condition of connective tissues affected by MPS.

Scientists hope that the already existing drug could provide a fast-tracked solution to a condition in dire need of treatments. Paradigm Biopharmaceuticals Ltd. will lead the repurposing and development efforts in the west, while ReqMed Company, Ltd. develops in Japan and Asia.

Animal tests are a very early stage in clinical testing, and are by no means an indicator of effectiveness in humans. Further study will be required before the drug is approved for use in MPS patients, and only then will it officially be “repurposed.”


How might drug repurposing be helpful for patients with no existing treatment options? Do you think this benefits consumers, pharmaceutical companies, or both? Share your thoughts with Patient Worthy!

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