Study on pentosan polysulfate / Elmiron® published in PLOS One

Pentosan Polysulfate: A Novel Therapy for the Mucopolysaccharidoses

January 2013: Dr. Edward Schuchman, et al, published an article in PLOS One. The article presents findings in MPS VI rats treated with pentosan polysulfate (PPS) and can be accessed in its entirety here.


The National MPS Society spoke with Drs. Simonaro and Schuchman who emphasized that they are focusing on safety and dosage trials of Elmiron® in animal models. Statement from Drs. Simonaro and Schuchman:

We are excited by the potential of pentosan polysulfate (PPS) for the treatment of MPS and other lysosomal storage disorders. Our primary goal remains continuing to evaluate the safety and effectiveness of this drug in MPS animal models and the hopeful initiation of clinical trials in MPS patients. Although PPS is approved by the FDA for use in adult women with “painful bladder syndrome” (interstitial cystitis) under the trade name Elmiron®, the appropriate dosing and formulation for MPS remains to be determined. In addition, the safety of the drug has not been studied in children. We will continue to keep the MPS patient community aware of these efforts through updates such as these, and thank you for your interest in our research.

Drs. Simonaro and Schuchman
Department of Genetics & Genomic Sciences
Icahn School of Medicine at Mount Sinai, NY

Update: March 2013 – Their work in MPS VI rats suggests that PPS “could be a simple and effective therapy for MPS that might provide significant clinical benefits alone and in combination with other therapies.”  The full text of the article and a statement by the authors is on our website in the Research section, under “research news”.  The National MPS Society, along with other patient advocacy groups, is in discussion with Janssen Research & Development, LLC, a research branch of Johnson & Johnson which licenses the FDA approved PPS, Elmiron®.  Janssen is establishing a group of MPS experts to begin discussions of the path forward toward a clinical trial.  Janssen has noted that their primary goal is to support patients and that they are open to a clinical trial.

Update: June 2013 – For information on the outcome of the expert discussions, please click here.

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