Mount Sinai Partners with Industry to Repurpose Drug to Treat MPS Diseases

Rare Daily Staff

The Icahn School of Medicine at Mount Sinai said it has entered into exclusive licenses with Paradigm Biopharmaceuticals of Australia and ReqMed of Japan to develop the drug pentosan polysulfate sodium for the treatment of mucopolysaccharidosis (MPS), a group of 11 progressive and deadly lysosomal storage diseases.

MPS is a group of metabolic disorders caused by genetic defects that affect the skeletal system, skin, heart, brain, and other organ systems and can lead to disability or death. These conditions occur in about 3.5 out of 100,000 births.

Pentosan polysulfate sodium is normally used as an anti-inflammatory for bladder pain, but Calogera Simonaro, a research professor in the department of genetics and genomic sciences at the Icahn School of Medicine, demonstrated the potential use of the drug to treat MPS through several proof-of-concept studies carried out in animal models of MPS.

Current therapies that exist for these diseases, such as enzyme replacement therapy and bone marrow transplantation, are costly, require highly invasive procedures, and can have limited efficacy. These therapies can slow, but often do not halt, progression of the disease.

“By using a drug that is already approved, we should be able to fast-track [pentosan polysulfate sodium] to patients and address an important unmet medical need,” said Edward Schuchman, professor of genetics and genomic sciences at the Icahn School of Medicine and the department’s vice chair for research. “Using one drug for several different lysosomal storage diseases changes the approach currently used for treating these diseases.”

Paradigm will develop the drug in the United States, the European Union, and other territories, and ReqMed will develop the drug in Japan and other select Asian territories. The financial backing and infrastructure provided by these partnerships will facilitate blinded studies for regulatory approval. Drs. Schuchman and Simonaro believe pentosan polysulfate sodium can also be used to treat other lysosomal storage diseases, including Fabry and Gaucher diseases.

January 23, 2019
photo: Calogera Simonaro, a research professor in the Department of Genetics and Genomic Sciences at the Icahn School of Medicine