Public Release: 

NYU study to examine investigational therapy for Gaucher disease

NYU Langone Medical Center / New York University School of Medicine

Enrollment underway for year-long trial

New York, NY, July 19, 2001- Researchers at NYU Medical Center have initiated a trial to study the efficacy of an investigational oral treatment for Type 1 Gaucher (go-SHAY) disease, an incurable rare genetic disorder affecting approximately 8,500 people in the U.S. The study is being conducted in the Gaucher clinic at NYU, one of the leading centers in the U.S. for the diagnosis and treatment of this disease.

"This drug has shown promise in earlier clinical evaluations conducted in Europe and Israel," said Dr. Gregory M. Pastores, Co-director of the Neurogenetics Unit at NYU School of Medicine, the study's principal investigator. "Our study was designed to provide additional information on how this new approach to managing Gaucher disease can fit in with the current treatment paradigm."

Type 1 Gaucher disease is the most common genetic disease affecting Jewish people of Eastern European (Ashkenazi Jewish) ancestry and is caused by an inherited enzyme deficiency that prevents the body from breaking down a lipid (or fat molecule) called glucocerebroside. This results in an enlargement of the liver and/or spleen, where the lipids accumulate, as well as blood-related disorders and severe bone disease. There are three known types of Gaucher disease, with Types 2 and 3 being extremely rare.

OGT-918 (Vevesca™) is an oral medication that works by inhibiting the production of the lipids associated with the disease. In contrast, the only currently approved treatment for Gaucher disease, enzyme replacement therapy (ERT), involves an intravenous (IV) infusion over the course of one to two hours usually twice per month.

OGT-918 has been studied in a total of 80 patients both in combination with ERT and as monotherapy. Oxford GlycoSciences Plc, who is developing the drug, will submit these data to the U.S. Food and Drug Administration (FDA) for marketing approval this year. The drug has received "fast track" status to expedite its review.

"The current therapy for Gaucher disease works very well for many people, but there are some who could benefit significantly from a second option," explained Robin Ely Berman, M.D., President, CEO and Medical Director of the National Gaucher Foundation (NGF).

The open-label trial, being conducted by Dr. Pastores and Dr. Edwin H. Kolodny, Chairman of the Department of Neurology at NYU, will involve 14 adult patients who have never received previous treatment with ERT or have stopped therapy for more than three months before enrollment in the study. Patients will take the medication three times a day for a period of one year and frequently be monitored to ensure the drug is safely and effectively managing their disease.


People with Gaucher disease who are interested in learning more about the trial should contact Dr. Gregory Pastores by phone at 212-263-8433 or e-mail at

About New York University Medical Center NYU Medical Center is a leader in patient care, physician education and scientific research. The Medical Center is internationally renowned for excellence in areas including women's health, cardiology and cardiac surgery, minimally invasive surgery, genetics, infertility, obstetrics and gynecology. All of these areas have benefited from groundbreaking research that has been applied to clinical care.

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