08/19/01 - Dr. Weinreb recently gave two presentations on Gaucher Disease
symptoms and developments in Trieste, Italy and Toronto, Canada. Both
presentations are now available on this website. Click here
to go directly to the page that allows access to these two presentations. Please
note that you must have Microsoft Powerpoint 97 or later installed in order to
view these presentations.
07/19/01 - NYU Study to Examine Investigational Therapy for Gaucher Disease; Enrollment Underway for Year-Long Trial Source
PR - PR Newswire
July 19, 2001
NEW YORK, July 19 /PRNewswire/ -- 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
withthe 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(TM)) 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) 263-8433or e-mail at Gregory.Pastores@med.nyu.edu.
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