Cytoclonal Pharmaceutics Files Patent for Production System That Offers Promise of Lower Cost Treatment for Gaucher's Disease
DALLAS--(BUSINESS WIRE)--May 7, 2001--Cytoclonal Pharmaceutics Inc. (NASDAQ: CYPH - news) announced today that it has filed a patent covering an enzyme production method that could help bring relief to Gaucher's Disease patients.
The new system includes features that have the potential to reduce the cost of treatment and make enzyme replacement therapy more available and affordable to the nearly 85% of patients who currently are not receiving treatment due to limited supplies and high cost.
Gaucher's Disease is a rare, inherited, potentially fatal disorder that results from a specific enzyme deficiency in the body (glucocerebrosidase), caused by a genetic mutation received from both parents. This disease can cause multi-organ dysfunction, including; enlarged liver and spleen, weakening of the skeleton and in some instances lung and kidney impairment.
Enzyme replacement therapy has become a viable treatment that has proven effective in many patients. Enzyme replacement therapy has successfully reversed many of the manifestations of the disorder. However, this form of therapy is very costly, estimated by the National Institutes of Health as $100,000 to $400,000 annually per patient. To optimize the return on its investment in this project, Cytoclonal is now exploring strategic alliances with other companies active in this field.
Cytoclonal's new production system has several benefits. First, the amount of the enzyme produced is higher than levels reported by other parties. Second, the enzyme is produced in cell culture and secreted into the production media, permitting more efficient purification. Third, the enzyme produced does not require the three additional costly steps to create the clinically effective form of the enzyme, which are used for the products presently on the market.
``It is very significant that the work done by our R&D group has resulted in unique, proprietary findings giving rise to this filing'' said Dr. Goode. ``This patent, when issued, will allow Cytoclonal to compete effectively in Gaucher's market, which is estimated at $500 million in 2000. But more importantly, this work offers the hope of bringing healing to those patients who presently have no hope for treatment because of the cost constraints of current therapies,'' continued Dr. Goode. In March 2001, Cytoclonal announced that it had completed work to identify a new expression system for glucocerebrosidase, to support enzyme replacement therapies.
There are three forms of Gaucher's Disease, Type 1,2 and 3 that are recognized. They are classified by age of onset (infantile, juvenile and adult) and the presence or absence of central nervous system involvement. Type 1 disease is the most commonly seen, affecting both children and adults. Type 2 disease usually presents in infancy with severe nervous system involvement. This form, in addition to spleen and bone marrow damage, causes seizures and damage to the central nervous system. Type 3 disease typically has mild central nervous system involvement and runs a more indolent course... |
