Treatment For Gaucher Disease From Protalix

Filed in archive Neurological Disorders , Studies , Treatment on July 4, 2009

Gaucher disease is a hereditary, genetic disorder with severe and debilitating symptoms. It is an inherited metabolic disorder in which harmful quantities of a fatty substance called glucocerebroside accumulate in the spleen, liver, lungs, bone marrow, and sometimes in the brain.

Now, a biopharmaceutical company focused on the development and commercialization of recombinant therapeutic proteins - Protalix - is currently producing proteins for the treatment of Gaucher disease using their proprietary plant-cell based expression technology.

Protalix has currently created over a dozen different proteins to treat other genetic disorders. Their plant-cell based technology enables a better gene uptake and longer half-life for their cells, creating a more potent treatment which may potentially cut in half the number of hospital visits a Gaucher patient must undergo for treatment.

Protalix is currently in phase III with this drug (currently referred to as prGCD), having completed trial enrollment in December 2008. The company expects to report results of the clinical trial in the next six months.

Additionally, because of the Genzyme virus, there is a possibility that the FDA will expedite the approval process for Protalix in order to get the drug to market as soon as possible. Through their clinical trials, there are currently a number Gaucher patients who have been successfully treated using Protalix's therapeutic proteins for over 2 years.

Read more about Gaucher Disease from the NINDS-NIH Fact Sheet.