Protalix BioTherapeutics, an Israeli biotech company based in Carmiel has developed a new, cheaper enzyme replacement therapy for patients with Gaucher disease.

Many have never heard of Gaucher disease. It is a lysosomal storage disorder where patients are deficient in an enzyme that breaks down specific fat molecules, allowing fat cells to accumulate in organs such as the liver, brain, spleen, lungs, and bone marrow.

The condition can be not only painful, but lethal.

Gaucher disease afflicts approximately 10,000 people worldwide. A disproportionate number of these sufferers are Jews of Eastern European ancestry.

The National Gaucher Foundation says around 1 in 100 people in the general U.S. population is a carrier for Type 1 Gaucher's disease -- a prevalence of 1 in 40,000. Among Ashkenazi Jews the rate of carriers is considerably higher, at roughly 1 in 15.

Protalix says the current treatment for Gaucher disease, also an enzyme replacement therapy, was used to treat more than 5,200 patients in 2008. Patients receive regular transfusions of the deficient enzyme, usually for their lifetime. The average annual cost for treatment is approximately $250,000 per patient.

The new system developed by Protalix to create the necessary enzymes is called ProCellEx. It expresses a “recombinant Glucocerebrosidase enzyme,” known as prGCD, through the use of plant cells, more specifically carrot cells, as opposed to the current method of mammalian cell-expressed enzymes which are produced in vats. In essence, Protalix makes plants become the drug factory.

The concept of plant-made pharmaceuticals has been around for about twenty years, but few companies pursued this field of biotechnology due to past struggles. Protalix seems to have figured it out.

The company explains that the ProCellEx system produces the enzymes in “sterilized, large flexible plastic containers used for culturing and harvesting cells in consecutive cycles,” where they are more easily extracted and refined. Protalix expects their method to allow for cheaper production of the enzymes, significantly cutting costs for one of the world’s most expensive disease treatments.

Protalix says their “plant-based systems are capable of producing high quantities of complex proteins at a dramatically lower cost than mammalian cell culture systems. Such plant-based systems offer greater scalability than alternative systems and almost unlimited capacity, while incurring relatively small increases in cost as production ramps.” Additionally, the company says, “there are no known risks of contamination from animal, bacterial, or viral pathogens in a plant-based system,” which further reduces risk to patients.

The scientists at Protalix are not the only ones embracing the safety of the ProCellEx system and enzyme. The company says it has “reached an agreement with the United States Food and Drug Administration (FDA) on the final design of the pivotal Phase III clinical trial for prGCD through the FDA's Special Protocol Assessment process.”

Protalix estimates that results for the clinical trial will be available “in the second half of 2009.” The company expects to submit a New Drug Application (NDA) for prGCD to the FDA and other comparable regulatory agencies in other countries in the fourth quarter of 2009.

Protalix is pursuing other ventures as well. The company says that it is “developing other recombinant therapeutic proteins to be expressed by the ProCellEx protein expression system” in “specialty disease and therapeutic market segments.”

The company says one enzyme in early development is PRX-102, a therapeutic enzyme for the treatment of Fabry disease. Fabry disease is another rare genetic lysosomal storage disorder in humans. The condition is characterized by accumulation of lipids in the cells of the kidneys, heart, blood vessels, and other organs.

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