Plant-Derived Glucocerebrosidase for Gaucher Disease
Author Information
Author(s): Aviezer David, Brill-Almon Einat, Shaaltiel Yoseph, Hashmueli Sharon, Bartfeld Daniel, Mizrachi Sarah, Liberman Yael, Freeman Arnold, Zimran Ari, Galun Eithan
Primary Institution: Protalix Biotherapeutics, Science Park, Carmiel, Israel
Hypothesis
The study evaluates the safety and pharmacokinetics of a novel human recombinant glucocerebrosidase enzyme expressed in transformed plant cells.
Conclusion
The study found that the plant-derived glucocerebrosidase enzyme is safe and well-tolerated in both primates and human volunteers.
Supporting Evidence
- The enzyme was well tolerated with no serious adverse events reported.
- No anti-prGCD antibodies were detected in the volunteers.
- The pharmacokinetic profile showed a prolonged half-life compared to the commercial enzyme.
Takeaway
Researchers tested a new medicine made from plants to help people with a disease called Gaucher disease, and it was found to be safe.
Methodology
The study involved safety and pharmacokinetics assessments in primates and a Phase I clinical trial with healthy volunteers receiving escalating doses of the enzyme.
Limitations
The study was conducted with a small number of participants and for a short duration.
Participant Demographics
Six healthy volunteers, 3 males and 3 females, aged 19-36, all Caucasian.
Digital Object Identifier (DOI)
Want to read the original?
Access the complete publication on the publisher's website