An Emerging New Enzyme Treatment for Type 1 Gaucher Disease


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In the last edition of Gauchers News, Prof Ari Zimran, Director of the Gaucher Clinic at Shaare Zedek Medical Center in Israel reported a new enzyme preparation for patients with Type 1 Gaucher disease, produced by Shire Genetic Therapies. Prof Zimran provides a further update on the results at nine months.


Shire Human Genetic Therapies Inc has recently completed a 9-month Phase I/II trial with Gene-Activated human glucocerebrosidase (GAGCB) at the Gaucher Clinic at Shaare Zedek Medical Center.

GA-GCB is human glucocerebrosidase which is produced in a continuous human cell line using proprietary Gene-Activated technology. Unlike Cerezyme, GAGCB has an identical amino acid sequence to the naturally occurring human enzyme, which may be an advantage. This innovative technology has been used to develop an alternative enzyme replacement therapeutic option for patients with Gaucher disease.

'The purpose of the Phase I/II study was to assess the safety and efficacy of GA-GCB in adult patients with Type 1 Gaucher disease. Twelve patients received GA-GCB every other week for 9 months. Of 12 patients treated, 11 patients completed the study.

There were no drug-related serious adverse events and no patient discontinued participation because of adverse events. Infusion related reactions were limited, mild and transient. None of the treated patients developed anti-GA-GCB antibodies.

The results with GA-GCB were very impressive. Notably, the GA-GCB study did not allow children to enrol, who typically respond better to treatment, yet similar results were obtained to the trial of Ceredase, where eight out of 12 patients were younger than 18 years of age. In the GA-GCB trial, there were significant increases in hemoglobin (on average 2.24g/dL; mean increase of 19.2% from baseline), in platelet counts (on average 40,600/mm3; mean increase of 67.6% from baseline) and also significant decreases in spleen and liver volume (by 49.5% and 18.2% from baseline, respectively).

Similarly, there were significant decreases in the biomarkers chitotriosidase (by 74.2%)and CCL18 (by 57.1%). Improvements in some parameters were apparent as early as three months after the start of the therapy.

These results suggest that Gene-Activated humanglucocerebrosidase (GA-GCB), holds promise as a new enzyme replacement therapy for Gaucher disease and warrants further evaluation in clinical studies in a broader population of patients.


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Source: Gauchers News June 2006.
© Copyright Gauchers Association 2006