Potential new treatment for Gaucher disease developed by scientists at Scripps Research Institute

us drawbacks.

The enzyme has to be infused intravenously or through a surgically implanted catheter--usually in a doctor's office--a process that takes several hours and must be repeated every one or two weeks. Enzyme replacement therapy is also extremely expensive, costing between $100,000 and $750,000 per year per patient. And the therapy is not effective at treating neurological complications of Gaucher disease.

Now Kelly, Beutler, and their colleagues at TSRI report positive results on a radically different approach that may address some of the problems with enzyme replacement therapy. Rather than replacing the mutant enzyme, says Kelly, "We use a small molecule to partially correct [its] imperfections."

Their small molecule "chaperone" stabilizes the mutant beta-glucosidase enzyme, helping it to fold properly and find its way to the lysosome, where it can degrade the fatty substance.

The chaperone targets the most common Gaucher mutation, which is referred to as "1226G" or "N370S." Virtually every Jewish patient with Gaucher disease has this beta-glucosidase mutation.

A therapy based on these chaperones has the potential to be much more convenient and less costly than enzyme replacement, because small molecules could be taken orally and would be cheaper to mass-produce than whole enzymes. In addition, small molecules can be found that cross the blood-brain barrier, and perhaps address the neurological complications of Gaucher disease, if they are caused by mutants that respond to chaperone therapy.

The article, "Chemical chaperones increase the cellular activity of N370S beta-glucosidase: A therapeutic strategy for Gaucher disease" was authored by Anu R. Sawkar, Wei-Chieh Cheng, Ernest Beutler, Chi-Huey Wong, William E. Balch, and Jeffrey W. Kelly, and appears in the online edition of the journal Proceedings of the National Academy of Sciences the week of November 4, 2002. The article will appear in print la

Contact: Jason Bardi
Scripps Research Institute

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