Toward a less expensive, more convenient treatment of Gaucher's disease

Prospects for eventual development of a less costly and more convenient treatment for Gaucher's disease have brightened with new research findings reported in the May issue of ACS Chemical Biology.

The existing treatment costs up to $750,000 annually for a single patient, continues for life, and must be given intravenously rather than by mouth. An oral treatment based on the new research could cut those costs by 100-fold.

Gaucher's (go-SHAYZ) disease is rare, but ranks as the most common lysosomal storage disorder and genetic disorder affecting Jewish people of Eastern European ancestry. Individuals with Gaucher's disease, which can be fatal, produce a defective form of GC, a critical enzyme that breaks down a fatty substance called glucosylceramide.

In the new report, scientists have confirmed experiments they reported initially in 2002 that "chemical chaperones" can partially correct the genetic defect responsible for most cases of Gaucher's disease. Like aspirin, penicillin and most other existing drugs, chemical chaperones are small molecules -- natural and synthetic substances with a low molecular weight.

Using patient-derived cell lines, researchers have extended those earlier studies to provide new insights into the defect and how chaperones correct it. The defect involves protein misfolding and prevents a key enzyme, glucocerebrosidase (GC), from reaching the location in cells where it normally functions. Jeffrey W. Kelly, Ph.D., of the Scripps Research Institute (TSRI) in La Jolla, Calif., headed the research team.

"Gaucher's disease patients can now be treated with enzyme replacement therapy," Kelly explained. "The hope is that this current strategy could be replaced with a small molecule chemical chaperone therapy wherein the cost would be reduced by at least 100-fold." Although enzyme replacement therapy is highly effective in treating Gaucher's disease, treatment often costs $100,000-$750,00

Contact: Michael Bernstein
American Chemical Society

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