Nature Study Suggests New Therapy for Gaucher Disease

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    Scientists propose in Nature blocking a molecule that drives inflammation and organ damage in Gaucher and maybe other lysosomal storage diseases as a possible treatment with fewer risks and lower costs than current therapies.
    An international research team led by Cincinnati Children’s Hospital Medical Center, which also included investigators from the University of Lübeck in Germany, report their data Feb. 22. The study was conducted in mouse models of lysosomal storage disease and in cells from blood samples donated by people with Gaucher disease.
    Current treatments for Gaucher and other lysosomal storage diseases (LSDs) include enzyme replacement therapy or substrate reduction therapy. These break down or prevent the accumulation of certain fatty molecules and other waste particles that clog cells to cause inflammation, cell and organ damage and, in some cases, death. People with LSDs lack enzymes that break down used-up proteins and other spent particles, preventing their cells from shedding these waste materials and functioning normally.
    Individually, the 50 genetic diseases characterized as LSDs are considered rare. But collectively they have a frequency of one in 8,000 births, making LSDs a major challenge for the healthcare system, according to information from the National Institutes of Health. Study authors stress there is a need for new therapies.
    “Current enzyme replacement and substrate reduction therapies are expensive and still associated with inflammation, increased risk of malignancies and Parkinson’s disease,” says Manoj Pandey, PhD, study first author and a scientist in the Division of Human Genetics at Cincinnati Children’s. “We suggest that targeting a molecule called C5aR1 may serve as a viable treatment option for patients with Gaucher disease and possibly other LSDs.”
    Pandey is co-corresponding author on the paper along with Jörg Köhl, MD, director of the Institute for Systemic Inflammation Research at the University of Lübeck, and adjunct professor in the Division of Immunobiology at Cincinnati Children’s.

    Source: Cincinnati Children’s

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