New Quality Control Method Treats Neimann-Pick Disease

Wei Zheng, PhD, National Center for Advancing Translational Sciences, National Institutes of Health (Bethesda, MD) and coauthors from the NIH, Poochon Scientific (Frederick, MD), and Washington University School of Medicine (St. Louis, MO), developed an analytical method that they used to characterize three preparations of MβCD.

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The researchers found differences in average molecular weight and side chain methylation in MβCD samples from different vendors and batches.

They discuss the potential impact of these differences on the ability of MβCD to reduce lysosomal cholesterol accumulation in the article entitled "Analytical Characterization of Methyl-β-Cyclodextrin for Pharmacological Activity to Reduce Lysosomal Cholesterol Accumulation in Niemann-Pick Disease Type C1 Cells."

"This scientific disclosure highlights the importance of consistency in batch production of MβCD. Without this technology, there would be little promise to develop a clinical treatment for these patients, many of whom are young children," says ASSAY and Drug Development Technologies Editor-in-Chief Bruce Melancon, PhD, Managing Director of the Chemical Synthesis and Drug Discovery facility at the University of Notre Dame.



Source: Eurekalert