New quality control method to select effective MβCD for treating Neimann-Pick disease
New Rochelle, September 20, 2017-Researchers have developed a quality control method to evaluate the pharmacological activity and potential effectiveness of different preparations of the therapeutic agent methyl-β-cyclodextrin (MβCD). Distinct batches of MβCD produced by different commercial laboratories may be more or less effective in reducing the cholesterol that accumulates in the fibroblasts of patients with the lysosomal storage disorder Niemann-Pick disease type C1. This new set of methods for selecting an optimal cyclodextrin preparation is described in ASSAY and Drug Development Technologies, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. The article is available free on the ASSAY and Drug Development Technologies website.
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. 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.
About the Journal
ASSAY and Drug Development Technologies is an authoritative peer-reviewed journal published 10 times a year online with open access options and in print. Led by Editor-in-Chief Bruce Melancon, PhD, Managing Director of the Chemical Synthesis and Drug Discovery facility at the University of Notre Dame, the Journal provides early-stage screening techniques and tools that enable identification and optimization of novel targets and lead compounds for new drug development. Complete tables of content and a sample issue may be viewed on the ASSAY and Drug Development Technologies website.
About the Publisher
Mary Ann Liebert, Inc., publishers is a privately held, fully integrated media company known for establishing authoritative peer-reviewed journals in many promising areas of science and biomedical research, including Applied In Vitro Toxicology, OMICS: A Journal of Integrative Biology, and Genetic Testing and Molecular Biomarkers. Its biotechnology trade magazine, GEN (Genetic Engineering & Biotechnology News), was the first in its field and is today the industry's most widely read publication worldwide. A complete list of the firm's 80 journals, books, and newsmagazines is on the Mary Ann Liebert, Inc., publishers website. (http://www.liebertpub.com/)
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