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Cyclodextrins as Therapeutics for Lysosomal Storage Disorders

Posted Nov 05 2012 7:00pm

Description of Invention:

Cyclodextrins (CD), alone or in combination with other agents (e.g., vitamin E), as therapeutics for the treatment of lysosomal storage disorders (LSDs) caused by the accumulation of non-cholesterol lipids.

CDs are sugar molecules in a ring form. The alpha-CD (6 sugars), beta-CD (7 sugars) and gamma-CD (8 sugars) are commonly used cyclodextrins. The hydroxypropyl-beta cyclodextrin (HPbCD) has been approved for pharmaceutical use. Recent reports show that beta-cyclodextrin including HPbCD and beta-methyl-cyclodextrin reduced cholesterol accumulation and neuronal cell loss in the mouse model of NPC1 disease.

NCATS investigators found that CD (alpha-, beta- and gamma-CDs) increased intracellular Ca2+ and lysosomal exocytosis in both wild type cells and cells with Wolman disease, and reduced the size of enlarged lysosomes in six patient cell lines with LSDs. Further, CD in combination with tocopherol synergistically/additively reduced cholesterol accumulation in cells of NPC and Wolman diseases. Based on these results, they propose treatment of LSDs with cyclodextrins (such as alpha and gamma forms) alone or in combination with Vitamin E and its analogues for better efficacy and less side effects.

  • treatment of lysosomal storage diseases
  • treatment of disorders caused by accumulation of non-cholesterol lipids

  • use of cyclodextrins in combination with vitamin-E (e.g., delta-tocopherol) provides additive therapeutic effect
  • less side effects than cyclodextrin only or vitamin E only for LSDs because of reduced doses for both compounds in combination

Development Status:
  • Early-stage
  • Pre-clinical
  • In vitro data available

Wei Zheng (NCATS)
Juan J Marugan (NCATS)
John C McKew (NCATS)
Miao Xu (NHGRI)
Manju Swaroop (NHGRI)

Patent Status:
HHS, Reference No. E-050-2012/0
US, Application No. 61/679,668 filed 03 Aug 2012

Related Technologies:
PCT, Application No. PCT/US2011/044590 filed 19 Jul 2011, Reference No. E-294-2009/0 , entitled" “Use of Delta Tocopherol for the Treatment of Lysosomal Storage Disorders” (Wei Zheng et al., NCATS)
US, Application No. 61/365,712 filed 19 Jul 2010, Reference No. E-294-2009/0

Collaborative Research Opportunity:
The National Center for Advancing Translational Sciences is seeking statements of capability or interest from parties interested in collaborative research to further develop, evaluate or commercialize this technology. For collaboration opportunities, please contact Dr. Juan Marugan at .

For Licensing Information Please Contact:
Suryanarayana Vepa Ph.D.
NIH Office of Technology Transfer
6011 Executive Blvd. Suite 325,
Rockville, MD 20852
United States
Phone: 301-435-5020
Fax: 301-402-0220

Ref No: 2494

Updated: 11/2012

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