Cyclodextrins as Therapeutics for Lysosomal Storage Disorders

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.

Potential Commercial Applications: Competitive Advantages:
  • 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 Stage:
  • Early-stage
  • Pre-clinical
  • In vitro data available


John McKew (NCATS)  ➽ more inventions...

Wei Zheng (NCATS)  ➽ more inventions...

Manju Swaroop (NCATS)  ➽ more inventions...

Miao Xu (NCATS)  ➽ more inventions...

Juan Marugan (NCATS)  ➽ more inventions...

Intellectual Property:
US Pat: - issued -
US Application No. 61/679,668 filed on 2012-08-03
PCT Application No. PCT/US2013/053527 filed on 2013-08-03
US Application No. 15/620,753 filed on 2017-06-12

Swaroop M, et al. PMID 22923786
Xu M, et al. PMID 23035117

Collaboration 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

Licensing Contact:
Suryanarayana Vepa, Ph.D.
Phone: 301-827-7181

OTT Reference No: E-050-2012-0
Updated: Nov 18, 2015