Technology ID

Cyclodextrins as Therapeutics for Lysosomal Storage Disorders

Lead Inventor
McKew, John (National Human Genome Research Institute (NIH/NHGRI))
Zheng, Wei (National Human Genome Research Institute (NIH/NHGRI))
Xu, Miao (National Human Genome Research Institute (NIH/NHGRI))
Swaroop, Manju (National Human Genome Research Institute (NIH/NHGRI))
Marugan, Juan (National Human Genome Research Institute (NIH/NHGRI))
Research Materials
Therapeutic Areas
Reproductive Health
Development Status
  • Early-stage
  • Pre-clinical
  • In vitro data available
Lead IC
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.
Commercial Applications
  • treatment of lysosomal storage diseases
  • treatment of disorders caused by accumulation of non-cholesterol lipids
Competitive Advantages
  • 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
Licensing Contact:
Vepa, Suryanarayana