Posted by Cyclo Therapeutics, Inc., a clinical stage biotechnology company dedicated to developing life-changing medicines through science and innovation for patients and families living with diseases, announced it has submitted its initial investigational new drug (“IND”) application with the U.S. Food and Drug Administration (“FDA”) for a Phase 2 study of Trappsol® Cyclo™ for the treatment of early Alzheimer’s Disease (AD).
Trappsol® Cyclo™ is a proprietary formulation of hydroxypropyl beta cyclodextrin and in multiple clinical studies has shown encouraging results to effectively manage the transportation of cholesterol. Many of the known risk factors for AD are associated with cholesterol metabolism. Cholesterol imbalance in AD patients is well known, and significant research exists suggesting these imbalances are responsible for Aβ and tau accumulation. Furthermore, neurons, because of their high metabolic demands, experience an increased level of oxidative stress. Oxidative stress has also been linked to abnormal cholesterol accumulation and processing. AD shares features with NPC-1, a neurovisceral, genetic disease in which cholesterol accumulates in lysosomes, including progressive decline in cognitive ability, amyloid beta plaques in the CNS, and increased levels of tau in the cerebrospinal fluid (CSF). Cyclo Therapeutics is currently testing the same investigational Trappsol® Cyclo™ drug in clinical trials for the treatment of Niemann-Pick Disease Type C1 (NPC-1). Taking the place of the defective NPC-1 protein, Trappsol® Cyclo™, with its cyclic structure, facilitates the transport of accumulated cholesterol out of cellular lysosomes so it can be further processed and excreted out of cells.