Heart disease research

CAVADEX® developed by Cholrem Pty Ltd

The CAVADEX® molecule


CAVADEX® is a form of Hydroxypropyl beta cyclodextrin (HPßCD) that is comprised of seven sugar molecules bound together in a ring (cyclic oligosaccharide).

The ring-shaped, three-dimensional structure has a hydrophobic cavity in its center, which is capable of trapping cholesterol and lipids. It has been used extensively as an excipient in the food, deodorant, and drug industry for the past century, and it is generally recognized as safe (GRAS).

As an active ingredient, CAVADEX® entraps and removes intracellular cholesterol and lipids that can cause injury to the kidneys and other organs, including the brain and liver.

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Source: Lopez CA, de Vries AH, Marrink SJ (2011) Molecular Mechanism of Cyclodextrin Mediated Cholesterol Extraction. PLoS Comput Biol 7(3): e1002020. doi:10.1371/journal.pcbi.1002020.

CAVADEX® has shown promising pre-clinical results in kidney disease. Progression of kidney disease was prevented in mouse models of focal segmental glomerulosclerosis (FSGS) and Alport Syndrome (AS), and diabetic kidney disease was prevented or partially prevented in two pre-clinical studies3.

Promising pre-clinical results have also been demonstrated in non-renal indications, including Alzheimer’s disease, atherosclerosis, non-alcoholic fatty liver disease, and Stargardt’s disease. A successful Phase 1 trial has been completed with HPßCD in patients with Niemann Pick, Type C1, an orphan condition associated with lipid accumulation in the brain and liver.

CAVADEX® has successfully been administered to patients with advanced atherosclerosis and has shown to substantially reduce arterial plaque buildup in as little as 4 weeks of treatment.

CAVADEX® is not orally bioavailable due to its high molecular weight and resistance to digestive enzymes, and therefore must be administered intravenously or as an enema. Once administered, CAVADEX® is distributed to various tissues, with most accumulating in the kidney for excretion into the urine.
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Our first patient results after 4 weeks of CAVADEX® treatment

CAVADEX® melts away plaque


Our first patient had 12 intravenous CAVADEX® treatments over 4 weeks. The before and after angiogram images show CAVADEX® has removed a substantial amount of the patients plaque from his arteries allowing increased blood flow to the heart.




CAVADEX® melts away plaque and cholesterol

plaque reduced in just 4 weeks

A recent study by the University Hospital in Bonn, Germany discovered that cyclodextrin melts away cholesterol crystals (the main cause of arterial plaque that clogs arteries) and reduced existing plaque within just 4 weeks. The cyclodextrin bound to and dissolved the cholesterol crystals from the plaque of mice, leading to transport of the dissolved cholesterol away from the plaques. The same effects were seen in human plaque samples treated with cyclodextrin.

Article : Cyclodextrin promotes atherosclerosis regression via macrophage reprogramming

Improve Liver function

Another study by the University of Texas Southwestern Medical Center, Dallas, Texas, shows the treatment of Npc1-deficient mice slows cholesterol sequestration in major organs and improves liver function. This study shows that cyclodextrin is effective in mobilizing entrapped cholesterol in late stage NPC disease leading to improved liver function.

Article : Systemic administration of 2-hydroxypropyl-B-cyclodextrin to symptomatic Npc1-deficient mice slows cholesterol sequestration in the major organs and improves liver function.




Cyclodextrin Publications


About Cyclodextrins

Molecular mechanism of cyclodextrin mediated cholesterol extraction


Rationale for Therapeutic Use of Lipid Transport Mediators in Renal Disease

Local TNF causes NFATc1-dependent cholesterol-mediated podocyte injury.

Systemic and renal lipids in kidney disease development and progression 

Metabolism, energetics, and lipid biology in the podocyte - cellular cholesterol-mediated glomerular injury.

Lipid biology of the podocyte--new perspectives offer new opportunities.

Renal lipid metabolism and lipotoxicity. 


Cyclodextrins and Non-renal Diseases

Cyclodextrin promotes atherosclerosis regression via macrophage reprogramming.

Weekly Treatment of 2-Hydroxypropyl-β-cyclodextrin Improves Intracellular Cholesterol Levels in LDL Receptor Knockout Mice.

Beta cyclodextrins bind, stabilize, and remove lipofuscin bisretinoids from retinal pigment epithelium.

Neuroprotection by cyclodextrin in cell and mouse models of Alzheimer disease.


Cyclodextrins and Renal Disease

Cyclodextrin Protects Podocytes in Focal Segmental Glomerulosclerosis (FSGS)

Hydroxypropyl-β-cyclodextrin protects from kidney disease in experimental Alport syndrome and focal segmental glomerulosclerosis.

Cyclodextrin improves renal function in experimental alport syndrome.

Cyclodextrin protects podocytes in diabetic kidney disease.