GM1 Cyclodextrin Pilot Study Initiated

The Cure GM1 Foundation is pleased to announce a pilot study in GM1 mice in collaboration with Dr. Miguel Sena-Esteves in the Department of Neurology at the University of Massachusetts Medical School (Worcester, MA) on the possible therapeutic impact of cyclodextrin on GM1-gangliosidosis.

Cyclodextrin has been studied extensively for another lysosomal storage disease called Niemann-Pick C (NPC) 1.  NPC is caused by the accumulation of cholesterol in the body, and specifically in the nervous system. In January 2016, Vtesse Pharmaceutical’s VTS-270, a specific cyclodextrin treatment, received the FDA’s breakthrough therapy designation for NPC.  In NPC, cyclodextrin is used to drive excess cholesterol out of the cell.  In GM1-gangliosidosis another lipid called GM1-ganglioside accumulates in the nervous system where it causes loss of neurons (neurodegeneration) over time.  A recent study in vitro suggests that cyclodextrin may help patient cells eliminate stored GM1 ganglioside 2.

The study being initiated in GM1 mice will test whether prolonged administration of cyclodextrin into the brain via the cerebral spinal fluid will reduce the amount of GM1 ganglioside in the nervous system. If the study reveals a benefit in GM1 mice, it may be possible to accelerate cyclodextrin as a GM1 therapy, given that human clinical trials are already ongoing for NPC 3, 4.

However, the results of this study have yet to be seen.  As the study progresses and the results are learned, updates will be provided.  Further studies may be required depending on the results.

Thank you for your ongoing support and your belief in the value of GM1 medical research and hope.

Literature cited:

1. Vite, C.H. et al. Intracisternal cyclodextrin prevents cerebellar dysfunction and Purkinje cell death in feline Niemann-Pick type C1 disease. Sci Transl Med 7, 276ra26 (2015)

2. Maeda, Y. et al. Effects of cyclodextrins on GM1-gangliosides in fibroblasts from GM1-gangliosidosis patients. J Pharm Pharmacol 67, 1133-42 (2015).

3. Maarup, T.J. et al. Intrathecal 2-hydroxypropyl-beta-cyclodextrin in a single patient with Niemann-Pick C1. Mol Genet Metab 116, 75-9 (2015).


“Study of 2-hydroxypropyl-β-cyclodextrin (VTS-270) to Treat Niemann-Pick Type C1 (NPC1) Disease” ( Identifier: NCT02534844)

GM1 Cyclodextrin Pilot Study Initiated
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