Nobel Prize Winning Cholesterol Researchers Brown and Goldstein Enter Cyclodextrin Field Of Study

I wonder how many smart cholesterol researchers have Google Alerts set up on Brown and Goldstein Labs?  If they do they might want to read this post about the Nobel Prize winning labs release of a very important cholesterol paper involving cyclodextrin and its modulation of cellular cholesterol metabolism.

With their amazing new research work, Brown and Goldstein have shown that cyclodextrin (hydroxy propel beta cyclodextrin or HPBCD) reverses the block in cholesterol movement out of the lysosome in cultured cells of both the mouse and human Niemann Pick Type C fibroblasts.  The papers are here:

Brown and Goldstein Paper – Cyclodextrin overcomes deficient lysosome-to-endoplasmic reticulum transport in Niemann-Pick type C cells

Brown and Goldstein Supplemental Information on Niemann Pick Type C, Cholesterol and Cyclodextrin

For those of you who have no idea what I am talking about, this is very big news in the world of cholesterol research. It’s not every day that Nobel Prize winning cholesterol scientists publish papers on a non toxic sugar compound that is the main ingredient in Febreze air freshener and  fat free food products like butter.

How on earth can cyclodextrin be entering the cell and the lysosome and reversing the Niemann Pick Type C cholesterol genetic defect that is trying to kill our twin daughters?

In this paper, Brown and Goldstein say that the mode of action of cyclodextrin is still unknown. This is remarkable to me because the most famous cholesterol researchers on the planet have been unable to figure out this scientific puzzle. When this is all sorted out, I think a whole new wave of drugs will be developed with this compound that everyone has thought is benign!  I hope it happens fast because this compound could save my girls’ lives.

In conjunction with this news from Brown and Goldstein, Addi and Cassi’s lysosomes are shrinking in size since receiving their cyclodextrin IV infusions.  Measurements of Addi and Cassi’s lysosomes were done with a special assay developed at Oxford University. But is cyclodextrin really crossing the cell membrane and going into the cells in living people?   The previous research on cyclodextrin would say it acts outside the cell membrane.

Addi and Cassi are now six months into their cyclodextrin infusions which were approved by the FDA under “compassionate use” IND applications.  Addi and Cassi are doing quite well with no apparent side effects (except positive effects like better balance and coordination and increased concentration and awareness). But how can this be happening if cyclodextrin does not get through the blood brain barrier?

The data in the twins’ lysosomes  indicates what Brown and Goldstein has found in mice and in cell culture/ fibroblasts could be happening inside Addi and Cassi’s systems as well.  Our next step will be to apply for Orphan Drug Status with the FDA for Cyclodextrin after Thanksgiving.  I am also working with doctors on ways to get cyclodextrin across the blood brain barrier.  I am currently pursing a path to get Cyclodextrin into the CSF of Addi and Cassi.  I think if we can do this we have a better chance to save them from this horrible cholesterol disease that is destroying their brains and causing dementia.

We are going to apply for a FDA grant in February 2010. Cyclodextrin kills the HIV-AIDS virus too.  If we can get the goverment to keep and open mind and get more researchers studying this compound, the possibilities are endless.