How Cyclodextrin Solution Is Made For Addi and Cassi’s Intravenous Cyclodextrin Therapy

A number of people from around the world who have children diagnosed with the fatal cholesterol disease called Niemann Pick Type C  have asked me how our we prepare our cyclodextrin solution for Addi and Cassi’s bi-weekly IV infusions of the compound.

All hydroxy propel beta cyclodextrin (HPBCD) is not the same. It’s very important that you make sure that the cyclodextrin that is purchased is endotoxin controlled and does not have propylene glycol in it or other impurities. Essentially propylene glycol is anti-freeze – not exactly what you want to put into kids!

We purchase our cyclodextrin from CTD, Inc., which is located in High Springs, Florida. The owner of CTD is extremely knowledgable when it comes to cyclodextrins and sells many different types of cyclodextrin products.  The cost for his HPBCD is about $5 USD per gram.

I am still working to try and make a solution that can be bottled and sent worldwide. As you can imagine, this takes time since we have no pharmaceutical company helping us with this effort.

When the girls receive their infusions, there is a basic filter that the cyclodextrin funnels through (this is simply as a precaution).  The filter is attached to the infusion line and as the cyclodextrin drips through the plastic line it goes through this little blue filter. I will try and find out the name of the filter we use.

We are currently looking at different ways to get cyclodextrin into the body — possibly through regular subcutaneous injections as well as intrathecal methods to get cyclodextrin directly into the brain through the spinal canal.  We are actively pursuing both ideas now on how this would be possible for Addi and Cassi.

I continue to be very happy with how the infusions are going.  The most difficult part so far has not been giving them cyclodextrin — it is being at the hospital for 2x a week all day long just sitting there watching cartoons. I am hoping the FDA will approve us to do infusions in a home setting (hopefully at night when they are sleeping) as we have not experienced any side effects.  We are at 2500 (yes, 2500) mg per Kg BW and the infusion lasts 8 hours.

Addi and Cassi seem to be a lot more alert and aware and have better balance and head control!   I pray we can speed up research on cyclodextrin and determine the mechanism for how it’s working.

For more information on Addi and Cassi’s cyclodexrin protocol, you can view our FDA compassionate use filings here.