Crossing The Blood Brain Barrier – Does Cyclodextrin Make Its Way Into The Brain?

Dr. David Begley, one of the world’s leading blood brain barrier experts at Kings College London is working on a research project we are currently funding on cyclodextrin and the blood brain barrier. We want to answer the following question. Does hydroxy propel beta cyclodextrin (HPBCD) cross the blood brain barrier? Since less than 5% of drugs (made up of very small molecules) are able to cross the barrier and cyclodextrin is not considered a small molecule nor a drug, the possibility of cyclodextrin crossing into the brain would be remarkable.

Addi and Cassi, my 5 year old identical twins, who have a cellular cholesterol metabolism disease called Niemann Pick Type C (often referred to as the Childhood Alzheimer’s) and are being treated with infusions of the sugar compound cyclodextrin. When we started Addi and Cassi’s first few rounds of cyclodextrin infusions three weeks ago, I honestly did not expect to see much of a change because we started with a low dose. I certainly did not think my girls would start saying words again.  

To put this story in context, prior to starting the cyclodextrin infusions, Addi and Cassi had both lost their ability to talk. Addi was still trying to talk by making grunting sounds and came out with an occasional word here and there and Cassi was virtually mute. However, since starting the cyclodextrin infusions, Addi has started repeating sentences again. This type of language is called echolalia and it’s something Addi did before she stopped talking. Cassi has become more vocal as well.

In the last 36 hours, Addi has repeated the following: Good morning, That’s great, That feels better, Rosie and Gilbert (characters from a cartoon), Let’s go walking, Let’s do it, Daddy’s here, Alright, Bye Tia (to our nurse), There’s Martha (in reference to our nanny), No, Me, We, More, Where’s Addison, That’s mine, I can do it, Let’s put them in the garbage can, Open, I Love You and Let’s have breakfast. Cassi has only managed a few words over the past few weeks but is making a lot more sounds with different pitches instead of a single low hum.  (Note: Cassi has never talked as much as Addi and her speech was lost a few months before Addi’s).

I can’t express in words what it’s like to hear your child talk again. When my husband walked into the hospital room and Addi repeated ‘Daddy’s here" his eyes welled up with tears. Our nanny Martha has been with our girls since birth and it’s been six months since Addi has said her name. Yesterday, Addi clearly said "Martha" twice. In addition to the spike in speech, the girls also seem happier, appear to have a slight improvement in head control (when rested) and their eye contact appears better.

I have noticed a few more "stare off" spells with Addi (possibly absent seizures?) but I am not sure if these have increased or if I am just paying more attention and noticing them more. The girls have experienced speech improvements previously when starting antibiotics (Amoxicillin and Septra). But the improvements did not last. There seemed to be a honeymoon period after starting the antibiotics and then the improvements stopped after 3-4 weeks. I have never received an answer from a doctor or researcher as to why antibiotics had a short term benefit for my girls, but they did.

To everyone’s delight, Addi and Cassi are experiencing neurological improvements on cyclodextrin. Since they are identical twins and are both improving, this leads me to the conclusion that cyclodextrin (HPBCD) is having some sort of effect on cellular cholesterol accumulation — either it’s crossing the BBB or somehow creating a siphon effect in the body and pulling cholesterol out of the brain? 

Dr. Begley will need to explain to the research world what cyclodextrin is actually doing and I can’t wait for his research work to finish. Cyclodextrin is very exciting and promising, not only for Addi and Cassi and other kids impacted with Niemann Pick Type C but for the scientific community in general.   I am starting to wonder what cyclodextrin could do for people suffering from atherosclerosis and if it would help eliminate the build up of plaques in the arteries?  Also, several lines of evidence have implicated a role for cholesterol in Alzheimer’s disease. I urge scientists working on diseases involving cholesterol pathways to spin up experiments with cyclodextrin (HPBCD).