May 2nd, 2009
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.
Less than 5% of Drugs can cross the Blood Brain Barrier: Click to Enlarge
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) right away.
Filed under Blood Brain Barrier, Cholesterol, Cyclodextrin, Niemann Pick Type C | Comment (0)
April 1st, 2009
Millions of children in Africa like Rosine are needlessly contracting HIV/AIDS when an inexpensive and non toxic sugar compound called Cyclodextrin (or HPBCD) kills the HIV/AIDS virus and could literally stop the spread of this horrible deadly disease from mother to child (not to mention adults!). Now that the world is going to find out how important the Niemann Pick Type C GENE and proteins (located on Chromosome 18) are to the HIV/AIDS viruses ability to create itself and replicate in an infected person’s body, there may be some significant breakthroughs on the horizon.
Rachel and Rosine - Africa Children With HIV/AIDS
Rosine’s HIV/AIDS infection is being spread through her body with the helps of the Niemann Pick Type C proteins which help the HIV/AIDS virus makes itself inside a person’s cells. Cyclodextrin sugar is having miraculous effect on the HIV/AIDS virus (it’s killing the virus in animals!). Millions of children in Africa contract HIV/AIDS through the birth canal. Imagine the possibilities of pregnant mothers in Africa having an inexpensive and safe cream applied inside the birth canal prior to giving birth? The virus could be stopped - today! Cyclodextrin is readily available and simply needs to be tested in humans and deployed to see if it can work as effectively on people as it does in animals.
Addi & Cassi Hempel - Twins with Niemann Pick Type C
Cyclodextrin treatment is being tried as a potential therapy in Niemann Pick Type C DISEASE, which afflicts 5 year old identical twins, Addi and Cassi Hempel. Addi and Cassi were born with two “genetic defects” or inherited “mutations” from their parents on the Niemann Pick Type C GENE. These mutations on the GENE cause a fatal cholesterol metabolism DISEASE that is often referred to as “Childhood Alzheimer’s.” The FDA recently approved a “compassionate use” IND to allow the twins to receive cyclodextrin infusions directly into their bloodstreams. With cyclodextrin having such a profound effect on cellular cholesterol in mice studies and on viruses like HIV/AIDS attach to cholesterol when they enter the body like HIV/AIDS, imagine what else it might do if more research was done on this compound?
Thanks to Kresta King Cutcher Venning for sharing her beautiful pictures of children in Africa who are suffering from HIV/AIDS. This picture of Rosine was taken in 2006 and she is still battling her HIV/AIDS infection. Together we must bring the story of children and families in different parts of the world who are fighting what seems to be entirely different fatal diseases, but are really connected through cholesterol, to the world!
Filed under Cholesterol, Cyclodextrin, News | Comment (0)
March 22nd, 2009
With the FDA approval now in hand and mediports installed, we are working towards starting intravenous infusions of Hydroxy Propel Beta Cyclodextrin on Addi and Cassi. We are currently capturing a full set of baseline testing on the twins (blood, urine and stool samples) for our doctors and researchers to study. Intravenous cyclodextrin treatments have never been attempted before to remove cholesterol from the cells and organs of living people, we have had to set up an entirely new testing and safety protocol from scratch. This has taken months of work.
Since Cyclodextrin is not a drug, is non-toxic and is already going into the human body through a drug called Sporanox, we were able to make the argument with the FDA to allow us to treat Addi and Cassi under “compassionate use.” Essentially, we have created our own personal clinical trial with our identical twins in order to try this sugar compound to see if it can extend or save their lives.
Addi and Cassi recently underwent neuropsychological exams by Dr. Seth Ubody at Children’s Hospital Oakland to capture neurological baselines. We will also be doing spleen and liver measurements and a whole host of other tests prior to starting the cyclodextrin infusions. Once we have all the baselines in place, the infusions will begin. Baseline samples can then be compared to samples collected following cyclodextrin treatment to see if there are any measurable changes.
The current plan is to admit Addi and Cassi to Renown Regional Medical Center where the girls will undergo 24 hour continuous infusions of cyclodextrin for 4 straight days (Note: If anyone reading this blog has extra children’s videos they don’t need, please send them to us!) After receiving 4 days of continuous infusions of cyclodextrin, the girls will become out patients and receive infusions 1x per week for 8 hours. We plan to carve out Friday’s for these infusions and we’ll be spending a lot of time at the hospital. Our goal is to eventually administer cyclodextrin infusions at home with the help of a home health nurse.
Right now, we have no idea what dose of cyclodextrin might have a therapeutic effect or if it will move cholesterol out of Addi and Cassi cells and bodies like it does in Niemann Pick Type C mice. We must start at a low level for safety purposes and then begin to scale up cyclodextrin doses. After 3 months, we plan to go back to the FDA and ask them to allow us to give Addi and Cassi slightly different doses of cyclodextrin so we can accelerate finding the ideal therapeutic dose. Since the girls are identical twins, they can work together to find the perfect dose.
To read more about Cyclodextrin, please visit Addi and Cassi’s website:
FDA Approves Addi and Cassi’s Cyclodextrin Infusions For Twins Who Suffer From Niemann Pick Type C:
http://addiandcassi.com/np-c/fda-approves-first-ever-cyclodextrin-infusion-treatment-for-twin-girls-suffering-from-fatal-cholesterol-disease-called-niemann-pick-type-c/
Johnson and Johnson Provides Cyclodextrin Safety Data to the FDA To Help Twins, Addi and Cassi Hempel:
http://addiandcassi.com/np-c/thank-you-johnson-johnson-for-helping-sick-kids/
http://addiandcassi.com/np-c/fda-reviewing-application-to-give-cyclodextrin-sugar-compound-to-5-year-old-twins-stricken-with-fatal-cholesterol-disorder/
Filed under Cholesterol, Cyclodextrin, Niemann Pick Type C | Comment (0)
