Cyclodextrin Sugar Killing HIV AIDS? Who Would Have Imagined This?

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!

Addi and Cassi Preparing For Intravenous Cyclodextrin Treatment

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.

 

FDA Approves First Ever Cyclodextrin Infusion Treatment For Twin Girls Suffering From Fatal Cholesterol Disease

After months of research, document preparation, discussions with the FDA and an immense amount of stress, it’s finally official.  The FDA has approved our “compassionate use” IND request to give Addi and Cassi infusions of a non toxic sugar compound called 2-Hydroxypropyl-beta-Cyclodextrin or HPBCD.

This Wednesday, March 18, 2009, at 7:30am and 8:30am, Addi and Cassi will have medi-ports surgically placed into their chests at Children’s Hospital in Oakland, California. The placements of these medi-ports (similar to ports used to administer chemotherapy drugs) will allow our doctors to start direct infusions of 2-Hydroxypropyl-beta-Cyclodextrin directly into Addi and Cassi’s bloodstreams.  The goal of the infusions (which will take place at Renown Regional Medical Center in Reno, Nevada) is to try and move trapped cellular cholesterol out of Addi and Cassi’s spleens, livers and ultimately their brains.

From what we understand, this will be the second time in the United States that cyclodextrin alone has been used in an attempt treat a fatal disease. Over 20 years ago, cyclodextrin was used in a medical case involving a boy with severe hypervitaminosis A and cyclodextrin saved the child’s life. In Addi and Cassi’s case, we are trying to save the girls from Niemann Pick Type C disease, a fatal cholesterol metabolism disorder that is often referred to as the “Childhood Alzheimer’s.”  There is no cure for NPC and only one experimental drug treatment.

Now this promising sugar compound that is not a considered a drug provides our family with a glimmer of hope. We would like to thank the following doctors, researchers and companies for their support: Dr. Caroline Hastings, Dr. Ron Browne, Dr. Susan Sorensen, Children’s Hospital Oakland, Renown Regional Medical Center, CollabRx, Johnson & Johnson for providing critical safety data on cyclodextrin to the FDA, the FDA for approving our request, researchers Benny Liu and Steve Walkley for their amazing work on cyclodextrin and Niemann Pick Type C mouse models, cyclodextrin expert Rick Stratton at CTD, Inc. and all of our friends and family who have provided us with the emotional support to forge ahead and never give up on finding therapies for our daughters.

Cyclodextrin is made by sugar angels. The sugar angels are hard at work to bring the world more amazing medical news on cyclodextrin and Niemann Pick Type C disease.

Thank You Johnson & Johnson For Helping Sick Kids

I am happy to report to everyone that Johnson & Johnson really does care about children, especially Addi and Cassi and kids suffering from Niemann Pick Type C disease. After I posted my previous blog and contacted Johnson & Johnson’s PR department about our situation with the FDA and obtaining cyclodextrin safety data from their company, they responded over the weekend and offered to help our family. If all corporations were like Johnson & Johnson, the world would be a better place!

Johnson & Johnson’s regulatory contacts agreed to send a letter to the FDA offering to open up relevant portions of their drug master files on cyclodextrin to assist us with Addi and Cassi’s compassionate use treatment case.

They also apologized to me for the response I received from Janssen’s global clinical operations group which has restored my faith in pharmaceutical companies. The challenge now will be to get the FDA to allow us to give Addi and Cassi this potentially life-extending non-toxic sugar compound via IV that could have far reaching implications beyond Niemann Pick Type C disease.

Dear Johnson & Johnson, do kids really matter to you?

After weeks of going back and forth with the U.S. Food and Drug Administration (FDA) on our request to give Addi and Cassi hydroxypropyl-ß-cyclodextrin (HP-ß-CD) or "CYCLO" infusions, we finally have a Type A meeting scheduled on February 26, 2009, from Noon-1pm to discuss the clinical hold the FDA put on our "compassionate use" treatment request.

The FDA has requested that we change our original protocol for cyclodextrin infusions and take the dosage down to 1/50th of the amount we believe will be therapeutic.  They have asked us to provide more "safety data" on CYCLO and we now need to try and convince the FDA in person to allow us to treat our twins with higher does of cyclodextin with or without the safety data.

We know that the sugar compound cyclodextrin has been infused into the bloodstream of children and adults and there is safety data available.  There is a drug called Intravenous Intraconazole that is given to children to treat fungal infections and it contains cyclodextrin which helps solubalize the drug and deliver it more effectively in the body.   This is the same cyclodextrin we propose to infuse into Addi and Cassi.  The pharmaceutical company Janssen makes this drug and has drug master files (DMFs) which contain safety data on CYCLOs effect in animals and humans taking intraconazole.  Intravenous intraconazole could not have been approved by the FDA without some amount of safety data.

A few months ago as we were gearing up to write our cyclodextrin protocol for the FDA, I contacted Janssen to get assistance in the hopes of accessing their DMFs on cyclodextrin.   I managed to reach the drug department and spoke to the woman in charge who obviously was on a power trip.  I explain that children like Addi and Cassi are dying and have no treatments and that a simple sugar compound called cyclodextrin could possibly save their lives. She refused to help me and essentially said I was out of luck. I tried to explain that cyclodextrin is used only as a delivery agent and that I was not requesting patentable information or trade secrets on the drug. She was unsympathetic. Sarcastically, I asked her if a letter from our doctors or even the President would help. She laughed at me and said "No." After I slammed down the phone, I started to cry.   How can this be happening?   All I need is safety data on a sugar compound that is already being eaten by people in fat free butter and being used to enhance the delivery of some drugs on the market.  How hard can this be?

I found out that Janssen’s parent company is Johnson & Johnson.    Here is a quote from their website:

Your family’s health and well-being is our passion. That’s why our companies offer the world’s broadest range of health care products. Whether you have a skin blemish or sniffles or a serious medical condition, you and the health professionals you trust can turn to our companies’ products for comfort and care.

As I was giving Addi and Cassi a bath and washing their hair tonight (with Johnson & Johnson shampoo!), I decided to finish the letter I started to Johnson & Johnson’s management team and board of directors.  Will Johnson & Johnson also be unwilling to provide human safety data to us on a non toxic sugar compound? All we need from them is to provide our doctors and the FDA with more information from their files that could help save the lives of Addi and Cassi and possibly 500 children around the world afflicted with Niemann Pick Type C disease.

I guess the world will soon find how much Johnson & Johnson cares about kids who are dying. Let’s hope the values they claim are so important to their company really hold true.

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