- Addi and Cassi’s FDA Cyclodextrin Submission
- Meet Addi and Cassi
- What is Niemann Pick Type C disease?
- Cyclodextrin and Drug Delivery
- Cyclodextrin and Odor Prevention
- Cyclodextrin on Wikipedia
- Cyclodextrin Overview - List of Uses and Drugs
- Cyclodextrin Scientific Papers
- Procter & Gamble Cyclodextrin Overview
- Society Of Cyclodextrins
- Sporanox: Approved Drug Containing Cyclodextrin
- CoQ10 and Cyclodextrin
- Cyclodextrin Releases Trapped Cholesterol
- KTVU Story On The Power of Cyclodextrin
- Take Drug Additive, Not Drug?
- Washington DC HIV/AIDs Epidemic
- Dr. James E. K. Hildreth, HIV/AIDS
- Dr. John Dietschy, Niemann Pick Type C
- Dr. Lajos Szente
- Dr. Len Kritharides, Vascular & Cholesterol Research
- Dr. Steven Walkley, Niemann Pick Type C
Featured Articles
Wacker Chemie Expands US Based Cyclodextrin Facility To Meet Increase In Worldwide Demand For Sugar Molecule
Wacker Chemie, the Munich-based chemical company, announced that is has expanded its US based cyclodextrin facility in Eddyville, Iowa. According the the press release issued by Wacker, the new cyclodextrin facility increases the company's capacity for alpha (α) and beta (β) cyclodextrins by 50 percent and doubles its capacity for gamma (γ) cyclodextrins. Investment in the entire facility totaled over $21 million and will enable Wacker to produce up to 7,500 metric tons of cyclodextrins a year. The extra capacity is needed to meet the worldwide rise in cyclodextrin demand. According to the press release, "the ability to reversibly enclose other substances makes cyclodextrins invaluable in many products such as pharmaceuticals, cosmetics, textiles and food, not to mention in the household-care, personal-care and construction sectors." What about entrapping cholesterol in the human body and helping get rid of it? Interestingly, Wacker's press release does not mention hydroxy propel beta cyclodextrin (HPBCD) and its potential health benefits. This is the type of cyclodextrin we are giving via intravenous infusions to Addi and Cassi for their fatal cholesterol metabolism disease, Niemann Pick Type C (otherwise known as the "childhood Alzhiemer's.") Hydroxy propel beta cyclodextrin is somehow grabbing the stuck cholesterol and taking it out of ...
Anthrax Bacteria Killed By Simple Sugar Compound Called Cyclodextrin. Is CDC Looking Into This?
We all remember the Anthrax-laced letters that killed five people and severely rattled the country post-9/11. Just when you thought there might not be a way to stop this lethal infectious disease along comes beta cyclodextrin, a non toxic sugar compound. A researcher by the name of Vladimir Karginov at a company called Innovative Biologics is working with beta cyclodextrin and Anthrax. Karginov has designed and synthesized a number of beta-cyclodextrin derivatives and evaluated their ability to inhibit the lethal toxin action of Anthrax. Several compounds displayed anti-toxin activity at low micromolar concentrations in cell-based assays and preliminary toxicity and efficacy studies in rodents produced very promising results. You can read about the research project here. Anthrax is a highly lethal and infectious disease caused by the bacterium Bacillus anthracis, a bacteria that forms spores, or dormant cells, which can come to life under the right temperature, nutrients and other conditions to allow growth. Anthrax occurs in humans after exposure to an infected animal or infected animal tissue or when anthrax spores are used as a bioterrorist weapon. There are some effective vaccines against anthrax, and some forms of the disease respond well to antibiotic treatment shortly after exposure. But there is ...
Journal Of Virology Reports On Link Between HIV and Niemann Pick Cholesterol Gene: Intact Intracellular Cholesterol Trafficking Pathways Mediated by NPC1 are Needed for Efficient HIV-1 Production
Journal of Virology May 27, 2009 Deficiency of Niemann-Pick type C-1 Protein Impairs HIV-1 Release and Results in Gag Accumulation in Late Endosomal/Lysosomal Compartments Tang Y, Leao IC, Coleman EM, Broughton RS, Hildreth JE. Human immunodeficiency virus type 1 (HIV-1) relies on cholesterol-laden lipid raft membrane microdomains for entry into and egress out of susceptible cells. In the present study, we examine the need for intracellular cholesterol trafficking pathways with respect to HIV-1 biogenesis using Niemann-Pick type C1 deficient cells (NPCD) wherein these pathways are severely compromised, causing massive accumulation of cholesterol in late endosomal/lysosomal (LE/L) compartments. We have found that induction of an NPC disease-like phenotype through treatment of various cell types with the commonly-used hydrophobic amine drug U18666A resulted in profound suppression of HIV-1 release. Further, NPCD EBV-transformed B lymphocytes and fibroblasts from patients with NPC disease infected with a CD4-independent strain of HIV-1 or transfected with an HIV-1 proviral clone, respectively, replicated HIV-1 poorly when compared to normal cells. Infection of the NPCD fibroblasts with a VSV-G pseudotyped strain of HIV-1 produced similar results, suggesting a post-entry block to HIV-1 replication in these cells. Examination of these cells using confocal microscopy showed an accumulation and stabilization of Gag in LE/L compartments. Additionally, normal HIV-1 ...
A Promising Compound That Could Stop HIV AIDS. Why Is It Not Being Supported?
In chapter 18 of a book by Stefano Bertozzi referenced by famous health economist Robert H. Topel in his article in the Journal of Political Economy, several insightful comments about HIV research funding and needs for prevention in the face of a rapidly increasing HIV infection rate are highlighted. The points made by Bertozzi et all about the lack of funding for research into preventive treatments for HIV are directly applicable to the difficulties I am facing obtaining funding and support from for a cheap sugar compound called cyclodextrin that has great potential to help stop the spread of HIV/AIDS. Even though the U.S. President’s Emergency Plan for AIDS Relief and the Gates Foundation are funneling a great deal of money into AIDS research, introduction of ameliorative therapy projects based on simple and available non toxic compounds such as hydroxypropyl beta cyclodextrin have not gotten past the initial screener. Why is further research into this simple sugar compound being held back? Bertozzi et al attribute such resistance to compounds like cyclodextrin to the perception that preventive research is viewed as “less innovative scientifically” and “typically less experimental” by funding organizations. They suggest earmarking such ameliorative therapy approaches to redress this imbalance. The ameliorative therapy ...
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. [caption id="attachment_376" align="alignright" width="163" caption="Less than 5% of Drugs can cross the Blood Brain Barrier: Click to Enlarge"][/caption] 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 ...
Cyclodextrins Can Reduce Side Effects of Cancer Drug Treatments
Each day, I learn more about the amazing benefits of cyclodextrins -- novel excipients of unexplored potential. Research studies in both humans and animals have shown that cyclodextrins and their derivatives can be used to improve the drug delivery system for almost any type of drug formulation from anti-cancer drugs to anti -viral drugs. In the pharmaceutical industry, cyclodextrins are used as complexing agents to increase the aqueous solubility of poorly soluble drugs and to increase their bioavailability and stability. When some cancer drugs are combined with cyclodextrins, their bioavailability increases. If a drug’s bioavailability can be controlled in the bloodstream and acceptable drug levels are reached more effectively and precisely, it takes far less of a drug to produce cancer killing effects. Cyclodextrins can help make cancer treatment far less debilitating for a patient undergoing chemotherapy treatment -- less of a toxic drug in a person's system makes for less side effects. Cyclodextrins were discovered over 100 years ago and the first patent on cyclodextrins and their complexes was registered in 1953. Here is an excellent paper outlining all the benefits of cyclodextrins which I believe has much greater potential than anyone ever imagined: http://www.pharmainfo.net/reviews/cyclodextrins-drug-delivery-systems-update.
Cyclodextrin As A Therapeutic “Drug” For HIV AIDS, Niemann Pick Type C and other Viruses
Addi and Cassi's first round of cyclodextrin infusions have been going smoothly at Renown Regional Medical Center in Reno, Nevada. We're now into our third day of continuous infusions of hydroxy propel beta cyclodextrin (HPBCD) into the girls' bloodstreams and they don't seem to be experiencing any negative side effects. I feel as if it's having a positive and immediate effect. Addi was talking yesterday afternoon and stringing together more than one word -- "I like my toys, I'm brave enough, I need your help, "Bye" to Dr. Hastings, and "Ad" for her name Addison. Even Cassi came out with a few words - "Mommy, No." This is quite encouraging to us but we're not yet sure if it's a result of the cyclodextrin treatment. [caption id="attachment_341" align="aligncenter" width="411" caption="Addi and Cassi resting during cyclodextrin infusions"][/caption] Addi and Cassi's blood work-ups have come back "normal" following the cyclodextrin infusions. There was a slight elevation in both girls' eosinophils after 24 hours but it was minor. We also had to change Addi's port access needle as it was not working properly and we were unable to draw blood. Unfortunately, we had to re-install the needle on her chest without any numbing ...
The Wall Street Journal Reports On FDA Approval of Addi and Cassi’s Cyclodextrin Treatment
By AMY DOCKSER MARCUS April 3, 2009 A Mom Brokers Treatment for Her Twins' Fatal Illness Bucking Scientific Convention, Ms. Hempel Gets Researchers From Different Fields to Share Data on Potential Therapy From the moment her twin daughters, Addison and Cassidy, were diagnosed with a fatal genetic disease in October 2007, Chris Hempel has been searching for a drug that might save their lives. The 5-year-old girls were diagnosed with a devastating cholesterol metabolism disorder known as Niemann-Pick Type C, which is ultimately fatal. Soon after, Ms. Hempel learned that researchers found that a form of a compound called cyclodextrin extended the lives of affected mice. Ms. Hempel set out to gather as much data as possible. She got a list of all major cyclodextrin distributors and connected with one in Florida, who shared scientific studies and other information with her. She found a short report in the medical literature about a doctor who had treated a child with a different disease using cyclodextrin and tracked him down. She became increasingly hopeful that, although cyclodextrin isn't approved as a drug in the U.S., she might get the Food and Drug Administration to allow her to give cyclodextrin infusions to her girls as an experimental treatment. Her search ...
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 ...
Featured Video
Bay Area Television station KTVU Channel 2 reports on the remarkable story of how two deadly diseases, one extremely rare and one a global epidemic, are connected by a cholesterol metabolism gene called Niemann Pick Type C located on Chromosome 18.
The story details how five year old identical twins, Addi and Cassi Hempel, who were born with a genetic defect on this gene, have obtained FDA approval to receive infusions of a non toxic sugar compound called cyclodextrin to help treat their rare and fatal cholesterol disorder. Dr. James Hildreth, a leading HIV AIDS researcher discusses how HIV AIDS is connected to cholesterol and how cyclodextrin punches holes into the virus and kills it.







