This is a follow up on the post earlier about Cyclodextrin. Addi & Cassi are twin girls that live in Reno, NV and their parents Chris & Hugh Hempel are great people doing a lot of great things with the abilities they have been given.
The research is early and the talented people working on this don’t exactly know how it is working or how it might ultimately turn out in children with NPC but it is a good risk to take from their perspective.
The girls will have mediports installed so they can inject Cyclodextrin right into their bloodstreams. Of course they will be heavily monitored for any progress or digression. This is great news. Our prayers and wishes are with them.
Here is the blog post on Addi & Cassi website:
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 celluar 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 them 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 us 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. Stay tuned.
Posted by Chris (Mom)
March 25th, 2009 at 3:57 pm
I am Lajos Szente co-founder and CEO of a cyclodextrin company in Budapest, Hungary. In 1981 I worked at NIH, in Baltimore with Dr. Josef Pitha. We tried to save life of mice having a toxic lipophile in their bloodstream. We used a kind of cyclodextrin to help the animals’ body distribute and eliminate this toxic comopund. We succeded! Years later in Boston Children’s Hospital, Thomas Carpenter M.D. saved the life of a tween boy suffered from A-hypervitaminosis, by the same mode of administration of hydroxypropylbetacyclodextrin. Again a success! The working principle is the very same, and I am personally convinced that this, finally FDA-approved, treatment will help! My very best wishes to the little ladies and the family. If somebody knows how powerful cyclodextrins can be, it is myself. I am not only touched and amazed about everything you have done in this matter.
very truly yours,
Lajos Szente,Ph.D., D.Sc.
March 25th, 2009 at 3:59 pm
I am Lajos Szente co-founder and CEO of a cyclodextrin company in Budapest, Hungary. In 1981 I worked at NIH, in Baltimore with Dr. Josef Pitha. We tried to save life of mice having a toxic lipophile in their bloodstream. We used a kind of cyclodextrin to help the animals’ body distribute and eliminate this toxic comopund. We succeded! Years later in Boston Children’s Hospital, Thomas Carpenter M.D. saved the life of a tween boy suffered from A-hypervitaminosis, by the same mode of administration of hydroxypropylbetacyclodextrin. Again a success! The working principle is the very same, and I am personally convinced that this, finally FDA-approved, treatment will help! My very best wishes to the little ladies and the family. If somebody knows how powerful cyclodextrins can be, it is myself. I am touched and amazed about everything you have done in this matter.
very truly yours,
Lajos Szente,Ph.D., D.Sc.