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.