FDA Approves First Ever Cyclodextrin Infusion Treatment For Twin Girls Suffering From Fatal Cholesterol Disease
March 15, 2009 by Chris Hempel
Filed under Cyclodextrin
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.
I SEEN YOUR STORY ON T.V. AND HOPE FOR THE BEST OF YOUR DAUGHTERS. THIS IS PRETTY INTRIGING STUFF, AND HOPEFULLY WILL PULL THEM THROUGH THEIR FIGHT. I ALSO AM COPING WITH THAT WHACKY DISEASE ALZHEIMER’S. MY MOM HAS LIVED WITH ME FOR FOUR YEARS AND LATELY IT’S BEEN BEATING ME DOWN PHYSICALLY AND MENTALLY. BEING A SINGLE GUY, NEVER MARRIED, IT’S DRAINING TO BE A 24HR. CARE TAKER. I’M NOT LOOKING FOR SYMPATHY, JUST TRYING TO FIGURE OUT IF THIS TREATMENT HAS EVER BEEN USED ON ALZHEIMERS PATIENTS? GOOD LUCK AND I’M GLAD YOU’RE HERE TO PUSH THROUGH THE RED TAPE FOR KIDS AND ELDERS ALIKE. IT’S A TRUE PITY THAT OUR KIDS AND ELDERLY HAVE TO SUFFER IN OUR COUNTRY. WHILE OUR HARD EARNED TAXES GO FOR LESS DESERVING, AND TRIVIAL PROGRAMS! IT REALLY CHOKES ME THAT MORE MONEY IS SPENT ON ILLIGALS AND GAYS AND WHAT EVER. THAN OUR HARD WORKING ELDERLY PARENTS AND YOUNGSTERS THAT NEED A FIGHTING CHANCE! KEEP UP THE GOOD FIGHT CHRIS AND THANKS AGAIN FOR CUTTING THROUGH THE BUREAUCRATIC MESS! TOM FULLER, CASTLE ROCK, WA.
Thanks for your message Tom. It’s so hard to be a caretaker, it’s a 24 hour a day job and you’re a saint for taking care of your Mom! As far as Alzheimer’s goes, I don’t know if cyclodextrin can work — it depends really if they figure out if Alzheimer’s is connected to cholesterol and calcium disruptions. It may be.
We know that both Alzheimer’s and NPC have tangles in the brain and many other connection points. Why? This is the key question — why would children get and Alzheimer’s like condition because of a genetic cholesterol metabolism disorder? Cholesterol pathways must be involved in Alzheimer’s — when will the reseachers figure this out?
I know cyclodextrin can work for HIV AIDS — many viruses utilize cholesterol when they enter the body and the NPC gene on Chromosome 18 helps viruses assemble in the human body. This Niemann Pick Type C gene is critical to all human survival and disruptions on this gene are catastrophic as witnessed by what is happening with Addi and Cassi.
Please stay in touch!
Chris
Chris, You are a Warrior Mom! As mom of a child on the autistic spectrum with numerous complex health issues, I am in awe of what you have accomplished in the midst of caring for your beautiful girls. When traditional medicine offers no hope, us moms become medical researchers, pharmacists, naturopaths, nutritionists, nurses, teachers, OT’s and diagnosticians. Keep up your phenomenal work and know that you have fans and supporters in the biomedical autism community!!
Thank you Willsmomstacie! I have never heard of the term Warrior Mom befor but I am going to use it! I feel Autism might be connected to disruptions in cholesterol — did you know that children with the disease SLOS all have severe Autism (they do not make cholesterol — Addi and Cassi on the other hand can’t process it)? I would be very curious to know what type of research is going on with Autism and cholesterol.
Well wishes to you. i am writing a research paper for my human genetics class on Niemann-pick disease after i watched your story on the news one night and was instantly intrigued and fell in love with your 2 precious girls. i hope the cyclodextrin works and i hope you will keep posting how the treatment is going so i can check up on the results.
- kraig van kirk
Hi Kraig:
Thanks so much for you post — the girls are doing well on their infusions of cyclodextrin. Human genetics is fascinating — I never thought I would ever learn anything about DNA and the genome, especially my own. I am very worried about my health because I am a carrier of an NPC mutation — they don’t know much about what happens to carriers like me but I bet in the future they will determine that I an pre-disposed to health issues. My Mother who is the carrier of the faulty gene and passed it to me had her first heart attack at 49 and a triple bypass at 65 despite a good diet and statin drugs. Yet they tell me I have nothing to worry about. Hmmmm.
Quería darles la enhorabuena por el logro conseguido al iniciar sus hijas, que por cierto son preciosas, el tratamiento con Cyclo.
Yo soy madre de un niñoa afectado por NPC que falleció el pasado mes de diciembre y se lo que tienen que estar pasando.
Les deseo toda la suerte del mundo para que tengan resultados positivos, porque se lo merecen.
También me gustaría me informara si sabe si hay alguna manera de hacer llegar este medicamento a España ya que ahora me estoy dedicando a ayudar a familias que todavía tienen a sus hijos.
We will pray that Addi and Cassi will be healed, and eventually other children. We have a grand-daughter who is suffering from Niemann Pick Type C disease.
Ken and Connie:
I am so sorry you are going through this nightmare like we are. I am sending prayers back to you and I hope that this cyclodextrin helps not only Addi and Cassi but someday your granddaughter.
Love,
Chris