Sunday, November 19, 2017

Dear British Media – Feel Free To Call or Email!

March 26, 2009 by  
Filed under Addi and Cassi News

London Big Ben

Dear British Media:

Thank you so much for your interest in our daughters, Addi and Cassi, and their fight against Niemann Pick Type C disease, a fatal cholesterol disorder that is often referred to as the “childhood Alzheimer’s.”  There have been two stories that were written today regarding Addi and Cassi’s cyclodextrin sugar  treatment and we can’t thank you enough for bringing this important health story to the attention of people in the UK.

Unfortunately, there are some incorrect facts in the stories (see below) that are now appearing online.  It also would have been courteous to contact us and conduct fact checking.  We realize that it’s a competitive media environment and you are in a world of meeting daily deadlines but we do not appreciate being quoted in stories when words did not come directly from us. We are more than happy to tell our story to the world (we are trying to do this all the time through our website!) and you can call or email us any time day or night as our contact information is on our website.

Again British media, thank you for getting the word out on Niemann Pick Type C disease.   While considered a “rare disease,” Niemann Pick Type C disease could have implications for millions of people.  The Niemann Pick Type C gene on Chromosome 18 is a very important gene in the human body – we all have this gene and it appears to regulate people’s cholesterol at a cellular level.  We hope that more discoveries regarding the NPC gene will come out soon.

The Evening Standard:

http://www.thisislondon.co.uk/standard/article-23667508-details/The+twins+aged+five+who+suffer+from+Alzheimer+s/article.do

Correct facts:

• Our names are Chris and Hugh Hempel. Chris is Mom, not Dad, as you have reported in your article

• Our children’s names are Addison and Cassidy, we refer to them as Addi and Cassi – no “e’s” at the end of their names

• The US FDA did not give the approval to us today to start these treatments on our children – this was approved a few weeks ago and we are in the process of gearing up for the treatments

• Children’s Hospital of Oakland California is not conducting the actual infusions – mediports for the sugar infusions were surgically installed at this hospital. Cyclodextrin infusions will take place at Renown Regional Medical Center in Reno, Nevada, USA. We are working towards the infusions right now and hope to start in a week or so

• We have no idea if cyclodextrin can help Alzheimer’s patients as was quoted in your story. However, this is an interesting idea and cyclodextrins amazing properties should be looked into further

• Doctors did not discover cyclodextrin, researchers at UT Southwestern University in the United States have reported on cyclodextrins effects in NPC mice

Telegraph:

http://www.telegraph.co.uk/health/healthnews/5055351/World-first-treatment-may-offer-hope-to-twins-with-childhood-Alzheimers..html

Correct facts:

• Niemann Pick Type C does not only impact children, it impacts adults too and there are a number of adult cases in the UK.  Children experience dementia, tangles and other symptoms that are similar symptoms seen in Alzheimer’s disease.  Possibly the two diseases are related to disruptions in lysosomal function in the cell?  Why are Alzheimer’s researchers not looking more at NPC and Lysosomal Storage diseases??

• The proper spelling for the disease is Niemann Pick Type C, not Niemann Pick’s

• Did you find quotes from Chris from previous videos on our website?  Feel free to contact us and we will be happy to give direct quotes that are current

• This is not the world’s first treatment – we actually provided all of the cyclodextrin information we submitted to the US FDA to a family in India and Lebanon. Cyclodextin infusions have been started on two children in these countries based on our ground work.  Due to the fact that these countries do not have the same types of restrictions that we have in the United States, these families have been able to start over 1 month ago!  This will be the first time in the US this will be tried

Again, thank you for starting the education process on Niemann Pick Type C disease.   Please pray for all who have this horrible and devastating illness that is stealing their minds.

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Addi and Cassi Preparing For Intravenous Cyclodextrin Treatment

March 22, 2009 by  
Filed under Cyclodextrin

Cholesterol and Kids - Addi and Cassi Fight Neimann Pick Type C

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.

 

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President Obama, Please Meet Addi and Cassi To Understand Why Your Jay Leno Joke Is No Laughing Matter

March 21, 2009 by  
Filed under Opinion, Rare Disease

Mediports Addi and Cassi Cyclodextrin Infusions

President Obama’s Special Olympics joke on Jay Leno may not seem like a big deal to some Americans but for others the gaffe is disappointing. Our twins, Addi and Cassi, are afflicted with a rare and fatal cholesterol disease called Niemann Pick Type C and they are considered “special needs.” We have all made mistakes with words or put our foot in our mouth from time to time. President Obama is human too and must be forgiven by the special needs community for his off hand joke. Of all people, our President understands what it’s like to live in a society where stereotypes abound and hopefully he will turn this negative situation into a positive.

What’s frustrating to me is that all special needs children seem to get lumped into one category — “retarded” (a word that should be forever removed from our vocabulary). Children with Ataxia-Telangiectasia are born healthy but completely lose their entire motor coordination control center in the cerebellum.  Their minds remain intact but they progress to wheelchairs and often die in their teens. Someone on the street would think a child with A-T is intellectually challenged when they are not. Many children who fit into the special needs category have life threatening and debilitating diseases like Addi and Cassi – they are called Rare Diseases.

Millions of people, primarily children, are afflicted with Rare Diseases that are often genetic in origin. President Obama needs to educate himself quickly and learn what it truly means to be special in America.  He will then learn to laugh with us and not at us. More importantly, President Obama can not only help us overcome stereotypes but he can invest in much needed medical research that can keep our special kids alive.

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

March 15, 2009 by  
Filed under Cyclodextrin

Addi-and-Cassi-Hempel

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.

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CBS Evening News Covers Dillon Papier’s and His Army’s Fight To Save His Life

March 4, 2009 by  
Filed under Videos

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