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The twins aged five who suffer from Alzheimer’s
Mark Prigg26 Mar 2009
A radical new treatment for children with a devastating disease known as childhood Alzheimer's is to be tested on five-year-old American twins.
American doctors hope the new drug, a sugar compound called cyclodextrin, will help children such as five-year-old Addison and Cassie Hempel.
The twins, from Reno, Nevada, suffer from Niemann Pick Type-C which means they will slowly forget all the things they have learned and have a shortened life span.
The disease affects 500 children worldwide, including 60 in Britain.
Today the US Food and Drug Administration gave the go-ahead for the trial after a “compassionate use” request from the twin's parents.
The treatment being offered to Addison and Cassie at Oakland Children's Hospital in California, could bring hope to scores of other families.
Lat year Britons Josh Cullip, two, and Hollie Carter, three, both from Milton Keynes, took part in a study at Maryland's National Institute of Health. It is expected they will die within 10-12 years if no treatment is found.
Chris Hempel, the father of the American twins who has been working on the trial with doctors and geneticists for more than a year, said: “If this works it could be a massive breakthrough for this rare condition.
“The sugar compound has just been approved by the US Food and Drug Administration. It could also offer hope for the condition of Alzheimer's as well.”
Niemann Pick is a genetic condition which means children cannot process cholesterol, which slowly builds up in the body, causing cells to die and leading to organ failure.
Because the neurons in a child's brain cannot process cholesterol, brain cells break down, leading to symptoms of dementia.
Doctors discovered that cyclodextrin, which is used in the food processing industry to extract cholesterol and make fat-free products, may help children with the condition.
Addison and Cassie have had tubes placed into their chests so the sugar compound can be infused directly into their bloodstreams.
It is hoped the infusions will move trapped cellular cholesterol out of their spleens, livers and ultimately their brains.
Reader views (2)
I have read the WSJ article and the involvement of CTD Holdings (OTCBB: CTDH) My prayers continue with you and the girls. I am anxiously hoping the results remain positive!
- Darrell Peterson, Vero Beach Florida, 09/05/2009 15:23
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Our hopes and prayers are with you Chris (the mom) and Hugh (the dad) for cyclodextrin to improve Addi and Cassi's condition! You are working tirelessly to renew hope for our children and we love you for it.
My own daughter shares this disease, that is more correctly named Niemann Pick type C. She had a later onset of the disease and she is 22 years old.
God bless, Nancy Trombley
- Nancy Trombley, Minneapolis, MN, 29/03/2009 05:31
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