The twins aged five who suffer from Alzheimer’s - News - Evening Standard
       

The twins aged five who suffer from Alzheimer’s

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.

Comments

Don't Miss
Rock star: Erin Wasson

Rock star

Erin Wasson is the ultimate anti-supermodel
Maybe it’s because she’s a Londoner … Happy anniversary, Ma’am

Happy anniversary

The monarchy has become stronger and more respected in the past 60 years
Victoria Coren: My obsession with children, five proposals a week and why David and I are no power couple

Victoria Coren

David Mitchell and I are no power couple
The Royal Academy of Arts Summer Exhibition preview party

Summer party

Stars at the The Royal Academy of Arts
London gets ready for the Diamond Jubilee - in pictures

Diamond Jubilee

London gets ready - in pictures
The Glamour Awards - stars turn on the style

Glamour Awards

Stars turn on the style
Duchess of Cambridge is pretty in pink at her first Buckingham Palace garden party

Garden party

Duchess of Cambridge is pretty in pink
FIRST review of Ridley Scott's latest sci-fi blockbuster Prometheus

First review

Is Ridley Scott's Prometheus any good?
Fair-weather goths

Fair-weather goths

The sultry shades of summer darks are coming out of the shadows
Dog save the Queen: Corgis surge in popularity

Dog save the Queen

Corgis surge in popularity