James Meek, science correspondent 

Baby cells give hope to brain disease victims

Gaye Woolford was so in love with her renovated brain that she jumped up from the sofa to demonstrate how things had improved since the drill went in.
  
  


Gaye Woolford was so in love with her renovated brain that she jumped up from the sofa to demonstrate how things had improved since the drill went in.

"The hands are so much better. The coordination is so much better," she said. "It's silly little things, like, my head would droop down, just like that, or when you bend down to pick something up your hand would clutch for it a dozen times, and you couldn't get it."

Gaye has been marked twice - once by a terrible inherited disease which was killing her, just as it killed her father and is killing her brother, and once by a pioneering operation which, she believes, will save her.

Gaye, 52, suffers from Huntington's disease, a fatal brain disease passed from parents to their children.

Until now, there has been no cure. Then, 15 months ago, Gaye became one of the first four British Huntington's patients to undergo a revolutionary treatment at Addenbrooke's hospital in Cambridge.

A hole was drilled in her skull and, over two hours, 6m cells extracted from the undeveloped brain of an aborted foetus were inserted into the right hand area of a part of her brain called the striatum.

The hope is that the transplanted cells, which do not carry the mutant gene that causes the disease and are at an early enough stage of development to be flexible, will attach themselves to the host brain and thrive there, replacing the damaged tissue and rebuilding shattered nerve circuits.

The story of the attempt to beat Huntington's is told in a BBC documentary tomorrow night.

The British scientists carrying out the research are cautious, warning that Gaye and the three other British patients will need a second, similar operation on the left hand part of the striatum and another year of observation before they can be sure the treatment is working.

But Gaye and her husband, John, are already convinced she is improving. "My legs used to buckle whenever I got up," said Gaye. "That doesn't happen now. When I made coffee I used to get more on the table than in the cup and it's still improving."

Without treatment Gaye's brain would continue to deteriorate, with movement becoming ever more difficult and speech and memory ever more confused until she lapsed into complete dependence on full-time nursing care and, ultimately, death.

That was the fate she saw overtake her father as a girl growing up. Her brother is now dying of the disease; her son, daughter and grandchild may have the killer gene.

Gaye said: "I'm very grateful for these cells that have been donated," she said. "It means Huntington's patients can have the life and hope that other people have."

Roger Barker, a neurologist at the Cambridge Centre for Brain Repair and one of the members of the scientific team carrying out the trials, said that women were asked to donate foetal material only after they had decided to have an abortion.

One of the problems for researchers is that, unlike a standard clinical trial, they have not been able to set up a control group to counter the placebo effect, where patients are given a psychological boost by the mere idea of a new treatment.

It is easy to give patients a placebo when the treatment is a pill or an injection - not so easy where brain surgery is involved.

Since getting his cell transplant another of the British patients has seen his swallowing problems disappear and has been able to button his shirt again.

But Stephen Dunnett, a Cardiff University neurobiologist who has done much of the fundamental research into the treatment, said: "I think we have to be cautious. We are optimistic, like they are. Of course one's hoping but we need time to be sure that these are changes in the right direction rather than just fluctuations."

The British programme is part of a Europe-wide study into foetal cell grafts for Huntington's, and additional hope for the treatment comes from operations on five French patients, all of whom have had grafts on both sides of their brain.

Three have improved, and brain scans show the grafts have taken. The lack of any change in the other two may have to do with failure to take the prescribed immuno-suppressant drugs.

For years, Gaye and John kept the nature of Gaye's illness from their son. "That was extremely difficult," she said. "I didn't tell him until filming for the BBC programme had almost started, and if I hadn't had the operation, I wouldn't have told him then. It's totally devastating for children to know what's in store."

A Cruel Inheritance will be shown on BBC1 tomorrow at 10.35 pm. Gaye and John's surname has been changed to protect their son from possible discrimination by employers.
Huntington's - Path of a disease

Huntington's disease is a fatal, so far incurable, brain disease which affects about 6,000 people in the UK.

The illness is inherited. It is caused by a mutation in a single gene. There is a 50% chance the carrier of a gene will pass it on to each of their offspring. If children have the gene they are certain to develop the symptoms of the disease.

In a way not yet understood, chemicals expressed by the faulty gene cause damage to areas of the brain responsible for movement, speech and memory. The first signs are slight, uncontrollable muscle movements, clumsiness, lack of concentration, short-term memory lapses, depression and mood changes.

The symptoms usually appear between the ages of 30 and 50. Later, involuntary movements increase and there may be difficulty talking and swallowing.

After an average of 15 years of worsening brain damage, the sufferer dies, often of secondary causes such as pneumonia.

 

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