http://news.scotsman.com/index.cfm?id=2128062005

RAYMOND HAINEY

STEM CELLS from aborted foetuses are to be injected directly into human brains in a procedure that could open the door to treating a host of neurological disorders such as Alzheimer's and motor neurone disease.

Scientists in the United States have been given the go-ahead to transplant stem cells into six children who suffer from Batten disease, a rare but fatal genetic disorder.

Batten disease is a group of degenerative brain conditions which leaves young victims blind, speechless and paralysed before killing them. Few victims survive into their teens.

The stem cells to be transplanted aren't human embryonic stem cells, which are derived from days-old embryos. Instead, the cells are immature neural cells that are destined to turn into the mature cells that make up a fully formed brain.

Parkinson's disease patients and stroke victims have received transplants of fully formed brain cells before, but the brain cells involved here have never been implanted.

Batten disease is caused by a defective gene that fails to create an enzyme needed in the brain to help dispose of cellular waste. The waste piles up and kills healthy cells until the patient dies.

The idea is to inject the sick children with healthy, immature neural stem cells that will "engraft" in a brain and turn into cells able to produce the missing enzyme.

Such an experiment showed promise in Batten-afflicted mice, but such an ethically charged test has never been tried in humans.

"I'm sure there is no threat to anyone's identity," said Arthur Caplan, director of the University of Pennsylvania's Centre for Bioethics. "But we are starting down that road."

What's more, some of the brain cells to be implanted will be derived from aborted foetuses, which Mr Caplan said raised ethical concerns.

Jonathan Cooper, of the Institute of Psychiatry at King's College, London, told The Scotsman: "This is potentially very important.

"It may be far more wide-ranging than this particular disease. We will be watching it with great interest."

Dr Cooper added: "People in various places have been doing foetal embryonic stem cell research, but that is not the same as taking cells, isolating them in a dish and then putting them in people's brains.

"It's potentially very exciting. Whether it will work, we don't know, but they've convinced the FDA that it's working to the point that they're prepared to approve this and move it into the clinic."

Dr Cooper added that researchers would have to get approval from UK regulators to use foetal tissue for stem cell research.

"It's a big step forward - but, at present, it's a step into the unknown."

Dr Jo Mountford, lecturer in stem cell technology at Glasgow University, said: "This is entirely new - it's analogous to blood cell transplantations for leukaemia. They're going to take stem cells and put them into the damaged brain and see if it will grow fresh, healthy brain.

"Instead of using cells which can be everything, they're using cells which want to be brain. It's very clever. It's certainly never been done in this country."

Robin Lovell-Badge, of the National Institute for Medical Research in London, added: "I would give these trials cautious approval - I hope good information will be obtained, not just for patients with Batten disease, but for patients with other neuro-degenerative diseases."

Stanford University Medical Centre chief neurosurgeon Dr Stephen Huhn said he would bore small holes in the children's skulls and inject the cells directly into the brain.

The young patients will be given drugs to ensure that they do not reject the new cells, and they will be closely monitored for a year.

Dr Huhn added that the trial would help doctors test whether receiving millions of new brain cells was safe, and stressed that further tests with more patients over several years would be needed to determine whether the stem cell treatment was effective for sufferers of Batten disease.

But he said: "This may be what the future holds for regenerative medicine."

Martin McGlynn, chief executive of Stem Cells Inc - the biotechnology company developing the treatment - said Batten disease was chosen because mice genetically engineered with the disease were readily available and because it's a brutal, fatal disease with no cure - a fact the US Food and Drug Administration took into account when considering approval for such a novel experiment.

Mr McGlynn said: "This endeavour is unique. It's pioneering, and no-one has ever proposed to do what we are attempting. Once you put these stem cells in, you can't get them back."

He added that the stem cells had to be tested in humans. "You cannot ask a mouse how it's feeling," he said.

Marcus Kerner, whose son Daniel, aged five, suffers from Batten disease, said the experimental procedure was the family's last hope.

He added: "It is a horrific, terrible way to watch a child die, and there is currently nothing available to stop it. I think this is going to be a major medical breakthrough that will save Daniel's life."

Disorder is rare - but no cure has been found

BATTEN Disease is a highly aggressive group of degenerative brain disorders affecting around one in 20,000 children.

Between eight and ten children in the UK are diagnosed with the condition every year.

A flaw in a single piece of DNA means that the brain fails to produce an enzyme or protein which carries out vital roles in helping to dispose of brain cell waste.

That means the waste material accumulates in the brain and, for reasons scientists do not yet understand fully, healthy brain cells begin to die.

Batten Disease is genetically transmitted when paired genes from the mother and the father are both faulty. Affected children suffer agonising seizures, go blind, lose the ability to speak and become paralysed. There is no cure.

Early symptoms include clumsiness, difficulty with school work and deteriorating vision, with complete loss of sight within a few years.

Many victims of some types of the disease fail to reach their teenage years. Some victims, however, live until their 30s and other types of the disease do not strike until later in life.