(forwarded from a friend who eats bd food!)

http://www.newscientist.com/news/news.jsp?id=ns99992851


Lorenzo's oil finally proven to work
  17:55 26 September 02

David Concar


The controversial do-it-yourself medicine that inspired the heart-rending
movie Lorenzo's Oil has finally been proved to work. The new research ends
years of uncertainty about the treatment and demolishes the claims of
experts who repeatedly said it was a worthless quack remedy.

New Scientist has learned that Hugo Moser, the neurologist and doctor played
by Peter Ustinov in the film, will on Saturday unveil the positive
conclusions of a 10-year investigation into the oil's effects on a group of
boys affected with the same genetic condition as Lorenzo.

Normally carriers of the genetic defect are at high risk of developing
adrenoleukodystrophy, causing them to progressively lose the ability to
move, hear, speak and - finally - breathe. Some victims, like Lorenzo Odone
portrayed in the film, get the childhood form which usually kills within
just two years. Others get the adult form of the disease, which strikes
people in their late twenties and acts more slowly.

The new study shows that the boys whose parents scrupulously administered
the oil - often in the face of scepticism from the medical establishment -
were much less likely to go on to develop symptoms in childhood than boys
who did not get the oil on a regular basis.

Boys carrying the defect have extremely high levels of very long chain
saturated fatty acids in their blood. The oil brings these levels back down
to normal in a way that can be tested, and this enabled the researchers to
monitor which boys were getting the treatment on a regular basis.


"Dramatic vindication"

Between 1989 and 1999 two teams, one led by Moser at the Kennedy Krieger
Institute in Baltimore, Maryland, the other based in Europe, looked at the
progress of 104 young boys with the defect. At the outset all were under six
years of age and none had begun to develop symptoms - their movement,
hearing and MRI brain scans were normal.

By the end of the study 76 per cent of the 68 boys getting the oil were
still healthy and producing normal brain MRI scans. The same was true of no
more than about one in three of the 36 boys who did not regularly get the
oil.

Augusto Odone, Lorenzo's father and inventor of the oil, told New Scientist
that the protection, though not complete, was "dramatic" and would "come as
a shock to the medical community". After years trying to persuade everyone
to take it seriously, he says he feels "vindicated".

The big remaining uncertainty is whether those boys in the study who have
escaped the childhood version of the disease will also be spared the adult
form. With many of the boys only just approaching their twenties this may
not be known for years.


Urgent treatment

In a message to parents of affected boys, Odone, told New Scientist : "Give
the oil as soon as you know your son carries the genetic defect. If you wait
the symptoms might come and then you are in a different ballpark. We are not
sure the oil is useful after symptoms have developed."

The results will be presented at an international meeting at the University
of Ghent. Moser, himself once a sceptic, will tell the conference that the
oil should now be routinely given to boys carrying the genetic defect.

The oil works by blocking the enzymes required to synthesise the very long
chain fatty acids, but how this prevents the devastating symptoms is
uncertain. These are caused by the progressive loss of myelin, the fatty
sheath that insulates nerve fibres, enabling them to conduct impulses
properly.

It was 17 years ago that Odone's son, Lorenzo, started bumping into
furniture and was diagnosed as having just two years to live. Racing against
the clock, Odone and his wife pestered scientists, devoured neurology
textbooks and learned the language of biochemistry.

The result was Lorenzo's oil, an unpalatable mixture of oleic and erucic
acids, and a hit movie. Lorenzo did not receive the oil until after symptoms
appeared. Today, he is alive but unable to move and being cared for at the
family home.
  17:55 26 September 02



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