Lorenzo’s Oil prevents rare disease in boys -study

WASHINGTON (Reuters) – A treatment called Lorenzo’s Oil can
prevent the onset of a rare but devastating neurological
disease in young boys, U.S. researchers reported on Monday.

The study is the first to use validated scientific methods
to test whether the substance, a combination of two fats
extracted from olive oil and canola or rapeseed oil, actually
works, the researchers said.

They tested the oil on 89 boys who had been diagnosed with
X-linked adrenoleukodystrophy or X-ALD, but who had not yet
begun to show symptoms.

Writing in the Archives of Neurology, Dr. Hugo Moser of the
Kennedy Krieger Institute in Baltimore and colleagues said it
prevented symptoms from ever beginning in most of the boys.

“This clinical study clearly demonstrates that the use of
Lorenzo’s Oil can prevent the onset of the rapidly progressive
and devastating form of the brain disease that affects 50
percent of boys with X-ALD,” Moser said in a statement.

His team tested 89 boys age 7 or younger who had an
abnormal gene that identifies X-ALD.

For seven years the boys got daily oral doses of the
substance, the basis for the 1992 movie “Lorenzo’s Oil,” and
moderated what kinds of fat they ate.

Subsequently, 74 percent of the boys showed no signs of
disease progression, the researchers found.

“The results of this trial offer clinical support for
treating the many young boys identified at high-risk for the
disease,” said Dr. Gary Goldstein, chief executive officer of
the Kennedy Krieger Institute.

X-ALD affects 16,000 patients in the United States.

Patients cannot metabolize big fat molecules called
long-chain fatty acids — either made inside the body or eaten
in food. The condition causes the breakdown of myelin, the
fatty substance that coats and insulates nerve fibers.

The most dangerous type is the childhood cerebral form, in
which brain cells are destroyed. Up to 40 percent of cases are
this type, which usually appears between 4 and 8 years of age.

Symptoms include a loss of the ability to speak, reduced
strength and coordination and, eventually, complete breakdown
of bodily function and death.

There is no cure, but potential treatments include the
cholesterol drug lovastatin and bone marrow transplants.

Then there is Lorenzo’s Oil, a treatment invented by
Augusto and Michaela Odone after their son, Lorenzo, was
diagnosed with ALD.

Lorenzo’s Oil normalizes the concentrations of very long
chain fatty acids. The Odones said the treatment stopped the
progression of Lorenzo’s disease and their son, now 27, is
alive though severely disabled.