Newsday – July 12, 2005

Vindication for 'Lorenzo's oil'
Doctors find that mix of olive and rapeseed oils can help prevent a severe form of a rare disease affecting boys

BY JAMIE TALAN, STAFF WRITER
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When Lorenzo Odone's father helped developed a treatment for his son's rare genetic disease, doctors were skeptical - as portrayed in the movie "Lorenzo's Oil."

Today, Odone, although severely disabled, just marked his 27th birthday, and Johns Hopkins doctors have proved that the treatment can prevent the severe form of the disease in a new generation of boys if treated before symptoms appear.

Odone has adrenoleukodystrophy, known as ALD, a condition that triggers a build-up of saturated very long-chain fatty acids in the brain. While these fatty acids can be identified in the blood at birth, those with the more severe form go from being normal 4-year-olds to totally disabled within two years.

In 1989, word spread that Augusto and Michaela Odone had developed an oil that saved their son, Lorenzo, from certain death.

ALD is marked by destruction of the myelin sheath, the protective coating around nerve cells that allows electrical impulses to flow normally from cell to cell.

Lorenzo was 6 years old when diagnosed. He was given nine months to live. His parents pursued every possible lead, and ultimately found a small citation by a French scientist who found that rapeseed oil was protective in stopping the accumulation of these fatty substances.

Today, the formula is a 4-1 ratio of olive and rapeseed oils.

Families of others with ALD began the treatment that seemed to slow the disease in patients already handicapped by the condition. It doesn't reverse brain damage, but prevents accumulation of the fatty substance. Lorenzo began treatment at age 11.

Dr. Hugo W. Moser, director of neurogenetics at the Kennedy Krieger Institute and professor of neurology and pediatrics at Hopkins, was among the early skeptics. But he began offering "Lorenzo's oil" to patients and soon began research to see whether it worked.

It did. In 1989, he set out to identify infants and toddlers with the genetic defect, finding 89 patients.

He gave them the treatment oil in conjunction with moderate low-fat diet.

The severe form of the disease was stalled, according to a study in the latest issue of the Archives of Neurology.

As of this year, 66 of the 89 boys remain well.

Twenty-one others showed abnormalities on a brain scan and 10 had neurological symptoms.

"We've shown it has a preventative effect," said Moser. "It's very exciting."

Four years ago, Lorenzo's mother died of lung cancer. His father, Augusto, is now 72 and lives with his son in Fairfax, Va.

"Now, so many boys will be saved from ALD," Augusto said yesterday. It's taken time, however, he added. "The medical profession has its own way of going about things."

ALD affects one in 17,000 people. In the U.S., there are about 12,000 to 16,000 patients with ALD. Half have the severe form, like Lorenzo.

There is also an adult form of the illness that is less severe but quite disabling. It is often mistaken for multiple sclerosis. Both men and women can have this adult form of ALD.