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The mixed legacy of 'Lorenzo's Oil'

Film's immediate impact was alienation between parents and researchers, but today, research advances on the mysterious disease

Tuesday, May 08, 2001

By Christopher Snowbeck, Post-Gazette Staff Writer

When the film "Lorenzo's Oil" came out nearly a decade ago, Augusto and Michaela Odone were depicted as crusading parents who fought the medical establishment to discover a life-saving dietary oil for boys who had the same rare disease as their son, Lorenzo.

 
   

For more information

These Web sites can provide information and resources about adrenoleukodystrophy, the Lorenzo's Oil disease:

www.ulf.org for information from the United Leukodystrophy Foundation.

www.myelin.org for information from Augusto Odone's Myelin Project.

 
 

Today, Lorenzo is a 22-year-old man still confined to his bed and unable to speak. Augusto Odone mourns the loss of Michaela, who died of lung cancer last year, and continues his work on research into the condition that disabled his son.

And Lorenzo's Oil itself?

It has not proved to be a cure for adrenoleukodystrophy, but a major study scheduled to end this year may tell doctors whether ingesting the oil can prevent or delay the disorder's devastating symptoms in some patients.

In the 1992 film made in Pittsburgh, the oil was depicted as a breakthrough that protected children against the horrors of ALD, a genetic disorder characterized by the deterioration or loss of the myelin sheath surrounding nerve cells in the brain. The condition, which occurs only in males, destroys the nervous system and often leaves children totally disabled. They may die between a year and 10 years after the onset of symptoms, although in a few cases, they live much longer.

That's been true for Lorenzo Odone, the Fairfax, Va., boy diagnosed with the disease in 1984, when he was just 5.

But even though his life has been extended, Lorenzo's condition hasn't changed much from what was depicted at the end of the film. Still unable to see and requiring constant care, he lies in bed and enjoys being read to and listening to music, said Augusto Odone.

The brain damage occurs because the enzyme that breaks down very long chain fatty acids in the body is impaired in those with ALD. Because of that, the body can't properly metabolize the acids and they accumulate and become toxic to the central nervous system.

The film depicted how Lorenzo's parents -- played by Nick Nolte and Susan Sarandon -- battled the medical community to develop a treatment for the disease that combined two monounsaturated dietary oils. The treatment changed the fatty acid composition of Lorenzo's blood, and Lorenzo then gained the ability to communicate by rolling his eyes and twitching a finger.

While scientific studies have verified that the oil normalizes the level of very long chain fatty acids in the blood, it doesn't appear to affect brain chemistry in children who already have developed the crippling symptoms of the disease.

Nearer to an answer

But the question that might finally be answered by the end of this year is whether the oil can prevent boys from developing symptoms -- a hope raised at the end of the film.

"It doesn't prevent ALD absolutely," said Dr. Hugo Moser, a professor of neurology and pediatrics at Johns Hopkins University who was portrayed in the film by Peter Ustinov under the name Professor Nikolais.

Moser noted that some children taking Lorenzo's Oil still develop symptoms. "The question we are trying to determine is whether it reduces the frequency of later neurological developments."

His study involves about 250 boys. In most clinical trials, part of the group takes a placebo, but in this study, all of the patients are taking Lorenzo's Oil because parents demanded that the boys all have access to the treatment.

To evaluate whether the oil is having a preventive effect, Moser will compare the group with historical estimates that about 35 percent of people with ALD will develop symptoms by 10 years of age.

There have been other benefits from the Odones' work. As head of a foundation that funds research into diseases that destroy myelin, Augusto Odone is helping pay for a new, small study at Yale University in which doctors will transplant Schwann cells, which create myelin sheaths for peripheral nerves, into patients with multiple sclerosis.

MS also involves a loss of myelin, and while its effects usually aren't as devastating as those of ALD, it strikes far more people -- an estimated 250,000 to 350,000 in the U.S. -- and can cause progressive loss of motor functions, balance and memory.

If the Schwann cell transplants help MS patients, they could conceivably help ALD patients as well.

In addition, Moser has used some of the Odone group's money to create genetically engineered mice with ALD symptoms, and has submitted a funding request to the National Institutes of Health to study an experimental treatment for ALD, based on the mouse work.

Since the movie was released, scientists have developed a better understanding of how many people are affected by ALD, Moser said.

Some patients get severe symptoms similar to those shown in the film, in which boys by age 10 are bedridden, blind and unable to swallow. But others have a milder form of the illness and can live to 70 or 80. The problem is, no one can predict which patients will get which form. This is especially confusing because different members of the same family can get different forms of the disease, Moser said.

The film ended with several interviews of boys who were symptom-free and claimed to have been saved by drinking Lorenzo's Oil, which is a combination of the oleic and erucic acids found in cooking oils. But Moser said those boys, some of whom have remained well, might not have been destined to have the severe form of the disease.

One issue ALD researchers have promoted is generalized newborn screening that would test for the presence of abnormal fatty acids in the blood. Some experts used to think that generalized screening for ALD would be unrealistically costly because of how rare the disorder is, but scientists now believe 1 in 17,000 boys gets the disorder, compared with the previous estimate of 1 in 50,000 or 100,000.

If screening were to be done, though, it would take a new technique. Infants currently are tested for 32 inherited conditions by having a blood sample blotted on filter paper. The trouble with using that method for ALD, though, is that the filter paper itself contains the very fatty acids that are markers for the disease.

Damaged relationships

When the movie "Lorenzo's Oil" came out, it strained the relationship between the Odones and Moser, and it has taken years for that tension to ease.

Ustinov's depiction of Moser was hurtful to the researcher, said Dr. John Barranger, a geneticist at the University of Pittsburgh who is also a board member of the United Leukodystrophies Foundation, a support group for patients with ALD and similar conditions.

"They made him out to be sort of a bumbling, not very caring person," Barranger said. "That conflict in the movie between the community and the bad research physician is something that sells movies. It's unfortunate because it kind of broke his heart."

The film also outraged parents of children who were taking the oil but not seeing good results, said Paula Brazeal, who runs the ULF and was also depicted negatively in the film, although her character had another name.

"The movie didn't last long because every community they went into, the parents were up in arms with the press saying, "Here's my child, he's not cured,' " said Brazeal, of Sycamore, Ill. Conversely, she said, "there are children who have lived as long as Lorenzo or maybe longer and never had a drop of the oil."

By now, the controversy that surrounded the film has mostly given way to shared hopes in research.

Dr. Timothy Vollmer, director of the Multiple Sclerosis Research Center at Yale University, is on the verge of beginning the Schwann cell study. The study, which is limited to a small number of patients who live near Yale, will look at whether Schwann cells that make myelin in nerve cells of the ankle can be transplanted into the brain or spinal cord to create myelin in the central nervous system.

"This is just to see if it is safe and if the Schwann cells survive," Vollmer said.

Odone proudly points to Vollmer's project as one example of research backed by the Myelin Project, an international group of families struck by demyelinating diseases that have come together to spur research efforts. With headquarters in Washington D.C., the project has spent around $3 million to finance 21 experiments since 1990.

In the film, the Odones are depicted bringing together a work group of international scientists to discuss approaches to ALD and related diseases. The Myelin Project continues to bring these researchers together -- the next meeting is in September.

"The Myelin Project has redirected one small area of research from theoretical to practical and from competitive to team research," Odone said.

The flow of research money also has helped build bridges between Odone and Moser -- the Myelin Project provided some of the funding he used to genetically engineer mice that carry the disorder, Moser said.

One bleak spot in Odone's life has been Michaela's death, but he said he has been helped by an old friend, Oumouri Hassane, the young African man depicted in the film as coming to the United States to help care for Lorenzo.

The Odones met Hassane when the family lived in the Comoro Islands off the coast of East Africa as part of Augusto's job as an economist with the World Bank. Hassane has taken over as primary caregiver for Lorenzo.

"He's the only person in the world that I would entrust Lorenzo to," Odone said.

Odone retired from the World Bank in 1987 because caring for Lorenzo precluded the travel his work required. He spends about 70 percent of his time on the Myelin Project and the other 30 percent doing work for his own consulting firm.

Moser doesn't dwell on his past conflicts with the Odones when talking about the history of Lorenzo's Oil.

"It's a very complicated situation, but on the whole the movie has been a benefit," he said.

Odone also has kind words for Moser, but he still believes medical researchers have wrongly dismissed his and his wife's contributions to treating ALD.

"The tendency of medical people is to say Lorenzo's Oil doesn't work because it was a layman who invented it," Odone said. "When we first appeared on the scene, everyone and his brother was trying to normalize very long chain fatty acid levels" in the blood. "I was the first to succeed."

He believes the movie also showed how consumers of health care can have an impact on a disease.

"The real value of the movie has been to show people that in cases where you have a disease in the family or yourself, you have to be proactive -- don't wait for doctors to tell you what the remedies are," he said. "It might be there is a doctor in Australia or France who has the answer, but you never know if you don't get in touch with them. I think the movie contributed to the changing relationship between doctors and patients."



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