It is a rare, 20-letters-and-eight-syllables-long disease, a lonely orphan in a medical system that tends to devote its big money and brainpower toward curing more widespread maladies. But now Hollywood has turned its golden light on adrenoleukodystrophy, casting Susan Sarandon and Nick Nolte as the real-life Washington-area couple who developed their own treatment for the incurable disease that doctors said would kill their 5-year-old son within two years.
Lorenzo Odone is now 14.
Why, then, has the movie about his story, "Lorenzo's Oil," which opened to generally laudatory reviews last week, received an angry thumbs-down from a renown Baltimore-based expert on adrenoleukodystrophy, or ALD, and a support group for its victims and their family members?
It is a conflict familiar to AIDS activists. With a terminal disease, whose timetable do you follow: the patient who is watching sand flow ever faster down his personal hourglass, or the researcher who must follow a rigorous, time-consuming scientific process of testing new treatments before prescribing them to patients?
"We appreciate the scientific method that has been carried on since Descartes. But we really couldn't wait for Lorenzo," said his mother, Michaela Odone, played in the movie with mother-bear ferocity by Ms. Sarandon. "What are the options for the parents of children who have been handed this sentence?"
What Mrs. Odone and her husband, Augusto, did was refuse to accept that sentence. While the couple, former Chevy Chase residents now living in Fairfax, Va., are both highly intelligent -- she is a linguist and he a former economist with the World Bank -- neither has any medical background nor training. Still, they delved through arcane medical journals for clues to the mysterious disease stalking their son, organized the first-ever ALD symposium -- which brought researchers from around the world to Baltimore in 1984 -- and, perhaps most of all, they didn't waste time worrying about stepping on any number of toes and egos as they sought a cure.
The so-called "Lorenzo's Oil" that they came up with and fed their son -- without going through the usual scientific experimentation of new treatments -- has slowed his mental and physical deterioration. (The oil reduces the levels of "very long chain fatty acids" in the blood, which in ALD sufferers accumulates and leads to neurological damage.) The Food and Drug Administration has since assigned Dr. Hugo Moser, a pediatric neurologist at the Kennedy Krieger Institute and Johns Hopkins Hospital, to test "Lorenzo's Oil" for its use in treating ALD; he is in the third year of a five-year trial involving 257 patients to date.
Dr. Moser, who in 1981 developed a blood test for identifying the disease, believes the movie offers "premature" hope to those suffering from ALD. (The defective gene which can cause ALD occurs in about one in every 25,000 male babies. Women are carriers of the gene, but it is their male offspring who can develop the disease.)
"It would be great if it were a cure. But it's certainly not a cure," said Dr. Moser, who is the basis for the movie's Dr. Gus Nikolais, played by Peter Ustinov. "We are having only moderate success with it."
Dr. Moser said that no conclusions can be drawn until the trial runs its entire, five-year course, but "Lorenzo's Oil" appears to be of greater benefit to those with moderate rather than more severe forms of ALD, he said. The greatest hope, he said, is that the oil will help boys who are currently asymptomatic from ever developing the disease, but that remains to be seen.
He is also concerned about the oil's apparent side effect discovered in some 40 percent of the patients -- a reduction in their blood platelet counts which can result in abnormal bruising or bleeding. (Mrs. Odone said she believes this is an acceptable risk, especially when weighed against the terminal disease the oil may help ease.)
While the movie pits the Odones against the medical establishment, in real life, the couple and the doctor have no personal fight.
"They are only to be admired. They're highly intelligent, energetic and totally committed to the well-being of their son," Dr. Moser said of the Odones.
"I like Dr. Moser. I hold him in esteem. If there's something wrong with my child, he is the one I will call," Mrs. Odone said.
But when it comes to the movie, or the Odones' current effort, the Myelin Project, which supports research into the regeneration of the nerve sheathing damaged by ALD, the doctor and the parents diverge.
"The movie overemphasizes the contributions they made and minimizes the contributions others have made," said Dr. Moser, who has treated Lorenzo and helped the Odones organize the Baltimore symposium on ALD. "To put me as a person who sits around diddling with statistics instead of helping people is incorrect."
In the movie, Professor Nikolais is a sort of stand-in for a medical establishment that moved too slowly for the Odones' needs, faced as they were with saving their beautiful and precocious son from the rapid deterioration caused by ALD.
But while the medical community takes its hits in the movie, the most negative portrayal is directed at a group based on the United Leukodystrophy Foundation, which offers support to victims and parents and raises funds for research.
"It goes beyond poetic license," said Ron Brazeal, executive director of ULF and a father who lost both his sons to ALD. "It's going to undermine something we've worked years and years to build.
"I'm not an egotist. I'm just a country boy out here and what people in Baltimore or Houston or whatever think about me isn't going to make the corn grow any faster here in Illinois," said Mr. Brazeal, who believes he and his wife are the model for the Muscatines, the couple in the movie who run the group.
The movie portrays a couple heading the group as rather petty people, stuck on the rules of order for conducting their meetings and unwilling to let the Odones spread the news of the oil that had helped their child.
Conflicts are inherent when a couple like the Odones, single-mindedly searching for a cure for their still viable son, join a group that focuses much of its efforts on providing grief support for parents of sons already lost.
Mr. Brazeal's main concern is that the negative portrayal of the foundation will hurt the group's fund-raising, and thus the amount of research it can help finance and the support it can provide to the afflicted and their families. Additionally, he and Dr. Moser believe the Odones' Myelin Project -- which is promoted with an 800-number at the end of the film -- will siphon money that instead could go toward other ALD research.
"We're in the here and now. There's no form of research that doesn't deserve attention, but some deserve it more than others," Mr. Brazeal said. "The Myelin Project is not going to help anyone who is alive now."
Mr. Brazeal is more hopeful about other possible treatments for ALD such as bone marrow transplantation; immunosuppression, much like chemotherapy is used for cancer; and genetic therapy, especially now that the defective gene that causes the disease has been isolated.
But his here-and-now argument is also the Odones' argument: They believe myelin regeneration research is advancing quickly enough that human trials can begin within a year on patients with less severe forms of the disease and within two years on children like Lorenzo.
As for the way the movie portrays Dr. Moser and the United Leukodystrophy Foundation, Mrs. Odone said she can't answer for Hollywood and its use of symbolic representations rather than documentary accuracy. (And even Mrs. Odone will say she isn't portrayed to a T, saying she never threw her sister out of the house or slapped her husband as Susan Sarandon does in the movie.) But she agrees that she and the foundation have different approaches.
"Did we have time for the scientific establishment to go through its orderly pace? Did this parents group want to adhere to the needs of science, or envision a broader role?" she asks rhetorically. "We were raising issues; we continue to raise them. Do you really want to use the same methods for testing a therapy for a terminal disease as you use for a . . . cold?"
It is an issue that goes beyond ALD. Mrs. Odone draws parallels to AIDS research and the need to offer experimental treatments to those without the time to wait for protracted experiments.
At the heart of the Odones' work, however, is their own son, Lorenzo. Mrs. Odone proudly notes that he can communicate through finger wiggles, eye blinks and head turns -- they've developed an entire vocabulary based on which finger he wiggles and how vigorously he blinks his eyes. To keep him mentally alert, Mrs. Odone will ask him questions in French (the family once lived in a French-speaking island off the coast of East Africa) and continually challenge him to greater and greater skills.
"We're two exhausted, determined people, but we're going to keep moving on," Mrs. Odone said of herself and her husband, who retired from the World Bank and now has an international consulting firm. "If Lorenzo can do it, so can we."