Posted on 09/27/2002 4:32:30 PM PDT by blam
Lorenzo's oil finally proven to work
17:55 26 September 02
NewScientist.com news service
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.
David Concar
This week it was mentioned that the "fat mice" are reponding to a gene therapy which mitigates their obesity. Maybe this will solve the obesity problem although it won't make the trial laywers very happy.
BTW the mice who were bread for obesity also developed diabetes. the gene therapy whups up on both the conditions.
As a Type II, I look forward to the treatment. Back in '96 one of Newt's prognostications was that if you could treat diabetes you would be helping to reduce the Medicare costs by 25% of more since diabetes and related cardiovascular, kidney and amputation issues consumed that much of each year's Medicare expenditures. Sounds like pre-emption to me!
Sorry can't answer that. I barely remember that I saw the movie years ago.
In the movie they fed it to him. Also, note that in the article it's referred to as "unpalatable".
Go the the 'Helpful Background' portion to read up on the particulars/details. I'm not sure if this is entirely accurate, since I'm not a Biochemist.
For huge sums of cash, I could be persuaded to play a Biochemist on TV though. ;)
The oil is fed to the genetically damaged offspring. It essentially keeps the enzyme busy.
After consulting with The Myelin Project Work Group, we have decided to fund a proposal submitted by Dr. Su-Chun Zhang of the University of Wisconsin-Madison. One of the most advanced researchers on stem cells, Dr. Zhang proposes to derive transplantable oligodendrocyte precursors from undifferentiated human stem cells. Dr. Zhang has already succeeded in coaxing embryonic stem cells into becoming neural cells.
Well, after using the oil, they want to do remyelination, and they have started funding stem cell research. Answers your question about killing unborn children.
The way I see it, and I'll be the one to be heartless about it, these genetically damaged kids should not be given the oil. Lets see, they get the oil, live a semi-normal life, get remyelination, start having kids, and now you have the defective gene spreading around in the gene pool.
Yep, I'm cruel and inconsiderate, and I'm a terrible person. But isn't what I just said what the people against genetically modified foods are for? They always say, DO NOT TINKER WITH NATURE.
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