The true story on the oil made to treat boys with ALD[Adrenoleukodystrophy]...?I have to write a one page essay on this.. my topic is-"Would it have been appropriate for Medical researchers to take the kind of chances with their patients that the Odones took with Lorenzo?Lorenzos Oil has side effects. The oil reduces blood platelet count[thromboytopaenia]-what medical problems would this produce?"
I would love any ideas at all!It just needs to be a page,and I need 3 reasons that support the theory.I would really like some help understanding this section of the movie.And perhaps some ideas to answer this question. Please help!
2007-03-21 13:08:06 · 6 answers · asked by ♥ 4 in Health ➔ Diseases & Conditions ➔ Other - Diseases
Hope this gives you some insight to get started...it sucks but you can do it!
Adrenoleukodystrophy (ALD) is a rare inherited metabolic disorder characterized by the loss of the fatty covering (myelin sheath) on nerve fibers within the brain and progressive degeneration of the adrenal gland. The basic defect is the impaired capacity to degrade very long chain fatty acids that are found in the blood plasma and tissues of the body. These fatty acids accumulate in the brain (cerebral white matter) and the adrenal glands.
ALD is an X-linked recessive inheritance of a syndrome of Addison disease and cerebral sclerosis. It only affects males although neonatal autosomal ALD may also affect females. This neonatal form is clearly separate from the X-linked ALD. Symptoms often appear in boys between the ages of 4 and 8 and may include behavioral changes such as poor memory, loss of emotional control and dementia. Other symptoms may include muscle weakness, difficulties with hearing, speech and vision.
The symptoms of ALD may develop due to abnormal or absent peroxisomes (microbodies that participate in the metabolism of fats) in the liver. This causes a disturbance of fatty acid metabolism and results in the abnormal accumulation of very long chain fatty acids. The exact enzyme deficiency that prevents the breakdown of VLCFAs is not known.
ALD is an allelic mutation. This disease is caused by a single mutation at the locus Xq28. There is a close linkage of ALD to the cluster of color-blindness genes. This single mutation causes deficient activity of the necessary enzyme ligase. There is a 50% chance the disease will be passed from mother to offspring. The male will express the disease and the female will be a carrier.
Diagnosis can be made from cultured skin fibroblasts or amniotic fluid cells and allows prenatal diagnosis and carrier identification of 90% of obligate heterozygotes. More recently it has been shown that the biochemical diagnosis can be performed on chorionic villi biopsy.
This disease can be prevented with a combined approach of oleic acid (Lorenzo's Oil) and a restricted diet minus very long fatty acids. Also some ALD patients have been treated experimentally with Glycerol Trioleate. More research is needed to determine the long-term safety and effectiveness of these substances.
The prognosis is excellent for those individuals who are identified prenatally or shortly after birth. There can be normal functions for those individuals who take the prescribed dose of oil and follow the restricted diet regimen.
If the myelin can be restored, patients like Lorenzo can lead a normal life. The conclusion relates that Lorenzo can communicate by using a computer and his father, Augusto Odone, has been awarded an honorary Ph.D. for his pioneering work in researching and discovering a significant treatment for ALD.
2007-03-21 13:26:46 · answer #1 · answered by Anonymous · 0⤊ 0⤋
The movie was based upon true events. I was a pediatric neuro-nurse when the movie came out. All the pediatric neurologist were talking critiquing the movie with positive reviews. There are side effects associated with Lorenzo’s Oil. It causes thrombocytopenia - low platelets. Platelets help our blood to clog. Low platelets can cause severe bruising, bleeding gums and nosebleeds. See the link below for details.
As far did the researchers take chances on the patients by having control groups: you have to have an understanding on how to appropriately do scientific testing. Control groups are important to get a true result of what % responds to the medication. There could be environmental factors that impact drug testing. Not just local factors i.e. dust but sunlight. In the name of science controlled testing is important. Would I have wanted my child in the controlled group… no way. Good luck on your document.
2007-03-21 13:57:11 · answer #2 · answered by Nanni 2 · 0⤊ 0⤋
Yes, it's been such a long time since I've seen the movie, but I recall it to be a very moving movie. My response isn't going to be that helpful because it's been a while since I've seen the movie, and I know very little about ALD. Honestly, it must depend on the situation. In some cases when life is surely slipping and virtually no alternative exist, at least sure alternatives, taking great risks as taken in Lorenzo's Oil may be an appealing option, especially if the risk has some chance of preserving life, and especially leading to a future cure that may prevent and preserve future cases. Ideally, I still believe it is best if we first experiment on animals until we determine how safe the alternative is on human life.
2007-03-21 13:17:38 · answer #3 · answered by Anonymous · 0⤊ 0⤋
Yeah, that movie gave me a really bad headache lol. I love Will Ferrel, he's super funny.
2016-03-28 22:52:07 · answer #4 · answered by Anonymous · 0⤊ 0⤋
Yes, it's an amazing story. Sorry, can't help you with the medical part.
2007-03-21 13:16:52 · answer #5 · answered by Lydia 7 · 0⤊ 0⤋
There is a film called that... saw it on TV a couple of years ago, but can't help beyond that info... sorry!
2007-03-21 13:11:09 · answer #6 · answered by waynebudd 6 · 0⤊ 0⤋