Lorenzo’s Oil: Myth or Miracle?
By Heather Denkhaus
Course: Biology 30
Unit 1: Systems Regulating Change in Human Organisms
1. The human organism regulates physiological processes using electrochemical control systems
·
Explaining
the significance of sex chromosomes
·
Predicting
sex-linked inheritance data
·
STS
connections: treatment of human genetic disorders
In 1984, Lorenzo Odone, a happy and healthy six-year
old boy, mysteriously begins to fall down, slur his words, and have
difficulties hearing. After a series of tests, his parents Augusto and Michaela
learn the horrifying truth: their son has Adrenoleukodystrophy (ALD), a rare
and fatal sex-linked recessive disorder. The doctors give their son just two
years to live.
The Odones refused to believe that ALD was incurable and set about to educate themselves and to find the cure that would save their son. Neither Micaela, an editor, nor Augusto, an economist, had “an ounce of medical knowledge”(Romney, 1993) and often felt scorned by the medical community. Michaela recalls that she and her husband were told by doctors that no layman could possibly understand the medical research (Adler & Hager, 1987).
Despite the considerable challenge, Augusto and
Michaela read everything they could and learned about the disease that was
killing their son. The disease attacks the myelin, a fatty covering which
insulates nerve fibers, slowing and eventually stopping transmissions in the
nervous system. ALD patients have a high concentration of very long chain
saturated fatty acid molecules in their blood. These saturated VLCFA molecules
are found in the diet and are synthesized by the body. The enzyme that breaks
down these molecules in healthy individuals appears to be impaired in patients
with ALD (Adler & Hager, 1987). These saturated VLCFA’s destroys the
patient’s myelin.
The Odones continued to research ALD and came to realize that the handful of experts in the field were working in isolation and had never come together to share their knowledge with one another. Augusto poignently described the difference between himself and the researchers, “they were looking at ALD as a route to the Nobel Prize 20 years down the road, while I was watching my son die.” (Adler & Hager, 1987). In October 1984 the Odone’s brought together researchers from around the world for the first world ALD conference.
Through their research the Odone’s discovered that there are “bad” saturated VLCFA’s and “safe” unsaturated VLCFA’s and that the same enzyme synthesizes both. Augusto and Michaela were inspired. They found an edible oil that if ingested would keep the enzyme busy creating the “safe” unsaturated VLCFA’s hoping that that would reduce the amount of “bad” saturated VLCFA’s in Lorenzo’s blood.
They had to find a suitable source of oil and convince the doctors and themselves that the oil was safe to ingest (it had been shown to cause heart disease in animals but not humans) so they tested the oil on Lorenzo’s aunt, a carrier of the disease who had elevated levels of saturated VLCFA’s in her blood. The oil did not harm her and did in fact lower her saturated VLCFA levels. Treatment of their son using this oil later dubbed “Lorenzo’s Oil” began.
Lorenzo turned 22 years old in 2000 (Kugler). He has not recovered in a large way but he is still alive and can communicate with winks and hand signals, something that was not possible before treatment with “Lorenzo’s Oil”. However the oil may not be the miracle it appeared to be, medical research has shown that for 96 boys suffering from ALD like Lorenzo the oil does not alter the course of their disease (Coleman & Concar 1993). There is some promise that the oil helps people suffering from a milder form of ALD called adrenomyeloneuropathy and that it may help patients if treated before any symptoms appear.
“Lorenzo’s Oil” may also be harmful. The taste is described as unpleasant, much like engine oil (Coleman & Concar 1993). The oil also hinders blood clotting in about half of the patients who take it leading to slow-healing cuts and bruises. Many ALD researchers condemn the portrayal of the Odone’s story in the movie “Lorenzo’s Oil” as creating false hope and doctors say they are pressured by parents to provide the Odone’s oil treatment despite knowing that it will not halt the disease.
There continues to be progress in ALD research. The Odone’s have created “The Myelin Project” an organization that “refuses to accept the conventional view that science can not be hurried” and that “aims to accelerate the research on myelin repair” (The Myelin Project Web site). For example, Scientists can now predict with 99% accuracy whether or not a woman is a carrier of ALD (ScienceDaily Magazine, 1998).
1)
Adrenoleukodystrophy
(ALD) is an X-linked recessive disorder. What are the genotypes of Augusto,
Michaela, and Lorenzo with respect to this disorder?
2)
What
are the advantages and disadvantages of having someone personally affected by a
disease spearhead research to find a cure for that disease?
3)
Is
it reasonable / ethical that less money is available to fund research for rare
diseases than for more common and / or more visible diseases? What would the
impact of “Lorenzo’s Oil”, a high-profile Hollywood movie about ALD, have on
research funding for ALD? Is that impact reasonable / ethical?
4)
Would
you want to know whether or not you / your wife was a carrier of ALD? If you /
your wife was a carrier of ALD would you still have children?
5)
If
you had a son afflicted with ALD would you want him to receive “Lorenzo’s Oil”?
Adler,
J., & Hager, M. (1987). They won’t let their son die. Newsweek, 110(20),
98-99.
Adler,
J., & Hager, M. (1993). Loving Lorenzo. Newsweek, 121(4), 48-49.
Coleman,
J., & Concar, D. (1993). Pouring cold water on Lorenzo’s Oil. New
Scientist, 137(1863), 23-24.
Kugler, M. (n.d.). ALD and Lorenzo’s Oil: Disease still not fully understood. Retrieved Jan 20, 2002 from Rare / Orphan diseases Web site: http://rarediseases.about.com/library/weekly/aa041301a.htm
“Lorenzo’s Oil.” (1992). MCA Universal Studio.
The Myelin Project. (n.d.). Retrieved January 20, 2002, from http://www.myelin.org/
New test spots carriers of ALD – Disease featured in “Lorenzo’s Oil” – with near perfect accuracy. (1998). Retrieved Jan 20, 2002 from ScienceDaily Magazine Web site: http://www.sciencedaily.com/print/1998/10/981028120620.htm
Romney, J. (1993). Medical nemesis. New Statesman & Society, 6(241), 33-34.
Two Web sites
that focus on “Lorenzo’s Oil” as a teaching tool
Gostinger, L. (1994). Adrenoleukodystropy (ALD): A case study using the film “Lorenzo’s Oil”. Retrieved Jan 20, 2002, from Woodrow Wilson Biology Institute Web site: http://www.accessexcellence.org/AE/AEPC/WWC/1994/adreno.html
Stith, B. (n.d.) The use of the movie “Lorenzo’s Oil” as a teaching tool. Retrieved Jan 20, 2002 from University of Colorado Web site: http://carbon.cudenver.edu/~bstith/loren.htm