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Plantilla, Ryan Gabriel G.

3Chem
Lorenzos Oil

Adrenoleukodystrophy (ALD) is a rare x-linked genetic brain disease


that is caused by the mutation in the ABCD1 gene that is located in the
Xq28; this disease roughly affects 1:15000 people. ALD has a defect in the
peroxisomal ATPase Binding Cassette Protein (ALDP) resulting in defective
peroxisomal beta oxidation and the accumulation of very long chain fatty
acids; it usually affects the myelin, adrenal cortex and Leydig cells in the
testes. The movie started by showing a healthy boy named Lorenzo, but as
time passed by Lorenzo began to show unusual behaviors and symptoms
that are bothering. His parents then decided to consult doctors about
Lorenzo. After several and multiple tests, they found out that Lorenzo has
ALD. Lorenzo was signed-up by his parents in clinical trials in order for
doctors to observe and study the disease. A restricted very low long fatty
acid diet was imposed on Lorenzo to lessen the accumulation of the long
fatty chains in his brain; but this diet has only minimal effects. The Odones
tried to research on their own on how to lower the long fatty chain without
harming the function of the body. They researched all day and all night in the
libraries in search for journals and articles that can help them, until one day
they found an article that is almost similar however rats were used in the
experiment. They managed to create a symposium with the top doctors and
scientist all over the globe to discuss what treatments could be done and
would collaboration be possible. The symposium lead to the use of oleic acid
in pure form, when the Odones tried it to Lorenzo his long chain fatty acid
declined up to 50%; however this is not enough to stabilize Lorenzo. A call for
further research was conducted and they found that erucic acid was also
helpful; they hoped that when oleic and erucic acid when mixed in right
proportions it will help to stabilize the long chain fatty acid of Lorenzo.
Unfortunately, erucic acid is not commercially available since there is no
market for it plus it is not FDA approved. They reached out to hundreds of oil
companies but all rejected the project until one chemist accepted the project
and isolated erucic acid from rapeseed oil. When erucic acid was produced,
the Odones mixed it with oleic acid and lucky it stabilized the long chain fatty
acids of Lorenzo. This oil was named Lorenzos Oil; it is now an accepted
treatment for ALD; however it must be taken under strict observation of a
physician because it is still not FDA approved. This movie showed us the
commitment of the Odones in finding a treatment for their sick child. It also

gives us the idea that when different branches of science works and
collaborates together a great result will happen. Like what the scientific
community wants, we must promote open science to achieve the impossible
and to cure the incurable. The main lesson of the story is that you dont have
to become a doctor or a scientist to make a difference, you just need to
believe, trust and love what you do and do what you love in order to make a
difference.

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