Practical Session 3 – Group 2

The keys to this practical session can be found at this link.

Lorenzo's Oil (1992)

The movie Lorenzo's Oil is based on true events and is an extraordinarily useful material to think about the ethical issues raised regarding the treatment of adrenoleukodystrophy (ALD), for which there is no known therapy. In the following video, the disease and its symptoms are briefly explained:

Augusto Odone and Michaela Teresa Murphy Odone are Lorenzo's parents, the child affected by ALD and star of the film. Augusto and Michaela refused to accept the rapid fatal outcome of their child's illness and began a desperate seek for a treatment. After many hours of research and study, The Odones discovered an oil blend which slows the degenerative process of the disease. The treatment is called since then "Lorenzo's Oil". In recognition of the work of Augusto and Michaela, Augusto received an Honorary Doctorate from the University of Stirling (Stirling, Scotland, UK). In this tender picture we can see Lorenzo with his father, Augusto:

ALD treatment

1. Lorenzo’s oil with a diet low in very-long chain fatty acids (VLCFA). VLCFAs decrease in blood and prevents disease in a large number of cases. Its effectiveness is greatly reduced as the disease progresses.

2. Bone marrow transplantation. This treatment may be beneficial in patients with moderate neurological symptoms. It has been successful in children with early symptoms of cerebral demyelination.

3. Immunostimulatory therapy. The treatment involves applying high doses of intravenous immunoglobulins with dietary therapy. The results to date have not been very encouraging to stop the progression of the disease.

4. Immunosuppressive therapy. The aim of this treatment is to reduce inflammation of the white matter caused by the disease, which seems to be caused by pro-inflammatory cytokines. This inflammation appears to mediate the rapid progression of neurological symptoms. However, the treatment does not seem to stop the symptoms of the disease.

5. Statins. Statins are drugs that lower cholesterol levels. Its efficacy to stop the progress of the disease has been shown to be very limited.

6. Gene therapy. This therapy is intended to incorporate, by gene therapy, the gene encoding the protein ABCD1. In combination with bone marrow transplantation it represents the great hope for the treatment of patients with ALD.

 Image taken from Cartier et al. Hematopoietic Stem Cell Gene Therapy with a Lentiviral Vector in X-Linked Adrenoleukodystrophy. Science 2009;326:818-823.

Super Size Me (2004)

Many of the obesity-associated diseases are described in the film. Furthermore, we can see how some of them are progressively developed in the protagonist of the documentary.

Both overweight and obesity are associated with an increased risk of type 2 diabetes mellitus, hypertension, dyslipidemia, stroke, other cardiovascular derangements, fatty liver, musculoskeletal disorders, sleep apnea syndrome, other respiratory disorders and development of certain types of cancer. These associations translate into decreased life expectancy associated with excess adiposity and increased mortality rates.

In this link you can see an animated infographic from the Department of Endocrinology and Nutrition - Clinica Universidad de Navarra (Pamplona, Spain), which explains very clearly some aspects of obesity. In addition, you can download the illustrative chart, shown below: