Lorenzo’s Oil is a movie about Augusto and Michaela Odone’s experience with their struggle to find treatment for their son, Lorenzo, after he was diagnosed with a rare disorder called adrenoleukodystrophy (ALD) at the age of 6. There was little available research and knowledge about how to treat it when he was diagnosed. ALD causes demyelination of the nerve cells of the brain which results in a loss of brain function.
The movie presents two parallel stories. First – what would you do if your child was given two years to live? In this case, the Odone’s pursued, and discovered, an oil that helped children with ALD.
The second theme in the movie which presents a compelling and relevant issue for us to consider – if there is an intervention for a patient with a serious medical condition, should it receive testing in a clinical trial before being offered, especially to children, on a widespread basis? In this case, the intervention was an oil, which seemed especially safe, but the intervention was in a pediatric population.
A more modern-day example was the case of bone marrow transplants for women with breast cancer – doctors provided bone marrow transplants to women with advanced breast cancer (and patients insisted that they receive them) before all of the data were in about their efficacy. Avastin in breast cancer is another example.
In Lorenzo’s Oil, a scientist is on trial for advocating a clinical trial to test the oil, and for pursuing a test for ALD. He is criticized for pursuing the scientific method, and for not moving fast enough to help boys suffering with the disease.
You can read about this scientist here, and what happened regarding his discovery.
The challenge we have to help patients now, and pursue research to help patients in the future is very real. The fact that most research dollars go to help very few diseases means that when a scientist is funded the challenge to best manage their research funding is a huge responsibility.