Lorenzo's oil

Lorenzo's Oil is a 1992 drama film directed by George Miller. It is based on a true story of Augusto and Michaela Odone, two parents in a relentless search for a cure for their son Lorenzo's adrenoleukodystrophy (ALD). The film was nominated for two Academy Awards.

Failing to find a doctor capable of treating their young son Lorenzo's rare disease, Augusto and Michaela Odone sought their own cure. They set out on a mission to find a treatment to save their child. In their quest, the Odones clashed with doctors, scientists, and support groups, who were skeptical that anything could be done about ALD, much less by laypeople. But they persisted, setting up camp in medical libraries, reviewing animal experiments, badgering researchers, questioning top doctors all over the world, and even organizing an international symposium about the disease.

Despite dead ends of research, the horror of watching their son's health decline, and being surrounded by skeptics (including the coordinators of the support group they attended), they persisted until they finally hit upon a therapy involving adding a certain kind of oil (actually an oil containing two specific long chain fatty acids), both isolated from rapeseed oil and olive oil to their son's diet. They contacted over 100 firms around the world until they found an elderly British chemist (Don Suddaby) working for Croda International who was willing to take on the challenge of distilling the proper formula. It proved successful in normalizing the accumulation of the very long chain fatty acids in the brain that had been causing their son's steady decline, thereby halting the progression of the disease. There was still a great deal of neurological damage remaining which could not be reversed until new treatments were found to regenerate the myelin sheath (a lipid insulator) around the nerves.

The film ends with Lorenzo at the age of 14 showing definite improvement (he could swallow for himself and answer yes or no questions by blinking) but indicating more medical research is still needed. The end credits of the film note that Lorenzo also regained his sight and was learning to use a computer.

http://en.wikipedia.org/wiki/Lorenzo's_Oil

http://rarediseases.about.com/cs/ald/a/041301.htm