A clinical trial evaluates the utility of genetically altered autologous hematopoietic stem cells for the treatment of childhood cerebral adrenoleukodystrophy, a deadly disease portrayed in the movie Lorenzo’s oil. The interim results are encouraging.
In 1984, at the age of six, Lorenzo Odone was a happy, bright, precocious kid, who was fluent in English, French and Italian and loved Greek mythology. However, suddenly that year he appeared to become hard of hearing, started to stumble a lot, and displayed mood swings. Concerned, his parents took him to a doctor who quickly ordered a brain scan. The result was devastating – Lorenzo was diagnosed with childhood cerebral adrenoleukodystrophy (CCALD), a disease that is rare, fatal, and untreatable. Doctors gave him two more years to live. The courageous struggle of his parents to find a cure was portrayed in the 1992 movie Lorenzo’s oil. The real life Lorenzo beat the odds by remaining alive until 2008. However, his disease never relented.