Baby's brain disease is 'cured' by untried drug
Friday November 06 2009
A BABY girl has become the first person to recover from a rare brain poisoning disorder after her parents begged doctors in Australia to scour the world for a cure.
The child, known as Baby Z to protect her privacy, suffered from molybdenum cofactor deficiency, which causes a build-up of toxic sulphite and began dissolving her brain shortly after she was born in May 2008.
She was given little hope but her parents refused to give up and pleaded with doctors at Melbourne's Monash Children's facility to look for a treatment.
The team searched through medical literature and discovered research into an experimental drug in a paper written by a German professor.
Prof Guenter Schwartz worked on the drug, cPMP, for 15 years but had tested it only on laboratory mice. He claimed his treatment might be able to combat the metabolic disorder which leads to brain damage.
Within hours of receiving the first dose of the untested compound, her sulphite levels fell by more than two-thirds and were at normal levels within three days. Baby Z will require injections of the drug every day for the rest of her life. (© Daily Telegraph, London
- Bonnie Malkin in Sydney
Irish Independent
