An Australian doctor, who used a German experimental drug that had been tested only on mice to save a baby’s life, left to brief US and European medical authorities about the amazing success, on Friday.
In a race against time, doctors at the Monash Medical Centre argued before their hospital’s ethics committee and finally the law courts to allow them to use the experimental drug on the baby.
The baby, known only as Baby Z, as the parents want to remain anonymous, suffered from a rare metabolic brain disease which had no known cure and her condition was worsening by the hour.
The disease stops the body expelling sulphite and causes babies to suffer increasingly severe seizures and brain damage until they die.
Baby Z was born on May 1, 2008, with the metabolic disorder which affects one in 500,000 babies and started having seizures 60 hours after her birth.
She was deteriorating fast even as doctors desperately searched medical literature in the hope that somewhere in the world there was help.
They found a medical paper written by German Plant Biologist Professor, Guenter Schwartz of Cologne University, who had been working on an experimental drug called cPMP for 15 years, but had tested it only on lab mice, the Australian Associated Press said.
It normally takes years for new drugs to get approval but Baby Z’s parents refused to let their baby die without a fight. They urged doctors to break normal medical procedures and let their baby be the human guinea pig for the unapproved drug.
The doctors contacted Schwartz, who immediately sent all his available compounds to Australia.
The parents argued before the hospital’s ethics board that their baby was about to die an agonizing death, so there was nothing to lose in giving the drug.
But the hospital insisted they also get approval from a court. A court hearing was hastily arranged and after a tense day of arguing, the judge allowed use of the unapproved drug.
The doctors, led by neo-natal specialist Dr. Alex Veldman, raced back to the hospital as the baby was fast deteriorating.
“The hospital pharmacist sprinted up the stairs with the substance in his hand and we started to treat the baby on that afternoon, actually about 40 minutes after we got the court order,” Dr. Veldman told reporters in Melbourne.
“What then happened was really amazing, because the baby was waking up, she was starting to move around, starting to look around, she was starting to drink milk from a bottle and she just improved massively.” Dr. Veldman said. The girl is now 18 months old and is doing well although she has some problems and has to be injected with Schwartz’s compound every day. “She is such a delightful little lady and the parents love her and she has a very happy life,” he said.
“The amazing thing is that I spoke to her mum and she said that she never believed that her baby would die, she always knew that she would fight for it until we find something.” Baby Z’s mother told reporters, through a telephone link-up that it was the most difficult thing anyone could go through.
“It was the most challenging and most traumatic time of our lives,” she said. She never doubted her baby should be the human guinea pig for the untried German compound. “There was courage and there was death - we opted for courage,” she said. “If she wasn’t treated, she would have experienced a very painful death.” she added.
Dr. Veldman left Australia on Friday, to present his findings to the United States Food and Drug Administration and European regulators - next week.