'We Cannot Stand By and Watch Them Die': Doctors Use Untested Medication for Deadly E. Coli
US drug manufacturer Alexion is sending free medicine to Germany to help doctors battling the most severe cases in the current E. coli outbreak. But the extent to which Eculizumab can help remains unclear.
Doctors in Germany have administered the largely untested drug, Soliris, to their most severe cases.
There is no effective treatment for E. coli patients who are currently suffering from epileptic seizures, kidney failure or strokes. So far, doctors in Germany have primarily used dialysis in an attempt to remove the bacterial toxins from the body as they seek to treat the widespread outbreak of a particularly deadly version of the E. Coli bacteria.
The hope surrounding this remedy is largely due to the work of Dr. Franz Schäfer, a nephrologist at the University of Heidelberg. Last fall, the condition of one of his young patients, three-year-old Sophie, was getting progressively worse. The girl was infected with E. coli, suffering from seizures and hemiplegia, and blood plasma exchanges offered no improvement.
"Finally, we placed all our hopes on one possible solution and gave her Eculizumab," Schäfer says.
The drug is known among kidney researchers, but has been neither tested nor approved for treating E. coli infections. Schäfer was surprised at how well young Sophie responded: Within 24 hours, the girl's condition had improved dramatically. After three days, dialysis was no longer necessary, and after nine days Sophie was discharged from the hospital.
"I'm still in touch with the family," says Schäfer. "Sophie is attending preschool just like any other child her age."
Doctors Desperate for a Cure
Schäfer was so impressed with Eculizumab that he offered to write an article about the case for the prestigious New England Journal of Medicine. The magazine had scheduled to publish Sophie's case, along with two similar stories about patients from Paris and Montreal, in a few weeks. But then came the E. coli alarm in Germany, and the editors decided to immediately put Schäfer's report online on May 25.
Many doctors currently treating patients infected with E. coli are desperately clinging to this medical article -- although the description of a cure for three children does not allow for more generalized statements about the drug's effectiveness. Nonetheless, it is currently being used in all hospitals with severe cases. At Hamburg's University Medical Center (UKE) last Friday, 49 patients were treated with Eculizumab, while 28 in Kiel and 20 in Hanover were administered the drug.
Professor Rolf Stahl at UKE says: "We are treating the patients with this drug because we cannot simply stand by and watch them die, suffer damage to their central nervous systems, or lose their kidney function." Stahl and his colleagues in Kiel and Hanover, however, are not observing such resounding successes as reported by their colleague Schäfer in Heidelberg with the three-year-old Sophie. A marked improvement after 24 hours? Stahl merely shakes his head in disbelief.
A Risky Medical Experiment
Eculizumab is one of the most expensive drugs in the world. A month of treatment in Germany costs over 37,000 ($54,000) -- per patient. The drug is only approved for a rare blood disease called paroxysmal nocturnal hemoglobinuria (PNH). All severely ill E. coli patients who want to receive this medicine have to be advised that they are participating in a risky medical experiment.
Eculizumab is produced by the US pharmaceutical company Alexion, which reported sales of over $540 million (372 million) last year with the product, a 40-percent increase over the previous year. After the outbreak of the current E. coli crisis, the company agreed to supply Eculizumab to Germany for free. Alexion Vice President Thomas Bock tells SPIEGEL that if the numbers of patients were to sharply rise in Germany, even a fivefold increase in the amount delivered would be no problem.
"We are currently communicating on a daily basis with the physicians and authorities in Germany, but we also don't know what is ideal," Bock admits.
Bock justified the medicine's high price with the company's extremely high-risk strategy of only developing drugs for rare and hopeless diseases.
Eculizumab, however, is the only drug that Alexion markets.
Translated from the German by Paul Cohen.
© SPIEGEL ONLINE 2011
All Rights Reserved
Reproduction only allowed with the permission of SPIEGELnet GmbH
The rotavirus is the most common cause of viral gastrointestinal disease in children. Ninety percent of children catch the virus before age three. Life-threatening dehydration is a possible effect. Other causes for gastrointestinal illnesses are salmonella and staphylococci.