'We Cannot Stand By and Watch Them Die': Doctors Use Untested Medication for Deadly E. Coli

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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. Zoom
DPA

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.

For several days now, hospitals have been experimenting with a largely untested drug called Eculizumab, which has the brand name Soliris. It remains unclear whether it can help.

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 says that the effect on blood vessels can last for months, even if patients with life-threatening hemolytic-uremic syndrome (HUS) are apparently free of symptoms. About one-third of those infected in the E. coli outbreak are suffering from HUS, which can lead to kidney failure. When asked whether free deliveries of the drug would also apply for treatments that last for months, Bock replied: "Our commitment is to do whatever is necessary to overcome the crisis."

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.

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Info Graphic
DER SPIEGEL
Unleashed Aggressor: How the E. coli serotype O104:H4 attacks the body
What to Know about E. Coli
Which E. coli strain is causing the outbreak?
There are hundreds of different types of Escherichia Coli, E. Coli, strains, most of which are harmless for humans. Strains are differentiated by their so-called serotypes -- variations within a subspecies of bacteria -- usually with the designations "O" and "H". "O" indicates the lipopolysaccharides, a type of sugar-fat molecule, found on the outer surface of the bacteria; "H" stands for the type of flagella found on the bacteria (flagella are the hairs the bacteria uses to move itself). E. coli bacteria are designated as enterohemorrhagic when they lead to bloody diarrhea in humans. This occurs because some subtypes, like the one currently causing panic in Germany, produce a certain poison, so-called Shiga toxins. The E. coli strain linked to the outbreak in Germany is being identified as E. Coli serotype 0104:H4. Experts have been especially surprised by the strain's aggressive nature and rapid spread. They first thought the strain to be a mutation of serotype 0104:H4. But initial genetic analysis suggests that the bacterium in question is actually a unique variant of the strain: According to the World Health Organization, this type of Enterohemmorhagic E. Coli (EHEC) has been seen in humans before but has never before been linked to an outbreak.
How can the risk of disease be reduced?
There is no vaccine currently available and treatment with antibiotics can be problematic. The reason: the E. coli bacteria in question release a dangerous (Shiga) toxin into the human body, and antibiotics can actually increase the amount of poison released. Good hygiene is one way to significantly decrease the risk of E. coli infection. Facial contact should be avoided after having handled animals or touched the floor. Raw meat and easily-spoiled foodstuffs should be stored at appropriate temperatures in a refrigerator or freezer. When cooking, foodstuffs should be cooked through in order to kill bacteria (at least 10 minutes at 70 degrees Celsius, 158 Fahrenheit). Wash hands regularly. Cutting boards, dishes and utensils should all be washed thoroughly. Avoid unpasteurized milk and unpasteurized milk products.
How can I tell if I've been infected by the E. coli bacteria?
The most common symptom of E. coli infection is heavy, and possibly bloody, diarrhea. Other possible symptoms include nausea, vomiting and, in rare cases, fever.
What can doctors do in the case of 0104:H4 infection?
The incubation period of O104:H4 is 10 to 13 days. Characteristic symptoms of the strain include bloody diarrhea, kidney failure and anemia. Experts have linked O104:H4 to haemolytic uraemic syndrome (HUS), which can lead to kidney failure and even death. With current knowledge, experts do not yet have the means necessary to effectively combat the O104:H4 pathogen. They are therefore treating the typical complications and symptoms as a whole, for instance through plasmapheresis, essentially a blood plasma transfusion. Some of those who are currently infected are also undergoing an experimental therapy of antibody treatment.
What should be done if symptoms arise?
In the case of serious diarrhea, saline- and fluid-loss need to be compensated for. If serious symptoms appear, medical treatment should be sought. In order to counteract potential complications as quickly as possible, those who are in extreme danger -- infants, small children or the elderly -- should be treated in a hospital.
What are other causes of gastrointestinal illnesses?
Gastrointestinal illnesses are among the most common infectious diseases in the world, and can be caused by germs other than E. coli. The norovirus circulates throughout the year and reaches its peak during the winter months. Its incubation period, at least six hours, is short. The norovirus also causes abdominal pains and fatigue, and is primarily spread via humans. During peak periods of illness, the victims are highly contagious.
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.


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