Kentucky-made drug sparks medical ethics debate

Los Angeles TimesAugust 7, 2014 

  • The threat to Kentuckians

    With the long incubation time and frightful toll of the Ebola virus, many people in the United States are increasingly asking whether the disease is likely to spread here.

    "The average Kentuckian is not at risk unless they do some traveling outside of the United States," said Dr. Kraig Humbaugh, director of epidemiology and health planning at the Kentucky Department for Public Health. He said epidemiologists across the state, including those at local health departments and hospitals, are aware of the cases in Atlanta and are on the lookout for unusual diseases that could cause a significant threat.

    In the long-shot scenario that there were a case of Ebola in Kentucky, he said, the state in conjunction with the CDC would let the public know and suggest the best ways to avoid contracting the disease.

    He said health officials in Kentucky are working behind the scenes to monitor the situation and keep the public safe.

    MARY MEEHAN

Two American aid workers were gravely ill, fighting to survive infection with the deadly Ebola virus. A San Diego drug company and its Owensboro, Ky., facility had three doses of an experimental Ebola medicine that showed promise in monkeys but had never been tested in humans.

Getting the medication to the two patients in Liberia seemed like the obvious thing to do. The Centers for Disease Control and Prevention, the National Institutes of Health and the Christian aid organization Samaritan's Purse worked together to make it happen.

Scrambling to get ahead of the Ebola crisis, the World Health Organization announced Wednesday that it was considering the declaration of an international public health emergency and would convene a panel of experts in coming days to explore the use of experimental treatments for the incurable disease.

The organization also announced 108 new Ebola cases recorded from Saturday to Monday, bringing the total to 1,711, with 932 deaths. Nearly all are in the three afflicted West African countries at the epicenter of the outbreak: Guinea, Liberia and Sierra Leone.

Patient advocates who think that the Kentucky-made drug is helpful have asked when it can be made available to the hundreds of West Africans who are ill.

But what looks like a simple case of humanitarian goodwill could lead to some unintended and negative consequences, experts said this week.

Although there could be a short-term gain for a dying patient, in the long run, it would undermine scientists' ability to determine whether the drug really was safe and effective.

"I don't think we want to say these drug companies are obligated to suddenly mass-produce these drugs," said Jennifer Blumenthal-Barby, a medical ethicist at Baylor College of Medicine in Houston. "That would subvert the whole FDA-regulated process of trying to do solid research on these drugs."

The Food and Drug Administration has elaborate rules for evaluating drugs before they are approved for widespread use. The process can take years, involving hundreds or thousands of patients and costing drug companies millions of dollars.

The rules are designed to make sure that a medication doesn't make patients more sick than they are and that it fights the disease it was created to fight. They are also used to figure out the minimum dose needed to get the desired effect.

The centerpiece of these rules is the clinical trial, which allows researchers to show that patients who took the drug fared better than patients who didn't.

In this case, there will be no way to tell whether Dr. Kent Brantly and hygienist Nancy Writebol were helped by the experimental Ebola drug, said Dr. Anthony Fauci, head of the National Institute of Allergy and Infectious Diseases.

The two aid workers are being treated in a specialized isolation ward at Emory University Hospital in Atlanta. If they survive their infections, doctors won't know whether it's because of the drug or the other care they receive. The only thing they'll be able to say with certainty is that the drug didn't kill them.

"I do hope that it was effective, but when you're dealing with medicine and all the vicissitudes, you can't say definitively," Fauci said.

Ebola is a virus that causes flu-like symptoms such as fever, vomiting, aches and intense weakness. As it progresses, patients may suffer serious bleeding, as well as kidney or liver failure.

The experimental drug given to Brantly and Writebol is a cocktail of three monoclonal antibodies designed to prevent the Ebola virus from latching onto and inserting itself into a host cell. If the virus does enter the cell, it begins to mass-produce copies of itself.

University of Minnesota professor Michael Osterholm said he fears that tried-and-true methods of clinical trials and drug approvals could be overshadowed by misunderstanding about any availability of the experimental drug.

"If the Americans had this serum all the time, why didn't they send it to us Africans to help save lives?" said Winston Ojukutu Macauley, a social commentator in Sierra Leone.

"How many times have we found magic therapies that ended up ... doing more harm than good?" said Osterholm, who advises the U.S. government on infectious disease threats.

The Kentucky drug, called ZMapp, is one of several under development to fight Ebola. Allowing any of them to be given to patients without proper vetting would be problematic, said Arthur Caplan, director of the medical ethics division at the New York University Langone Medical Center.

"There's a fairly good chance that it could do more harm than good," Caplan said. "The drug could kill you faster, or make you die more miserably."

And because Ebola isn't 100 percent fatal, he said, some patients could wind up dying after taking a drug that later proves to be unsafe.

Even if it seems that patients have nothing to lose, the FDA has argued that its clinical trial system ultimately benefits more patients.

In 2003, a patients' rights group went to court seeking expanded access to experimental drugs for terminally ill patients. The Abigail Alliance argued that patients with "desperate diagnoses" had a constitutional right to potentially lifesaving treatments that had passed an initial round of safety testing.

But that would have removed a powerful incentive for patients to participate in clinical trials, FDA backers argued. A federal appeals court sided with the FDA, and the Supreme Court declined to hear the case.

There are other problems too. Monoclonal antibody drugs such as ZMapp are very expensive to produce, so determining the smallest dose that's effective is important, Caplan said. That requires proper testing.

Even if the drug were deemed safe for immediate use and a donor were found to pay for it, it could take months or longer to produce enough to treat everyone who wanted it.

"You'll be in shortage right away and you'll have some hard choices to make about who goes first," Caplan said.

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