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A life-saving drug that treats a rare infection is almost impossible to find

cdc-says-it-is-too-late-to-make-new-flu-vaccine-for-this-season naegleria-fowleri-amoeba-130815 rtr3frnwBy Lindzi Wessel, STAT From Business Insider

Hospitals are stocked with lots of vital drugs.

But there’s one that is time-sensitive, life-saving, and an utter necessity in treating a rare kind of infection — but you’ll find it almost nowhere in the US.

The drug is called miltefosine. It is a microbe-killing drug that can sometimes save the lives of people infected with a brain-eating amoeba, if given immediately upon diagnosis.

The problem is that, until recently, the only place the drug came from was CDC headquarters; when an infection was reported, the drug would have to be flown or driven from Atlanta, adding hours of delay.

Only four Americans have ever survived the infection, and in three of those cases patients received miltefosine at early stages of treatment.

Now, in an effort to change these dire odds, a small drug company is testing out a novel approach — making use of a tactic originally created as a financial workaround to get the drug on shelves before disaster strikes.

So far this year, five US children and teens have been infected by the amoeba Naegleria fowleri, and four have died. The amoeba, which grows in warm water, most often strikes children and teens as they play in lakes or ponds during the summer.

The infections are extremely rare but almost always deadly. When carried into the brain in water forced up the nose, the amoeba swap their normal diet of bacteria for consumption of brain tissue.

Miltefosine is an antiparasitic, and it’s the most promising treatment that exists for the infection. Before 2013, when the CDC obtained special approval to keep miltefosine within US borders, doctors had to get special permission to request the drug from overseas sources, and it could take days for the drug to be flown to the hospital in need.

Only one person survived during that period — a 1978 survivor who is thought to have been infected with a less deadly strain of the amoeba.

But in 2013, the CDC started storing and distributing miltefosine as an investigational drug, and that year two Americans received the medication and lived through the infection. Then, in March of this year, a small drug company called Profounda, based in Orlando, Fla., became the first US company to distribute the drug commercially.

Since then the company has already distributed the drug to multiple patients, and earlier this summer another child survived.

Doctors can’t be certain that miltefosine is what made the difference — with so few cases, controlled clinical trials are impossible. New advances in brain injury treatments are also likely a factor.

It’s also unclear how soon after infection the drug has to be administered. Not every patient who receives miltefosine has survived, and delays in diagnosis or drug delivery increase the risk.

Those factors may have contributed in a case this year, when an 11-year-old girl in South Carolina was infected on Aug. 2. Profounda got the call from the hospital at 10 p.m. that night, too late to make a shipment by flight, so a courier drove the drug six hours to Charleston’s Medical University Hospital where Hannah Collins was being treated. She died three days later.

Now the CEO of Profounda wants to do away with shipment delays entirely, by putting the drug on hospital shelves throughout the nation.

“We’re trying to ensure that hospitals across the country will actually have it in stock,” said CEO Todd MacLaughlan, who recently led Profounda in acquiring licenses to the drug. “Then we cut out the transportation time all together.”

That is a hard sell, given that a full course of miltefosine costs $48,000 and any given hospital is almost certain not to need it. So earlier this year the company began offering hospitals to stock the drug for free and charge them only if they wind up using it.

Profounda is not the first to use such a consignment model, something that has become more popular in the last five years as hospitals have looked for new ways to cut costs, said Michelle Kromelis, senior director of pharmacy services at Children’s Health in Dallas.

She estimated that the hospital uses this method for about 10 percent of the drugs they stock, including miltefosine. In most cases, the goal is just to help offset upfront costs, and drug companies assume the hospitals will use the drugs eventually, she said.

Miltefosine is different. MacLaughlan assumes the opposite. He’s pretty sure most of the company’s product will not be used. Still, he doesn’t expect this approach to hurt his business. In the United States, drug companies are required to demonstrate that they can consistently produce their drugs every year, so Profounda will be producing about 100,000 miltefosine capsules, whether they are used or not. (The drug is also used to treat a parasitic infection called leishmaniasis, which is also rare but is more common than amoeba infections.)

“Rather than expiring at the warehouse they can be at the hospital and we can exchange them out so that it’s always there,” he said. “We know that 95 percent of them will probably never use it, but it’s on stock, it’s there for them if they need it.”

Profounda has so far this year gotten the drug into eight hospitals — in Dallas and Fort Worth, Texas; Orlando, Tampa, and St. Petersburg, Fla.; Charlotte N.C.; Columbia, S.C.; and Pittsburgh, Pa. — and is in conversations with three more. MacLaughlan’s goal is to have miltefosine in 50 to 100 hospitals dispersed across the country, so that no hospital will be far from the drug.

Dr. Jennifer Cope, a medical epidemiologist and the CDC’s point-person for brain-eating amoeba infections, said the CDC is supporting the initiative to get the drug in more hospitals.

“We just know with how serious of an infection this is, the sooner the better,” she said of access to the drug. “We think the more widely available the drug is the better.”

Profounda’s conversations with hospitals also help make the facilities aware that the drug exists, MacLaughlan said, and may also make them more likely to correctly diagnose the infection in the first place. Just this month, a 19-year-old woman in New York died after doctors initially misdiagnosed her and sent her home without substantial treatment.

The ideal would look a lot more like what happened in August. That time the call for miltefosine came from within Orlando, the city where Profounda is headquartered.

A pharmacist from the Florida Hospital for Children called the company with an urgent request for the drug to save a 16-year old boy — and it quickly became clear that the fastest method of delivery would be a personal one. Michael MacLaughlan, who is Todd’s son and the operations manager of the company, jumped into his car still in pajamas, handing the medicine to a nurse outside the hospital less than 30 minutes later.

Sebastian DeLeon, who had also been quickly diagnosed, was the first in history to take miltefosine with a glass of water, while still conscious, before being put in a medically induced coma.

Last week, he appeared by video at a national summit for brain-eating amoeba awareness, smiling and speaking normally, to thank the doctors and medical staff that saved his life.

DeLeon is home and planning to return to school soon. In the meantime, the Florida hospital worked out an agreement with Profounda to keep miltefosine on their shelves.

Read the original article on STAT. Copyright 2016. Follow STAT on Twitter.

pharmaceutical research Reuters/Ina Fassbender

A general view of the Centers for Disease Control and Prevention (CDC) headquarters in Atlanta, Georgia September 30, 2014. REUTERS/Tami Chappell

naegleria fowleri amoeba 130815 Center for Disease Control


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