Beaufort News

Experimental treatment has had some success fighting brain-amoeba illness

This image of Impavido (miltefosine), provided by Profunda, was rushed to Charleston in an attempt to treat a patient with primary amebic meningoencephalitis (PAM). The drug was originally developed in the 1980s by German scientists as a breast cancer treatment.
This image of Impavido (miltefosine), provided by Profunda, was rushed to Charleston in an attempt to treat a patient with primary amebic meningoencephalitis (PAM). The drug was originally developed in the 1980s by German scientists as a breast cancer treatment. Profunda

Todd MacLaughlan scrolled frantically through flight schedules Tuesday night, looking for a plane headed from Orlando to Charleston.

There weren’t any until Wednesday morning.

That wasn’t soon enough.

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It was about 10 p.m. and MacLaughlan, CEO of the Orlando-based drug company Profounda, had just hung up with a representative from the Medical University of South Carolina, who got his contact information from the Centers for Disease Control.

There was a patient in South Carolina sick with a deadly disease caused by a brain-eating amoeba — and Profounda is the only company in the United States that manufactures a drug that has twice proved successful in treating it.

Doctors in Charleston needed that drug — marketed by Profounda as Impavido and known generically as miltefosine — and they needed it fast.

“Time is absolutely critical — every hour matters,” MacLaughlan said Thursday morning. “The sooner (the patient receives the medicine), the better.”

Terry C. Dixon, MD, PhD – Division of Infectious Diseases at the Medical University of South Carolina Children's Hospital - Pediatrics, discusses diagnosing and treating Primary Amebic Meningoencephalitis (PAM) on Aug. 4, 2016.

With no plane to jump on, MacLaughlan scrambled to come up with a Plan B.

Within a half-hour of the call from the hospital, he had arrangements in place with the UPS Critical Express courier service.

Six hours later, the courier arrived in Charleston with the drugs.

Jason Mills, MUSC’s pharmacy supply chain manager, confirmed in an email Wedenesday that MUSC was “able to procure miltefosine by working directly with Profounda.”

Miltefosine was originally developed in the 1980s by German scientists as a breast cancer treatment.

Doctors and researchers soon discovered the drug was an effective treatment for leishmaniasis, a tropical disease transmitted by sand fleas that causes skin sores and ulcers, MacLaughlan said.

Profounda began producing miltefosine earlier this year to treat the roughly 30 annual cases of leishmaniasis in the United States, he said.

Jeremy Lewis of Midlothian, Texas, who in 2010 lost his 7-year-old son Kyle to Primary Amoebic Meningoencephalitis (PAM) - known colloquially as "brain-eating amoeba" - spoke to The Island Packet & Beaufort Gazette on Thursday, August 4, 2016, abo

In recent years, the drug has been used in emergency situations to attempt to treat primary amebic meningoencephalitis (PAM), the disease contracted recently by the South Carolina patient who came into contact with the brain-eating amoeba known as Naegleria fowleri.

“Recently, two people with Naegleria infection survived after being treated ... (with) miltefosine that was given along with other drugs and aggressive management of brain swelling,” according to a CDC report.

“The drug is being used off-label right now under direction from the CDC,” MacLaughlan said.

Off-label refers to the use of a drug in a manner not officially approved by the U.S. Food and Drug Administration.

The drug comes in capsule form, and a round of treatment, which takes a month to complete, costs $48,000.

Insurance companies typically pay for most of that cost. But if that is not the case with the South Carolina patient, MacLaughlan said he will consider waiving the fee.

Profounda offers miltefosine to hospitals on consignment, MacLaughlan said.

The company will send the drug to directly to the hospital to store and use as needed, eliminating the need to wait on the drug to be shipped. Hospitals or patients would pay for the drugs only if they are used.

MUSC is currently working to “obtain approval to be a recognized consignment site” for miltefosine, Mill said.

Profounda has been called by the CDC once before to help treat a PAM case.

“Since we have had (the drug), we’ve treated one patient in Texas,” MacLaughlan said. “The patient didn’t make it.”

But miltefosine treatments have been successful in the past.

In 2013, a 12-year-old Arkansas girl was diagnosed with PAM after a trip to a local water park, said Dr. Jennifer Cope, an infectious disease physician with the CDC.

The patient was treated with miltefosine along with a cocktail of antibotics and antifungal medications.

The disease “has a very high mortality rate. Most people die of this infection,” she said. “So, we kind of throw the kitchen sink at it.”

After more than three weeks in the hospital, the patient was released and has since made a full recovery.

Because that patient’s successful drug cocktail included miltefosine, the CDC now recommends that doctors use the medicine to treat all PAM cases, Cope said.

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