(CBS News) The “brain-eating” amoeba may have finally met its match. Government health officials are offering doctors an experimental new drug that may treat deadly infections like primary amebic meningoencephalitis (PAM), which two children appear to have survived this summer.
PAM is an often fatal infection caused by the Naegleria fowleri amoeba, a microbe found in warm fresh waters (mostly in the South) that, if ingested through the nose, can destroy the brain tissue and lead to rapid death.
There have been 128 known infections in the U.S. from 1962 to 2012, with only one documented case of survival in addition to a case of survival in Mexico.
Called miltefosine, the experimental drug is not approved for use in the United States, but has been prescribed on a case by case basis to treat a parasitic infection called leishmaniasis, and sometimes breast cancer. Since 2009, the CDC has also administered the drug for amebic infections only in emergency situations, with permission from the Food and Drug Administration (FDA). Other so-called free-living amoeba infections include granulomatous amebic encephalitis, a serious infection of the brain and spinal cord that often strikes people with weakened immune systems, and is caused by Balamuthia mandrillaris and Acanthamoeba species.
Dr. Jennifer Cope, a medical epidemiologist at the CDC who authored the new report on the investigational drug, confirmed to CBSNews.com that the drug was involved in two rare cases of children surviving the infection this month: A 12-year-old Ark. girl, Kali Hardig, and a 12-year-old Fla. boy, Zachary Reyna.
“We’re hoping that we reach clinicians and physicians taking care of these patients (with PAM),” Cope said of the decision to release news of the drug expansion in the Aug. 22 issue of the CDC’s journal, Morbidity and Mortality Weekly report. The CDC also alerted doctors of the drug’s expanded availability through its Health Alert Network and patients through the media.
Cope explained that miltefosine is a German drug that is not licensed in the U.S. for any indication, which meant any time a doctor wanted to use it on a patient, he or she would have to fill out paperwork, specifically an Investigational New Drug (IND) application. Once approved by the FDA, the physician would then have to wait for the drug to come over from Germany.
“It got to be somewhat cumbersome,” she said.
The process could take up to a week, and high-risk patients could die before the medication arrives.
That’s what led the CDC to pursue an expanded access IND, said Cope, which would allow doctors to get the approval for more than one patient. Not only that, the expanded access allows the CDC to stockpile the experimental drug at its Atlanta headquarters and directly send it to hospitals.
The CDC was able to get the drug to Kali Hardig within 24 hours, while Zachary Reyna received it in about seven hours, according to Cope.