“Repurposed drug battles ‘brain-eating’ amoeba”
by Katherine Kornei
Science, February 3, 2023
“One of the most deadly and devastating diagnoses that we can give to any patient.”
Those are the words of Dr. Natasha Spottiswoode, an infectious diseases specialist at the University of California, San Francisco Medical Center. More than 90% of patients who contract granulomatous amebic encephalitis, a rare disease of the central nervous system caused by an amoeba called Balamuthia mandrillaris, are dead within a few months.
COVID-19 pales in lethality, as does even bubonic plague. The CDC, not one to mince words, includes this statement on its website: “Most cases of Balamuthia are diagnosed right before death or after the patient has died.”
But a Balamuthia patient recently made an astounding recovery at the University of California, San Francisco Medical Center. Dr. Spottiswoode and her colleagues, who oversaw the case, attribute the unexpected success to the novel use of a drug never intended to treat the disease.
In Asia and Europe, nitroxoline has been used for over 50 years to cure urinary tract infections. But it’s remarkably effective at inhibiting Balamuthia mandrillaris as well, researchers discovered in 2018 when they screened more than 2,000 drugs in the laboratory in the search for a Balamuthia antidote. Now, nitroxoline’s potency has been demonstrated for the first time in a patient.
When a man in his 50s living in California was diagnosed with Balamuthia, doctors started him on the classic cocktail of drugs typically used to treat the disease. But the drug combination soon proved to be toxic to his vital organs, a common outcome. The patient and his family were desperate enough to try nitroxolene, and Dr. Spottiswoode and her team coordinated an emergency use authorization with the FDA to obtain the drug. Within a week of starting nitroxoline, the abscesses in the patient’s brain began to shrink. Now, over a year since his diagnosis, the patient is still alive and is living at home. “I think he’s very much on his way to being one of the survivors of this disease,” Dr. Spottiswoode told a group of UCSF colleagues in November.
How about a story? I’m envisioning a short feature that highlights this case study and introduces readers to Balamuthia–I haven’t found any mention of this disease in Science’s archives.