A University of Minnesota trial of an experimental antifungal drug offered promising results Tuesday and hope of a new weapon against a growing source of infections worldwide.
Researchers have been looking for a less toxic treatment for cryptococcal meningitis, a fungal brain infection that is a leading cause of death worldwide in people with HIV. It's currently treated with intravenous amphotericin, which is so harsh that it is nicknamed "ampho-terrible," said Dr. David Boulware, a U infectious disease expert leading the trial.
The research showed a liquid form of the drug, taken orally, is equally effective with fewer side effects.
"If it works for the most severe life-threatening fungal infection, it should work for other fungal infections," Boulware said. "That's the key. It's a potential revolution in fighting fungal infections."
Fungal infections in the U.S. involving Candida auris strains have been growing in recent years and showing resistance to existing drugs. Clinical cases increased from 173 in 2017 to 2,377 in 2022, according to the Centers for Disease Control and Prevention. Three cases have been reported in Minnesota.
Most people suffer no health problems when exposed to cryptococcus fungal strains, but they can be deadly to people with HIV or other conditions that weaken their immune systems.
Tuesday's results in the journal Clinical Infectious Diseases compared the effectiveness of oral and intravenous amphotericin on 141 people with HIV who developed cryptococcal meningitis.
The U's federally funded drug study recruited participants in Africa through a partnership with the Infectious Diseases Institute of Makerere University in Uganda.
About 90% of infected patients taking the oral liquid drug were still alive after four months, which was comparable to the survival rate with the intravenous drug, the study showed. Boulware called the results "amazing," because about a third of patients hospitalized with this infection in Africa die within two weeks.
Another, larger trial is still needed before U.S. regulators will consider approving the drug, which is being developed by Matinas BioPharma. Boulware said additional follow-up studies could then prove the drug's utility against other fungal strains.
Fungal outbreaks remain rare in the U.S., generally involving contaminated medical products that are given to already-weakened patients.
Contaminated steroid shots made by a Massachusetts compounding pharmacy led to 753 fungal infections in 2012 and 64 deaths, according to the CDC. The tainted shots were delivered to six pain clinics in Minnesota, which reported 12 infections and one death.