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Brazilian courts tussle over unproven cancer treatment

Patients demand access to compound despite lack of clinical testing. A court in the Brazilian state of São Paulo has cut off distribution of a compound that is hailed by some as a miracle cancer cure — even though it has never been formally tested in humans. On 11 November, to the relief of many cancer researchers, a state court overturned earlier court orders that had obliged the nation’s largest university to provide the compound to hundreds of people with terminal cancer.

The compound, phosphoethanolamine, has been shown to kill tumor cells only in lab dishes and in mice (A. K. Ferreira et al. Anticancer Res. 32, 95–104; 2012). Drugs that seem promising in lab and animal studies have a notoriously high failure rate in human trials. Despite this, some chemists at the University of São Paulo’s campus in São Carlos have manufactured the compound for years and distributed it to people with cancer. A few of those patients have claimed remarkable recoveries, perpetuating the compound’s reputation as a miracle cure.

The Brazilian constitution guarantees universal access to health care, and it is common in Brazil for patients to turn to the courts to access drugs that the state healthcare system does not dispense because of their cost. But phosphoethanolamine presents a different situation because it is not really a ‘drug’ at all. It is not approved by Brazil’s National Health Surveillance Agency.

Those who argue that people who are terminally ill have a right to try experimental medicines saw a decision in favor of a patient in October 2015 as a significant victory. But to the university administration, drug regulators and cancer researchers, it showed blatant disregard for the basic scientific principle that a drug should be demonstrated to be safe and effective before being given to patients outside of a clinical trial.