Patients demand access to compounddespite lack of clinical testing.
A court in the Brazilian state of SãoPaulo has cut off distribution of a compoundthat is hailed by some as a miracle cancercure — even though it has never beenformally tested in humans. On 11 November,to the relief of many cancer researchers, astate court overturned earlier court ordersthat had obliged the nation’s largestuniversity to provide the compound tohundreds of people with terminal cancer.
The compound, phosphoethanolamine,has been shown to kill tumor cells only in labdishes and in mice (A. K. Ferreira et al.Anticancer Res. 32, 95–104; 2012). Drugsthat seem promising in lab and animalstudies have a notoriously high failure rate inhuman trials. Despite this, some chemists atthe University of São Paulo’s campus in SãoCarlos have manufactured the compound foryears and distributed it to people withcancer. A few of those patients have claimedremarkable recoveries, perpetuating thecompound’s reputation as a miracle cure.
The Brazilian constitution guaranteesuniversal access to health care, and it iscommon in Brazil for patients to turn to thecourts to access drugs that the state healthcare system does not dispense because oftheir cost. But phosphoethanolaminepresents a different situation because it isnot really a ‘drug’ at all. It is not approved byBrazil’s National Health Surveillance Agency.
Those who argue that people who areterminally ill have a right to try experimentalmedicines saw a decision in favor of a patient in October 2015 as a significantvictory. But to the university administration,drug regulators and cancer researchers, itshowed blatant disregard for the basicscientific principle that a drug should bedemonstrated to be safe and effective beforebeing given to patients outside of a clinicaltrial.
Source: Nature 527, 420–421 (adapted).http://www.nature.com/news/brazilian-courts-tussleover-unproven-cancer-treatment-1.18864. Access:March 21st, 2016.