A furious debate that is raging in Brazil pits the nation’s largest university against hundreds of cancer patients who want access to a compound that some have branded a miracle cure.
But whether the compound holds any benefits at all remains to be seen: it has never been evaluated in human trials. The conflict is an extreme version of a debate that has gone on in the United States and elsewhere, as terminally ill people whose diseases have withstood modern medicine’s proved arsenal have demanded access to untested treatments.
As we report on page 420, courts in Brazil have previously sympathized with those demands, ordering the University of São Paulo to provide a compound called phosphoethanolamine to hundreds of patients. People on both sides of this debate are armed with good intentions. The university argues that the drug is untested, and should not be used to give false hope — and unknown side effects — to vulnerable patients. On the other side, it is understandable that people with little hope may prefer the uncertainty of an untested drug to the certainty of a terminal illness.
But there are also concerning reports that some people with cancer are not taking their prescribed medications, for fear that scientifically proven medicine may interfere with the supposed miracle of phosphoethanolamine. The tenor of the debate has also been harmful at times, with some phosphoethanolamine advocates accusing the government or the pharmaceutical industry of actively suppressing further development of the drug.
The sad truth is that the drug is unlikely to be a miracle. In the United States, for example, only one in ten drugs that make it to phase I clinical trials are destined to gain approval from the US Food and Drug Administration (FDA). And phosphoethanolaminehas not even made it that far: its promise is backed up by a few publications based on lab and animal tests.
Even so, terminally ill patients may be willing to try a treatment with only the slimmest odds of success. In the United States, several states have passed laws that, to varying degrees, grant such patients the right to try experimental drugs outside the purview of the FDA. The laws have triggered debates of their own, and have come under fire for offering false hope and for potentially leading patients away from other, more promising avenues.
The situation in Brazil is more extreme. A university laboratory is neither a pharmaceutical plant nor a pharmacy; it is not required to follow good manufacturing protocols. There is no oversight to certify what is going into the blue-and-white phosphoethanolamine capsules produced at the University of São Paulo. Neither the compound’s side effects nor its efficacy are systematically monitored. To order a university to supply a drug is to show a disregard for the importance of all these safety measures.
The hope of phosphoethanolamine lies in further research. Federal funders in Brazil have said that they will support further preclinical studies of the drug. Researchers are pursuing options for moving the compound into clinical trials, should those animal studies succeed; patients who are interested in pursuing phosphoethanolamine treatment could enrol in the clinical tests. In the meantime, the courts should liberate patients from the legal tug-of-war and uphold the latest decision to halt distribution of phosphoethanolamine until its potential is better understood.
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