Responding to political pressure and popular demand for a largely untested cancer drug, Brazilian President Dilma Rousseff signed into law today a measure that allows the renegade compound—synthetic phosphoethanolamine—to be produced and sold legally as a cancer therapy in Brazil.
Scientists poured scorn on the decision, contending it puts patients at risk and undermines the authority of the Brazilian Health Surveillance Agency (the equivalent of the U.S. Food and Drug Administration) to regulate research and approval of new drugs based on internationally accepted safety and efficacy protocols. This was a “political decision inspired by a messianic surge of pseudoscience,” says Gustavo Fernandes, president of the Brazilian Society of Clinical Oncology in Brasília. “It was the worst possible way of dealing with this problem.”
The “cancer pill” sparked a national debate last year after Brazilian media carried testimonials of patients claiming that it relieved symptoms or even cured their cancer. The compound was developed in the early 1990s by Gilberto Chierice, an analytical chemist at the University of São Paulo whose lab distributed it to patients free of charge for several years, without any regulatory approval or clinical oversight.
Apart from a few studies in mouse models and in cell lines, there is no laboratory evidence that synthetic phosphoethanolamine works as a cancer drug. The university tried to shut down Chierice’s operation in June 2014, but since then more than 15,000 people sued the university, forcing it to continue providing them with the pills. Advocacy groups, meanwhile, pressured politicians and health authorities to legalize use of the compound as a cancer drug. Brazil’s congress passed a bill to do just that last month.
Officials in the ministries of health, industry, and science advised Rousseff to veto the bill, according to O Estado de S. Paulo newspaper. But with Rousseff fighting for her political life—the congress is attempting to impeach her over allegations of fiscal improprieties—her executive office recommended signing the bill, sources say.
The law authorizes production, prescription, and consumption of synthetic phosphoethanolamine as a cancer therapy “independently” of registration with the Brazilian Health Surveillance Agency. To acquire the pills, consumers must show medical proof that they have a malignant tumor and sign a consent form.
Under the new law, the substance can only be produced and distributed by “licensed agents.” On 1 April, the University of São Paulo closed Chierice’s former research lab, which had until then been producing the pills under court orders. The only place the compound is currently being produced is a private lab contracted by the São Paulo state government to supply it for an upcoming clinical trial. The lab is preparing the pills according to a formula patented by Chierice and six colleagues; they claim their preparation is different from the synthetic phosphoethanolamine available on the international market as a dietary supplement.
Late last year, the science ministry committed to spending nearly $3 million on preclinical studies of synthetic phosphoethanolamine. Initial results, made public last month, were not promising. According to the experiments, conducted at four academic and private labs, the pills produced by Chierice’s group contained only 30% synthetic phosphoethanolamine, and the substance failed to kill cancer cells. It doesn’t appear to be toxic.
At a 5 April hearing in Brazil’s Federal Senate, Chierice charged that the government-sponsored studies are being done in “bad faith,” and stated that his group is obtaining clinical data from overseas. The University of São Paulo in 2015 lodged a complaint with the police accusing Chierice of “curandeirismo,” or illegally dispensing unproven medical treatments. A criminal investigation is underway, according to news reports. “Maybe [Chierice] was well intentioned, but he did a lot of wrongs,” Fernandes says.
The new law may please desperate cancer patients, but it’s an unfortunate move, says biochemist Luiz Fernando Lima Reis, research director at Hospital Sírio-Libanês in São Paulo, where high-profile politicians have been treated for cancer, including Rousseff in 2009 and former President Luiz Inácio Lula da Silva in 2011. “Decisions like this should be based on scientific evidence,” Reis says.