Brazilian Society of Clinical Oncology Makes Last Stand Against "Miracle" Cancer Drug

Brazilian Society of Clinical Oncology Makes Last Stand Against "Miracle" Cancer Drug

Gilberto Lopes, MD, FASCO, MBA

May 23, 2016

 "You see, boys forget what their country means by just reading The Land of the Free in history books. Then they get to be men they forget even more. Liberty's too precious a thing to be buried in books, Miss Saunders. Men should hold it up in front of them every single day of their lives and say: I'M FREE TO THINK AND SPEAK. My ancestors couldn't, I can, and my children will. Boys ought to grow up remembering that."
—Jefferson Smith,
Mr. Smith Goes to Washington

In Frank Capra's 1939 motion picture, James Stewart plays a naive man who is nominated to the United States Senate but quickly finds himself in trouble for being  an idealist in a dog-eat-dog environment where pork-barreling is commonplace and special interests dictate the agenda. 

Given the number of lobbyists in suits aboard our plane, you would be forgiven for thinking our destination was Reagan or Dulles, DC’s nearby airports, but we actually landed at Juscelino Kubitschek Airport, one of the few significant legacies left in the nation's capital from the Football (Soccer) World Cup in 2014. The airport is named after the president who built Brasilia in just a few years in what was the middle of nowhere in the 1950s. Today,  the richest federative unit of the country with a per capita GDP of nearly US$30,000, the Federal District is the only part of Brazil that would be considered developed by that yardstick.

The shiny new contemporary glass and steel terminals contrast nicely with Congonhas, from which we departed two hours earlier. Christened after the Viscount who became the first president of the province of São Paulo in an independent Imperial Brazil (and who actually owned the land where it was later built), the commuter airport was inaugurated in 1936 with an Art Deco façade and has since gained a Modernist central hall and other eclectic additions as it tries to keep up with the sprawling city.

In the last chapter (yes, sorry, the synthetic phosphoethanolamine soap opera goes on), the Brazilian Congress had just passed a bill that allowed marketing of the substance and we were hoping that a presidential veto would restore normalcy in the drug approval process.

 (If you missed my previous ASCO Connection posts on this subject, “An Industry of False Hopes” and “Every Day a New Chapter,” please take a moment to catch up on the story and then come back. We'll wait).

You now remember that this supposedly miraculous cancer treatment came to life in the chemistry institute at the University of São Paulo in the city of São Carlos, and was given to thousands of patients without any documentation, detailed informed consent, or control  over 2 decades. Despite that, mainly because of popular clamor due to unsubstantiated reports that phosphoethanolamine had cured many patients, the legislative and executive branches of government decided that hope without proof was enough for official approval.

In the midst of an impeachment process, one of the many populist Hail Mary passes that Dilma Rousseff attempted, hoping to regain a modicum of popularity, was to approve phosphoethanolamine without proper evaluation by the medicines agency, ANVISA. Just before the Senate voted to suspend her from presidential duties and to investigate the illegal use of funds from state banks to balance the federal budget during her campaign for reelection, fewer than 10% of polled Brazilians supported her.  

As for our nemesis, phosphoethanolamine, new and independent studies, financed by the Ministry of Science and Technology, determined that only 30% of the content of the pills was actually phosphoethanolamine. (Not an unexpected finding when a "medication" is produced artisanally at a backyard laboratory with disregard for good manufacturing practices).

Moreover, and more importantly, initial sensitivity studies in preclinical models did not show significant activity for the parent compound and only minor shrinkage of tumors with one of its metabolites (at doses that are too high to be feasibly given to patients). These data were already available when the Senate approved the bill and the now-suspended President Rousseff signed it into law, but that did not dissuade the politicians mired in the largest corruption scandal Brazil has ever seen from choosing populism over due process and public safety.

Chased by a bloodhound-like Federal Police and prosecuted by a fiercely independent judiciary, it is becoming clear to the nation that the Workers Party has become an organized crime ring that will do whatever it takes to hold onto power. (Most of the money from graft has been used for advertising in elections and to buy political support). Petrobras (the state oil company) alone faces bribery charges amounting to an estimated US$20 billion (yes, that's a B) and several members of Congress have lost their mandates and are being prosecuted. Many of Ms. Rousseff's closest allies have been implicated in wrongdoing.

Sergio Moro, the leading federal judge in these proceedings, chosen by Time as one of the most influential leaders in the world, has even briefly detained  and interrogated Luiz Inacio Lula da Silva, the once widely popular president of Brazil. Lula, as he is better known, finished his second term 5 years ago with  impressive 80% approval ratings and easily elected his successor, who up to that time had never been voted into public office. They rode high on a commodities boom and on increased social spending which had been made possible by the financial stabilization their predecessor, Fernando Henrique Cardoso, fostered when he ended hyperinflation in the 1990s with sensible orthodox economic policies. Like-minded action is now sorely needed to reduce government spending and borrowing (public debt soared by 20% last year).

When Dilma nominated Lula to become her Chief of Staff (in practice to function almost like a Prime Minister), bringing with it freedom from prosecution except by the Supreme Court, protests again erupted from north to south, increasing the calls to remove her from office.

In the midst of all this, the medical community gathered for a last stand against the barbarian hordes of witchcraft and ignorance which have taken the country by storm and tricked countless of patients and families into believing that phosphoethanolamine might be active.

While we can't definitively confirm or refute its efficacy as clinical trials are still ongoing, there's circumstantial evidence that phosphoethanolamine is not active and may indeed be harmful. The Brazilian Society of Clinical Oncology (SBOC in Portuguese) surveyed 398 of its 1,200 members. Of those surveyed, approximately a third had had patients who took the agent, and none had seen or could provide objective evidence of response. Moreover, many reported possible adverse events related with its use.

With this information at hand and hoping to prevent setting a dangerous precedent, the Brazilian Medical Association (AMB), which plays a combination of the functions that the American Medical Association and the American Boards of Specialties do, supported by SBOC and by the Federal Medical Council (CFM, which would be the equivalent of a federal medical board in the U.S.), went to the Supreme Court alleging that the law was unconstitutional and hurt due process in drug approval, potentially harming patients who stop medical treatments to try their luck with phosphoethanolamine.

That brought my colleagues and me to Brasilia. As we drove by the aptly named Three-Powers Plaza, which is surrounded by the National Congress, the Presidential Palace, and the Supreme Court building, I was still incredulous that in a few minutes we would be meeting the country's Chief Justice to discuss a matter that—to physicians and scientists—is so crystal clear that it would not merit a pub discussion.

But that is what democracy is all about.  The diverging opinion of many Brazilians was reflected a day after our audience in the Supreme Court, when four of the justices, arguing that the popular call for alternatives and the need for compassionate options for terminal patients and an individual's right to make decisions about their own care and body made the law valid and voted to strike out our request.

Luckily, the six other justices (one of the eleven was on leave) sided with our motion  and, at least temporarily, placed the law on hold. When the matter comes back to the court for full consideration (which is likely to take many months or a few years), we hope that the ongoing trials will be finished and put the matter to rest. Or they could prove us wrong, at which point we would gladly change our minds.

This is what a democracy is supposed to do best: hear the cacophony of voices we have in public discourse and balance the disparate points of view that citizens may have on the same issue. Brazil's New Republic is young. It has barely been 30 years since the military returned to the barracks and became subordinate to civilian government. In this period we have elected our representatives directly, but have also become entangled with a political system that brings 27 parties to Congress and forces illegal pacts and corruption to become the mechanism of governance.

The Brazilian people have taken to the streets over and over again and in greater numbers at each turn. We marched and obtained the right to vote and a new democratic constitution in the 1980s. We protested again in the 1990s when it became clear that Fernando Collor de Mello, the first directly elected president in a generation, was corrupt and we forced Congress to impeach him. (Ostracized for 8 years, Mr. Collor is now a senator and may be receiving kickbacks again). Millions have now come to the streets and we are about to impeach another president, making that twice during my lifetime (Dilma has 180 days from the beginning of her suspension to defend herself before being definitively ousted).

We can't, however, hope to become a normal country (if such a thing actually exists) until we reform our political system. We will keep trying until we do it right! Expect more bumps—and posts—ahead.


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