Trump’s fetishization of The Great Man Theory
Authored by Benjamin Wolfson
By misunderstanding the role of regulation in science and drug discovery, Trump puts the public at risk.
At his first speech in front of both houses of Congress last night, President Trump used Megan Crowley, a survivor of Pompe’s Disease, as a prop to promote his goal of cutting regulations across the Government.
As infants, Megan and her brother Patrick were both diagnosed with the neuromuscular disorder Pompe’s Disease. Frustrated with the lack of treatment options, their father John Crowley left his management position at Bristol-Myers Squibb to be the CEO of Novazyme Pharmaceuticals, a company researching treatment of the disease. When Novazyme was acquired by Genzyme Corporation, Crowley was in charge of Genzyme’s global Pompe research program, which was the largest singular research effort in the company’s history. In 2003, Megan and Patrick received the new treatment they had developed, and as President Trump explained during his speech Megan is now a sophomore in college, having far outlived doctors initial prediction of 5 years.
This is an extremely attractive story, and one that we’ve seen before. A child is deathly ill, and when doctor’s are stymied a parent does everything they can to discover the cure. We know that this is a great story because when it works Hollywood is quick to turn it into a movie. Crowley’s story was dramatized in the 2010 movie Extraordinary Measures, starring Brendan Fraser and Harrison Ford. 20 years earlier, we had a similar story with 1992s Lorenzo’s oil starring Nick Nolte and Susan Sarandon.
President Trump is trying to use this story to sell the public on reduced regulation. He is selling a false hope to patients with incurable diseases and their loved ones, that a great man, now unburdened by federal regulation, will save them.
This can happen. John Crowley was able to lead a biotech company to discover a cure for the disease his children had. But it’s not always so successful. Lorenzo’s oil, the miracle cure for Adrenoleukodystrophy, has not proven so effective. 30 years later it is still under clinical trial, and while there are some positive results its use remains controversial.
And therein lies the rub. Only 9.6% of drugs that reach Phase 1 clinical trials (treatment of <100 healthy volunteers to identify drug tolerance and adverse effects) get approved by the FDA. This is not due to strict regulations by the FDA, but because science is hard. Cell and animal models often do not match human biology as well as we would like, and clinical trials play a crucial role in the production of safe and effective drugs.
Federal regulation is necessary to protect patient safety. Just look at what has happened to the field of dietary supplements. Supplements are largely unregulated by the FDA, and are the cause of an estimated 23,000 emergency room visits per year.
Last night, President Trump stated:
“If we slash the restraints, not just at the FDA but across our Government, then we will be blessed with far more miracles like Megan [Crowley]. In fact, our children will grow up in a Nation of miracles.“
If he truly wants to bless America with miracles, the focus should be on increasing scientific funding, supporting research, and recruiting the brightest minds from around the world (not banning them). Cutting regulations may result in more drugs in the hands of patients, but too many of them will be monkey’s paws.
Originally published at: https://medium.com/@brwolfson/trumps-fetishization-of-the-great-man-theory-85408dde4a3d#.rbyzqo1da.