Originally published on Medium
It is an extremely attractive story, and one that we’ve seen before. A child is deathly ill, and when doctors are stymied a parent does everything they can to discover the cure. In fact, Hollywood has been quick to turn this tale into movies. In 1992, Nick Nolte and Susan Sarandon starred in Lorenzo’s Oil, a film about a miracle cure for Adrenoleukodystrophy. Nearly 20 years later, Brendan Fraser and Harrison Ford starred in Extraordinary Measures. This incredible true story dramatizes John Crowley’s journey when his infant children Megan and Patrick were both diagnosed with the neuromuscular disorder Pompe’s Disease. Frustrated with the lack of treatment options, John Crowley left his management position at Bristol-Myers Squibb to cofound Novazyme Pharmaceuticals and research 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. Now Megan Crowley is a sophomore n college, having far outlived doctors’ initial diagnosis of only 5 years.
During his first speech in front of both houses of Congress, President Trump used Megan Crowley as a prop to promote his goal of cutting regulations across the Government. By misunderstanding the role of regulation in science and drug discovery, Trump puts the public at risk. 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.”
President Trump is trying to use the Crowleys’ 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, once unburdened by federal regulation, will come to 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. The miracle cure for Adrenoleukodystrophy portrayed in Lorenzo’s Oil has not proven as effective. 30 years after its development the medicine is still under clinical trial, and while there are some positive results its use remains controversial.
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 stri
ct FDA regulations, 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 with dietary supplements. Supplements are largely unregulated and are the cause of an estimated 23,000 emergency room visits each year.
If President Trump 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.