President Trump introduced the country Tuesday night to a young woman in a white jacket seated next to her father — a man who once aspired to be a fighter pilot, but for nearly two decades has made a career out of trying to cure his daughter and son of a rare, life-threatening disease.
In broad strokes, the president sketched the outlines of their story: As a baby, Megan Crowley was diagnosed with Pompe disease, a genetic disease that causes progressive muscle weakness. Her father, John, partnered with a scientist to found a small biotech company to find a cure. That company was acquired by a bigger one, eventually leading to the drug that has allowed Megan to defy doctors’ expectations that she’d die in childhood.
Today Megan is a sophomore at the University of Notre Dame and John is at the helm of a different venture, Amicus Therapeutics, working to develop an even better treatment for her disease. It’s an inspiring tale of innovation, invention and healing that was chronicled in a series of Wall Street Journal articles and a book, which inspired a movie called “Extraordinary Measures” starring Harrison Ford and Brendan Fraser.
“Megan can be quite a fierce and passionate advocate,” John Crowley said in an interview Wednesday, between meetings with lawmakers to advocate for more attention on rare-disease therapies. “She's a very, very smart and caring human being.”
The short vignette, embedded in a lengthy address to Congress, was part of a call to slash regulations. But it was notable as one of the most inspiring stories about American business and innovation that Trump has told in his presidency, embodying a vision of business and economics that is almost precisely the opposite of the one he's been selling to his supporters. Against Trump's promises of an American economic renewal fueled by protectionism and trade barriers, the full story of Megan’s miracle highlights the way critical innovations tend to emerge from a highly globalized network of manufacturing, knowledge-sharing and immigration — that could be disrupted by Trump.
As Trump said, Megan's presence is “about the unbounded power of a father's love for a daughter.” But dig deeper and the tale is about how Crowley's ingenuity and grit also depended on Genzyme, a Massachusetts company that grew into one of the biggest success stories in the American biotechnology industry under the leadership of Henri Termeer, a Dutch-born businessman.
The drug that saved Megan's life is a quintessential example of how American capital and research infrastructure draws cutting-edge scientists and talent from around the world, and how making progress depends on international and academic research collaborations.
And a president who has promised to slash drug prices may not have realized he was praising a treatment that costs an astonishing $298,000 per year, per patient, and that without that high price might never have been created at all, because there are so few patients — fewer than 3,000 people in the world take the drug today.
A father's search for a cure
John Crowley co-founded Novazyme Pharmaceuticals, a small biotech company based in Oklahoma, in 2000 with the scientist William Canfield, after Megan and her younger brother, Patrick, now 18, were diagnosed with Pompe. The disease eventually leaves patients confined to a wheelchair and threatens their ability to breathe.
In 2001, Novazyme, which had grown to 80 employees, was acquired by Genzyme, a biotechnology company from Massachusetts that had already struck major success in getting a rare-disease drug into the hands of patients. In the American-dominated biopharmaceutical industry, Genzyme rose to success under the guidance of its longtime (now former) chief executive, Henri Termeer. Termeer was born in the Netherlands, and he recently joined more than 100 colleagues in signing a letter that was highly critical of Trump’s immigration ban and the effect it could have on American innovation.
“The United States has led the world in medicine production for decades, not only because of its ability to finance drug discovery, but also because, more than any other country, the United States represents opportunity regardless of borders, gender, race, sexual orientation or political cast. This has enabled our industry to attract the best talent, wherever it is found,” Termeer and other executives wrote in a letter to the editor of the science journal, Nature Biotechnology, in February. “At a stroke, the new administration has compromised years of investment in this national treasure.”
Though Genzyme bought Novazyme, it was not ultimately that company's version of the drug that was approved. Researchers compared several experimental therapies head-to-head in the search for the best drug. The most effective was one that Genzyme had developed itself, with collaboration from academic researchers in the Netherlands and at Duke University.
That drug, then called Myozyme and today called Lumizyme, was first approved for patients in 2006.
“That was Genzyme's leadership. Genzyme took that through the FDA and I give them great credit for it,” Crowley said. “There were challenges, but the leadership was very, very helpful. They realized it was not a perfect drug, by any means. But it was something, and it helped. And it needed to get to as many patients as soon as possible.”
The reality of the ‘slow and burdensome’ drug approval process
Trump said in his speech that the onerous regulatory process was holding up the development of new cures.
“Our slow and burdensome approval process at the Food and Drug Administration keeps too many advances, like the one that saved Megan's life, from reaching those in need,” Trump said. “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.”
Drug approvals have actually sped up. It took, on average, 19 months to get a drug approved in 1993, compared to less than eight months in 2016, according to the agency's data. And a regulatory expert noted that Megan’s story is, in many ways, a textbook example of how federal officials can exercise flexibility when faced with a life-threatening disease for which there is no therapy.
“It is false to say that FDA was slow and burdensome in the review of Myozyme,” said David Kessler, a former FDA commissioner. “FDA approved the drug in nine months based on information in a few dozen patients, provided expanded access to the drug while it was conducting the review — and caught a life-threatening adverse reaction.”
Crowley also saw the Myozyme approval process as a successful one: “I think it was a very strong example of FDA being flexible, putting the patient view at the center.”
Crowley believes that the FDA plays an essential role in determining that drugs are safe and effective. But he argues that in recent years, the FDA has reached an inflection point and become more conservative in some areas, so reforms are needed that will allow more flexibility for rare-disease drug development. John and Megan met with the president and vice president on Tuesday afternoon and spoke about the need for more medicines for rare diseases. Crowley declined to give any specifics about the meeting, other than to say that Trump and Pence were “intensely focused” during the meeting.
“It's an example of a growing disconnect in certain of these rare diseases among regulators in the world,” Crowley said. “We told the FDA we would do that study. It's going to take us several years to complete.”
The complex heritage of American innovation
There’s no question that the Crowleys' story is one of American innovation: a testament to the hard work and love of a desperate New Jersey father, the vision of a Dutch CEO who found his best business-building opportunity in America, the success of a Massachusetts company in working together with researchers abroad.
But the drug Trump held up as a triumph of American medicine is no longer even an American product. In 2011, the French pharmaceutical company Sanofi bought Genzyme for more than $20 billion. The drug is manufactured in Belgium.
That wasn’t a tax dodge; it was a business decision that allowed the company to acquire a partially-built facility years ago, said Bo Piela, a spokesman for Sanofi Genzyme. Genzyme's Dutch collaborator was in receivership in 2001, so the American company acquired some of its assets, including infrastructure to produce the drug in Belgium. That gave the company a leg up in producing the drug faster and, ultimately, getting it to patients. In 2011, Genzyme built an additional $350 million plant in Belgium to expand its production capacity.
Trump’s example really is a testament to how American innovation works — not the easy version, but the complicated way that high-value American companies harvest knowledge and talent to help patients, agnostic about where insight arises, to benefit both business and patients. Despite the fact that only about 3,000 patients receive Sanofi Genzyme's therapy, it accounts for more than a half-billion dollars in revenue each year.