So, This Is What a Biotech Tycoon Looks Like

One of the biotech tycoon's most trusted advisers is his mom. You'd have to arrive at Genzyme headquarters as early as he does—before 7 each morning—to see Termeer seated in the indoor garden not far from his office atop the building. (Like its waterless urinals, its gardens, 18 of them in all, are part of what makes it a paragon of eco‑architecture, a place celebrated as one of the greenest office buildings in the world.) But if you found Termeer here, chances are good that he'd be on his cell phone and that he'd be talking to his mom, six hours ahead of him, in Holland.

At 93, she still keeps a close eye on her son, and on his company. (She's become a frequent visitor and a sort of mascot at Genzyme's plant in Belgium, not far from where she lives.) The two speak at least once a week and the conversation is rarely laden with idle pleasantries. Mrs. Termeer has a point when she calls, and she tends to get right to it. The impulse is the same when the family gathers for its annual fall reunion along the Dutch coast, in the province of Zeeland. Tradition dictates that the Termeers each rise at the table to regale the clan with a little speech. Without fail, the matriarch's chats are the most, well, weighty. "We all cringe a little when she speaks," Termeer says. "There's usually a big moral angle to it. She's quite serious."

So Termeer wasn't terribly surprised by the call he received a few years back. His mom sounded agitated. She had read about Dutch patients who were relying on free infusions of an experimental precursor to Myozyme to treat their Pompe disease, a rare disorder that shrinks sufferers' muscles, eventually crippling their heart and lungs. The news suggested that Genzyme had plans to stop the program. These were Termeer's countrymen, and such apparent callousness was red meat for the Dutch politicians who denounced him in parliament. "She wanted answers. She wanted to know what I was going to do," Termeer says.

Termeer told his mother the story was simply wrong: No patient was in danger. She was pleased but confused. If the news was incorrect, she expected her son to stand up and shout that, to defend his position. She raised her six kids to be tough, sure, but not to gladly suffer a bludgeoning like this. "I told her that for a society to have concerns is completely okay. To even ask questions in a somewhat awkward way, that's okay," Termeer says. What he tells his mom, and others who ask, is that steering Genzyme where he does takes patience and sense enough to anticipate the ethical concerns his work raises. "If I can't take it, I need to get out of this business."

The biotech tycoon's immodest goal is to change healthcare. That is what he's trying to do, after all. That's part of why he doesn't sweat the bad press, which he regards as the penance of the innovator. His therapies for ultrarare diseases, he says, point the way forward, toward a day when very targeted drugs cure ailments perfectly, precisely. Don't think of his niche therapies as being used by tiny, statistically inconsequential groups; think of them as being deployed in ways that get results every time. Now contrast this with the trial-and-error approach that dominates medicine as it's practiced today, in which doctors pick and choose from the menu of drugs available and calibrate dosages until finally, hopefully, they land on what works best for that particular person. What if instead every condition had a drug that was the smart bomb that Cerezyme is for Gaucher's?

As more and more of their patents expire, the profits that can be wrung from conventional, scientifically simple (and therefore comparatively cheap-to- develop) drugs are drying up. The business is adjusting. "The part of the system that's funding the research and development is getting smaller, meaning that we have less to fund more," Termeer says. "So to fund this R&D engine, the drugs have to be very expensive. So they have to be very good. So they have to work every time." As a result, he says, "we're moving toward the kinds of things that we have done in treating rare diseases, but in treating diseases that aren't rare."

Across the harbor from Termeer's living room, the sun is setting behind the hills of Marblehead. Termeer doesn't seem to notice that it's left us sitting in the gloomy gray of dusk. What he's saying is more important than getting up to turn on the lights. He's caught up in explaining what he sees as the real purpose of Genzyme's superlatively expensive drugs: to fund the research to create new perfect solutions for more and more common diseases and benefit larger and larger groups of patients. That therapies like his company's cost so much, he says, is merely proof of how new biotechnology still is. Computers, after all, were plenty pricey when we first started making them.

He brings up the scolding New York Times editorial, and its argument that if the future of medicine is going to come with such steep price tags, we are in trouble. "My response is, let's not be afraid," he says. "This is a fantastic problem to have. Today people die from diseases. Pricing the solution is not the problem. Creating the solution, now that's the problem."