Trump’s story of U.S. in­no­va­tion de­pends on the rest of the world

The Washington Post Sunday - - BUSINESS - BY CAROLYN Y. JOHN­SON

Pres­i­dent Trump in­tro­duced the coun­try Tues­day 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 raise money and 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 Crowley to defy doc­tors’ ex­pec­ta­tions that she would die in child­hood.

To­day Crowley is a sopho­more at the Univer­sity of Notre Dame and her fa­ther is at the helm of a dif­fer­ent ven­ture, Am­i­cus Ther­a­peu­tics, work­ing to de­velop an even bet­ter treat­ment for her dis­ease — they hope, a cure. 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 Har-

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.”

Trump’s 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 Crowley’s mir­a­cle high­lights the way crit­i­cal innovations tend to emerge from a highly glob­al­ized net­work of man­u­fac­tur­ing, knowl­edge- shar­ing and im­mi­gra­tion — one that could be dis­rupted by Trump.

As Trump said, Me­gan Crowley’s pres­ence is “about the un­bounded power of a fa­ther’s love for a daugh­ter.” But dig deeper and the tale is about how John Crowley’s in­ge­nu­ity and grit also de­pended on Gen­zyme, a Mas­sachusetts com­pany that grew into one of the big­gest suc­cess sto­ries in the U.S. biotech­nol­ogy in­dus­try un­der Henri Ter­meer, a Dutch-born busi­ness­man.

The drug that saved Me­gan Crowley’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.

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 Ter­meer, its long­time (now former) chief ex­ec­u­tive. 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 in­vestri­son in this na­tional trea­sure.”

Though Gen­zyme bought No­vazyme, it was not ul­ti­mately Crowley’s com­pany’s ver­sion of the drug that was ap­proved. Re­searchers com­pared sev­eral ex­per­i­men­tal ther­a­pies head-to­head 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 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.”

‘Slow and bur­den­some’

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 with less than eight months in 2016, ac­cord­ing to the agency’s data. And a reg­u­la­tory ex­pert noted that Me­gan Crowley’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 ex­ment panded ac­cess to the drug while it was con­duct­ing the re­view — and caught a life-threat­en­ing ad­verse re­ac­tion.”

John 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,” he said.

Crowley says 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 Crowley met with Trump and Vice Pres­i­dent Pence on Tues­day af­ter­noon and spoke about the need for more medicines for rare dis­eases. John Crowley de­clined to give any specifics about the meet­ing, other than to say that the pres­i­dent and vice pres­i­dent were “in­tensely fo­cused” dur­ing the meet­ing.

Crowley’s new com­pany, Am­i­cus, has got­ten a drug ap­proved for the rare Fabry dis­ease in Europe, but U.S. reg­u­la­tors have asked for more ev­i­dence.

“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 com­plex­ity of 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 the United States, the suc­cess of a Mas­sachusetts com­pany in work­ing 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 in­vested $350 mil­lion to ex­pand that fa­cil­ity.

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 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.

JONATHAN NEW­TON/THE WASH­ING­TON POST

John Crowley, left, head of Am­i­cus Ther­a­peu­tics, and daugh­ter Me­gan Crowley watch Pres­i­dent Trump’s ad­dress to Congress on Tues­day. Me­gan has a rare ge­netic dis­ease called Pompe dis­ease.

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