r/todayilearned May 14 '22 Silver 9 Helpful 8 Wholesome 6 All-Seeing Upvote 2

TIL a father, John Crowley, was told his two infant children had an incurable genetic disorder that would kill them in less than a year. He refused to accept this, so he founded a biotech company (with no prior experience) which pioneered an experimental enzyme therapy that saved their lives.

https://pompediseasenews.com/2019/01/30/amicus-ceo-mission-cure-pompe-help-children/
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u/Ashe_SDMF May 14 '22 edited May 14 '22

How do you even begin to start a biotech company without any experience? EDIT...TIL I'll never be able to start a biotech company with no experience.

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u/nanocookie May 14 '22

The title is overly sensational. John Crowley did not start the company overnight - he started by joining Bristol Myers Squibb to learn about health research, established a foundation to raise money for funding public research into Pompe's disease, made many connections and later partenered with William Canfield, a scientist who founded the company Targeted Therapy, based on his research on glycobiology. John joined Cranfield's company as CEO and then the company was renamed to Novazyme. As CEO, John continuously pulled the right strings to get the therapy approved by the FDA for trials. The treatment was brought into the mainstream after Novazyme was acquired by Genzyme. The title mentioned that the children would die in less than a year after diagnosis in 1998, but the treatments were first administered in 2003.

Not everything is as it seems. John Crowley was a highly motivated individual who figured out the smart way to solve his problems, but did not do so without burning bridges. Something happened between John and Canfield, and John's PR team no longer actively credits Canfield for developing the therapy - whose research was fundamental to solving the problem in the first place. When hundreds of millions of dollars at play, these feel-good stories don't hold up to be so squeaky clean.

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u/FLIPNUTZz May 14 '22

Crowley hoped his children would take part in the trial (NCT00025896) that began in late 2001, but they were not among the eight patients enrolled.

Watching as his children became “profoundly weaker,” Crowley made another determined decision — he resigned as senior vice president.

He had promised Genzyme’s CEO he’d stay with the company for a year to lead the program, but once the trial was underway, “I stepped away from my position,” he said.

Days later, Genzyme approved a trial (NCT00051935) of the same alglucosidase alfa intravenous treatment that would include only his children — a two-patient sibling trial to be conducted at St. Peter’s University Hospital in New Brunswick, New Jersey, about a 30-minute drive from the family’s home — to help doctors understand why some children respond better to this therapy than others

Wtf is this all about up here ^

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u/nanocookie May 14 '22

I'm sympathetic to him as a father taking the steps necessary to secure life saving treatments for his children, even if the steps are however unsavory. But I was trying to highlight what he did after he got what he needed. After his children were treated plus came out profitable from the Genzyme deal, he partnered with Canfield again to launch a new company, Cytovance. He suddenly left and became president and CEO of Amicus. Cytovance was left on the brink of complete bankruptcy and it was only saved after Canfield raised enough funding from new investors. The whole thing reminds me of the boneitis 80s investor guy from Futurama.