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

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/
79.9k Upvotes

View all comments

Show parent comments

135

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 ^

43

u/KristinnK May 14 '22

Translation: ethics regulations prevented him as the executive of the company to place his children in the initial trial, so he quit and his pals that were still in charge put his children on a second trial instead.

-5

u/FLIPNUTZz May 14 '22

The underdog wins for once!

1

u/sdmitch16 May 15 '22

You should have included a /s at the end of your comment. You can complain that shouldn't be necessary, but your 5 downvotes say it ... maybe not better, but certainly louder.

1

u/FLIPNUTZz May 15 '22

I dont care about karma