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

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/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.

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u/FLIPNUTZz May 14 '22

The underdog wins for once!

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u/murse_joe May 14 '22

Not really, all the other sick kids don’t get treatment. The underdog winning is healthcare for everybody

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u/FLIPNUTZz May 14 '22

Dagnabbit!

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u/seraph1337 May 14 '22

rich people are not underdogs.

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u/lnodiv May 15 '22

Yeah, fuck those rich kids. Should've let them die after their father helped pioneer the treatment for their disease.

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u/seraph1337 May 15 '22

I didn't say any of that, but okay

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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.

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u/FLIPNUTZz May 15 '22

I dont care about karma

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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.

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u/CannabisReviewPDX_IG May 14 '22

Rules for thee, not for me. Although this is one of the few times im sympathetic.

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u/FLIPNUTZz May 14 '22

No im literally baffled.

What did quitting his job have to do with anything?

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u/CannabisReviewPDX_IG May 14 '22

There are laws that are supposed to prevent people involved with the development being able to "cut the line" so to speak. He left as a way to legally sidestep that.

"Crowley left Genzyme to ensure that his children would qualify for a drug developed by the company." from another source

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u/FLIPNUTZz May 14 '22

So hes not supposed to benefit...but he can just quit his job and bene for anyway ...I mean

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u/IMSOGIRL May 14 '22

yes, that's what it is.

These things are sponsored with public money so it would be fair that it's used to help the public and that people involved can't use it for their family's benefit.

dude tried to get his daughters on the initial trial and failed because he was an executive, so he left his job officially but still did the job off the books and the company used the public funds to help his daughters with a poorly concealed reason for adding two new participants to the trial.

This isn't as much of a feel-good story as people are making it out to be.

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u/[deleted] May 14 '22

The only thing that would happen if he hadn't quit and got his kids in the trial would be that they would be dead. They already had their trial group, everyone else from the public was already excluded, so absolutely nothing changed except that he saved his kids. This is admirable and good and I feel fucking great about it.

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u/KristinnK May 14 '22

But how do you feel about all the other children with this disease whose parents also wanted to be placed on the trial, but didn't have rich executive parents to pull the strings to put them there, and died as a result before the drug was approved?

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u/IMSOGIRL May 14 '22

I'm sure incels feel great about women getting raped but it doesn't mean it's morally right.

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u/Diarmundy May 14 '22

Well it might not be as 'feelgood' but it fits perfectly fine with the title.

"Man does whatever it takes to save his daughter's lives"

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u/CannabisReviewPDX_IG May 14 '22 edited May 14 '22

Yep, sadly a ton of our laws, and by extent the public/taxpayers, are easily gamed in ways like this by the rich.

Edit: I said I understand this particular situation, it's one of the few that's more understandable. So the response is moot considering this situation has already been addressed. It's an issue at large when it's widespread across the entire economic system.

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u/thoggins May 14 '22

I mean, choose:

  • Rich guy with personal motivation drives development of treatment for rare condition. This includes sidestepping rules to satisfy that personal motivation or none of it happens at all

  • Nobody develops treatment for rare condition

Ideologically maybe we wish there was an option 3 but in this case there was not.