Astral Codex Ten

Boston Children’s Hospital

9 min read

Boston Children’s Hospital

Article

Boston Children’s Hospital is a recurring venue in the Astral Codex Ten archive, appearing 2 times across 2 issues between August 05, 2021 and August 06, 2021. The archive places it in contexts such as “Around 2010, Boston Children’s Hospital found some loophole”; “approved the Investigational New Drug application that let Boston Children’s Hospital keep using it”. It most often appears alongside Europe, FDA, ACT UP San Francisco.

Metadata

  • Category: Venues
  • Mention count: 2
  • Issue count: 2
  • First seen: August 05, 2021
  • Last seen: August 06, 2021

Appears In

Source Context

Recovered passages from the original issue text. When the raw archive preserved outbound links inside the source passage, they are listed directly under the quote.

Adumbrations Of Aducanumab
August 05, 2021 · Original source
A while back I learned about cholestasis in infant Short Bowel Syndrome, a rare condition with only a few hundred cases nationwide. Babies cannot digest food effectively, but you can save their lives by using an IV line to direct nutrients directly into their veins. But you need to use the right nutrient fluid. The FDA approved one version of the nutrient fluid, but it caused some problems, especially liver damage. Drawing on European research, some scientists suggested that a version with fish oil would cause less liver damage - but the fish oil version wasn’t FDA-approved. A bunch of babies kept getting liver damage, and everyone knew how to stop it, but if anyone did the FDA would take away their licenses and shut them down. Around 2010, Boston Children’s Hospital found some loophole that let them add fish oil to their nutrient fluid on site, and infants with short bowel syndrome at that one hospital stopped getting liver damage, and the FDA grudgingly agreed to permit it but banned them from distributing their formulation or letting it cross state lines - so for a while if you wanted your baby to get decent treatment for this condition you had to have them spend their infancy in one specific hospital in Massachusetts. Around 2015 the FDA said that if your doctor applied for a special exemption, they would let you import the fish-oil nutritional fluid from Europe, but you were only able to apply after your baby was getting liver damage, and the FDA might just say no. Finally in 2018 the FDA got around to approving the corrected nutritional fluid and now babies with short bowel syndrome do fine, after twenty years of easily preventable state-mandated damage and death. And it’s not just this and coronavirus, I CANNOT STRESS ENOUGH HOW TYPICAL THIS IS OF EVERYTHING THE FDA DOES ALL THE TIME.
Inline links: Finally in 2018
Details Of The Infant Fish Oil Story
August 06, 2021 · Original source
In 2002, Boston Children’s Hospital needed to give IV nutrition to a kid with a soy allergy. The hospital only had soybean-based fluid, and tasked our hero Dr. Gura with finding a solution. She asked a bunch of experts and “put the question on various nutrition listserves”, and finally some nutritionist who attended European conferences mentioned that Europe had a fish-oil based fluid, Omegaven. If they could get permission to import Omegaven from Europe, they could maybe save this kid. They asked the FDA for permission, filled out the relevant forms, and (to its credit) the got approved within 48 hours. They were able to get an emergency shipment of Omegaven from Europe and the kid got better.
After this, Boston Children’s Hospital and the FDA kind of became pen pals - every time BCH got a new PNALD patient, they would ask the FDA for permission to import Omegaven from Europe, the FDA would grant it, and the patient would make a miraculous recovery. Eventually they worked together with the FDA to get something called an IND, which meant their hospital only was allowed to use Omegaven in the context of investigating and studying it.
It didn’t mention some potentially exculpatory factors, like that there weren’t that many studies in favor (also, although I didn’t get into this above, some people thought the fish oil based fluid might cause bleeding, although this turned out not to be true). I’m sorry for these errors and will add a link to this post on my Mistakes page. Still, I think the basic structure was right - the new fluid was better than the old fluid, this was pretty clear in terms of miraculous recoveries, and it took 14 years between the first patient saved and full FDA approval. You’ll notice that in Dr. Gura’s story (whose tone I tried to reproduce in Part II) it’s not obvious that this is an anti-FDA morality tale at all. Dr. Gura gently criticizes other doctors and hospitals for not realizing the value of Omegaven fast enough. She criticizes funding agencies for refusing to fund Omegaven studies, and journals for refusing to publish Omegaven articles. She even criticizes Fresenius, the pharma company behind Omegaven, for failing to advocate hard enough for its own product. The FDA, in comparison, comes out looking pretty good. They approved the first few original single-patient exemptions to import Omegaven from Europe. They (eventually, after a lot of work) approved the Investigational New Drug application that let Boston Children’s Hospital keep using it (I’m still unclear whether any other hospitals got INDs for this). They even contributed a little funding to get one small study done. It wasn’t enough, but the FDA is not primarily a funding body, actual funding bodies dropped the ball on this one, and so it’s really impressive that the FDA went above and beyond to try to move Omegaven through the pipeline. Dr. Gura seems to have been left with the impression that the FDA was one of her few allies during this fight, which I think is fair. I mentioned in a section of my recent post, “Sympathy For The Devil”, that I think the FDA as an agency is often quite good. They’re smart, caring people, and they usually carry out their mandate well - so well that the few exceptions, like aducanumab, are highly newsworthy. I have no objection to Dr. Gura’s mostly-positive portrayal of them. My problem is: doing anything in medicine is illegal until you clear a giant hurdle. To clear the hurdle, you have to pay millions (sometimes billions) of dollars, fill in thousands of pages of forms, conduct a bunch of studies that can be sabotaged for reasons like “this drug is too good so it would be unethical to have a control group”, and wait approximately ten years. You have to clear this hurdle to do anything, even the most obviously correct actions. Everything starts out illegal, and then a tiny set of possible actions is exempted from the general illegality. The easiest name for this hurdle is “the FDA”, since they’re the agency charged with enforcing it. Given the existence of the hurdle, various people come off looking like villains, eg: Yes, funding agencies look bad, insofar as they refused to provide enough millions of dollars to clear the hurdle. But there’s no funding agency so rich that they can can give hurdle-clearing amounts of money to every promising new treatment. Yes, the pharma company involved looks bad, insofar as it balked at the time and expense involved in starting the approval process for its product. But who made the hurdle so high that even a pharma company - one with a great drug and the chance to make millions of dollars off it - took one look at the amount of time/energy/expense involved and said “no thanks”? And if pharma companies are intimidated by the process, what hope does anyone else have? Yes, the other doctors who didn’t really care about this potentially life-saving treatment look bad. But in their defense, they would have had to go through the FDA special-exemption import process to even begin testing this on their own patients. I once had a patient who needed an unusual European drug not available in this country. briefly considered the FDA special-exemption import process, and decided to do the reasonable thing and walk my patient through the process of ordering it illegally on the Internet. I am not super proud of this, but at least the patient got their drug, which is more than you can say about most of the times this kind of thing happens. …and given the existence of the hurdle, the FDA (as an agency) comes off looking good. They helped coach Dr. Gura through the process of filling out forms. They helped fund Dr. Gura’s study. They worked extra hard to process the Omegaven application as fast as they could. Good for them. But as long as this giant hurdle sits in the path of medical practice, lots of people will inevitably be villains, and lots of other people will need to display an unusual and not-fair-to-expect-of-them level of heroism to get anything approved at all. Partly this is about Dr. Gura having to move heaven and earth (and become something of a political activist) to gather the coalition that eventually got Omegaven approved. But partly it’s about - did you notice how unlikely the discovery of Omegaven was? It required: Dr. Baker, a BCH nutritionist, was in touch with the European medical establishment and had learned about their alternative nutritional fluid.
Inline links: Mistakes page