
Sweet drug clears cholesterol, reverses heart disease–and was found by parents - gmac
http://arstechnica.co.uk/science/2016/04/drug-clears-cholesterol-reverses-heart-disease/
======
dang
[https://news.ycombinator.com/item?id=11450451](https://news.ycombinator.com/item?id=11450451)

------
baldfat
Since that was yesterday's HN I want to just say that as a parent who's son
died of cancer at 12 that this shows how little resources in time and money is
spent on children with "Small populations" of patients.

This includes every single childhood cancer out there. The kids get 4% of the
federal research money and $0.01 from every dollar from American Cancer
Society (Light the Night) (Notice they don't have any pictures of children for
4 years now due to parents being so mad at being the poster child that got the
least.

Childhood cancers are different then adult cancers and adult cancer treatments
don't work for the VAST majority of the kids due to their whole body is
growing as fast as the cancer. Adult treatments kills kids. So we now have 1
new drug int he past 20+ years! Ok enough of a rant.

~~~
cmdrfred
Only about 60%[0] of the American Cancer Society revenue goes to charity to
begin with so that is to be expected. Most of the cancer charities I've seen
(Susan G Komen et all) are rather bad.

[0]
[http://www.charitynavigator.org/index.cfm?bay=search.summary...](http://www.charitynavigator.org/index.cfm?bay=search.summary&orgid=6495)

~~~
baldfat
Well the issue specifically is Childhood Cancer Research funding.

This link will state a good pissed off parents stance on ACS. They raised
funds for their daughter and asked people to donate to ACS instead of flowers.
Then they realized where their money was going after ACS pulled funding of
summer camps across the nation with out any warning.

[http://curechildhoodcancer.ning.com/profiles/blogs/my-
though...](http://curechildhoodcancer.ning.com/profiles/blogs/my-thoughts-
about-the-american-cancer-society)

~~~
melling
ok, can someone explain the best places to donate? I've donated to Fred Hutch
in the past:

[http://www.fredhutch.org/en.html](http://www.fredhutch.org/en.html)

Who's effective with the money and doing great research?

~~~
cmdrfred
I like this article myself:
[http://www.theatlantic.com/business/archive/2015/06/what-
is-...](http://www.theatlantic.com/business/archive/2015/06/what-is-the-
greatest-good/395768/)

~~~
melling
That article has nothing to do with cancer research.

------
randcraw
The paper in Science Translational Medicine:

[http://stm.sciencemag.org/content/8/333/333ra50](http://stm.sciencemag.org/content/8/333/333ra50)

This does sound promising. The compound (2-Hydroxypropyl-beta-cyclodextrin, or
CD) was approved by the FDA in 2009, but only for compassionate use in
children for treating Niemann-Pick disease intravenously (IV). Clinical trials
are ongoing for non-compassionate access (again, only for treating Niemann-
Pick).

However intramuscular (IM) use for treatment of atherosclerosis in the general
population probably will have to go through the entire Phase I-II-III clinical
test cycle before CD will become available to that end. That's likely to take
another 5-10 years, since such trials have not yet begun. I can't guess
whether the Niemann-Pick trials might expedite acceptance of CD for a
secondary target using a different delivery method (IM vs IV). Maybe.

------
maxerickson
Small discussion from yesterday:

[https://news.ycombinator.com/item?id=11450451](https://news.ycombinator.com/item?id=11450451)

------
logfromblammo
The parents want to try a novel method of delivery to treat their kids' shared
disease.

Their _twin_ kids.

You see where I'm going with this? I'm going to go sit in the corner now, and
think about why the first thought that came to my mind was so horrible.

~~~
maxerickson
Another way to look at it is only risking harming one of them, so I'm not so
sure it is horrible to consider starting out only treating one of them.

(In this case the drug doesn't seem to be a big risk though)

------
melling
Here's a great PBS series to watch if you want to learn more about cancer.

[http://www.pbs.org/show/story-cancer-emperor-all-
maladies/](http://www.pbs.org/show/story-cancer-emperor-all-maladies/)

It's based on this book, which I have not read: [http://www.amazon.com/The-
Emperor-All-Maladies-Biography/dp/...](http://www.amazon.com/The-Emperor-All-
Maladies-Biography/dp/1439170916)

------
dmritard96
Seems like in many cases, just trying many things on many tissues is whats
needed to find solutions. I know there are groups like Opentrons and there are
groups out there working on snythetic tissue generation, but I wonder is the
bottle neck tissue availability, researcher examination time or studying long
term effects. Seems like we need to open all these bottle necks up to really
iterate.

------
tmaly
I would like to know what the side effects of using this as an anti-
cholesterol treatment are.

It seems like this drug could be a general benefit to the population.

~~~
brad0
From the sounds of things there's nothing really when taken orally, though I'm
not sure if taking this orally helps with cardiovascular issues.

------
B1FF_PSUVM
> the parents kept digging into the research

That wasn't probably in the cards when it meant spending days or weeks in
libraries, with fewer chances of hitting gold.

It's almost enough to pay for all the rest of the "net experience".

------
api
I wonder if simple compounds like this get forgotten because they are not
patentable, so there is no monetary drive to do the research.

~~~
giarc
It likely wasn't picked up by Big Pharma because the research was for a
disease that affects a couple hundred people in the states. There's no money
in that disease, but if the research had been in cardiovascular disease, Big
Pharma would have been all over it (which it looks like is now the case).

------
marz0
How many more compounds are out there with valuable research behind them that
we haven't realized?

~~~
djcapelis
Many! Science exists because the world is not yet fully known and isn't likely
to be anytime soon!

