Sweet drug clears cholesterol, reverses heart disease–and was found by parents

What a heart-breaking introduction to the world of cancer research. I personally know an immensely bright person working on a vicious type of childhood sarcoma cancer, and his work is innovative and may, in time, lead to a highly specific form of treating cancer that won't have the same long-term side effects as the "one size fits all" type of chemo approach. It's important work, and he has received both private and public funding for his lab because he's making progress. It's slow.

One thing I have learned though, is that once a parent discovers their child has a diagnosis of that type of cancer, they go online and look it up. Eventually they find his name if they look long enough. Some of those parents write pleading, begging letters to him looking for some kind of treatment - anything. Other than giving them a name for the best clinic in the US in his opinion, there is nothing he can do to help but continue his work. Such letters do appear to take a toll on him, emotionally, because he does care.

As a person with a rare, permanent genetic condition myself, I have come to accept and live peacably with the outlook that people, as a group, simply do not find ways to care about other people's problems with a sliver of intensity that they do once one of those problems becomes their problem. It's just the way our species seems to be wired for now.

I never knew that kids cancers cannot be treated in the same way as adult cancers. I just assumed that the advances in cancer treatment applied to all ages.

This message needs to get out.

Truly sorry for your loss.

Just wanted to add my perspective as a US medical student--

First I would like to say that I'm sorry for your loss, and please don't take my comment as defending or justifying our current research system.

But the explanation as to why there is so little medical research for children has to do in part with the federal laws regarding Protection of Human Research Participants (PHRP), specifically the "vulnerable populations" subsections, which define additional protections that are required to perform research on such groups (for children, it is Subpart D).

I won't go into detail, but the gist is that we err far on the side of caution. Whether that's for the best is another matter.

There's a huge amount of red tape/obstacles for performing clinical trials on competent adults, and much more if your study includes children or other "vulnerable populations". There is a whole list of exclusion criteria that you have to get past before your study can be approved and you can start seeking funding (some of which may be difficult to prove or justify to the IRB), you need parental permission and the child's assent (which also may be difficult to prove, and doubles your chances of losing a subject), risk assessment and the associate requirements/protections are handled differently, and so on.

Here are some resources if you want more details:

http://www.hhs.gov/ohrp/humansubjects/guidance/45cfr46.html#...

http://grants.nih.gov/grants/guide/notice-files/not98-024.ht...

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...

Terribly sorry for your loss. My younger brother passed away due to leukemia and I feel your frustration.

I do want to give some credit to Light the Night though. It is run by Leukemia & Lymphoma Society, not ACS. LLS has an 84% score on Charity Navigator, and while that score could be better, they do spend 73% of their expenses on the programs they exist to deliver, which is very good.

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)

I see your point. And I don't think it's quite as horrible, and might actually be somewhat more responsible to only give the drug to one kid to start with.

Assuming, of course, the kids aren't at risk of death any time soon (they seem fairly healthy in that picture), and the results could be measured fairly readily.

Kid A could be the control group? It's done for a lot of other important research, and they could stop the trial and give Kid A the drug once they prove it makes a difference.

> 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.

But what about Christmas?: http://www.girlgeniusonline.com/comic.php?date=20090706

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.

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).

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