Should the FDA prevent dying patients from trying experimental drugs?

[joeglow3]

Yes, I know the source, but I found the article intersting. Additionally, I included links to pages set up for child to see the personal side. Frankly, he is a hulluva cute kid and it pisses me off that we won't try anything and just leave him to die a horrible death:

"The January 31, 2014, Boston Globe front page included two life-and-death stories. One announced that the U.S. Department of Justice would seek the death penalty for Dzhokhar Tsarnaev, who is facing trial for the Boston Marathon bombing. Animated debate about the proper penalty for Tsarnaev continues around Boston.

The other story snapped my head back because it involved a death penalty for Jack Fowler, a 6-year-old boy dying of the more virulent form of Hunter syndrome (also known as MPS-II). The drug he needs to survive, which he is being denied, is one that I had first proposed in 2003 when I was CEO of a small biotech company focused on rare diseases. In 2005, after two years of developing this drug, Shire Pharmaceuticals bought my company over my objections, and I lost touch with the program. Jack’s situation is particularly compelling, but hardly unique.

Hunter syndrome is a simple disease that, absent a spontaneous mutation, kills only boys because the genetic defect occurs in the male chromosome. It is in a class called lysosomal storage diseases in which the absence of one gene on a chromosome means the body cannot produce an enzyme that clears certain cells of a substance that eventually kills those cells. The timing and details of a patient’s deterioration depend on which types of cells the particular disease damages, but without treatment in almost all cases the missing enzyme causes a painful death, usually at a young age.

Every program to address a lysosomal storage disease with enzyme replacement therapy has been at least partially successful, with minimal safety risks. These days it is not a big deal from a technical perspective to make the missing enzyme and then infuse a patient with the enzyme. Shire has been hugely successful with Eleprase, an enzyme for Hunter taken through Phase III trials by my old company."


http://www.weeklystandard.com/articles/can-boy-s-life-be-saved_784914.html


https://www.theisaacfoundation.com/category/jack-fowler/

http://heresjack.com/hunter-syndrome/

[MannyGoldstein]

1. I haven't read the articles, but I know that FDA issues compassionate-use exemptions

For similar situations. Has this been tried?

[joeglow3]

3. That is what the article is about

The family has had no such luck with that route.

The author talked about when he worked for a drug company how often he got calls from politicians who pushed through exemptions for their family and friends, but how hard it can be for someone not politically connected.

[Archae]

2. Too much opportunity for abuse.

If a drug is still unapproved, it's for a reason.

The US had an unapproved drug scandal drug scandal back in the 1960's, a drug manufacturer in Europe was pressuring the FDA and congress so they could sell Thalidomide here in the US.

We all remember how that turned out, right?

[joeglow3]

4. You can't see the difference?

In your example, it was marketed and used to "treat" non fatal issues. In the example I cited, it is a ONE HUNDRED PERCENT GUARANTEE that this kid will die (and die a painful death). What is your concern as it relates to the example you cited?

[Archae]

5. Because we don't know if the drug will work.

[joeglow3]

6. What is the worst case scenario

What could be worse than the kid dying a horrible death in the next couple years?

[pnwmom]

7. Of course not. The FDA approves a lot of testing involving dying patients. n/t