Slashdot Mirror

Show me a drug which cures Alzheimers. Show me a cure for Type 1 diabetes, or celiac, or Parkinsons or Huntingdons. Or arthritis. Show me a new antibiotic that works on pandrug-resistant staph.

Miracles all around and you don't even know it. Shit's crazy.

Can't give you cures for the specific diseases you've listed, but if you want a big-scary-incurable disease, how about VRTX's Kalydeco, the first drug to actually addresses the root cause of Cystic Fibrosis?

If you take a look at VRTX's stock chart this year, you'll see it's been quite a wild ride. That's because curing CF is just one of the things they've been fiddling with...

Show me a cure for any viral infection, or even an effective palliative for the common cold.

Incivek can't cure the common cold, but Hepatitis C is definitely viral. Incivek is VRTX's cure for HepC, and is effective in about half the time of the previous leading therapy. Incivek was was approved last May, and may be rendered obsolete in less than 2-3 years VRUS's PSI-7977 (still in clinical trials) treatment for HepC, which promises a pill-based cure. Cures as fast as Incivek, but no more interferon injections. The tech was promising enough that VRUS got bought out by GILD for $137 two weeks ago. VRUS's stock was in single digits less than a year ago.

I see miracles all around me,
Do due diligence, it's all astoundin',
VRUS, VRTX, don't mean to be curt,
Fuckin' biotech, how does it work?
(And I don't wanna talk to a stock analyst,
Those motherfuckers lyin', and gettin' me pissed. :)

Sorry, but this is *not* any innovative science. Rather, it is a computational reproduction of facts already well known. Nothing more than a typical molecular modeling class assignment during a graduate chemistry education.

He did not invent any new drugs - the really breakthrough was by the researchers of Vertex Pharmaceuticals, see for example VX-770.

He did not discover the mechanism of action of the drugs. Rather, he took published protein structures and published compounds and re-ran some docking studies (of the same type Vertex and other pharmaceutical companies probably spend hundreds of thousands of processor hours on, with the difficulty that they had to check tens of thousands of compounds, not just two already known to work).

He was not the first to notice that different promising compounds in clinical trials have different points of interaction with the defective proteins of CF. Thinking that a drug combination may be useful is not exactly a new and brilliant insight, and this was for example even discussed a couple of months ago in CE&N (the general chemistry member journal of the American Chemical Society). I am very confident that is has been evaluated before, and probably there are patents already filed.

The only interesting point here is that the guy is 16,not 20 or 22 like the normal chemistry student. But then pressing the right buttons in a molecular modeling software is really not that difficult, especially when you already know the outcome you want to reproduce.

So where's your results? Where's your research follow-up of the drug combinations that have failed? Why is this kid having different results than what is documented already? If this work has been done already then this 16 year old's discoveries wouldn't be newsworthy now would they?

The fact is this may have been overlooked due to competition between drug companies rather than looking at the big picture. The cure. Leave it to someone who isn't out to make money to get real results.

There are about 30,000 CF patients in the US. I've seen numbers that say 1 in 3500 are born with CF, but a number possibly die shortly after birth. Note that numbers vary by ethnicity, so statistics may vary by how categorized.

http://www.cff.org/AboutCF/

30,000 is a very small number to build a market on when compared to diseases/syndromes like MS (400,000), breast cancer (200,000+ new cases in 2010), HIV (over 1,000,000). (US only)

Sorry, but this is *not* any innovative science. Rather, it is a computational reproduction of facts already well known. Nothing more than a typical molecular modeling class assignment during a graduate chemistry education.

He did not invent any new drugs - the really breakthrough was by the researchers of Vertex Pharmaceuticals, see for example VX-770.

He did not discover the mechanism of action of the drugs. Rather, he took published protein structures and published compounds and re-ran some docking studies (of the same type Vertex and other pharmaceutical companies probably spend hundreds of thousands of processor hours on, with the difficulty that they had to check tens of thousands of compounds, not just two already known to work).

He was not the first to notice that different promising compounds in clinical trials have different points of interaction with the defective proteins of CF. Thinking that a drug combination may be useful is not exactly a new and brilliant insight, and this was for example even discussed a couple of months ago in CE&N (the general chemistry member journal of the American Chemical Society). I am very confident that is has been evaluated before, and probably there are patents already filed.

The only interesting point here is that the guy is 16,not 20 or 22 like the normal chemistry student. But then pressing the right buttons in a molecular modeling software is really not that difficult, especially when you already know the outcome you want to reproduce.

Right on. Look at the two articles published the other day in the New England Journal of Medicine regarding the use of hypertonic saline in patients with Cystic Fibrosis. Both of those studies were supported by the Cystic Fibrosis Foundation which has done a tremendous job marshalling resources toward finding a cure and new treatments for a rare disease.

New England Journal of Medicine: http://content.nejm.org/
Cystic Fibrosis Foundation: http://www.cff.org/