PLX4032

Jes, it is just for people with a melanoma primary, though?

I have only read page one....

Sounds like any cancer that has the specific mutation on which this drug acts, but it usually starts as melanoma.

As I understand it, metastases are still considered to be the same type as the original cancer.

(Hopefully someone will disavow me of that belief if it is wrong.)


So.....some non-melanomas have the mutation, you think?

That's how I understand it as well.



The article did not state definitively one way or the other, but I gathered that this particular mutation is unlikely or unknown to begin as other forms of cancer.

Yeah...that was the best I could make of it, though I am not clear at all, either.

Still...if this really helps with some melanomas!!!!

I didn't get what the mechanism was for how it worked, but I hope a similar approach can be used on other cancers.

Indeed.

It's so hard to know whether the research will proceed in the same slow, painful way, or if there is going to be some sort of sudden cascade of critical information that makes a huge difference for many cancers all at once!

The article is very vague on details and talks mainly about particular people's experiences, rather than the specific mechanism the drug uses to work.

External Source:

Cool...thanks!

My impression is that even though this particular drug targets only a particular gene/cancer, that the general methodology may be applicable to other gene sequences if they can be isolated.

I guess the challenge now would be to isolate other cancer specific genes and modify the drug to target them.

And then of course it'll be an endless battle to keep up with new cancer mutations.

The methodology seems promising. Score one for the value of genetic research.

I just think the whole thing is astounding, even if it just saves one life that we would've otherwise, well, just kinda written off. There's a guy mentioned -- they show his scans -- his cancer had spread to his bones and the docs all thought, "death sentence". He takes this stuff and suddenly 2 months later his tumors have shrunk.

I really, really hope this methodology can be used for a lot of different types of cancers. I mean, if it can work -- and be proven to be continuing to work over a long (ten years? five? I dunno, whatever the standard is) term, then we are talking Nobel Prize here. Hell, we're talking about the doctors being knighted, about people naming their kids after the doctors.

The article is typical of the repositioning of the New York Times as a quasi-tabloid. The drug's specific mechanisms have been known for some time >

http://www.sciencecodex.com/trial_of_new_treatment_for_advanced_melanoma_shows_rapid_shrinking_of_tumors
> and there are many caveats. Btw, Interleukin 2 (mentioned in this excerpt) was a previous "cancer cure" which nearly bankrupted both Boston University's endowment and its then president. However he obviously believed in its curative effects since he had his own son (ill with AIDS) treated with it rather than with other AIDS drugs. It's still around, though the original biotech company that came up with it went backrupt. But drug uses can be found even for older formulations - e.g. Thalidomide is still in use as a cancer drug. This new compound, PLX4032, is promising, but blocks only:

well

Well I do know something about the methodology. It's a product of the ability to sequence large amounts of human DNA in a short amount of time.

The inventors of the drug/molecule would have had to sequence or rely on information about people who sequenced many many types of cancer cells and found what genes mutated causing the cell to divide uncontrollably. Once they found the gene they had to figure out an attack plan. Change the mutation back to normal, change it to something harmless, disable its ability to be expressed, etc. In this case I think they chose the ladder by creating a binding molecule (not easy) that would somehow disrupt the expression chain.

The details of the disruption are probably not going to be divulged so readily but the most astounding thing is that whatever they did required an incredible understanding of genetics and hopefully little luck. This means that the level of understanding of genetics, thanks to stem cell research, human genome projects, etc, is at a point know where really useful drugs can be created.
It's definately something to be excited about! The methodology can definately be used on many types of diseases and hopefully we will see many more types of drugs like this on many hated diseases.

They are going to have drawbacks of course as molecules that bind to certain genes are bound to have wierd effects on other similar proteins in the body but hopefully that can be mitigated and/or eliminated by improving the attack.

So if you are rich and you want to invest in profoundly important research that will help billions of people, invest in this.
If you are not rich and you want to donate to profoundly important research ...., donate to this research.

Sorry I'm just excited for the people suffering from cancer. I hope and pray there are no untreatable side effects. Chemotherapy sucks. May God forbid that any child ever has to go through that in the near future.

I've been doing a little research as I may want to get involved in the clinical trial, now at stage 3, which means that the drug has shown promise and does not have serious side effects, like death.

You may have seen the incredible pictures of the tumors that disapppeared. The drug only works on tumors which have the B-RAF genetic mutation, which is about 1/2 of melanomas and about 6% to 10% of other tumors. The drug (PLX4032) seems to have this dramatic effect on the tumors for about 6 months, and then, unfortunately, they recur. The important take-away is that this drug does have this dramatic impact on the B-RAF tumors, which indicates that this type of treatment, called "targeted therapy," is a potentially important new way of treating cancer. Before, there was only chemotherapy, which is basically poison that kills good and bad cells alike and is mostly very toxic, and immunotherapy, which attempts to get your own immune system to recognize and kill cancer. This als has a lot of potential.

Bottom line: an exciting new line fo research.