Omacetaxine

[Guest guest]

Dear Group,

" ChemGenex is currently developing omacetaxine as a potential treatment for a

range of blood malignancies, including CML, and is completing a pivotal study in

CML patients who harbor the T315I mutation. The company expects to complete its

filing of a New Drug Application for use of omacetaxine in that patient

population to the U.S. Food and Drug Administration by mid-year.

" Greg Collier, Ph.D., Managing Director and Chief Executive Officer of ChemGenex

added, " The results of Dr. Li's animal study are very encouraging and we are

currently collaborating with Professor Tessa Holyoake from the United Kingdom,

to carry out similar investigations in primary human stem and progenitor cells.

In the meantime, ChemGenex remains focused on our primary objective of

developing omacetaxine as a therapeutic option for CML patients who have

developed the T315I mutation and who are resistant to all first and second line

TKIs. This is the most pressing unmet medical need in the field of CML

management. "

http://www.eurekalert.org/pub_releases/2009-03/ka-dst032409.php

From my experience, this is an old drug with a new name. I was a patient in a

trial in 2000 when it was called HHT, then Homoharringtonine, then Ceflatonin®.

When I was in the trial, there was no mention of T315i, I took it in tandem with

Inteferon. Sometime later it was found to have some properties that killed the

T315i mutation. I believe Jackie Schneider is still on this drug of it's own,

not with any other combo. As far as I know she no longer has the T315i mutation

and is waiting to get into another trial. Maybe she could bring us up to date

on her current status with the drug and how well it is performing for her.

I personally believe that in the future we will have more combos, as in the

HHT-INF combo; Gleevec-Peg Intron Combo. The patients that I know who were on

the latter did extremely well and are now PCRU, but I know of no one in the same

HHT-INF combo that I was in, so I have no data to back up whether it was

successful or not. Evidently it was not for me, because I went to another

trial. The patients I met who were on INF also left for other trials.

I don't know if they changed the drug in any way to make it more potent, but it

certainly has a long life, as it keeps cropping up with different names. That's

just my opinion and my experience with the drug. Whether it makes the cut

remains to be seen in future trials, but I fail to see the benefit of getting

another patent with another name if it is the same drug, (Drugs are usually

prescribed off-label when doctors discover new uses for a drug. There's nothing

inherently wrong with this. Doctors may discover that a device or drug can be

beneficial in more instances than just those the FDA evaluated when it was first

developed.) May 4, 2009. http://www.mayoclinic.com/health/off-label/DI00088

More on off label drugs:

Off-label uses have not been formally evaluated, and evidence provided for one

clinical situation may not apply to others. As an area of controversy, off-label

use is subject to the contradictory expectations of various stakeholders,

including health care payers, the pharmaceutical industry, physicians, and

consumers. The FDA has a role in balancing these expectations, but it currently

does so primarily through regulating corporate marketing. Although there is a

strong rationale for greater FDA involvement in off-label use, it is moving

toward relinquishing control in its new draft guidelines.

Material copied from: http://content.nejm.org/cgi/content/full/358/14/1427 On

April 3, 2008.

FYI,

Lottie