Omacetaxine for CML

[Guest guest]

This is the drug that I took when it was called HHT or homoherringtonine, the

first trial I was in at MDACC. It has come full circle with another name and

been approved as an Orphan Drug by the FDA. Jackie Schneider also took it for

about 2 years to knock out her mutation. I took it in a central venous

catheter, but it is now administered with a hypodermic needle. There was a

discussion about this drug earlier this year, however, we have many newcomers

who might be interested in this venture by Chem-Genex. Sometimes things that

were once old are new again and this drug has been resurrected and found to

knock out the T-315i mutation.

Article Date: 03 Jun 2009

" Roswell Park investigators, under the direction of Dr. Wetzler, evaluated

omacetaxine in 60 patients who had failed at least two TKIs. Sixty-five CML

patients participated in this phase II clinical study. Dr. Wetzler and

colleagues report patients experienced treatment responses in both the white

blood cells (hematologic) and the marrow cells (cytogenetic).

" Eighty percent of the chronic-phase patients experienced a complete hematologic

response (CHR) rate and no disease progression for a median of 7.5 months. In

this same group, 20% had a major cytogenetic response rate, and median response

to disease progression was 2.7 months. For study patients in the accelerated

phase, a 60% CHR rate was observed, with a median of 8.9 months without disease

progression; and for blast phase patients, a CHR rate was 40%, with a median

duration of 5.7 months.

http://www.medicalnewstoday.com/articles/152346.php

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September 09, 2009 Press Release

ChemGenex to Present at Rodman & Renshaw Global Investment Conference in the

U.S.

September 09, 2009 Press Release

ChemGenex Submits New Drug Application for OMAPRO™ (Omacetaxine Mepesuccinate)

to U.S. FDA

September 09, 2009

" ChemGenex Pharmaceuticals Limited (ASX:CXS) announced today the completion of

its New Drug Application (NDA) submission to the U.S. Food & Drug Administration

(FDA) for OMAPRO™ (omacetaxine mepesuccinate). OMAPRO™ is being developed for

the treatment of patients with chronic myeloid leukemia (CML) who have failed

treatment with imatinib and who have developed the Bcr-Abl T315I mutation.

Imatinib, a tyrosine kinase inhibitor (TKI), is the first-line standard of care

for patients with CML. OMAPRO™ is a first-in-class cetaxine which works

differently from imatinib, and the second-line TKIs nilotinib and dasatinib.

" OMAPRO™ has received Orphan Drug designation in the U.S. and in the European

Union, and has received fast track status from the FDA. OMAPRO™ demonstrated

clinical benefit in the pivotal Study 202, in CML patients who had failed

imatinib and have the T315I mutation. Interim data were recently presented at

the American Society of Clinical Oncology Annual Meeting. "

http://www.ritabiotech.com/news.html?id=189

Details on the clinical trials can be accessed from the following websites:

http://www.clinicaltrials.gov/ct2/show/NCT00375219?term=homoharringtonine & rank=9

and http://www.tkiresistantcmltrials.com

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August 29, 2009 Good news for the Brits

" A new report, launched today by Leukaemia CARE, shows that 60 percent1 of

Primary Care Trusts (PCTs) and Local Health Boards (LHBs) surveyed are funding

National Institute of Health and Clinical Excellence (NICE) approved treatments

for blood and lymphatic cancers. However, despite there being marked

improvements in levels of care and treatments available to blood and lymphatic

cancer patients, significant obstacles in accessing treatment still exist. Tony

Gavin, Director of Cancer Campaigning and Advocacy, Leukaemia CARE, commented:

" One of our greatest concerns is that patients should have access to treatment.

This report shows that some cost effective treatments are not always being made

available to patients and that confusion over the stages at which treatments can

be made available is delaying vital treatment by as much as 90 days - days that

some patients just don't have. "

" The report is based on results from a survey titled, 2009 Haematology Survey,

by Leukaemia CARE. It investigates some of the unique challenges faced by people

with blood cancers in getting access to treatment and care. The survey examined

to what extent NICE guidance is being implemented, how and when PCTs and LHBs

are making treatments available to patients and the impact of the recent

Government reforms on patient access. "

http://www.medicalnewstoday.com/articles/161323.php

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(19 June 2008) " The existence of a small population of 'cancer-initiating

cells' responsible for tumour maintenance has been firmly demonstrated in

leukaemia. This concept is currently being tested in solid tumours.

Leukaemia-initiating cells, particularly those that are in a quiescent state,

are thought to be resistant to chemotherapy and targeted therapies..... " Read

the article in Nature.

http://www.nature.com/nature/journal/v453/n7198/full/nature07016.htmlI

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(2009, Sep 5) G-CFS - maybe not a good thing?????????

" Stimulating Factor (G-CSF), together with imatinib, has led to a significant

reduction in leukemic stem cells in vitro. In this paper, we design a novel

mathematical model of stem cell quiescence to investigate the treatment response

to imatinib and G-CSF. We find that the addition of G-CSF to an imatinib

treatment protocol leads to observable effects only if the majority of leukemic

stem cells are quiescent; otherwise it does not modulate the leukemic cell

burden. The latter scenario is in agreement with clinical findings in a pilot

study administering imatinib continuously or intermittently, with or without

G-CSF (GIMI trial). Furthermore, our model predicts that the addition of G-CSF

leads to a higher risk of resistance since it increases the production of

cycling leukemic stem cells. Although the pilot study did not include enough

patients to draw any conclusion with statistical significance, there were more

cases of progression in the experimental arms as compared to continuous

imatinib. Our results suggest that the additional use of G-CSF may be

detrimental to patients in the clinic. "

http://www.ncbi.nlm.nih.gov/pubmed/19749982?ordinalpos=1 & itool=EntrezSystem2.PEn\

trez.Pubmed.Pubmed_ResultsPanel.Pubmed_DefaultReportPanel.Pubmed_RVDocSum

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(Oct. 2009)Plately Recovery Treatments

" The National Heart, Lung and Blood Institute of the National Institutes of

Health has awarded a $16.8 million, seven-year grant to launch a bicoastal

research partnership between Fred Hutchinson Cancer Research Center/University

of Washington Cancer Consortium and Children's Hospital of Philadelphia. "

http://www.medicalnewstoday.com/articles/165900.php

Source: Dean Forbes, Fred Hutchinson Cancer Research Center

FYI,

Lottie Duthu