Hello Sandy,

I think what you are referring to is FLT-3 it is a kinase that is found 
to be active in certain types of leukemia, such as CML and AML.  Both c-
kit and FLT-3 are kinases that are active in CML.  Gleevec targets c-
kit, but not FLT-3, if I am not mistaken.  It just may be that this 
young person you mention here has had molecular or gene testing and his 
doctors feel that treatment with a drug that inhibits FLT-3 would be 
better suited to him.

I have pasted an article from a news release in November 2004 that 
indicates that a biotech company has signed a licensing agreement with 
Novartis to develop this molecule and bring it to market.  You should 
keep us posted on how well this works for this patient.  By the way, 
his mother donated "stem cells" for the BMT.  I am sorry that he has 
relapsed and hopefully this drug regimen will help him.

Best,
Cheryl-Anne

Novartis Selects Drug Candidate for Clinical Development from Protein 
Kinase Research Collaboration with Vertex

--Novel compound, VX-322, targets key mechanisms implicated in leukemia 
and other cancers--

Cambridge, MA, November 10, 2004 -- Vertex Pharmaceuticals Incorporated 
(Nasdaq: VRTX) today announced that Novartis has selected a small 
molecule protein kinase inhibitor, VX-322, from Vertex for clinical 
development for the treatment of cancer. VX-322 is the first drug 
candidate to be transferred from Vertex to Novartis under an amended 
agreement to discover and develop drug candidates directed at the 
protein kinase gene family, and represents a novel, highly targeted 
approach to the treatment of cancer. In conjunction with the selection 
of VX-322, Vertex will receive $10 million from Novartis, a portion of 
which will be recognized by Vertex as revenue in the fourth quarter of 
2004, with the remainder recognized through the term of the contract. 

"Vertex's delivery of this innovative drug candidate highlights our 
progress in drug discovery on many promising molecular targets in the 
kinase gene family, as well as the continued strength of the research 
collaboration between Novartis and Vertex," said Peter Mueller, Ph.D., 
Senior Vice President of Drug Discovery and Innovation and Chief 
Scientific Officer at Vertex. "VX-322 represents a novel approach to 
address key molecular mechanisms that drive tumor growth, and we are 
pleased that it has been selected for development by Novartis, an 
industry leader in cancer drug development."

VX-322 is a potent, selective, dual inhibitor of the Flt-3 and c-kit 
kinases, which function as molecular switches that regulate the growth 
of certain cancers. Flt-3 inhibition has attracted significant 
attention among cancer researchers as a highly targeted approach to the 
treatment of certain leukemias as well as other hematological 
malignancies and solid tumors. Flt-3 is abnormally activated or 
upregulated in a wide range of leukemias, including more than 70% to 
100% of patients with acute myelogenous leukemia (AML). 

Current treatment for AML generally involves aggressive chemotherapy 
with "non-specific" agents that cannot discriminate between healthy and 
diseased cells, which results in significant toxicity and limited 
efficacy. The five-year survival rate for AML patients is 14%. New 
targeted approaches hold the potential to transform the treatment of 
AML: reducing side effects, improving tolerability and increasing the 
efficacy of chemotherapeutic regimens.

In May 2000, Vertex and Novartis entered an alliance to discover, 
develop and commercialize small molecule drugs directed at targets in 
the kinase protein family. The agreement was amended in February 2004, 
providing for a more rapid and earlier stage transfer of the drug 
candidates discovered by Vertex to Novartis for clinical development. 
Upon selection, Novartis holds worldwide development and 
commercialization rights to VX-322. Vertex will receive milestone 
payments based on clinical advancement of VX-322, and will receive 
royalties based on commercial sales.

Background Information: Flt-3 and c-kit Kinases, and Discovery of VX-322
VX-322 was discovered in a joint effort by scientists in Vertex's San 
Diego and Cambridge research sites, leveraging expertise in protein 
biochemistry, structural biology, high throughput cell assays and 
medicinal chemistry. Earlier in 2004, Vertex researchers published the 
three-dimensional atomic crystal structure of Flt-3 in the scientific 
journal Molecular Cell1, a key scientific advance that provided insight 
into the mechanism by which mutated forms of the Flt-3 receptor can 
activate themselves, triggering uncontrolled proliferation of immature 
blood cells characteristic in several types of leukemia. 

Flt-3 kinase is abnormally activated or upregulated in the majority of 
patients with AML, as well as in patients with acute lymphoblastic 
leukemia (ALL) and chronic myelogenous leukemia (CML). Specific Flt-3 
mutations that are believed to be key drivers of cell proliferation are 
present in up to 40% of AML patients. In addition, the protein c-kit 
has been found at high levels in 60% to 80% of AML patients. 
Preclinical studies conducted at Vertex using cells isolated from AML 
patients have suggested that dual Flt-3/c-kit inhibition provides more 
potent reduction in cell proliferation compared to inhibitors of Flt-3 
or c-kit kinase alone. These data have enabled researchers to design 
molecules with optimized inhibitory profiles against Flt-3 and c-kit 
kinases.

Key Facts About Leukemia
Leukemia is a cancer of the blood characterized by uncontrolled growth 
and proliferation of cells in bone marrow, the tissue responsible for 
the production of all blood cells. According to the Leukemia and 
Lymphoma Society, an estimated 33,440 individuals will be diagnosed 
with leukemia in the U.S. in 2004, more than one third of whom will 
have acute myelogenous leukemia (AML), the most common form. 

--- In [email protected], Sandy Johnson <[EMAIL PROTECTED]> wrote:
>
> Hello,Doe's anyone know what fit 3 drug is? We know a 12 year old boy 
that is going to start on this in Feb.That is as soon as the Doctor can 
get it for him.He is in St.Judes.He had a bmt that failed.His Mother 
gave him some kind of cells not sure what that was,and they have him  
on hydrea for now.His name is Nolen Kane,he could use a lot of prayers 
right now.Sandy
> 
>               
> ---------------------------------
> Yahoo! Photos
>  Ring in the New Year with Photo Calendars. Add photos, events, 
holidays, whatever.
>






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  • [CML] fit 3 Sandy Johnson
    • [CML] Re: fit 3 - Sandy Long Post cher111376

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