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Scans and Plans.... Perifosine?


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Well, we got our CT scan results back today. Not so good.

The original tumors in the lungs have grown, and there are more there now. There's growth in the lymph nodes, the bones looked mostly the same--some growth particularly on one rib and maybe some on one of her vertebrae, and now there are mets in the liver...

So... Not so good at all.

BUT... The doctor wants to try a new drug called Perifosine. Has anybody heard of that one? I sure hadn't. I guess it was JUST approved for use with LC and is still in the trials phase. They don't have any placebos in the trial, they're just fooling around with dosage issues. The doc. thought it was worth a shot. After that he said we'd look at Tarceva.

The scans aren't the greatest news, BUT... Mom didn't even bat an eye at the idea of continuing with a new course of action. The things she had been saying about not wanting to do anymore, I guess were more a smokescreen of sorts than anything. So THAT encourages me.

I was impressed with the doc today (only my second time meeting him). Here we are in a podunk little town, and he's wanting to try something that sounds pretty new and cutting edge. I think he's agressive and up on his game.

So that's where we stand now. Please pray that she'll be approved to get the Perifosine and that it's just the ticket. That's what we're hoping.

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I can't help you with the drug I'm afraid. If it's new in the US then we won't be testing it on mice until about 2050 :lol:

I'm sorry to hear your Mum's bad news but it sounds as though as she's regained her spark and is ready to fight on which can only be a good thing. I like the sound of her doc too.


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I am sorry to hear of your mom's bad news. I have never heard of perifosine. I suppose it is worth a try. you might ask her onco doc about using avasitn in combination with other drugs such as taxol and carboplatin. That is a new recipe too. It has had good results for others.

Don M

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Here's what I found thru Goggle.

KRX-0401, or Perifosine, is the prototype of a new group of anti-cancer drugs referred to as alkylphosphocholines that appear to be potent inhibitors of the activation of Akt, a protein in the body associated with tumor survival and growth. Akt appears to be inherently activated in approximately 10-50% of most tumor types and is also believed to be activated by, and thus confers resistance to, most anti-cancer therapies. Once activated, Akt exerts its cell survival and cell proliferation functions. Based on its prevalence across cancer types and importance in the control of cell survival and cell proliferation, Akt is considered to be one of the most important cancer targets being researched today.

In pre-clinical models and early clinical trials, Perifosine displayed impressive anti-tumor activity, as well as anti-Akt activity. Treatment with Perifosine did not result in myelosuppression, or toxicity to the bone marrow, which is a major side effect of most traditional anti-cancer treatments. Accordingly, we believe that Perifosine is well suited for use in combination regimens with established anti-cancer therapies and, therefore, represents a significant market opportunity.

In proliferation assays, Perifosine inhibited the growth of a variety of human tumor cell lines. Perifosine was also tested in vivo in a number of animal models and shows significant single agent anti-tumor activity. Additionally, Perifosine strongly enhanced radiation-induced apoptosis in human leukemia cell lines, as well as the anti-tumor activity of cisplatin, Adriamycin, and cyclophosphamide in the DMBA-induced rat mammary carcinoma model. The combination regimens were superior to chemotherapy alone and the combinations were well tolerated.

Three Phase I studies of Perifosine have been completed in Europe and two other U.S.-based Phase I trials with the NCI pursuant to the CRADA arrangement have been completed or are nearing completion. Data from these trials demonstrates that Perifosine is a safe drug with a reasonable toxicity profile and no observed myelosuppression, or bone marrow suppression. The dose limiting toxicity in each Phase I study was gastrointestinal toxicity, primarily nausea and vomiting. Perifosine also displayed preliminary single agent activity as evidenced by a total of two partial responses and 16 disease stabilizations in these heavily pre-treated refractory cancer patients.

We believe this data confirms the anti-cancer activity of Perifosine and that Perifosine represents a new class of anti-tumor agents that promote apoptosis and block cell growth signals.

Please see "Clinical Trials" for more information.


Copyright © 2004 by Keryx Biopharmaceuticals, Inc.

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Sorry to hear the scans didn't go as well as hoped but glad to hear your mom is ready to pick up the fight. Haven't heard of the drug either but as you pointed out, the oncologist seems to be an up to date kind of doc and that's important.

Hope this drug is your mom's magic bullet.

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Hi Val,

Like the others, I'm really sorry to hear about the progression. I know that can be devastating news.

It sure was good that the doctor had an aggressive plan already in place when your mom got the news. I'm sure if you like and trust the doctor that your mom does, too.

I don't know anything about the drug, but thanks to Ginny's post, we all know a little more.

Prayers for your mom.



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