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Perifosine


Guest nstephe

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Guest nstephe

Anyone have any experience with this? My Dad's Dr has suggested a trial. Dad has advanced NSCLC and is not strong enough for surgery, chemo, or radiation. His breathing is very labored and he lost his voice about 2 months ago. Any info would be appreciated. Thanks.

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Perifosine was what was next up for my Mom before she became too weak for treatment. We learned that it came in a pill form to be taken daily. It's one of the inhibitor drugs (those are the ones that target the tumors and keep them from growing, right?).

I wish I could give you more first hadn information than that. Below is the information that Ginny so kindly scared up for me when we were looking at this option last year.

Let us know how it goes!

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.

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Guest nstephe

Thanks for taking the time to reply. I have read alomost everything out there regarding the trials. I am really looking for any first-hand anecdotal information.

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"nstephe"]Thanks for taking the time to reply. I have read alomost everything out there regarding the trials. I am really looking for any first-hand anecdotal information.

Unfortunately, from what I have observed, there aren't a whole lot of folks here with first-hand knowledge about Perifosine. So... you may be a pioneer for us!

I hope that it's just the thing for your Dad. Is he having any luck getting through the paperwork and such? That was such an agonizing thing for us.

As I said, keep us posted.

Val

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