Feeling in a RAD mood

I'm pretty sure I'll sign up for the trial.  I don't think there's much of a downside.  I had a few questions that I sent to Dr. Treon and his Nurse Practitioner (Trish Sheehy), but Ms. Sheehy cleared them up on the phone this afternoon.  Here are the questions and answers:


1.  In section B of the consent form, it says that "the purpose of this research study is to determine the safety of RAD001..."  Could you please confirm that the purpose is also to determine the efficacy in treating WM?

- the purpose of the trial is both safety and efficacy

2.  Is the definition of "the drug is working to reduce WM" (page 3, section D, second paragraph of the consent form) a decrease in IgM level, decrease in bone marrow involvement, and resolution or reduction of adenopathy/organomegaly?  In particular, if the IgM decreases and there is decreased bone marrow involvement, but my anemia doesn't improve, would the treatment be deemed successful for the purposes of the trial?

- this is correct: IgM levels, bone marrow involvement, and adenopathy/organomegaly are the indications.

3.  One of the "less likely" risks (page 10, 4th bullet) is anemia.  As I understand it, the reason that near-term therapy (as opposed to "watching & waiting") is recommended for me is my existing anemia.  Is there any reason to think that I'm at additional risk because of my anemia?

- my anemia is likely to worsen, at least initially.  If the HGB level goes below 7, they interrupt the trial and give me hormones to stimulate red blood cell growth or else a transfusion, and resume when the level recovers.  If this happens 3 times, they discontinue me.  Since my HGB is currently 8.5, they will monitor this closely.

4.  My family doctor had the concern that if I go on the trial, and it doesn't help, then I'll have lost some time on the more traditional therapy.  On the other hand, it's my understanding that the Rituxan-based therapy might fail as well.  My feeling is that since there's a risk of no-response either way, perhaps this isn't an important consideration.  Does this sound like a reasonable point of view?

- NP Sheehy considers this a reasonable point of view.  Either treatment could fail, and the alternative can be started.

And related to the disease in general rather than the trial specifically:

5.  What are the likely negative outcomes if the disease is left untreated?

- The anemia will likely get worse, and eventually start to damage organs including the heart.  The malignancy can also spread.

6.  What is the expected survival rate?  Much of the literature says 5-7 years, but at the same time some of it says that with therapy, the symptoms can be managed and the disease is not likely to be fatal.  What is the Bing Institute's view on this?

- At the Bing Institute (the WM research center at Dana-Farber), they currently view the survival rate as 12-19 years.  They can't say more than 19 years only because they haven't been studying it that long.  It sounds like, for all intents and purposes, the treatments can manage the disease.  NP Sheehy believes that the 5-7 numbers that are all over the Web haven't changed because the organizations that post them (e.g. American Cancer Society or National Cancer Institute) just haven't kept up with the latest research, because they're busy with the more common cancers like lung cancer and breast cancer.