FDA Approves Farydak (Panobinostat)

This is a victory for myeloma patients, and perhaps for all patients facing a life-threatening disease.

Farydak (panobinostat) is a new oral drug approved specifically for use with bortezomib (Velcade) and dexamethasone to treat patients who have previously undergone at least two prior regimens, including Velcade and an immunomodulatory agent (thalidomide, Revlimid, or Pomalyst).

It comes with this boxed warning:

In other words it can be very unpleasant and can put your life at risk.  Further, in addition to the symptoms listed in the box, it can cause a reduction in platelets, neutrophils, or red cells, and can cause other serious problems.

So why is this a victory for patients?  Because Farydak can extend the lives of patients taking Velcade by months, perhaps years, that's why.

Nevertheless, last November the FDA Oncologic Drugs Advisory Committee (ODAC) voted 5 to 2 against approval of Farydak, judging that the potential benefits did not outweigh the risks.  This is a panel of "experts" (not a patient among them) deciding by themselves what risks WE patients should be permitted to take, and attempting to block us from all access to this drug.  In a recent speech I said "for people in my shoes, the side effect of NOT having the drug is worse – we call it death"!  

All of the new FDA-approved drugs have a daunting list of possible side effects, but we take them anyway because we don't like the alternative.  And as a result the median survival for myeloma patients has doubled during the 12 years since my diagnosis.  In my own case the advertised side effects of my little magic pill have been mostly absent - I've run 58 marathons now in my seven years on Pomalyst.

I congratulate the FDA on their understanding of this issue and their willingness to let us, with our pretty damn smart doctors, make the life-determining decisions ourselves.  This seems new, and I hope it is a harbinger of things to come.

Farydak is new too.  It works on an entirely new principle - it's called a histone deacetylase (HDAC) inhibitor - with the potential to be combined with any of the drugs that work on other principles. There is much to be learned about Farydak.  I hope I never need it, but I'm glad to have it in my quiver.

For more about access to emerging treatment technologies, please visit Closing the Gap Now .

Pomalyst Study Ends

But I'm still on Pomalyst, 2 mg daily with no days off, thanks to insurance.

Somewhat to our surprise, Mayo Clinic abruptly cancelled my participation in the study at the visit which ended my 89th 28-day cycle.  As far as I know the drug manufacturer, Celgene, was prepared to continue the study, but Mayo explained that they needed the resources elsewhere.

I headed home from that last visit with no meds and wondering how or when I would get them, and also wondering if or when I would have any appointment.  Happily, answers to those questions came in a few days (after some phone calls) and treatment is back on track.  My copay is quite manageable, and of course I'm aware that there are organizations that will help with the copay if I seek help.

I've now had my first "regular" appointment at Mayo, where we established a new schedule:  I will continue to get myeloma markers checked every month, but will actually drive the 200-mile round trip to Mayo only every second or third month, otherwise mailing in the blood samples and getting the results online.  The next Mayo visit will be in April and we will do either an x-ray bone survey or a PET scan at that time, to be determined after this upcoming marathon.

This time Mayo chose to do my CBC in the morning, together with all of the other blood tests, and found that the neutrophil count was barely above the lower limit of 1.0 k/uL.  We decided to go back to our earlier practice of taking the CBC the afternoon before the visit, because the afternoon neutrophil count is usually double the morning reading.  The neutrophils are there in the morning too of course - they apparently just don't like to get up early.  There may be a better medical explanation for that.

Results from the last study cycle and this first regular cycle indicate that the myeloma is still stable, with an IgG of 1270 mg/dL and M-Spike of 1200 mg/dL (1.2 g/dL).  Both of these values are within the range of values seen recently.  The myeloma will probably take over someday, but not yet.

At a gluten-free restaurant called Nourish in Gilbert, AZ.
That dressing is lime-based, perfect for the salmon and the salad.