Why Patients Need to Be Heard At Medical Congresses on not just CLL (chronic lymphocytic leukemia) but on All Important Medical Issues

I was asked by friends to help with a campaign to ensure that the voice of informed patients be heard more loudly, more clearly and more often at large medical congresses such as ASH and ASCO. To that end, I shot a series of very short videos, starting with this one that introduces who I am and how I got involved in traveling the world, teaching my fellow patients and physicians about my disease, CLL and how patients approach their medical decisions.

Me in front of bleach painting by my son, Will Koffman

This video effort is directed at doctors, and is an important but smaller piece of my larger work that focuses on providing us patients with the best possible resources to educate and support ourselves in dealing with our largely incurable cancer.

So here is my first "ask".

Please complete this survey to help me and a team of patients, nurses and doctors across the world to meet the unmet needs of the CLL community.  It will take you less than 5 minutes to do.

We plan to start by constructing the most patient friendly, robust and relevant CLL specific website. 

We don't plan to step on the toes of the other great websites out there and replicate what already exists, but to make something different, deeper, and more accessible and searchable. Please remember that our goal is to meet the unmet needs of our community.

Next, we are going to build a facilitated network of CLL specific support groups across the country, linked by the mother website and a common goal to make sure we are all getting the best possible care for our particular circumstances.

We can't do this alone and we already have a strong team of folks in place: doctors, nurses, researchers, advocates and patients, many of whom you already know and respect from the videos hosted on this blog and/or their ubiquity in the CLL forums and other helpful sites.

Truth is that the CLL world has changed radically in the last few years, and what needs to done has outgrown the capacity of this humble blog. It will slowly morph back to my telling my own story and sharing my opinions, but in order to teach and support my fellow CLL patients, a IRS approved nonprofit 501(c)3, The CLL Society Inc. (EIN # 46-4131354) will largely be doing the heavy lifting, starting with designing a web presence from the ground up.

Stay strong. We are all in this together.

Non-Chemo Approaches to CLL in and out of Trials: Lenalidomide

Avoiding chemo, especially when it doesn't cure any of us with CLL/SLL, has always made good sense. My readers know my mantra of protecting our bone marrow and immune defenses for the long term battles aheads again against infections and secondary cancers.

Now with the promise of the approval of all these new agents in sight, many CLL gurus (Kipps, Keating, Byrd and others) hail the twilight of chemo as frontline treatment. Today there are even more reasons to consider non-chemo " bridge therapies" to buy us time until the magic CLL slayers are approved or there is a trial open that accepts someone with our profile (not always easy).

Besides the short term misery of the side effects in the infusion center and for the weeks that follow, why put ourselves at increased risk for secondary cancers (especially other hematological malignancies), bone marrow damage with resulting cytopenias (low blood counts), infections, and maybe even an increased risk of auto-immune issues. If we can hang on for a year or two, we can get a much less toxic therapy from our drugstore, assuming we can find a way to pay the $10,000 or more a month cost, but that's another subject. See my friend, Dr. Jeff Sharman's blog at http://www.cll-nhl.com/2013/05/how-expensive-are-new-drugs.html?m=0 for a nice discussion of that issue.

The good news is that there are already several good non-chemo options available in trials or in the clinic, for use as single agents or in combinations.

Over the next few weeks I will offer up several for your consideration.

I will start with an old friend, one of the choices that is both on the market already and available in some interesting trials.

Lenalidomide (Revlimid), an immunomodulator, is approved for multiple myeloma (MM) and some special cases of myelodysplastic syndrome (MDS). In CLL, the NCCN recommends it as 1st line therapy for those over 70 or sicker younger patients with co-morbidities. That can help you get it off label and with a little bit of effort, have your insurance to help pay for it. At an estimated $425 for each daily 10 mg tablet at Walmart that translates to more than $150,000 per year, we all need some help.

That is one real unsung advantage to a clinical trial: the trial drugs are free.

And Revlimid is also available in several promising trials for CLL. There are two in particular that are very much alike that have me excited: NCT01766583 and NCT01732861 where it is combined with a very specific BTK inhibitor (CC-292 AKA AVI-292 or AVL-292) that is behind the leader of that pack in development, ibrutinib.  More on that drug later. Both of these trials are for those with relapsed or refractory disease.  Read the details. Call the co-ordinator. Become your own expert.

If you go on to http://clinicaltrials.gov/ct2/results?term=lenalidomide+CLL&Search=Search you'll find an impressive list of 75 different trials with lenalidomide for CLLers. Many of these are closed or not recruiting, many are in combo with chemo, and many have restrictive inclusion and exclusion criteria, but take a look and you may find something that fits your need to control the CLL.

Lenalidomide has several advantages. It is an oral medication. It may work better the longer you take it. It is an IMiD that modulates the immune system, and CLL is a disease of the immune system. It doesn't damage DNA or target rapidly dividing cells as does most chemo. It make sense that it might boost the efficacy of other therapies from vaccines to rituximab (AKA R+R), steroids and more.

And as I said, it's not "chemo.

Not that it a free ride. It can suppress blood counts especially neutrophils and platelets, increase infection risk, cause a nasty and potentially dangerous tumor flare and tumor lysis syndrome, make us very sleepy, increases the risk of dangerous blood clots, should never be taken if you could get pregnant (it is closely related to thalidomide that also works in CLL) and it has been associated with more secondary cancers.

Still, today, I definitely sees many circumstances where I would take it ahead of the gold standard, FCR or the new hottie, BR.


Do your homework and check it out.

More soon on other non-chemo options including new and old TKIs and the next generation of MaBs and others.

Also part two of the Wiestner interview from ASH and more details on my recent visit to OSU.

"If I listened long enough to you" Tim Hardin

"If I gave you time to change my mind

I'd find a way just to leave the past behind

Knowing that you lied straight-faced while I cried

Still I'd look to find a reason to believe"

Tim Hardin

This post is pretty straight ahead medical. I will share some of my emotional and intellectual twisting in the wind later this week.

As you may recall we left me, our protagonist on a quest for more information.

When the experienced pathologist peered through his microscope at my bone marrow on the first of the month, when the hematology section of the lab went further and dug deeper using the fancy flow cytometry to search for one in 10,000 cancerous cells, they came up empty. No cancer. Nada! Rien de tout!

When they did my CT scan, the rad (the in term for radiologist), found rock solid stability in my cervical, axillary (armpits), inguinal, mediastinal (chest) nodes, BUT he discovered to our mutual dismay that my mesenteric (gut) nodes were "plumper".  True, I am trying to bulk up, but not nodally. Today I got a second opinion and had the digital files (x-ray on actual films are so 90s) reviewed by another rad. He found some growth not only in size, but also in number. Subtle. He called it suspicious. I call it bad news.

At ASH ( the 20,000 strong Hematology conference) I got a plethora of conflicting takes from the transplant and CLL gurus. More on my reflections on ASH later.

I met some wise doctors who were most generous with their time, and I asked very specific questions, about what matter most to me, that being me.

Question: Can CLL reoccur in the nodes with a clean marrow after transplant? 

Answer: Very rarely, OR " you betcha" especially with my risk factors, maybe a 1/4 of the time.

Question: Is the change in node size significant or are there benign causes for the fluctuation?

Answer: Maybe in the neck or groin, but you just don't see those changes in the gut OR they are still so small I wouldn't worry about it.

My best guess?  You must call them the way you see them, not the way you wish they were. Not much else would cause my nodes to swell. I assume it is the CLL, but I also think it is safe to wait 6 week to confirm with a follow up CT scan to be for sure for sure. There is much at stake. One CLL expert from Australia said I might be cured by the processes I have already been through. It was a minority opinion, but maybe he's right.

Question: Should I get a DLI (donor leucocyte infusion where I get donor white cells injected into my blood stream with no immunosuppression in a daring attempt to save my graft and knock out any residual cancer, but at a high risk of  GVHD or graft versus host disease)?

Answer: Most everyone said yes, but I better hurry. My doctor was not convinced as of Sunday, but he planned to get more opinions. I hope he's changed his mind.

Question: Should I complete the course of chemo with 3 more sets of FCR (the top chemo cocktail out there to treat CLL and the one that was heavily fortified for use in my pre-transplant preparation or conditioning)?

Answer: Finally a consensus: Don't do it. Turns out that a MRD- (minimal residual  disease negative) remission is just as predictive of good outcome no matter if the test is able to uncover only 1/1,000 cancer cells or 1/100.000. That would seem to suggest that striving for an even deeper remission once I've hit that sweet spot of being MRD-  by dousing me with more chemo wouldn't add much except more toxicity and immunosuppression. My doctor doesn't necessarily agree.

Here's my plan as of 12/8/2008 at 23:28:47. It could change 10 times in the next 10 days. Or ten seconds.

I want a DLI if possible, but it must be soon. If no DLI, then a second transplant now (now really means in 6-8 weeks in all probability if donor and insurance line up). I am also fine with waiting up to two months, but not longer and if the CT scan or bone marrow biopsy then shows any hint of a problem, cue the transplant music again. Details of that particular dance will be the thrust of another post. If I remain disease free, I just need close follow-up, every 90 days or so. That's a lot of rads (not the cool term for radiologists, but the hot term for absorbed cancer causing radiation energy), but I don't see much choice. You can't fight what you don't know

What I won't do is more chemo unless it is the first step of a two step process, with step two being transplant. Makes no sense to me.

But if I gave you time to change my mind....

"Shine a light" The Rolling Stones

And I'm wonderin' what it is I should do,

It's so hard to keep this smile from my face,

Losin' control, yeah I'm all over the place.

Clowns to the left of me,

Jokers to the right,

Here I am Stuck in the middle with you

Rafferty/Egan

It is not that I think of my doctors as clowns and jokers. It is just that we are all working in a herky jerky dark comedy as we roughly pull the biological levers of a graceful and delicate system that we are trying to force to obey our will. 

I will soon be making some major decisions, with less than perfect knowledge. So I have decided to get the best expert advice I can and read up on the latest thinking and most recent studies that apply to my circumstances. Be prepared. Shine a light.

I am off to ASH (American Society of Hematology) conference in San Francisco to meet the gurus and read the papers. As a bonus I get to stay with my daughter and son-in-law in Berkeley.

And then I will consult Dr. Kipps at UCSD and my local doctor, Sanjay Sharma before the year is out.

And probably a quick trip to the east coast to get third and fourth opinions from Kanti Rai and Ken Miller in January.

I will make several attempts to predict the best of all possible futures.

At least that's the plan for now. It may change after my bone marrow biopsy and CT scan next Monday. There is much at stake. As a friend said, I hear the drumroll. Show time! Let us hope there's no rimshot this go around.  I already have had my share of my cosmic humor: being too healthy with too robust a bone marrow and immune system to allow my graft to take hold. What hubris.

What does sadden me is how head long and deeply I have had to dive back into the murky waters of CLL and transplant medicine just when I thought I had reached the shore and was lazing on the beach in the sun.

As I do my research, I hope what I will find makes it hard to keep my smile from my face.  I will smile come what may, but it wouldn't it be great if it came easy.

"I gotta get a message to you" The BeeGees

I want someone to talk to me like Moses

Randi McMatthew

It is hard enough when I have all the facts to make a decision. Should I play the odds and hope that I get lucky? Will I be part of the 41% of those who despite rejecting their graft, go on to have durable remissions? Some theorize that the same kick ass immunity that is giving the boot to my donor, may also be pushing my misanthropic clone out of Dodge. The remission odds are even better if the cancer is chemo sensitive, which mine clearly was.

Tough decisions. 

Dennis Prager teaches that in order to be clear, you  first need to acknowledge what you want to be true.  Then look coldly at the evidence that confirms or denies what you so desire to be the reality of the situation. Admit your prejudice, and be so very cautious not to discount what you find that is counter to it.

I've said it  before. I so wish that an organic vegan diet and gobs of green tea would cure leukemia, but the facts say otherwise. Might help for sure. Cure it?  No way.

But what about when the facts aren't there? What if I need to decide when there are gaps in my knowledge big enough to hide a rock star's ego?  Gaps, not only about my own status, such as did I get my remission from the chemo or the donor's T cells doing their job of search and destroy, but gaps in the state of the knowledge out there. 

Many of the problems I want answers to have none, at least yet. The medical literature doesn't talk about the success of DLI salvage when your donor's monos and granulocytes are zero, but the donor T cells remain relatively high. Someone knows from their experience or from their deeper understanding of the basic science. I suspect the DLI is hopeless, based on my incomplete understanding of the biology, but I want the word from on high.

The medical journals I read often have only a few cases that are similar to my circumstances, providing very thin cover for venturing into a jungle of  known and unknown dangers.

So I will start reaching out to all the experts I can. Fly to the east coast and see Dr. Rai and Dr. Miller. Email and phone others.

Maybe go to the American Society of Hematology meeting http://www.hematology.org/meetings/2007/index.cfm in San Francisco in 2 weeks.

I am looking  for all the help I can get.

I am also writing up a storm and hope some of you including "CLL spouse" will email at bkoffmanMD@gmail.com if you can help with my book in any way. It would be much appreciated.