Ibrutinib, and other new miracle drugs..... do we know what we don't know?

Those who know me know that I've been reticent to wave flags over the next miracle drug.... I now find myself taking one, very reluctantly. Before you say I'm crazy, please hear me out.

The history of Cancer drugs has been promising us cures for as long as pharmaceutical companies have been in existence. There's gold in them there hills. Lots of it... more in the sizzle than the actual steak. The Markets rise and fall on the next announcement at ASH and other conferences. But that same road to Oz is littered with the corpses of the ones that never made it. Some even did very well for some people, but were dropped for lack of a market, or due to too many undesirable side effects or AE's, adverse events, a nice way of saying , bad stuff.

To understand why this happens over and over one must learn to read abstracts critically. Any trial is only as good as it's design.... how many patients, what sort of patients, age, condition, etc. Often they don't say, or they don't share that.... How much follow up and what sort,,, How many phases did it go thru before it was called a miracle and pushed in front of the FDA ? How many actual patients actually were treated and how did they do? We also must remember that those in trials are presumably watched by those who research the drug and tho they know very little about how it will or may perform, they sure know more than your local doctor. Many drugs make it to the market now in very short order... I realize it seems forever to those of us waiting. Often only a few hundred patients, very carefully selected have taken the drug. We who get it next are actually the real 'trial' subjects,,,, guinea pigs for the drug company, the researchers and those who will or won't get it later. Are we the lucky ones? don't be too quick to be jealous of us.

Ok the day arrives and you know you need to begin treatment, or do it yet again. You've heard or read about this drug and you are anxious... but depending on how realistic you are and how well prepared you are, you're also nervous. you read the list of side effect, and especially in the case of ibrutinib, it boggles your mind. Read that list critically as well. Most drugs that have been on the market for a while, some say five years is the very earliest one can feel a bit safe.... will show side effect of <10% or in some cases a bit more for one or two... rarely over that for any one effect. too many at high percentages, now that the drug has been given to more people, the drug will likely get pulled, or redesigned or blessed with black box warnings.... Ibrutinib has very high percentages for each of it's many side effects... largely that's due to the very small number of those taking it and possibly the fact that they may have been treated before, or are late stage, or older or other factors play in. The real trial comes now for those of us who are perhaps more middle of the road CLL patients or at least a different mix than those who were in the trials.

With so many side effects possible, if one has a bout of diarrhea the question arises ,,,, is it real or is it ibrutinib? Now lets say we get a clot.... possibly not related to taking ibrutinib..(clots are listed, as is dieahhreah, some really bad bouts and rashes) . or is it? do we reduce the dose, or stop the drug, or keep plugging on to see what happens. No local doctor will know. No local emergency room doctor will even want to hear about it... you're a bad dream to him... you're as much of a mystery as a kid brought in with a suspected drug overdose... does he treat with x or y or do nothing? might he make it worse? what else are you on? does he have access to your whole history? do you?

I'm not saying no to a new drug because I have few better options now, and I am far from a typical CLL patient. In fact I am again coming late to the table this time, PCR just in time last bout, and now stalled following the CLL due to a rare cancer found in my mandible requiring a major surgery, a jaw transplant, chemo and radiation... result, my marrow is again nearly 90% CLL... and as luck such would have it I came down with Lyme disease two weeks into ibrutinib.... doxycylcine is the fix for that we hope, but it too can cause diahhreah. you begin to see where I'm going? And the unknown also applies to the drugs that may or may not react differently with ibrutinib, or any other 'new' drug, even if it's not yet listed.

If you are going to take a new drug, or even enter a trial, PLEASE become the best self advocate you possibly can. read, study, and keep scrupulous records of what you are taking, what you have taken, do a full history and have that and your drug list on you at ALL times. either electronically or on paper.

The truth is with any new drug, we don't even know what we don't know..... don't be too upset if you are in a place where you are only offered an "older'" drug... those drugs have proven history in all types of people, and their quirks can be searched easily by even a triage doctor in your local ER. Not so with the new miracle pills. This is true of ALL new drugs, whether cancer treatment or not.

There is always another bus, and there's always another new drug around the corner..... the newest isn't always the best.. It may boil down to what choices you have and your tolerance for risk. But it's your body, your life. Get involved like your life depended on it, because it does.

Good luck to all, and stay alert. best, Beth

10 Replies

  • Thank you. A sobering post that will make all of us stop and think. Nothing like hearing the thoughts of other patients. Wishing you health and success on Ibrutinib and luck with the possible complications. Do let us know how you are getting on.

  • Thank you. My intent was not to frighten but to remind us to stay alert. Probably the toughest part of our disease. Sometimes feels like no rest for the weary. The paradigm of watch and wait seems at times designed to lull us toward the opposite, "don't worry we're watching" The truth is we worry but who's watching? For the most part, that's our job. I've found that diligent watching helps tone down the worry and pays off in better care.

    My best to you , beth

  • Although your post is scary it verbalises my feelings almost exactly. I wish that I had your way with words and your writing stamina! So, I agree it's what the professionals don't know that is a concern.

    Nevertheless, as humans, we mainly progress in knowledge by assessing how to move forward with limited and insufficient information.

    The journey ahead with Ibrutinib may be a bumpy one, the future is uncertain after all, but already on this journey my spleen and lymph nodes have returned to normal size and 18 months of diarrhoea is a problem solved so far. I trust the local haemotology team, love them to bits in fact, and am happy, content and confident to continue on this medical mystery journey.

    “You're missing the point. It's not that I believe in what Lee's saying; it's that I hope he's right. I really, really hope he's right, because what's the alternative?”

    ― Alexandra Bracken, The Darkest Minds

  • Beth,

    I don't disagree with anything in your post.

    But there are many of us that realize the downside of FCR Chemo and have decided to be pioneers- like most of our immigrant ancestors that moved to the USA without knowing what would happen (or even the Apollo astronauts).

    My first treatment was Rituxan monotherapy (when I was 62 years old) and it gave 6 months remission, but before and after those 6 months I felt, looked and behaved like and 80 year old man. I was unable to work effectively and even had problems driving a car.

    I started on the Phase 3 trial for Idelalisib in December 2012 and within 6 weeks had my stamina and strength back, able to work and enjoy life fully. I believe that about 360 other patients worldwide were in all the Idelalisib trials to that point. 80% of us had benefits from the drug, 20% did not or had serious side effects.

    After 30 months my CLL found a way to evolve around Idelalisib, and my strength & demeanor started to decline, so I switched to Ibrutinib. Within 8 weeks my wife said she knew it was working for me, since my appearance was changing for the better.

    So 7 years after diagnosis and now due to my 3 NON CHEMO treatments, I have a fully normal life (I do have minor autoimmune skin problems that started just before my CLL diagnosis but are treatable).

    So I will choose progress and taking reasonable risks to have the best treatment that medical science can offer for my CLL.

    BTW- when ABT-199 - Venetoclax gets approved (next year?) I hope to create my own clinical trial (with careful advice from my CLL expert doctor), to go for a triple combo of Imbruvica, Venetoclax and Gazyva, in hopes it will give me a very long remission, without daily expensive pills- better than I would get with FCR and far less permanent damage to my chromosomes.


  • I'm afraid you've missed my point. Most on this list will only have fcr as their option, or a trial, tho far from all. While you and I are USA.

    I am not against progress. Life is all about the unknown.

    My point is proceed, but always remember that the unknown IS what we face every day. 320 good results doesn't mean we will be one. Not automatically.

    Your path has been to steadfastly avoid the known. Let's not forget that many are alive today thanks to FCR and its kin. Many had long remissions with very few problems. Not everyone on FCR suffers the worst you feared. Far from it. There's also been a huge middle ground.

    What's right for each of us is what we have to decide. Often no matter how we plan the Indians circle the wagons and we must fight or die.

    I too have great hope for venetoclax or some combo or cocktail with it. That one actually looks to be a potential cure. Nothing else is yet. That is possibly the most important thing to remember. CLL cells are really stubborn, they just keep learning new tricks. We are making progress.

    Eyes wide open and forward. Be ready and consider all options. Accept that sometimes compromise is the best we can do Review critically and then proceed accepting that we don't know . I don't think I'm saying anything so different.


  • Very interesting topic and contributions....... watch this space!!!!!

  • The fact is that there are concerns and side effects with all sorts of drugs more with chemo drugs than let's say the simple aspirin but they still have their problems for some.

    When taking any drug for the first time or indeed over the long term side effects can develop over time but surely it's the balance between side effects and results on the condition the drug is treating. Problems escalate when you talk about chronic disease and the long term effect of a drug. With cll reacting and being such a personal condition not all will get side effects but many may do so.

    Every time I may be diagnosed with a condition and start a new drug for me I am concerned about potential side effects many can be life threatening when I read the documents with the drug.

    The problem we face as CLLers is that chemo treatment is very toxic and can cause serious problems during and after treatment. Any attempt to find a pill that works without the toxicity of chemo has got to be a positive move forward.

    What I am trying to say is that even well tried and tested drugs have their risks and we don't know how they will affect us before use or in long term use.We should, therefore be diligent when taking any drug for any condition. When you have a chronic life threatening condition such as cll there is a time when you have to take a leap of faith and hope that the medics are offering you the best treatment available.

    In emergency departments all over the world staff have little expertise when dealing with cll because they see few suffering from this condition this is why both GP and emergency staff need the support of cll experts and,indeed,why it is recommended patients see such specialists during treatment and diagnosis.

    Yes it's about what we don't know but that is the same whatever drug we are given for any condition its all a bit of a jump into the dark but at least some have the chance to take that leap and a positive outlook must assist when doing this.


  • I've never agreed with both sides of a discussion before but I think everyone is actually saying the same thing and very eloquently too. Don't stop. This is great stuff. Thank you.


  • Dear Lapermone,

    I like the way you think and enjoyed the wisdom in the back and forth discussion from all on treatment options for CLL. I know as CLL'S we are all thinking about the possible side effects when we need treatment and hoping that when that day comes there will be great options out there for us. I agree we need to read and learn all we can like our life depends on it because it does. Don't forget there are doctors doing research on immunology using your own stem cells in your blood to fight off the cancer. (Brilliant). I wish there were more research going on with natural combinations. I am not against drugs far from it. No one wants to go back to a world without them because they save lives. But if the drug companies need to be able to patent natural combinations let them. The drug companies invest in years of research and should be rewarded when they find a cure and save lives. Can't there be an exception made in the case of a cancer cure?


  • Loved this, it expresses how I feel.I can't wrap my head around this new miracle drug.There are many unknowns and I feel as a CLL patient I am being pushed to take a drug that I'm just not comfortable with.I feel I am being pushed to be part of a huge experiment without wanting too.I have refused this drug so far because it had some major interactions with medication I'm already on.I'm glad some are seeing benefits, but I'm just not ready to be on a lifetime drug with some pretty serious risk.It has caused a huge distrust in my relationship with my doctor of eight years.I'm all for trying new things when the time is right and I'm ok with the risk, but the push by my cancer center to put everyone on this drug and dismiss the risks as nothing has been a huge setback emotionally

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