Venetoclax (ABT-199) Receives Breakthrough Therapy Designation In Relapsed Or Refractory CLL In Previously Treated 17p deletion Patients

This breakthrough designation - only for relapsed/refractory 17p deletion patients should mean the path to FDA approval will be smooth and short in the USA. This is very good news for my fellow American patients with 17p del. Hope the rest of the world stays in step.

We are only going to beat this cancer by giving it no backdoor escape routes and combos of non-chemo targeted therapies are already rolling out.

I am excited.

For the details, the official press release, and the background on the FDA breakthrough designation, visit our website (under the news section).

Stay strong.

We are all in this together.


18 Replies

  • Hi Brian,

    My husband also has 17p and is going in for a transplant in a few weeks. He's had an excellent response to Ibrutinib however his doctor still recommends a transplant due to his age (48). I read that you'd already had a transplant and just wondered if you are considering another? Our doctor's advice is not to stay on the Ibrutinib as the long-term effects aren't known and 17p patients are more likely to relapse on the treatment. Is your doctor advising you to have another transplant? Just wondering if we are going down the right path.



  • M,

    You are asking a tough question. Allo-HSCT still have a 10-20% treatment related mortality in the 1st year. And GVHD is always a concern and you can't undo a transplant. About 50% still alive at 5 years. But there is a significant risk of relapse w ibrutinib in 17p del patients, especially if they have been heavily treated before starting. With fewer prior treatments, 17p does significantly better w ibrut. No-one knows the right answer. I was diagnosed at 54 and will be 64 next month, so I am hoping to hold out for the perfect knock-out combination of targeted therapies rather than another HSCT.

  • Hi M

    I'm sorry to hear about your husband's situation. If you don't mind me asking where in Australia are you and do you mind sharing who your specialist is? I don't have 17P but relapsed at the age of 40 after having FCR at 39 so I'm now classed as Relapsed and Refractory and am hoping to either access a trial or get ibrutinib on compassionate access here in Melbourne. I see from one of your earlier posts that you have had to pay $10k a month for ibrutinib which I imagine is because you were using it as frontline treatment - that's unsustainable for anyone and I'm sorry you had to do this. Ibrutinib is before the PBAC in July for listing on the PBS on 1 August - if you haven't done a submission it would help other Australian patients enormously if you were to do so. It only takes 5 minutes - here is the link-

    Does your husband not qualify for the ABT-199 trial for those with 17P deletion? There is a trial for previously untreated with 17P deletion - not sure where the ibrutinib therapy would come in for that - here is the link

    Don't be afraid to ask to be referred for 2nd, 3rd an 4th opinions at this stage. I see David Ritchie at the Royal Melbourne who would be my transplanter and Con Tam at the Peter Mac who is my CLL expert and who I refer to for trials etc. It's your husband's life and, while their egos might not be thrilled, Drs do understand you wanting other opinions. If your husband hasn't seen Con at the Peter Mac he can see him as I do almost instantly at St Vincents if you pay privately - I would highly recommend him at this critical stage -

    Deciding to go for transplant is a massive step as you know. I was diagnosed at 38 so have pretty much been living with the fear of that for 3 and a half years and I'm fortunate to have a 10/10 sibling match. I have just returned from the US where every specialist I met told me not to proceed to transplant unless I developed a richters transformation. To be fair Con has been against me having a transplant at the moment and always said I should try clinical trials first.

    The novel therapies are still working for those with 17P and the combinations are looking really promising but there are no guarantees so you're in the invidious position of having to make a decision which no one wants to make. It's really hard because if a transplant works yourhusband gets his life back if he's very lucky but I'm sad to say I've lost friends who've gone through transplant and I will be exhausting other therapies before going along this route. Sorry you're having to go through this.


  • Hi Deb,

    We are in Sydney at St Vincents, however my husband has flown to Melbourne to see Dr Tam. Dr Tam has always encouraged him to have a stem cell transplant due to his age and the 17p. They seem to think that if he stays on Ibrutinib he will end up relapsing and miss the opportunity for a transplant. Unfortunately, he was never able to get onto any kind of trial because he hadn't had a failed treatment. A transplant does seem an enormous gamble to take when he is currently well and living a normal life. Really don't know what to do.


  • Oh I'm sorry to hear that - I'd be really torn in your position. I trust Tam and the fact he's telling me not to have a transplant and your husband to must mean the 17P is a real determining factor. He has always said I may have to have a transplant but being younger I have time - I would have thought 48 is still young enough to have time to try other things first - really wish he could have a shot at ABT-199 - it's looking so promising even with 17P deletions. Please stay in touch and feel free to PM me and I'll give you my details. You're at a great hospital and if I had to have a transplant now I'd be very happy that I'm in Australia (unlike my lack of access to clinical trials here!). Thinking of you and your husband, such a scary time - once the decision is made I hope it gets easier. There are some good transplant stories on - you might want to follow Wanda, Flygirl, Chonette and Joe's stories which give us real hope. I'm feeling happier that you've seen Con. Thanks for sharing. Deb

  • Hi M,

    Deb's advice is solid. Always engage a true CLL expert as part of your team, even if means hopping on a plane. I am alive today because I moved to Ohio 2000 miles from my home in California to enroll in a clinical trial with ibrutinib 3 years ago

    Stay strong




  • Hi Brian

    Yes, we have flown to see Dr Tam in Melbourne who is considered the 17p expert in Australia. His advice has always been to have a transplant, however part of me wonders if we should just sit on Ibrutinib a bit longer. Just don't know what to do now. Thanks for your advice.


  • M just thought I would update you. I saw Dr Tam this week and, even without 17P, he is leaning towards transplant for me after trying a novel agent for my relapsed CLL.

    This has thrown me somewhat as none of the specialists in America thought transplant would be in my future. They are now also searching for an unrelated donor as my matched sister has health issues. I keep thinking of your husband - there are so few of us in Australia that actually have transplants for this disease. I hope all is going well and he (and you) have come to terms with it - I'm a million miles from that head space yet so am having to start reframing my thinking. Looking forward to seeing my actual specialist (who is a transplanter) on the 3rd June.

  • To m and Deb,

    Ibrutinib offers very durable responses when it is used frontline, less so if it is used for relapsed disease. Also the real high risk for relapse might be from having a complex karyotype, rather than deletion 17p, though the two tend to run together. Easy to test for. I have both and I have failed a transplant, yet I am 3 yrs out on ibrutinib, but we are all different. I am also older, turning 64 next month so less keen on a second transplant. Don't have a second good donor and there are special risks with using the first donor a second time.

    Stay strong


  • Thanks Brian - just checked my original paperwork and I have complex karyotype - haven't really considered this before, have just been hung up on being unmutated with the del 6q thing - I do get the impression that those who know my disease best here are more worried than I had been - it's been a bit of a shocking revelation to me to be honest and everything feels suddenly very overwhelming. So looking forward to seeing David Ritchie in a couple of weeks to get some clarity. M hope you and your husband are going well.

    Best wishes


  • Biology is not destiny. These are very tough decisions.

  • You're right Brian and I am a bit of a fatalist - I think the universe may have led Dr Gribben sit next to me at your conference in San Diego - I wasn't even looking at trials in London- it seems too much of a coincidence to ignore (particularly when Dr Tam said if he was in my position he'd go to London and enter Dr Gribben's trial). It's just such an upheaval but a necessary one I think at this stage. If I don't have a good response to ABT-199 i'd be more than a happy to jump on a plane and head back for a transplant, and would be grateful then that the opportunity is available to me in Australia.

  • Makes sense to me. Treatments keep getting better so ABT-199 may buy you time to the next best thing.

  • Hi Deb

    Yes, it is completely overwhelming and I find the entire situation quite surreal. We are still going ahead. Our biggest dilemma now is the transplant itself. It seems the global trend is towards reduced intensity conditioning, however our doctor thinks a myeloablative transplant would be best (with donor cells taken from the bone marrow). However the risk of death is much higher with this type of transplant due to the toxicity. On the other hand, our doctor thinks it decreases the chance of graft versus host disease and offers a better chance of success.

    Have you had any advice regarding the type of transplant you should have? We'd be really interested to know what your transplant doctor thought.

    Also, I'm not sure how to PM you?


  • Yes David Ritchie told me he'd be leaning towards the myeloblative rather than the reduced intensity because of the same reasons your husband's Dr said. Because we're younger we can cope with the additional to toxicity and the benefits are greater. Ho hum I know he's been seeing me every few months since diagnosis 3 and a half years ago but I'm still not ready for this to be getting so serious so quickly. Feeling for you - I can't work out the PM on here either (just tried it)



    Admin: Deb, I've removed your email address and sent you both a PM to get you started.

    Sending a PM

    Reading a PM

  • No data supporting a survival advantage for myeloablative transplant. it's the graft versus leukemia that saves your live, not the chemo. You need just enough to accept the graft. See for more

  • Thanks Brian - I'll ask David Ritchie about that when I see him next.

  • OncLive's report on this announcement:

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