BBC News - 18/04 - Breakthrough in leukaemia treatment (Prof Chris Pepper of Cardiff University on Ibrutinib)

Scientists helped to develop drugs which target the disease at a molecular level

By Carwyn Jones

BBC News (includes video of Prof Chris Pepper)

bbc.co.uk/news/uk-wales-270...

Scientists at Cardiff University say they have made a significant breakthrough in the treatment of the most common form of leukaemia.

Chronic lymphocytic leukaemia (CLL) affects 3,000 people in Wales and 20,000 in the UK.

A new drug has been developed that targets cancer cells, stopping them in their tracks before they have time to multiply and travel through the body.

Scientists said the disease would become less of a clinical problem.

A team at the institute of cancer and genetics at the University Hospital of Wales has been working on the project which has been led by Prof Chris Pepper, who has worked on the disease for 20 years.

"These drugs will transform the outlook for patients, without a doubt," he said.

"They are massively going to change the landscape for individual sufferers of this disease.

"I confidently predict that within 10 years certainly, and probably five years, this disease will become much less of a clinical problem.

"And people like me won't be working on this disease much longer. These new agents are likely to completely alter the clinical path of this disease."

The team's research has focused on three main elements of CLL:

• Understanding the basic biology of the disease - what makes the cancer cells survive and grow out of control.

• Understanding what makes some patients have a particularly aggressive form of the disease, while other patients have a strain of the disease that doesn't require immediate treatment.

• Using that knowledge to devise better treatment for patients with CLL.

That treatment has resulted in the development of a drug called Ibrutinib.

Cancer cells

It is already in advanced clinical trials in the UK and is expected to be licensed for use in Wales by the end of the year.

The drug targets cancer cells, stopping them in their tracks before they have time to multiply and travel throughout the body.

When the CLL cells circulate around the bloodstream, some of them burrow their way through the blood vessel wall and escape into the body's solid tissues, attaching themselves to the lymph nodes or bone marrow. That is when the cancer cells multiply and become a clinical problem.

Cardiff University's research has concluded that one particular molecule in the body - NF KappaB - plays a critical role in determining whether the tumour cells escape the blood vessel.

The drug Ibrutinib will target this molecule and get the cells to break free from the solid tissues and recirculation around the bloodstream.

Prof Pepper is confident that his team's research will change how CLL is treated in the coming years.

"I'm not suggesting that we're going to cure this disease or completely eradicate the leukaemia," he added.

"But what I'm telling you is that these drugs will effectively arrest the progression of the disease.

"So patients will be able to live with a normal age-adjusted life expectancy with their disease. That's something I could never have predicted even five years ago."

Admin: Added Prof Pepper and Ibrutinib into the subject for easy searching, plus added a link to the BBC on-line article which includes a video interview with Prof Pepper.

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24 Replies

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  • Thanks Marty R, I'm not quite sure how to respond to this article because I posed a question on here, 'Is Ibrutinib the Cure?' and it was suggested that it was not, but the doctor does not suggest it is curative, just that it changes the management of the condition. I await the comments of those more qualified than me.

  • This does sound brilliant but is there any possibly at all, in the view of those in this community who are more knowledgeable than I am, that here in the UK this drug will become routine treatment as the first line therapy or will cost prohibit this and we will continue to have to face FCR for the foreseeable future?

  • Hi Fileybay,

    From what I've read, Ibrutinib is being offered to those who have either relapsed after FCR or who are too frail for FCR. I am guessing that this will be how it will go, but, like you, I don't profess to be an expert. Even if this is the case, it looks like good news?

    This webpage seems to support my belief: ukmi.nhs.uk/applications/nd...

    Regards,

    zentangle

  • I've asked for a copy of this video, as I saw it too. Well done Marty for getting the content down in print.

    Bubnjay

  • You will see in the latest UK CLLSA Newsletter that there is an on-going campaign to raise awareness of the benefit that these Transformational Therapies in CLL with UK Members of Parliament, the UK regulatory bodies and the Pharmaceutical companies.

    If you would like to support this activity by writing to your MP then you will find the details in the recent CLLSA Newsletter. The issues will be availability and cost of these therapies at a time when all budgets will be increasingly under strain. The Patient Voice is needed now or nothing will change.

  • Thanks for the post Marty, I missed the BBC news item on TV, this is the link to the BBC on-line report to join the copy. bbc.co.uk/news/uk-wales-270...

    Great news that this new drug is likely to gain licensing this year and that compassionate use has come available in some parts of the UK under an extended access program for those who have run out of options to stay alive. Gaining approvals for mainstream use and availability with our cash strapped system is another matter.

    As Maudmari is stressing in these times of austerity and budget constraints unless we start preparing the ground now to raise awareness of the benefits of these new drugs nothing will change.

    The CLLSA newsletter has not be allocated a URL yet, this is the article that Maudmarie is referencing to, now is the time to start:

    TRANSFORMATIONAL THERAPIES in CLL

    The recent approval of Ibrutinib/IMBRUVICA in the US by the FDA has increased expectation and excitement in the UK CLL community for patients who can benefit from the emerging new therapies. The questions are how can these breakthrough therapies be accessed and how can they be afforded? To ensure that we gain a fair share of the UK cancer budget it is important to increase the profile of CLL among political decision makers.How can you help with this important work?

    THE PATIENT VOICE REALLY MATTERS!

    The campaign has been initiated by CLL patients in the Midlands and we need to roll this out nationally in order to be effective as an active voice. We need at least 40 patients/partners/carers nationally to join us. A standard/sample letter has been developed which we would like you to send to your MP. If you have a particularly urgent case yourself you may want to book a meeting at the MP's constituency surgery as well.

    So please let us know by email if you can lend your support to this vital campaign or send us a PM and we will contact you with an email address (it is available in the CLLSA newsletter if you have a copy).

    Nick

  • Is this available in the United States? If so is it approved? Does anyone have more info on it? This has really caught my attention.

  • Hi Extreme, From what I've seen, I believe it's available in the United States. If you click my link above, it suggests it's available here (UK) now. It is suggested that it is being prescribed in US for people who've had at least one round of chemo or are too frail for chemo. All the stuff I've read has suggested the trials have no "end point" i.e they haven't failed, once started, though some people don't respond to it. I'm waiting for comments of more experienced posters on here.

    Regards,

    Zentangle

  • I have been on W and W for nine years.

    Now on the borderline, waiting for drug trails.

    Where are the drug trails for Ibrutinib being held?

    Cut off here in Cornwall from the mainstream.

  • Hi hazelmark,

    I think the trials are over/closed, although I'm not entirely sure. They were in Birmingham, Harrow, Plymouth, Sheffield and Sutton. There is some talk on here of being able to get it in Scotland, but I don't know the facts.

    Regards,

    Zentangle

  • Hi zentangle. The FLAIR trial which compares FCR with Rituximab + Ibrutinib will open in the UK in July.

  • I predict a riot! Actually I don't, because the majority of patients at my hospital are elderly and one couple I spoke to had never heard of Ibrutinib. More media coverage needed. Any ideas?

  • Ibrutinib (Imbruvica) was approved in the US for use with some CLLPatients in February, which has meant that others have been able to get it "off label". a good place to read about it is Dr. Jeff Sharman's blog cll-NHL.com He has several entries about it.

    Pat

  • I live in San Francisco, California. Ibrutinib is already available in US. The cost is $12,000 for 30 days. Of course, most insurance companies cover the cost. I started my treatment on April 3rd (Imbruvica), no side effects. This pill is magical. My enlarged lymph nodes (size of an orange) gone in 4 days. I hope you guys can get this drug wherever you live.

  • I'm no expert on the politics or economics of pharmaceutical development and manufacture but I'm shocked and dismayed to hear of the cost of Ibrutinib. It equates to about £85,000 a year (minimum calculation) in the UK looking at Shazie's costing. (Delighted it's working so well for you by the way Shazie.) It's quite clear that the NHS would struggle and resist paying these kind of figures and as Hairbear has said, 'how can these breakthrough therapies be accessed and how can they be

    afforded?'

    Maybe it's naivety that makes me wonder why pharmaceutical companies pitch the costs so high even though I totally appreciate the enormous research and development costs. But what's the point if the end product then becomes prohibitively high. Why can't they take the 'long view' in terms of profitability?

    I agree that we must make our voices heard in terms of obtaining a fair share of the cancer budget in the UK but I can't help thinking that the companies must play their part and not price these drugs outside modern health systems. There must be a limit even in terms of private health insurance too?

    The only way to win political opinion is in costing out equivalent therapies with their resultant welfare benefit costs associated with unemployability and dependency then setting them against the cost of these new drugs. Having worked in the very tight social care sector with increasingly austere and savage cuts in public spending, I know sentimentality won't come into it. It will be which costed 'care/health package' will meet the minimum need. Depressing but true.

    Let's hope that if Ibrutinib is rolled out following trials, that the pricing structure actually becomes more realistic and achievable. I live in hope (a necessary requirement with CLL).

    Newdawn

  • Hi Newdawn,

    This is all new. I know I may be naïve, but I can't imagine politicians standing up and saying that THEY are allowing people to die because they will not sanction the buying of Ibrutinib. I am new to CLL and so new to this kind of debate, but someone posted that the same thing happened with AIDS treatments, and they are now readily available. The scenario of 20,000 people being consigned to a seemingly premature death would not make for good international news copy. Plus the cost of SCT is £250,000, the equivalent to three years on Ibrutinib. It still remains a huge concern though.

    Regards,

    zentangle

  • I'm certainly not suggesting that catastrophic scenario zentangle. Yikes, I'm also in the same boat and could become reliant on this drug at some point.

    The point I was making is the excessively high cost of Ibrutinib set against other treatments and the struggle health systems will have in funding them if the drug companies don't make them more affordable. I'm certainly not defending lack of funding for any reason but if you read Hairbear's post you'll see that the challenge in fighting for it has been recognised and we need to seize that.

    I worked in the field of Aids/HIV support and funding and am deeply heartened that antiretrovirals have become so available in the developed world and are saving and preserving so many lives. Regrettably the under-developed world still struggle to obtain them.

    I think if you read Hairbear's post the position will be explained much more clearly. I had no intention to alarm but the reality is we may have to fight to obtain widespread funding and all other cancer groups are having to do the same. But CLL has been a 'Cinderella' group for too long and I sincerely hope this is the drug we have all been waiting for and that it can be afforded widely as an alternative to more toxic therapies. Obviously much depends on long term results and suitability.

    But I'm afraid there are too many cases of drugs being denied funding to people due to cost and that's a reality I hate but cannot deny.

    Regards,

    Newdawn

  • Hi Newdawn,

    I did not mean to alarm anyone, just to illustrate a scenario no one would tolerate politically (let's not involve morals, just yet). Here's an interesting document, the last paragraph f which alludes to the fact it's all in NICE's hands now: google.co.uk/url?sa=t&rct=j...

  • The document won't open I'm afraid zentangle.

    Newdawn

  • Hi, If you Google "Will ibrutinib be available in UK?", go to Page 5 and you will see a document under heading "[DOC]

    Targeting BTK with Ibrutinib in Relapsed Chronic Lymphocytic ..." it should open, if you have Word.

  • Hi Newdawn,

    The other thing to consider is that people (understandably) talk about wanting Ibrutinib as a first line therapy, to avoid the toxicity of chemo. However, no one is suggesting using it in this way. The suggestion is FCR for those who can tolerate it and then possibly Ibrutinib or Ibrutinib for 17p deleted or other resistances/refractory patients or elderly. However, now, when FCR has lost its efficacy, surely the only available treatment will be Ibrutinib or SCT?

    Regards,

    zentangle

  • Thanks, shazie,

    Although I understand all evidence on here is purely anecdotal, I have yet to hear anything bad about Ibrutinib. There is the possibility, I guess, of becoming refractory to it, but the four years people have enjoyed may be a better prognosis than other treatments and we don't even know how "far out" (timewise) it can go.

  • I am glad my post brought so many responses - thanks also Nick for the link to the video - this brings the text to life ! As many of you may know I have just completed six months of Chemo - complimented by Idelaslib trial (a similar drug to Ibrutinib) In fact my Consultant said that if ever the Idelaslib was withdrawn from my trial ( planned for me to take it twice a day till 2017 !) then Ibrutinib will be a possible substitute! Regards Marty

  • Hi Marty, Glad to hear things are going well. So, just to confirm, your consultant has said Ibrutinib may be made available in UK, is that only as part of a trial?

    Regards,

    zentangle

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