Ponatinib (Iclusig) – GREAT NEWS!!!

I love news like this, especially when it’s been four-years in the making.

Iclusig (ponatinib) for the treatment of Chronic Myeloid Leukaemia (CML) in adult patients with chronic phase, accelerated phase, or blast phase CML who are resistant to dasatinib or nilotinib; who are intolerant to dasatinib or nilotinib and for whom subsequent treatment with imatinib is not clinically appropriate; or who have the T315I mutation.

For CML patient in England, this means we now have another safety net that is readily available for consultants to prescribe immediately. This is going to make a big difference to treatment options and the mental well-being of many patients.

Just a quick shout to all of the people who work for and are associated with Incyte (formally ARIAD) who’ve never given up on this and have worked so hard to get it to us. Congratulations and thank you.

NICE has also recommended ponatinib for treating Philadelphia-chromosome-positive acute lymphoblastic leukaemia in adults. Hit the link for more info on this: www.leukaemiacare.org.uk/news/NICE-recommends-ponatinib

The full press release follows.

Kris

 

NICE Issues Positive Final Recommendation for Iclusig (ponatinib) for Chronic Myeloid Leukaemia (CML) in England

CML patients across the UK who are resistant or intolerant to second generation tyrosine kinase inhibitor (TKI) therapies will now have equal access to Iclusig

LONDON, UK [28 April 2017] – Incyte Corporation (Nasdaq:INCY) announces that the National Institute for Health and Care Excellence (NICE) Technology Appraisal Committee (TAC) has published a positive Final Appraisal Determination (FAD) recommending Iclusig^â (ponatinib) for the treatment of Chronic Myeloid Leukaemia (CML) in adult patients with chronic phase, accelerated phase, or blast phase CML who are resistant to dasatinib or nilotinib; who are intolerant to dasatinib or nilotinib and for whom subsequent treatment with imatinib is not clinically appropriate; or who have the T315I mutation.[i]

The positive FAD from NICE brings CML patients in England in line with those in Wales and Scotland who have had full-access to Iclusig, according to its license,[ii]^,[iii] since 2015; providing patients with CML across the UK who have failed other treatments equal access to an additional and important option.

“Today’s decision is important for patients with CML who have failed other treatments, as well as for physicians in England, who will now have access to the clinical benefits of Iclusig,” commented Mark Tanner, General Manager of Incyte Bioscience UK. “Together with the CML community, we have worked very hard over the last four years to encourage NICE to reconsider their original evaluation and are delighted that NICE has acknowledged the unmet need and the value that Iclusig brings.”

CML is a rare blood cancer with around 700 new cases each year in the UK.[iv]  CML affects economically active people, with around 50 percent of UK cases in people aged under 65 years.^iv Many patients with a new diagnosis of CML have a prolonged clinical benefit from targeted therapy with tyrosine kinase inhibitors (TKIs). However, there has been a high unmet need and poor prognosis for patients whose advanced disease is resistant and intolerant to other therapies.[v] Once available treatment options are exhausted, the prognosis can be poor.^v  Despite advances in treatment, there remains a need for additional effective therapies for the management of CML.[vi] Iclusig fulfils an important need in the treatment pathway for CML patients and provides clinicians and patients with a full suite of treatment options for CML.

Professor Jane Apperley, Department of Haematology, Imperial College School of Medicine, Hammersmith Hospital, London said, “This is an exciting and long-awaited outcome, which allows physicians to manage patients in a logical and clinical-evidence based manner with the goals of improving long-term survival and providing a good quality of life.”

Iclusig was approved by the European Commission[vii] in 2013 as an orphan drug for the treatment of adults with chronic phase, accelerated phase, or blast phase CML who are resistant to dasatinib or nilotinib; who are intolerant to dasatinib or nilotinib and for whom subsequent treatment with imatinib is not clinically appropriate. In Ph+ALL (Philadelphia chromosome‒positive Acute Lymphoblastic Leukaemia) patients, Iclusig is licensed for adult patients with Ph+ ALL who are resistant to dasatinib; who are intolerant to dasatinib and for whom subsequent treatment with imatinib is not clinically appropriate. Iclusig is also licensed for people with CML and PH+ALL who have T315I mutation.[viii]

About CML and Ph+ ALL

CML is a cancer of the white blood cells that is diagnosed in approximately 7,000 patients each year in Europe.[ix] CML is characterized by an excessive and unregulated production of white blood cells by the bone marrow due to a genetic abnormality that produces the BCR-ABL protein. After a chronic phase of production of too many white blood cells, CML typically evolves to the more aggressive phases referred to as accelerated phase and blast crisis. Ph+ ALL is a subtype of acute lymphoblastic leukaemia that carries the Ph+ chromosome that produces BCR-ABL. It has a more aggressive course than CML and is often treated with a combination of chemotherapy and tyrosine kinase inhibitors. The BCR-ABL protein is expressed in both of these diseases.

About Iclusig^® (ponatinib) tablets

Iclusig targets not only native BCR-ABL but also its isoforms that carry mutations that confer resistance to treatment, including the T315I mutation, which has been associated with resistance to other approved TKIs.

In the EU, Iclusig is approved for the treatment of adult patients with chronic phase, accelerated phase or blast phase chronic myeloid leukemia (CML) who are resistant to dasatinib or nilotinib; who are intolerant to dasatinib or nilotinib and for whom subsequent treatment with imatinib is not clinically appropriate; or who have the T315I mutation, or the treatment of adult patients with Philadelphia-chromosome positive acute lymphoblastic leukemia (Ph+ ALL) who are resistant to dasatinib; who are intolerant to dasatinib and for whom subsequent treatment with imatinib is not clinically appropriate; or who have the T315I mutation.

Incyte has an exclusive license from ARIAD Pharmaceuticals, Inc, a wholly owned subsidiary of Takeda Pharmaceutical Company Limited, to develop and commercialize Iclusig in the European Union and 28 other countries, including Switzerland, Norway, Turkey, Israel and Russia.

About Incyte

Incyte Corporation is a U.S.-based biopharmaceutical company focused on the discovery, development and commercialization of proprietary therapeutics. For additional information on Incyte, please visit the Company’s website at www.incyte.com.

Follow @Incyte on Twitter at https://twitter.com/Incyte.

[i] NICE. 2017. Final Appraisal Determination: Ponatinib for treating chronic myeloid leukaemia and acute lymphoblastic leukaemia. Available at https://www.nice.org.uk/guidance/gid-ta10060/documents/final-appraisal-determination-document Last accessed 28 April 2017

[ii] All Wales Medicines Strategy Group. Ponatinib (Iclusig). Appraisals. Available at: http://www.awmsg.org/awmsgonline/app/appraisalinfo/1163. Last accessed 24 March 2017

[iii] Scottish Medicines Consortium. SMC Advice. Ponatinib (Iclusig). Available at: http://www.scottishmedicines.org.uk/SMC_Advice/Advice/1032_15_ponatinib_Iclusig/ponatinib_Iclusig. Last accessed April 2017.

[iv] CRUK. Chronic myeloid leukaemia (CML) incidence statistics. Available at: http://www.cancerresearchuk.org/cancer-info/cancerstats/types/leukaemia-cml/incidence/. Last accessed April 2017.

[v] Cortes JE, KimD-W, Pinilla-Ibarz J, et al. A Phase 2 Trial of Ponatinib in Philadelphia Chromosome–Positive Leukemias. N Engl J Med 2013;369: 1783-1796. Available at: http://www.nejm.org/doi/pdf/10.1056/NEJMoa1306494.

[vi] Woessner DW, Lim CS, Deininger MW. Development of an Effective Therapy for CML. Cancer J 2011;17(6):doi:10.1097/PPO.0b013e318237e5b7. Available at: http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3251313/pdf/nihms-332259.pdf. Last accessed April 2017.

[vii] EMA. Iclusig EPAR summary for the public.  Available at: http://www.ema.europa.eu/ema/index.jsp?curl=pages/medicines/human/medicines/002695/human_med_001656.jsp&mid=WC0b01ac058001d124. Last accessed April 2017.

[viii] Iclusig Summary of Product Characteristics. Available at: http://www.medicines.org.uk/emc/medicine/28145. Last accessed April 2017.

[ix] Rohrbacher M, Hasford J. Epidemiology of chronic myeloid leukaemia (CML). Best Pract Res Clin Haematol. 2009 Sep;22(3):295-302. Based on current estimate of population of Europe (738,199,000 in 2010).

 

(Another) open letter to Dr Fielden: generic imatinib

I don’t take any pleasure in having to follow-up letters that haven’t been replied to, especially letters about something so important. I also dislike having to copy senior people in to force a reply out of someone, it feels childish and wastes time.

But, I’m left with no other option. The letter speaks for itself and I’ve linked PDFs to the two letters that have gone unanswered. I hope that this provokes a dialogue that reassures patients about the generic process. Thanks, Kris

 

Dr Jonathan Fielden
Director of Specialised Commissioning – NHS England
xxxxxxxxx
xxxxxxxxxx
xxxxxxxxxx

17^th March 2017

Dear Dr Fielden,

RE: Imatinib – your reference JF 16-1201.1

Sorry to have to write again but it has been a month since I asked for a swift response to my letter dated 16^th February 2017. I should also note that the reason I sent the letter in February is because I didn’t receive a reply to the letter dated 21^st December 2016.

I am concerned by your lack of communication and so are the CML patients that I represent, numbering around 2,000. I have copied the Secretary of State for Health, the Chief Executive of NHS England and my local MP who I hope will encourage you into dialogue with me. I have attached my previous communication and if need be my local MP, Mark Garnier, will vouch for my credibility, he is aware of my advocacy and has been incredibly supportive over the years.

At a time when you are expecting blood cancer patients to make significant changes to their treatment, I am appalled by the lack of communication and care that has been shown. I hope that we can kick-start this relationship and give CML patients some reassurances that their questions and concerns are being taken seriously and explored. Given the amount of money that the switch to generics will save the NHS, it is surely the very least that can be done.

With stretched budgets, patient advocates and charities are under increasing pressure to fill gaps. I am proud to represent my fellow patients and I will not stand by and be done unto and I will not go away. We are a ground-breaking bunch of cancer survivors and I expect to be treated with the same amount of dignity and respect by NHS England that we are shown at all other touchpoints within the NHS.

I’m sure you appreciate how important it is for patients to have faith in the system, I look forward to answers in full for all my questions including this addition to my previous letter.

5. Some patients are reporting that consultants aren’t fully briefed on the switch. What measures have been put in place to ensure information has been disseminated and how is the data from new side-effects being monitored and centrally collated? Some patients are reporting new and different side-effects after switching.

The concerns I had in my previous letter still stand; the process that brought generic imatinib to market is flawed and this is now being realised. I hope that you can provide the reassurances that are needed.

Yours sincerely,

Kris Griffin (Mr)
078xxxxxxxx

CC: Secretary of State for Health, The Right Honourable Jeremy Hunt MP
CC: Chief Executive of NHS England, Simon Stevens
CC: MP for Wyre Forest, Mark Garnier
CC: CML-UK Facebook Group
CC: CML-Worldwide Facebook Group
CC: Access CML Drugs blog

 

Letter to Dr Fielden dated 21st December 2016 – PDF
Letter to Dr Fielden dated 16th February 2017 – PDF

 

Open Letter to Dr Fielden: generic imatinib

Due to a lack of response from Dr Fielden and the number of patients waiting for clarification on aspects of generic imatinib, I’ve taken the decision to make my most recent letter to Dr Fielden public. Let’s hope that he replies to this one. Kris

 

Dr Jonathan Fielden
Director of Specialised Commissioning
NHS England
xxxxx
xxxxxx
xxxxxx xxxxxx

16^th February 2017

Dear Dr Fielden,

RE: Imatinib – your reference xxxxxxx

I’m writing in response to my letter dated 21^st December 2016 to which I’ve yet to receive a reply. I would appreciate a swift response to this letter given the troubling nature of the contents and the outstanding queries from previous communication.

1. If a patient who has had a successful period on branded imatinib then switches to generic and cannot tolerate new side effects, are they able to revert to branded imatinib, considering it’ll likely to be cheaper than the other TKIs available?
2. In a previous letter, you listed Leukaemia Care as an organisation who have been involved in drafting the guidance. Leukaemia Care aren’t aware of any such involvement. Please can you clear this up?
3. Are you aware of a national shortage of Wockhardt (generic imatinib)? I’ve come across two instances now where patients have been without treatment. If you are aware, why has this happened?
4. Are you aware of the labelling issue with at least two form of generic imatinib? This is the issue that suggests that the drug, for adults, is for blast crisis only and not chronic or accelerated phases. This issue has the ability to mislead and worry patients. What action are you taking and how did this issue escape what I hope would be a rigorous checking procedure?

Unfortunately, my concern with the process that brought generic imatinib to market is now being realised and I’ve very much like some answers.

Yours sincerely,

 

Kris Griffin (Mr)

 

Speaking at the APPG on Blood Cancer (Westminster, London)

This week I was invited to the Houses of Parliament to speak at the inaugural meeting of the All Party Political Group (APPG) on Blood Cancer. It was a great honour.

I’m not sure it would be entirely fair to document the entire proceedings; the secretariat, Bloodwise, will be documenting the detail in due course. I can say that we heard from the Parliamentary Under Secretary of State for Community Health & Care, David Mowat MP and the meeting was chaired expertly by Henry Smith MP.

The ultimate aim of the APPG will be to conduct enquiries into blood cancer-related issues. I believe that the first enquiry will cover how well (or not so well) the national cancer strategy is working for blood cancer patients (given their specific needs). This would be a great place to start and I applaud the approach.

Kris talking at the APPG on Blood Cancer

Kris with the Leukaemia Care Team (Zach and Monica)

Kris and Ben

There were a number of people representing various blood cancer organisations and charities and I hope that we can all pull together, with our parliamentarian counterparts and make a real difference to the lives of blood cancer patients. I’ll keep you posted.

I’ve posted my speech below, it’s not every word but I’m sure that you’ll catch the gist.

All of my best wishes, Kris (Griffin)

SPEECH – Wednesday 18th January 2017, House of Commons

Thank you for inviting me along today. I feel very honoured to be in such distinguished company and to be able to provide the room a patient voice.

It’s been 10 years (next month) since my CML diagnosis.

As you can imagine I’ve got many stories to tell about my blood cancer…not all of them bad…

Whilst talking this speech over with a good friend, we discussed a number of things that have happened to me that I could share with you. Not necessarily all bad.

Three CML anecdotes: bat ears, sperm banking, grapefruit.

No, probably best to leave the funny stories well alone, get on with the more serious stuff.

So, I want to tell you about the lad who was helping me out cutting a hedge down in my garden. We had a conversation. I told him that I had leukaemia.
And as he ran down my drive he shouted back, “CAN I CATCH IT?”

The problem is, people just don’t get us. The trouble is that it’s not just about taking meds anymore.

My feeling is that with CML being a treatable disease (or controllable), our needs are very different to many other cancers. I don’t believe that the health sector really understands us. Not because they are uncaring, but because we have moved so fast and so far forward.

When our treatment goes well, our wellbeing needs to be looked after and managed. There is a psychological impact of having a cancer diagnosis. Some people manage this better than others. But, this is an invisible problem for us. As we start to treat other blood cancers in a similar way, these are issues that need addressing more and more. It’s why I constantly bang on about personalised treatment. A personalised approach does provide the best outcomes and the best quality of life. I’m sure we can all agree that this is the aim.

Because if we get it right, the economic impact is huge. Potentially you could have people who previously were unable to work, now able to work. 40% of them may not be receiving any treatment at all. 20% (and growing) may be taking generic drugs which are much cheaper.

We are trailblazers and I treat my disease like a superpower. It opens doors and allows me to help people, to ensure that they are represented, to provide a voice. I work everyday and am kept on my toes by an active 5-year-old.

Even though blood cancer is the fifth most common cancer, there are 250,000 people (and growing) living with it in the UK…it’s hard to define…too many types and generally nothing to cut out.

And whilst the treatment for CML…and some other forms of blood cancer are getting more straightforward and successful…we can be in danger of forgetting that there is a real person behind each diagnosis. We forget that people can react differently to news, information, approaches and misinformation.

When I told the members of the CML-UK Facebook group I was speaking today, many of the 2,000 members replied asking me to mention a variety of subjects. I’ll do my best to represent some of those views now:

GENERICS.
No consultation with patients. No formal information. But, imatinib patients are expected to simply switch tablets and accept what they are told…this after being told that patient power is a good thing. What about the months, sometimes years of getting to a good place only to be told to start again. No official reassurances. I’m still waiting to hear if you can go back to a drug that previously worked for you. MUST DO BETTER.

SUPPORT
The psychological scars run deep, as deep as any cancer patient. But, we are the ones with invisible problems, different needs. CML, in particular, is a disease, that doesn’t necessarily have to destroy your life. If the tablets go down well, carry on. I’ve not missed a day of work through CML since diagnosis. So, empower us. We can be your support network. Take a look at the Bloodwise Ambassador scheme and the CML-UK Facebook group for evidence of burgeoning, useful communities.

GENERAL AWARENESS
There are people in the medical profession who still don’t know enough about blood cancers. I know that Leukaemia Care is trying to address this. Just some basic awareness. I was diagnosed by accident, many blood cancers are. If my GP hadn’t been curious…I wouldn’t be here now. Which leads me to…
PERCEPTIONS
Many employers don’t get it. Many patients WANT to work and yet outdated, overly bureaucratic business practices stops them. How can you have cancer, you haven’t lost your hair? And many patients who are unable to work because of devastating side effects, cannot access the benefit system because CML is a “treatable” disease.

INSURANCE
There are some travel insurance policies that we can get. But not life cover. Why? Exclusions? They use the wrong data!
Many traditional cancer patients can get cover after being clear for a number of years. But us? No. Ongoing treatment. And no one wants to listen.

LIVING ON THE EDGE
No matter how many times you’ve gone through it, no matter how robust you are, no matter how long you’ve been undetectable. When you go and pick your bloods up, it still feels like you are rolling the dice. And that, every three months, every six months. In many cases, for a lifetime. It takes some getting your head around.

On one hand, we should congratulate ourselves, we’re getting our head around the science and as for CML, it feels like a winnable battle. But there are many types of blood cancers and we have to keep pushing hard on all fronts.

Blood cancer patients are the front line. In my lifetime I want to see most cancers treated in the same way that my cancer is treated (or better). Many of you have played a part in the journey so far and are certainly responsible for getting the right drugs and treatment to the right people at the right time.

Let’s make sure that these patients are empowered. Let’s not leave them to fight other battles on their own. Let’s fight for their employment rights, look after their well-being, consult them on changes, find insurance and keep breaking walls down.

I’m incredibly grateful that we now have an APPG on Blood Cancer, it has already brought together some fantastic people, here in this room. But, please, let’s keep patient needs central to the agenda.

It is serendipitous that, when my journey started, 10 years ago, after a long walk, Sir Ian Botham told me that I was going to be OK.

He was right. I feel more confident today than I was yesterday, passing the message along and telling my fellow blood cancer patients that it’ll be OK.

That’s what matters. Thank you.

Kris Griffin speaking at the APPG on Blood Cancer

CML Advocates launch Leukaemia App

CML Advocates Network has launched its new mobile Chronic Myeloid Leukaemia (CML) adherence app called CML Today. It’s for CML patients to help them support adherence to tyrosine kinase inhibitors (TKIs) as well as to track their polymerase chain reaction (PCR) results.

The mobile app is available for FREE in English, French, Spanish, German, Portuguese, Arabic and Hebrew. More languages will follow soon.

The App will allow patients to:

• track the regular intake of their medicine
• remind them to take their prescribed medication
• track their PCR test results and other laboratory parameters
• facilitate patients connect with local support groups in their country.

I’ve downloaded the App for my iPhone and have found it easy to use and very clear; the reminder function is excellent. Whilst there are several Apps that support CML patients this is the only one, to my knowledge, developed by a patient advocacy network. I know the people who have developed it and I trust them, very important with medical data of this nature. This App gets my seal of approval and a big thumbs-up from me. Well done CML Advocates Network.

Find CML Today in the Apple App Store (iphone) and in the Google PlayChronic (Android).

https://itunes.apple.com/de/app/cml-today/id1038000286?l=en&mt=8
https://play.google.com/store/apps/details?id=com.appropo.cml_adherence_app&hl=de

Thanks, Kris

Ponatinib: Scotland 1, Wales 1, England 0

In recent news I reported that Iclusig® (ponatinib) had been granted access to patients in Wales by NHS Wales. You can read the whole story here. I’m pleased to be able to write today to say that patients in Scotland have been given access to ponatinib too! Both countries are providing access for ALL phases of chronic myeloid leukaemia (CML).

In England ponatinib is only available on the Cancer Drugs Fund (CDF) IF the patient has the T315i mutation. We recently reported the majority of patients were being turned down in England after making individual funding requests; full story here.

Whilst you can read the full details of the appraisal by NHS Scotland I’ll draw your attention to this:

A non-comparative phase II study of ponatinib was conducted with primary outcomes of major cytogenetic response in patients with baseline chronic phase CML and major haematologic response in patients with baseline accelerated or blast phase CML or Ph+ALL. Ponatinib demonstrated efficacy in heavily pre-treated CML and Ph+ALL patients who had received dasatinib/nilotinib as second line or further line tyrosine kinase inhibitor therapy or who had the T315I mutation.

The studies of ponatinib show it to be very effective and NHS Scotland have recognised this, but NHS England remain steadfast and refuse to appraise ponatinib because the patient numbers are too low. We find ourselves in a situation where patients aren’t able to access a drug that could save their lives. How can the system be fair when, depending on which NHS authority you come under, will depend on the availability of a drug to you?

There is little that can be done at the moment but once Parliament reconvenes we’ll start to apply pressure and ask direct questions of the organisations that are allowing us to fall behind our counterparts in the United Kingdom.

Thanks, Kris

Advice: following a full submission considered under the orphan and end of life process:

ponatinib (Iclusig®) is accepted for use within NHS Scotland.

Indication under review: Adult patients with
• Chronic phase, accelerated phase, or blast phase chronic myeloid leukaemia (CML) who are resistant to dasatinib or nilotinib; who are intolerant to dasatinib or nilotinib and for whom subsequent treatment with imatinib is not clinically appropriate; or who have the T315I mutation.
• Philadelphia chromosome positive acute lymphoblastic leukaemia (Ph+ALL) who are resistant to dasatinib; who are intolerant to dasatinib and for whom subsequent treatment with imatinib is not clinically appropriate; or who have the T315I mutation.

A non-comparative phase II study of ponatinib was conducted with primary outcomes of major cytogenetic response in patients with baseline chronic phase CML and major haematologic response in patients with baseline accelerated or blast phase CML or Ph+ALL. Ponatinib demonstrated efficacy in heavily pre-treated CML and Ph+ALL patients who had received dasatinib/nilotinib as second line or further line tyrosine kinase inhibitor therapy or who had the T315I mutation.

This advice takes account of the views from a Patient and Clinician Engagement (PACE) meeting.

Taken from: www.scottishmedicines.org.uk/SMC_Advice/Advice/1032_15_ponatinib_Iclusig/ponatinib_Iclusig

Ponatinib Access: Denied

I recently sent in a Freedom of Information request to NHS England to find out how many patients in England had requested ponatinib for chronic myeloid leukaemia and who would not already be entitled to it on the NHS. Currently only patients with the T315l mutation are able to have the drug prescribed by their doctor, with other patients who want the drug having to get a clinician to make a special request (an Individual Funding Request, or IFR) to the Cancer Drugs Fund, which NHS England runs.

I was shocked by the response to my query, that of the 14 patients who requested ponatinib (from April 2013 to March 2015), just 2 of them were granted access to the drug and the other 12 were denied. It seems short-sighted of NHS England not to allow patients access to a drug which could benefit them when others have stopped working, and when the only other option is often a stem cell transplant.

With such small patient numbers NICE won’t even consider appraising ponatinib, the CDF is supposed to act as a support system for patients to access drugs for rarer cancers, but the system clearly currently isn’t working.

Patients in England are again missing out compared to their counterparts in Wales, where the drug is fully approved for all CML patients.

This excellent graphic clearly shows that in the ponatinib PACE trial, patients benefited from ponatinib after they had failed other TKIs at various stages of disease progression.

Ponatinib: Wales 1 England 0

After the bad news yesterday, where it was announced that 25 cancer drugs will be removed from the Cancer Drugs Fund (read the full story here), I come with better news. NHS Wales will be providing access to ponatinib for ALL phases of chronic myeloid leukaemia. In England ponatinib is only available on the Cancer Drugs Fund if the patient has the T315i mutation. This progressive step by NHS Wales, based on the Phase 2 PACE trial, gives patients in Wales another, much needed line of treatment against CML. I hope that authorities in England, Scotland and Northern Ireland take note of this step forward when they assess, or reassess, ponatinib for their respective countries.

Thanks, Kris
—

The positive assessment from the AWMSG is the first UK Health Technology Assessment (HTA) of Iclusig

Leatherhead, UK, 9 January 2015 — The Minister for Health and Social Services in Wales has ratified the recommendation from the All Wales Medicines Strategy Group (AWMSG) to approve Iclusig® (ponatinib) as a cost-effective treatment to be used in NHS Wales for the treatment of all phases of chronic myeloid leukaemia (CML) and Philadelphia chromosome positive acute lymphoblastic leukaemia (Ph+ ALL), in accordance with Iclusig’s licensed indication.

CML is a cancer of the white blood cells that is diagnosed in approximately 7,000 patients each year in Europe. The incidence of CML in Wales is 1.4 and 0.9 per 100,000 males and females, respectively. Iclusig is a targeted cancer medicine discovered and developed at ARIAD Pharmaceuticals, Inc.

“We are delighted that the Minister has endorsed the positive AWMSG recommendation, recognising the innovative nature of Iclusig and the potential benefit it can bring to cancer patients in Wales,” said Mark Tanner, General Manager, ARIAD UK. “Our goal is to deliver innovative solutions that address gaps in care for patients who are left with few clinical treatment options. Iclusig offers a new treatment option for many of these patients.”

The approval by the AWMSG was based on results from the pivotal Phase 2 PACE (Ponatinib Ph+ ALL and CML Evaluation) trial in patients with CML or Ph+ ALL who were resistant or intolerant to prior tyrosine kinase inhibitor (TKI) therapy, or who had the T315I mutation of BCR-ABL. In Europe, Iclusig was approved in July 2013 for the treatment of adult patients with:

• Chronic phase, accelerated phase, or blast phase chronic myeloid leukaemia (CML) who are resistant to dasatinib or nilotinib; who are intolerant to dasatinib or nilotinib and for whom subsequent treatment with imatinib is not clinically appropriate; or who have the T315I mutation;
• Philadelphia chromosome positive acute lymphoblastic leukaemia (Ph+ ALL) who are resistant to dasatinib; who are intolerant to dasatinib and for whom subsequent treatment with imatinib is not clinically appropriate; or who have the T315I mutation.

“We welcome the ratification of the recommendation from the AWMSG for the treatment of CML and Ph+ ALL with Iclusig in all its licensed indications,” commented David Ryner, Chair of the Chronic Myeloid Leukaemia Support Group. “Although the number of patients qualifying for treatment will be limited, access to Iclusig represents an opportunity to make a significant difference to their lives. We would like to see the same opportunity made available to all other qualifying patients, no matter where they live in the UK.”

About Iclusig® (ponatinib)
Iclusig is a kinase inhibitor. The primary target for Iclusig is BCR-ABL, an abnormal tyrosine kinase that is expressed in chronic myeloid leukemia (CML) and Philadelphia-chromosome positive acute lymphoblastic leukemia (Ph+ ALL). Iclusig was designed using ARIAD’s computational and structure-based drug design platform specifically to inhibit the activity of BCR-ABL. Iclusig targets not only native BCR-ABL but also its isoforms that carry mutations that confer resistance to treatment, including the T315I mutation, which has been associated with resistance to other approved TKIs.

About CML and Ph+ ALL
CML is a cancer of the white blood cells that is diagnosed in approximately 7,000 patients each year in Europe.Error: Reference source not found CML is characterised by an excessive and unregulated production of white blood cells by the bone marrow due to a genetic abnormality that produces the BCR-ABL protein. After a chronic phase of production of too many white blood cells, CML typically evolves to the more aggressive phases referred to as accelerated phase and blast crisis. Ph+ ALL is a subtype of acute lymphoblastic leukaemia that carries the Ph+ chromosome that produces BCR-ABL. It has a more aggressive course than CML and is often treated with a combination of chemotherapy and tyrosine kinase inhibitors. The BCR-ABL protein is expressed in both of these diseases.

About ARIAD
ARIAD Pharmaceuticals, Inc., headquartered in Cambridge, Massachusetts and Lausanne, Switzerland, is an integrated global oncology company focused on transforming the lives of cancer patients with breakthrough medicines.  ARIAD is working on new medicines to advance the treatment of various forms of chronic and acute leukaemia, lung cancer and other difficult-to-treat cancers.  ARIAD utilises computational and structural approaches to design small-molecule drugs that overcome resistance to existing cancer medicines.  For additional information, visit http://www.ariad.com or follow ARIAD on Twitter (@ARIADPharm).

Iclusig® is a registered trademark of ARIAD Pharmaceuticals, Inc.

You can download the original release here: AWMSG approval_FINAL release_090115

Geoff Thomas and Cure Leukaemia Launch Biggest Fundraiser

Ultimately, it’s about curing cancer and if that doesn’t grab your attention then nothing will.

Last night was the launch of the biggest fundraiser in the history of Cure Leukaemia, the Birmingham based charity founded by Professor Charlie Craddock and Graham Hampson Silk.

Deloitte in Brindley Place hosted the event where former England footballer Geoff Thomas told the audience he will hold two major cycling events in 2015 to support the work of Cure Leukaemia and Professor Charlie Craddock in Birmingham. This comes ten years after going into remission from leukaemia himself.

In 2003 Geoff was diagnosed with chronic myeloid leukaemia (CML) and was given less than three months to live. Following treatment from Cure Leukaemia co-founder Professor Charlie Craddock, including a bone marrow transplant from his sister, Geoff has been in remission since January 2005. Geoff now aims to raise £2million in two years by holding two unique cycling events for an exlsuive number of participants.

Firstly, in June 2015 Geoff will lead a closed group of 300 cyclists on a four-day, 500 kilometre cycling challenge: ‘London 2 Paris: Inspiring the Revolution’. The event is built for both keen cyclists and beginners looking for a new challenge – every aspect of the ride is planned with precision and will be a professional event for amateur riders. It might sound like an event only fit for cycling’s elite, but with four different speed groups, this is something anyone can achieve. It also boasts itself as one of the ONLY events that offers rolling road closures throughout France.

Six months after Geoff’s treatment finished in 2005 and inspired by cancer survivor and pro-cyclist Lance Armstrong, Geoff set himself the challenge of cycling the Tour de France 2005 route two days ahead of the race. Geoff succeeded in his 2005 challenge and his second major cycling event will revisit the challenge of cycling all twenty-one stages of the Tour de France; this time, just one day ahead of Le Tour 2015. Along with a closed group of only twenty participants, this will be a once in a lifetime opportunity to join Geoff in completing one of the toughest physical challenges around.

The event was a huge success with many signing up on the evening, Geoff was more energised than I’ve seen him for a long time and Charlie, with ususal humility, had a steely resolve that suggested these extra funds for the charity could be a real game-changer. We’ve already seen the Centre for Clinical Haematology at the QE Hospital in Birmingham become one of the leading centre in the world for the development of new drug and transplant treatments for patients with blood cancers. These new approaches to clinical care are providing a new economic force for the city – in life sciences. The focus on ‘cure’ from everyone at the charity is to be commended and the leadership from CEO, James McLaughlin has been outstanding.

Please visit the links and consider signing up and supporting these events.

www.l2prevolution.com
www.beforethetour.com
www.cureleukaemia.co.uk

Leukaemia & Lymphoma Research Impact Day 2014

It might seem crazy what I’m about to say
Sunshine she’s here, you can take a break
I’m a hot air balloon that could go to space
With the air, like I don’t care baby by the way

– Happy by Pharrell Williams

There was a definite happy vibe at Leukaemia & Lymphoma Research’s Impact Day yesterday at the Tower Hotel in London. Impact Day is the charity’s annual conference, with a difference. It’s clearly deigned to have an impact on the audience. It did just that! The delegates made up from fundraisers, patients, carers, researchers, Doctors, clinicians, nurses, family members and staff from the charity, were given a blast of progress. What progress! I lost count of the number of times the word ‘cure’ was used. There is a genuine belief that they are the cusp of greatness and whilst looking back at the roots of the charity it truly felt we were standing on the shoulders of giants.

The countless lost to blood cancer over the years has not been in vain, not a bucket rattle wasted and not a clinical trail regretted. We heard about the progress in Childhood Leukaemia (Professor Christine Harrison), Acute Myeloid Leukaemia (Professor Paresh Vyas and Advanced Nurse Practitioner Kirsty Crozier) and Chronic Lymphocytic Leukaemia and Lymphoma (Professor Chris Pepper). Matt Kaizer, Chris West and David Henderson all spoke, with great conviction, about the role of the charity in research, policy and insight respectively. It’s great to see Leukaemia & Lymphoma Research taking policy and insight so seriously, both done well will save lives, albeit in a different way to research; equally important though.

The audience, which was considerably larger than last year, hung on every statistic and anecdote. There were tears and there was admiration. Particular memories for me included the progress in childhood leukaemia where survival rates are now over 90% and the passion in which Kirsty Crozier spoke about her ‘calling’ as a Nurse. It was good to hear Professor Pyas say that the Cancer Drugs Fund has been, “transformational” and Professor Pepper suggest that the best way to make drugs affordable is to create competition in the marketplace. It certainly gives me renewed vigour in hearing that.

A chap called Kris Griffin (!) spoke about patient support and the new online patient services being offered by the charity and Dr Peter Campbell who is Head of Cancer Genetics and Genomics at the Wellcome Sanger Institute gave the audience an insight into individually tailored treatment plans. The progress is more than a little mind-blowing, costs are reducing as is the time taken to sequence; the impact on patient treatment immeasurable.

The day was hosted impeccably by Professor Chris Bunce, whose must surely be a shoo-in to replace Brucie on Strictly, such is his calm, smiling, brilliant demeanour. The day was rounded up perfectly with a closing address by Chief Executive Cathy Gilman who summed up with ease and grace. The hope. The joy. The memory of those that we have lost. The joy that we are step closer to a cure.

“Life is a gift. Use it wisely and live it fiercely.”
– Cathy Gilman

Each loss hurts more now. That we are so close means the pressure is truly on. I can hear buckets being rattled harder, more cakes being baked, running further, cycling harder. To hope. True impact. With heartfelt thanks to everyone, everyday. More happy days.

And all this from a charity who started, quite humbly, in 1960. A seemingly impossible task. This is just the start. Beware cancer, we’re coming for you!

Kris Griffin (middle) with the patient services team and CEO Cathy Gilman from Leukaemia & Lymphoma Research