CML Advocates launch Leukaemia App

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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

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A Day In Westminster

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I met with parliamentarians yesterday to ask for their support in helping chronic myeloid leukaemia patients access vital, life extending medicines and life insurance. Overall it was a very productive day.

The meetings, held with Mark Tami MP and Lord Avebury, provided an opportunity for me to highlight the fact that patients in England currently are missing out on innovative new drugs whilst patients in Wales have full access. I also explained the difficulties CML patients have getting life insurance cover.

New drugs such as ponatinib (Iclusig®) have the potential to offer patients another treatment option when others have failed and when the only option for many is a stem cell transplant. The system for appraising drugs in England, NICE, says that there are too few patients for them to even consider making ponatinib available on the NHS, they won’t even look at the figures. It doesn’t seem fair that across the border in Wales all patients can be prescribed the drug when here it’s not going to be looked at.

Both parliamentarians were also supportive of my bid to get insurance companies to offer CML patients life insurance, as many of us are being turned down. We’re not asking the insurers to pay out on CML related deaths, but many patients can’t get mortgages without life insurance, so it’s vital that we can be accepted with exclusions for our condition.

There’s plenty of follow up work to be done: letter writing and tabling of parliamentary questions before purdah, the pre-election period in the United Kingdom. I’ll also be meeting up with my local MP Mark Garnier, who has been incredibly supportive over the years, next week to ask for his support on both matters.

Once the dust settles on the general election in May, I’ll be going back down to Westminster to gather more support from MPs and peers. The landscape will be a little calmer by then (I hope) and we’ll put even more pressure on the insurance industry and NICE to give CML patients the access to drugs and services that we deserve. Yesterday was a great start.

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Trial Suggests Imatinib Discontinuation Safe, Some Responses Persist

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More positive news on World CML day – we need to keep working towards a cure. K

Trial Suggests Imatinib Discontinuation Safe, Some Responses Persist

Evidence continues to mount that discontinuing imatinib treatment for chronic myeloid leukemia (CML) in the chronic phase is safe. A new phase II Dutch and Belgian study showed only about two-thirds of patients relapsed after discontinuing treatment with imatinib and cytarabine, and all patients remained sensitive to imatinib after relapse.
Tyrosine kinase inhibitors including imatinib have revolutionized CML treatment in recent years, but the need to continue treatment indefinitely is limiting. Several recent studies have begun to suggest that alternate treatment schedules or discontinuation of therapy are feasible among patients with good molecular responses. In a study of the Dutch-Belgian Cooperative Trial for Haemato-Oncology, researchers led by Noortje Thielen, of the VU Medical Center in Amsterdam, 33 patients with a molecular response lasting at least 2 years on imatinib and cytarabine combination therapy were randomized to either continue (18 patients) or cease (15 patients) imatinib treatment. Results were published online ahead of print in July in the European Journal of Cancer.

After a median follow-up period of 36 months, three patients randomized to continue treatment (17%) and 10 patients in the discontinuation arm (67%) had a molecular relapse; all three of the former patients had stopped imatinib treatment after randomization.

The molecular relapse rate at 12 months was 0% in the continued therapy group and 53% in the discontinuation group; at 24 months, those rates were 6% and 67%. In an as-treated analysis (accounting for the patients who ceased treatment in spite of randomization to continue imatinib), the two-year rate was 61% for discontinued therapy and 0% for continued imatinib.
The five patients in the discontinuation group who did not relapse showed a stable molecular response. The 13 patients who relapsed all regained molecular response after a median of 6 months from the restart of imatinib or nilotinib treatment.

To our knowledge, this is the first randomized trial regarding the discontinuation of imatinib in first chronic phase CML patients who have achieved a durable and stable molecular response,” the authors wrote. “Our results are encouraging.” They noted that the addition of cytarabine to the initial regimen may have contributed to the persistence of response after therapy discontinuation, but that remains unclear.

Further studies on this issue will need to define predictive factors for successful discontinuation of imatinib, as well as of other TKIs including nilotinib and dasatinib, the researchers wrote. “Although imatinib treatment was previously expected to be life-long, our data suggest that, under close PCR monitoring, discontinuation of imatinib is safe in CML patients with a long-lasting molecular response,” they concluded.

http://www.cancernetwork.com/chronic-myeloid-leukemia/content/article/10165/2157668

NEW CML Resource & Knowledge Centre

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Trust those great people at CML Advocates Network to drop a brilliant new resource in our lap. This is well worth checking out. K

Over the next three years, CML patients in more and more countries will be facing the use of generic TKIs and copy drugs to treat CML.

To provide patient organizations with background information on this important issue which has so far been quite difficult to find, we have launched a Resource & Knowledge Center on CML generics, copy drugs and substandard drugs.

Please see new section here: http://www.cmladvocates.net/generics

It provides:

  • an unofficial directory listing all CML tyrosine kinase inhibitors (TKIs) that are – to our knowledge – available to date:  http://www.cmladvocates.net/generics/cml-drugs-register
  • the results of our survey on generics, copy drugs and substandard drugs in CML which was conducted by CML Advocates Network in collaboration with iCMLf in March 2013. The survey summarizes 86 responses from 55 countries – the data is also available for download.
  • the webstreams of our session on CML generics and substandard drugs at “CML Horizons 2013”, which featured a presentation of the WHO, of the CML Association of Serbia and the CML Advocates Network
  • an index of scientific articles on the use of generic drugs in CML treatment: http://www.cmladvocates.net/generics/generics-publications
  • our blog on CML generics. (This is only available after login for members of the CML Advocates Network to keep discussions private in the community) http://www.cmladvocates.net/generics/generics-blog
  • a glossary with key terms and definitions in the area of drugs and generics: http://www.cmladvocates.net/generics/glossary

We will soon complement this by a “Best Practice Toolbox” for advocacy on generics in a few weeks, which is currently under construction. The “toolbox” will provide useful advice and tools on how to address this topic as an advocacy organization when generics or copy drugs are being introduced in your country.

Finally, we would like to stress that the Resource & Knowledge Center on CML generics, copy drugs and substandard drugs is a purely patient-driven, non-commercial initiative. It has no interest to promote, or assess, any of the drugs. The only intent is to increase transparency in a confusing environment.

We hope that you will find this Resource & Knowledge Center very useful!
We are looking forward to your active participation in the blog and the new knowledge center, and are happy to receive your feedback!

Nicole, Giora and Jan
CML Advocates Network –
http://www.cmladvocates.net

Tom Watson MP joins our cause!

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I’m still trying to get my head around today! It may just be the most amazing thing to happen to the AccessCMLDrugs campaign to date. Tom Watson who is the Member of Parliament for West Bromwich East, Deputy Chair of the Labour Party and the Labour Party’s Campaign Co-ordinator has agreed to support our cause!

We met this afternoon in West Bromwich and spent some time going over the issues affecting CML patients, namely the unavailability of dasatinib. We discussed, in detail, the STOP trial data, the effectiveness of dasatinib, what the TKI (tyrosine kinase inhibitor) drugs mean to CML patients, survival rates and the cancer drug fund. I told him about our campaign so far and how hard it is get our voice heard, especially by NICE, the pharmaceuticals and his own party. He agreed that it is important to ensure that all CML drugs are available to patients, free at the point of delivery. He has told me that he will do all he can to support us, he looked me in the eye and he told me that he believed in what we are trying to do.

To say I was blown away is an understatement. Tom is one of the most powerful men in politics today and I’d gone prepared; I’d even taken my box of dasatinib to show him what this is all about. He was sharp, working out quickly how much the NHS could save if the STOP trials findings are conclusive and adopted in England. These being the ongoing trials where 40% of patients who achieve CMR (Complete Molecular Remission) over a period of time come off their drugs and don’t need them again. The other 60% take the drugs again and the leukaemia goes back into remission. Hopefully you never tire of me writing about this as a cure for cancer.

So that’s it in a nutshell. Tom wants me to put a report together and propose how I want him to help out. He was sincere, friendly and genuinely interested in leukaemia and blood cancer patients. I sat in my car for 20 minutes afterwards just taking everything in, it was emotional. The implication on what we are trying to achieve is huge and having someone like Tom behind us is simply fantastic (sadly that doesn’t feel like a big enough word).

I need to start gathering research and writing up that report but in the meantime I’m sure you will join me in thanking Tom for taking an interest in a handful of poorly people and their little fight to stop a big disease. Thank you.

Kris

Tom Watson MP and Kris Griffin

Tom Watson, Member of Parliament (MP) for West Bromwich East, Deputy Chair of the Labour Party and the Labour Party’s Campaign Co-ordinator, with CML campaigner and patient Kris Griffin

Front page of the Independent: Cost of CML Drugs

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Well, look here. Front page of The Independent yesterday with “the news” that CML drug prices are way too high. It’s something we’ve known for ages and it’s fantastic that the damaging price of these drugs is being brought to public attention.
I think it is important that we consider the other side of the argument for a moment though, and this is not an attempt to justify the prices and defend the pharmas. These drugs are saving lives and, in some cases, curing a form of cancer. What price on that? These drugs have revolutionised CML treatment and have certainly kept me alive. Do I care about the price? No. I care that they’ve saved my life and it helps that I live in the UK and don’t have to find that money. Do I care that there are people on this planet dying from CML. YES – and this is the issue.   Morally these pharmaceutical companies have an obligation to service each territory to ensure these drugs are available at an affordable price. There are plenty of opportunities to cover research costs but there is bigger picture stuff here…even bigger than the shareholders of these companies. Let’s not use these prices as a stick to beat the industry with, after-all they’ve come up with something truly amazing that is going to change the face of healthcare forever. Instead let’s appeal to their common decency, their moral compass and put the pressure on them to deliver a fair solution. This applies to you too BMS. Your failure to provide a Patient Access Scheme in the England has meant the NHS has not been able to deliver dasatinib as a standard treatment.
What a fantastic opportunity for people to do the right thing. Will it happen? Probably not but I must have faith in the human condition. We can put pressure on the pharmaceuticals,  we can write, ask the awkward questions and make sure they know every time a CML patient dies because they can’t afford the treatment they need. Let’s be brave about this, we thank the industry with all our heart, but the time has come to be bigger than the bottom line.  Kris

The wounded surgeon plies the steel
That questions the distempered part;
Beneath the bleeding hands we feel
The sharp compassion of the healer’s art
Resolving the enigma of the fever chart.
T.S. Eliot (1888-1965) – Four Quartets ‘East Coker’ (1940) pt. 4

The real cancer killer: rip-off prices for drugs set by ‘profiteering’ Big Pharma giants

An influential group of cancer experts has warned that the high prices charged by pharmaceutical companies for cancer drugs are effectively condemning patients to death.

The group of more than 100 leading cancer physicians from around the world, including nine from the UK, accuse the drug industry of “profiteering” – making a profit by unethical methods such as by raising the cost of grain after a natural disaster.

Of the 12 drugs approved by the Food and Drug Administration in the US in 2012, 11 were priced above $100,000 (£65,000) per patient per year. In addition the price of existing drugs of proven effectiveness has been increased by up to threefold.

The specialists say: “What determines a morally justifiable ‘just price’ for a cancer drug? A reasonable drug price  should maintain healthy pharmaceutical industry profits without being viewed as ‘profiteering’. This term [profiteering] may apply to the trend of high drug prices where a life threatening medical condition is the disaster.”

The high prices mean the drugs may not be approved by the National Institute for Clinical Excellence in the UK forcing doctors to fill in a 14 page application apply to the Cancer Drugs fund for British patients who could benefit from them.

In addition, the rising cost of existing drugs in a cash limited health service such as the NHS means treatment is denied to other patients with other conditions.

The authors of the article, published in the journal Blood, are all specialists in blood cancers such as leukaemia, where cancer drugs have proved most effective.

One of the best known – imatinib, whose brand name is Glivec – has proved so successful in chronic myeloid leukaemia that patients who a decade ago survived for a few years can now look forward to a near-normal life expectancy.

But the cost of Glivec has risen from £18,000 per patient per year to around £21,000 in the UK, and from $30,000 to $92,000 in the US. This is despite the fact that all research costs were covered by the original price, and the number of patients treated and the length of time they are on the drug have both vastly increased because of the drug’s success.

Daniel Vasella, former chairman and chief executive of Novartis, the manufacturer, said the original price charged for Glivec in 2001 was considered “high but worthwhile” and was estimated to yield annual revenues of $900 million, enough to cover its development cost in two years. A decade later Its annual revenues in 2012 were $4.7 billion (£3 billion).

The cancer specialists say the revenue earned by Glivec over the last ten years “represent generous profits to the company”. But this has put heavy pressure on those who have to foot the bill. “Grateful patients may have become the financial victims of the treatment success, having to pay the high price annually to stay alive”.

In the US even those with health insurance may pay an average of 20 per cent of drug prices out of pocket. Drug prices are the single most frequent cause of personal bankruptcies in the US.

Three new drugs have been approved for chronic myeloid leukaemia in the last year by the FDA but the prices are “astronomical” the authors say at up to $138,000 a year per patient.

Worldwide only about a quarter of the patients with chronic myeloid leukaemia who could benefit have access to drugs because of the cost. “A small fraction are rich enough to pay individually, and most are treated intermittently or not at all. The effects of these financial pressures on long term survival… are yet unknown.”

In the UK, patients are shielded from the “direct economic anxieties of illness”, the article says.  But Professor Jane Apperley, chair of the Department of Haematology at Imperial College, London, and one of the authors, said high drug prices were still a cause of harm in Britain .

“The price of a drug heavily influences the decision of NICE whether we can prescribe it on the NHS. I am chief of service at Imperial College and we are constantly being asked to reduce our spending. We have to look very carefully at the cost of the drugs we use.”

“Of course we need the pharmaceutical industry to go on developing new drugs. It is very exciting that a number of cancers are now becoming susceptible to these new drugs. But the rising cost is unsustainable. “

“The drugs are very effective at keeping people alive. But if they are priced out of what you can afford you know that you can keep people alive but you can’t afford to do so. It is completely unsustainable for the NHS because the costs are going up every year. We need a serious dialogue about whether we can sustain these costs.”

The authors of the article in Blood conclude: “We believe the unsustainable drug prices may be causing harm to patients. Advocating for lower drug prices is a necessity to save the lives of patients who cannot afford them. We believe drug prices should reflect objective measures of benefit, but should not exceed values that harm our patients and societies.”

The group say they intend to organise regular meetings and campaign for lower cancer drug prices.

A spokesperson for the UK charity Beating Blood Cancers said: “As a charity we want to see an ethical approach to drug pricing . There is no point in us investing in research if the pricing policy means drugs won’t be available to patients.”

In a statement to The Independent, Novartis said: “We recognize that sustainability of health care systems is a complex topic and we welcome the opportunity to be part of the dialogue.  Our critical role, as one of many parties working towards improving cancer care, is to discover and develop innovative treatments.”

“ Novartis innovation in chronic myeloid leukemia (CML) has changed the course of the disease. Before Glivec(imatinib)* and Tasigna (nilotinib), the five-year survival in CML was only 30 percent. Today, nine out of ten patients with CML have a normal lifespan and are leading productive lives.”

“Over the years, our programs have evolved to improve patient access to our medicines. We work together with government health care systems, charities and other payers to build successful cost-sharing models.”

Expert view: ‘Price of drugs is harming patients’

The following is an extract from an article, contributed to by more than 100 leading cancer physicians from around the world, including nine from the UK, published in the journal, Blood.

This perspective reflects the views of a large group of CML experts, who believe the current [high] prices of drugs may compromise access of needy patients to highly effective therapy, and are harmful to the sustainability of our national healthcare systems…

If drug price reflects value, then it should be proportional to the benefit to patients in objective measures, such as survival prolongation, degree of tumour shrinkage, or improved quality of life. For many tumours, drug prices do not reflect these endpoints, since most anti-cancer drugs provide minor survival benefits, if at all.

As physicians, we… believe the unsustainable drug prices in CML and cancer may be causing harm to patients. Advocating for lower drug prices is a necessity to save the lives of patients who cannot afford them … For CML, and for other cancers, we believe drug prices should reflect objective measures of benefit, but should also not exceed values that harm our patients and societies.”

An ethical price tag? Cancer drugs

Brands used for the treatment of chronic myeloid leukaemia

Imatinib (Glivec) £21,000 per patient per year – Novartis
Designed from first principles, it proved hugely effective and unexpectedly turned into a blockbuster, earning billions of pounds for its makers.

Nilotinib (Tasigna) £21,000 – Novartis
Designed for patients who fail to respond to Glivec, Novartis reduced the cost to get it past Nice, whilst increasing the cost of Glivec.

Dasatinib (Sprycel) £31,000 – Bristol Myers Squibb
Also designed for patients who cannot take Glivec. But it has not been approved by Nice for use on the NHS because of its high cost.

Bosutinib (Bosulif) £76,000 – Pfizer
For patients who suffer side-effects from the other drugs. It won approval in the US in 2012 but is awaiting a licence in the UK.

Omacetaxine (Synribo) £100,000 – Teva
For patients who cannot tolerate other drugs. Approved in US in 2012 but awaiting licence in the UK.

Ponatinib (Iclusig) £90,000 – Ariad
A third-generation drug which works in a different way. Approved in the US in 2012 but awaiting a licence in the UK.

JEREMY LAURANCE    MONDAY 29 APRIL 2013

http://www.independent.co.uk/news/uk/home-news/the-real-cancer-killer-ripoff-prices-for-drugs-set-by-profiteering-big-pharma-giants-8591825.html

TKI survey on behalf of CML Advocates Network

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A request from the CML Advocates Network who I fully endorse and would ask you complete the survey. Many thanks, Kris

—-

Imatinib, dasatinib and nilotinib are a tyrosine kinase inhibitors (TKIs) that have been established as effective therapies in chronic myeloid leukemia. While imatinib (marketed as Gleevec/Glivec), and in some markets nilotinib (marketed as Tasigna) as well as Dasatinib (marketed as Sprycel), have been available for some years, non-original copy versions of these drugs have also been provided to CML patients in a number of countries. With the patent ending on Glivec/Gleevec from 2013 in some markets, the use of generic TKIs in CML is becoming more and more widespread.

With the CML Horizons 2013 patient conference putting a spotlight on the situation of generics and copies in CML therapy, this survey intends to understand the availability of original TKIs and other non-original TKIs for the treatment in CML in countries all across the globe. It also aims to understand how CML patient groups are addressing the issue of quality assurance when generics and copies become available.

This survey is run by the CML Advocates Network without any commercial interest and without support from any pharmaceutical company. The results of this survey will be presented to the community at the CML Horizons 2013 meeting in May.

It should take 10-15 minutes of your time to complete the 10 questions. We would like to cover as many countries as possible – your contribution is greatly appreciated and will help us a lot!

Please respond to the survey here: http://www.cmladvocates.net/tkisurvey
(and please also respond if NO generics/copies are available in your country, which is equally important to know)

Can you please respond latest by 12 April 2013?

Many thanks and best wishes,

Jan (on behalf of the CML Advocates Network)

NICE reviewing Dasatinib!

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Okay. Let’s not get our hopes up TOO much but I’ve come across an agenda for the NICE Technology Appraisal Committee meeting on Tuesday 15 January 2013. Listed as point 6 on the agenda is:

6. Rapid review of TA251 – dasatinib, nilotinib and standard-dose imatinib for the first-line treatment of chronic myeloid leukaemia (part review of technology appraisal guidance 70)

This would indicate to me that something has changed, perhaps BMS have offered a Patient Access Scheme (discounted price) for dasatinib and that NICE are duty bound to review the guidance. This is NOT a formality but there is a chance that the guidance could change. It would be wonderful to think we all had a small part to play in this, but the big thanks has to go to Leukaemia Care and the group of charities who have supported and worked with them – their unwavering pressure has been vital…so far.

If I hear anything else then I will post here, but let’s keep the pressure up and keep our fingers crossed.

Original link here: http://www.nice.org.uk/newsroom/publicmeetings/TAC15January2013.jsp

Kris

Report: deaths from Chronic Myeloid Leukaemia (CML) have halved

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The National Cancer Intelligence Network (NCIN) have this week released a report which has shown that deaths from Chronic Myeloid Leukaemia have halved since TKIs were introduced in 2001.
The report states that “What’s even more promising is that, in the last four years, second and third generations of these drugs have been developed. We believe more and more CML patients have been receiving TKI’s and we’d predict that the improvements in survival should be even greater in the future” – However, we know of the challenges patients will face on long term access to dasatinib, especially with no firm long term commitment to the future of the CDF. If we take into account the success of these new drugs over the last 4 years I predict that survival figure will sky-rocket. More reason to ensure access to all of the drugs for everyone.
Kris

IMPROVED TREATMENTS FOR CHRONIC MYELOID LEUKAEMIA HAVE DRAMATICALLY INCREASED SURVIVAL

Survival for people diagnosed with Chronic Myeloid Leukaemia (CML) has risen by nearly half, with around 58 per cent of people surviving their disease for at least five years compared with only around 40 per cent in the late 1990s, according to a new report from the National Cancer Intelligence Network (NCIN), published today (Wednesday)*.

The improvements are largely down to a family of drugs called Tyrosine Kinase Inhibitors (TKIs) which have now become the standard treatment for the disease. The first of these was imatinib (Glivec), which was licensed in 2001.

The Northern and Yorkshire Cancer Registry and Information Service (NYCRIS), on behalf of the NCIN Haematology Site Specific Clinical Reference Group (SSCRG), looked at the rates of people in England getting, dying from and surviving a range of different blood cancers between 1995 and 2008. And it is the first national study in England to look at survival for different types of leukaemia.

For patients diagnosed with CML, researchers found that the chance of surviving the disease for at least five years after diagnosis rose from 41 per cent to 57 per cent in men and from 38 per cent to 59 per cent in women between the late 1990s and the early 2000s**.

CML is a relatively rare form of leukaemia*** that mostly affects older people, with around 700 cases diagnosed in the UK every year****.

Dr Robin Ireland, chair of the SSCRG at the NCIN, said: “It’s really exciting to see the enormous difference new drugs can make in treating cancer. And, as this new data shows, TKI’s can be considered a revolutionary treatment for Chronic Myeloid Leukaemia.”

“Basic research has given us a greater biological understanding of cancer tumours, which has led to the development of successful targeted cancer drugs that are now the first line treatment for CML. TKIs target cancer cells by blocking the molecules they make, which stops them from multiplying. These drugs have completely changed the outlook for patients with this disease and it’s the first example of our improved understanding of cell molecular biology leading to the design of a specific inhibitor of the disease.

”Dr Steven Oliver, Haematological Cancer Epidemiology Lead at NYCRIS and lead author of the report, said: “This report shows that, although the number of people developing Chronic Myeloid Leukaemia hasn’t changed much since 2001, survival from the disease has greatly improved.”

“What’s even more promising is that, in the last four years, second and third generations of these drugs have been developed. We believe more and more CML patients have been receiving TKI’s and we’d predict that the improvements in survival should be even greater in the future.”

“Chris Carrigan, head of the National Cancer Intelligence Network (NCIN), said: “Being able to link data on the diagnosis, treatment and outcomes for cancer patients allows us to identify where improved cancer care is having an effect on peoples lives. The improvements in survival demonstrated here highlight the difference that effective treatments can make.”

Notes

*Haematological Malignancies in England: cancers diagnosed 2001 – 2008.Note that analyses of deaths from blood cancers for 2001-2008 were also reported, along with 5-year survival figures for patients diagnosed with different blood cancers in 1995-1999 compared with those diagnosed in 2000-2003.

**Absolute change in CML 5-year survival rates, in England, 1995-1999 compared with 2000-2003. Male rates 40.7% and 56.9%; female rates 38.4% and 58.7%, in 1995-1999 and 2000-2003 respectively.

***Leukaemias are a group of malignant diseases that affect the production of white blood cells – the body’s guards against foreign cells and infections. The different types of leukaemia affect different organs and vary in how quickly they spread. Different types of leukaemia affect different age groups and there are some wide differences in survival found across the age groups in certain forms of the disease.

**** There were 710 cases of CML registered in 2010 in the UK (Cancer Research UK Statistical Information Team)The NICE guidance recommends standard-dose imatinib as the first-line treatment for CML.

Source: http://www.ncin.org.uk/news_and_events/improved_treatments_for_chronic_myeloid_leukaemia_have_dramatically_increased_survival.aspx

FDA approves Synribo for chronic myelogenous leukaemia

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More fantastic news regarding another drug to treat CML. More hope for those who fail on other TKIs. We must ensure our UK health system funds drugs like this as they become available. Kris

The U.S. Food and Drug Administration today approved Synribo (omacetaxine mepesuccinate) to treat adults with chronic myelogenous leukemia (CML), a blood and bone marrow disease.

An estimated 5,430 people will be diagnosed with CML in 2012, according to the National Institutes of Health. Synribo is intended to be used in patients whose cancer progressed after treatment with at least two drugs from a class called tyrosine kinase inhibitors (TKIs), also used to treat CML.

Synribo blocks certain proteins that promote the development of cancerous cells. It is injected just under the skin (subcutaneously) twice daily for 14 consecutive days over a 28-day cycle until white blood cell counts normalize (hematologic response). Synribo is then administered twice daily for seven consecutive days over a 28-day cycle as long as patients continue to clinically benefit from therapy.

“Today’s approval provides a new treatment option for patients who are resistant to or cannot tolerate other FDA-approved drugs for chronic or accelerated phases of CML,” said Richard Pazdur, M.D., director of the Office of Hematology and Oncology Products in FDA’s Center for Drug Evaluation and Research. “Synribo is the second drug approved to treat CML in the past two months.”

On Sept. 4, 2012, the FDA approved Bosulif (bosutinib) to treat patients with chronic, accelerated or blast phase Philadelphia chromosome positive CML who are resistant to or who cannot tolerate other therapies.

Synribo is approved under the FDA’s accelerated approval program, which allows the agency to approve a drug to treat a serious disease based on clinical data showing that the drug has an effect on a surrogate endpoint that is reasonably likely to predict a clinical benefit to patients. This program provides earlier patient access to promising new drugs while the company conducts additional clinical studies to confirm the drug’s clinical benefit and safe use. Synribo also received orphan-product designation by the FDA because it is intended to treat a rare disease or condition.

The effectiveness of Synribo was evaluated using a combined cohort of patients whose cancer progressed after previous treatment with two or more TKIs. All participants were treated with Synribo.

The drug’s effectiveness in chronic phase CML was demonstrated by a reduction in the percentage of cells expressing the Philadelphia chromosome genetic mutation found in most CML patients. Fourteen out of 76 patients (18.4 percent) achieved a reduction in an average time of 3.5 months. The median length of the reduction was 12.5 months.

In accelerated phase CML, Synribo’s effectiveness was determined by the number of patients who experienced a normalization of white blood cell counts or had no evidence of leukemia (major hematologic response, or MaHR). Results showed five out of 35 patients (14.3 percent) achieved MaHR in an average time of 2.3 months. The median duration of MaHR in these patients was 4.7 months.

The most common side effects reported during clinical studies include a low level of platelets in the blood (thrombocytopenia), low red blood cell count (anemia), a decrease in infection-fighting white blood cells (neutropenia) which may lead to infection and fever (febrile neutropenia), diarrhea, nausea, weakness and fatigue, injection site reaction, and a decrease in the number of lymphocytes in the blood (lymphopenia).

Synribo is marketed by Frazer, Pa.-based Teva Pharmaceuticals. Bosulif is marketed by New York City-based Pfizer.

For more information:

The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.

Source: http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm325895.htm

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