Interview with patient advocate Jan Geissler

Cancer survivor Jan Geissler, who lives in Germany, is an active patient advocate globally. In this interview, he shares the reasons he became an advocate and steps he feels others can take to have a voice in their own health care. He also talks about actions being taken in Europe and the progress being made there in giving more patients input into the clinical trial process. Geissler, a chronic myelogenous leukemia (CML) patient, points out the success made in developing new treatments for CML patients and the benefits that knowledge is providing for many other types of cancers. He says great progress has been made, but there is still much more to be done. He advises patients to become informed, join patient support groups and to seek second medical opinions when they feel they are needed.

http://www.patientpower.info/video/a-european-patient-advocate-speaks-out-advice-on-how-to-be-a-powerful-patient?autoplay=1

Dr Jorge Cortes explains latest on existing and emerging treatments

Dr. Jorge Cortes, a renowned expert on CML and Chair of the CML Section at MD Anderson Center explains the latest news on existing and emerging treatments. This includes encouraging long-term follow-up for CML patients taking second generation tyrosine kinase inhibitors (TKIs). Dr. Cortes also comments on whether patients who are doing well on the original TKI, Gleevec, should switch. He also explains the latest information on a promising treatment in trials, Ponatinib, and the hope it gives patients who become resistant to TKIs or who have the T315i mutation not treated effectively by the approved TKIs. Dr. Cortes also comments on other drugs up for FDA review, Bosutinub and Omacetaxine, and explains which patients might benefit.

From www.patientpower.info

CML Survey – please take part

As many of you may already know, the link between non adherence (previously referred to as non-compliance – that is forgetting to take or deliberately not taking oral medications as prescribed) to tyrosine kinase inhibitors (TKIs) and poor outcome or lose of response, has been proven in two big research studies one by the Hammersmith hospital London and the other Adagio study – Universitair Ziekenhuis (UZ) Gent, Gent, Belgium.

The Leukaemia Patients Advocates Foundation, and CML advocates network has now launched phase 2 of its International CML Adherence Survey. The survey will be available in 12 languages, The goal of this survey, which builds on a pilot survey that had already revealed invaluable insights, is to better understand patients’ attitudes to taking their oral medication for the treatment of CML. It aims to gain greater understanding of patient attitudes and behaviours surrounding adherence, and ultimately help support physicians and patients to improve compliance and to develop adherence tools.

The survey, which is completely anonymous, is available via this link, www.survey.euro.confirmit.com/wix/p929997428.aspx.  They want as many CML patients as possible to fill it in so they can identify differences between countries in adherence levels and attitudes and improve compliance and to develop adherence tools that may be country specific.

Anonymity and confidentiality of all responses will be assured, so results cannot be attributed to any individual patient. Participants in the survey need to be CML patients that are currently under TKI treatment and be at least 18 years old.

Please help with this very valuable project, and fill it in as honestly as you can.  The results will be published in peer reviewed journals, and will help guide physicians in the future treatment of all CML patients.

Thanks,

Kris

Dr. Brian Druker: My Vision for CML and the Road to a Cure

What better video to post the day before International CML Awareness Day from the man that gave us all hope and continues to push the boundaries of treatment. Thank you Doctor Druker. Kris

Dr. Brian Druker, a CML expert from OHSU’s Knight Cancer Institute and a key researcher responsible for the development of imatinib, gives his perspective about the progress that’s been made in research and treatment.  He is encouraged that, in most cases, patients can have their disease well managed on existing therapies, while newer agents–some still in trials–will provide even more options for those who are resistant or are experiencing side effects.  Dr. Druker explains that he believes CML research can, and should, begin focusing on eradicating the disease altogther, and provides his suggestion about how he believes that might happen.

FDA approves Pfizer leukeamia drug – Bosutinib / Bosulif

Whilst I desperately want to celebrate a new CML drug coming into the marketplace I find it hard when I think about the dasatinib debacle we currently find ourselves in here in the UK. If Pfizer do not offer a PAH we will find ourselves lagging even further behind in CML treatment, this being said even a PAH will not guarantee availability.

This does prove to us that developments are still being made and that we must do all we can to ensure patients get access to the drugs they need, regardless of where they live.

I’ve started off with an excellent video interview with Dr. Carlo Gambacorti-Passerini, a prominent CML researcher from the University of Milan:

In this video interview from ASH 2011, Dr. Carlo Gambacorti-Passerini, a prominent CML researcher from the University of Milan Bicocca, gives us an update on bosutinib, a newer Tyrosine Kinase Inhibitor (TKI) that he has studied in-depth.  Once approved, bosutinib may provide one more option for patients and their physicians to better treat the disease while managing side effects.

WASHINGTON | Tue Sep 4, 2012 5:33pm EDT – LINK TO ORIGINAL ARTICLE

(Reuters) – Health regulators on Tuesday approved a Pfizer Inc pill for a rare type of leukemia, another step in the company’s effort to expand its oncology business.

The medicine, called Bosulif, treats chronic myelogenous leukemia (CML), a blood and bone marrow disease that usually affects older adults. About 26,000 Americans live with the cancer, and 5,430 people in the United States expected to be diagnosed with it annually, the U.S. Food and Drug Administration said.

Most people with CML have a specific type of genetic mutation called the Philadelphia chromosome. This mutation causes bone marrow to make an enzyme that triggers the abnormal growth of white blood cells. Bosulif, known generically as bosutinib, blocks the enzyme’s signal that causes the white blood cells to grow.

“We are seeing improvements in the treatment of CML based on a better understanding of the molecular basis of the disease,” Dr. Richard Pazdur, head of the FDA’s cancer drugs center, said in a statement.

Pfizer’s drug is meant for people who have CML with the Philadelophia mutation who cannot tolerate other medicines, such as Novartis AG’s Gleevec, or whose cancer has stopped responding to the older treatments.

The FDA gave Bosulif orphan status, which means it treats a condition or disease that affects fewer than 200,000 people in the United States. The designation comes with a seven-year period of marketing exclusivity.

The medicine is expected to reach peak global sales of $341 million in 2016, according to the average forecast of analysts polled by Thomson Reuters.

Bosulif is the second Pfizer cancer drug to get the FDA’s nod this year, after its kidney cancer drug Inlyta. Investors are looking for signs of the company’s research productivity, to help replace lost revenue from its cholesterol fighter Lipitor. The world’s top-selling drug began facing generic competition last year.

Shares of Pfizer were largely flat at $23.85 in after-market trading on Tuesday.

(Reporting by Anna Yukhananov, additional reporting by Bangalore equities newsroom; Editing by Leslie Gevirtz)

New CML drug shows promising results – ponatinib

I always read stories about new drugs with great joy. That there are scientists and companies dedicated to developing drugs that keep us alive is very inspiring. The naysayers will quote share values and profit margins but all that matters to me are those 2 little tablets (dasatinib) that I take each morning that keep me alive.
If this product comes to market let’s hope NICE don’t stop access to this drug too.
Kris

Ariad Pharmaceuticals is to expand into Europe following promising results for an investigational cancer drug.

The US biotech firm will site a European HQ in Switzerland to support the planned filing of ponatinib, a pan-BCR-ABL inhibitor which has performed well in chronic myloid leukemia (CML) patients in clinical trials.

Talking to Pharmafocus, Ariad’s president of R&D and chief scientific officer Tim Clackson said the Swiss office would be open “imminently”.

Ponatinib is the Cambridge, Massachussetts-based oncology specialist’s lead candidate and is expected to be filed with US and European regulators before September this year.

If approved it would be up against tyrosine kinase inhibitors (TKIs) such as Novartis’ Glivec (imatinib), Novartis’ Tasigna (nilotinib) and Bristol-Myers Squibb’s Sprycel (dasatinib).

But Ariad is confident ponatinib’s mode of action is different from these brands as it bypasses T315I, the so-called ‘gatekeeper’ mutation in CML and can treat patients with all mutations – or none at all, a group Clackson says are “the most difficult to treat”.

The product was developed from the ground up with exactly that function in mind, part of Ariad’s use of computational chemistry approach which Clackson says brings in “the building blocks to sculpt the drug exactly as we want”.

Many CML sufferers develop resistance to TKIs over time, but in the recent PACE trial patients at all stages of the disease had a positive clinical response to ponatinib.

It saw 54% of patients achieving a major cytogenetic response (MCyR), with 44% a complete cytogenetic response (CCyR). The median follow-up for chronic CML patients is 10.1 months.

Of those patients with the T315I mutation, 70% achieved a MCyR with 66% a CCyR.

When it came to the higher bar of measurement, 30% of chronic-phase patients achieved a major molecular response (MMR), something 50% of chronic patients with the T315I mutation also attained.

A phase III trial pitting ponatinib against Glivec is planned for the third quarter of the year.

CML is a cancer of the white blood cells which affects around 7,000 patients each year in Europe. “T315I was a death sentence,” says Clackson. “Now there’s another approach.”

Switzerland chosen as European base

Ariad is currently building a sales force and commercial infrastructure in the US and will be mirroring it in Europe, with “small” sales teams in the UK and other key European markets.

There are no plans for any R&D to be based on the continent. “We’ll continue to manufacture at various third parties in the US and Europe,” Clackson said.

The Swiss facility will be in the canton of Vaud, where Ariad “will be looking to establish scientific connections with universities, research institutes and hospitals”.

Tax incentives were one of the key reasons, along with pharmacological expertise and a skilled workforce, for choosing Switzerland over other European locations, Clackson says.

Ariad’s most high profile drug discovery before this was oral mTOR inhibitor ridaforolimus, for which two years ago Merck acquired the exclusive license for oncology indications.

Ponatinib has also attracted the attention of other pharma companies but Ariad has decided to develop and commercialise the drug itself.

“We had incredible interest in partnering,” says Clackson. “We had very, very substantial offers on the table. But it was an unequivocal decision to go it alone.”

Ariad’s other pipeline products include AP26113, an oral, dual inhibitor of anaplastic lymphoma kinase (ALK) and epidermal growth factor receptor (EGFR), which is to be developed in non-small cell lung cancers.

The company also has AP1903, which is being evaluated in patients with prostate cancer and is licensed to Bellicum Pharmaceuticals.

Adam Hill
http://www.inpharm.com/news/173231/ariad-touts-next-generation-cml-treatment
Published on 29/06/12

6 year follow up data on dasatinib

Another study showing positive results for patients on dasatinib after 6 years who were resistant or intolerant to Glivec (imatinib). Yet more evidence showing that we need dasatinib to offer patients a complete range of treatment.

The original press release can be found here.

PARIS, June 15, 2012 /PRNewswire/

Results Presented at 17^th Congress of the European Hematology Association

Bristol-Myers Squibb Company (NYSE:BMY) and Otsuka Pharmaceutical Europe Ltd., today announced six-year follow-up results from a Phase 3 randomised, open-label, dose-optimisation study of SPRYCEL^® (dasatinib) in Philadelphia chromosome-positive (Ph+) chronic-phase chronic myeloid leukaemia (CP-CML) adult patients resistant or intolerant to Glivec^® (imatinib).

Long-term survival data

The six-year data shows progression-free survival of 49.3% and an overall survival of 71% for patients randomised to dasatinib 100 mg once daily (n=167), with 6% of patients (n=10) progressing to accelerated or blast phase on study at six years of follow-up.^[1]

Safety and tolerability data from patients randomized to the 100 mg arm during the six-year follow up are consistent with the previously reported safety profile of dasatinib 100 mg once daily. In this 100 mg QD arm, the most common grade 3/4 adverse events (AEs) were (cumulative 6 year occurrence): neutropenia (36%), thrombocytopaenia (24%), and anaemia (13%).^[1] The cumulative incidence rates of the most common non-haematological AEs of Grade 3/4 at six years of follow-up were: diarrhoea (4.3%), fatigue (4.3%), infections (6.1%) and pleural effusion (5.3%).^[2]

This is the longest reported follow-up of 2^nd generation Tyrosine Kinase Inhibitors for patients resistant or intolerant to imatinib.

Safety and Tolerability at Six Years

Safety and tolerability data from the six-year study are consistent with the previously reported safety profile of dasatinib 100 mg once daily. For full information on SPRYCEL (dasatinib) please refer to SmPC at http://www.ema.europa.eu/.

These data were presented today at the 17^th Congress of the European Hematology Association in Amsterdam. (Poster 0199).

About Study CA180-034

Study CA180-034 was designed to assess the efficacy and safety of dasatinib following intolerance or resistance to imatinib. The trial enrolled 670 CP-CML patients with resistance (n=497) or intolerance (n=173) to imatinibwho were randomised to one of four treatment arms: 100 mg once daily (n=167), 50 mg twice daily (n=168), 140 mg once daily (n=167) and 70 mg twice daily (n=168). In this pre-treated population, the median time from onset of CML to randomisation in patients on the 100 mg once daily arm was 55 months and 46% of these patients had more than three years of prior imatinib treatment. Data on the primary endpoint of the study, major cytogenetic response with a minimum follow up of 6 months in imatinib-resistant patients, have been previously reported. Thirty-one percent of patients randomised to receive dasatinib 100 mg once daily remained on treatment at 6 years.^[1]

About SPRYCEL^®

Discovered and developed by Bristol-Myers Squibb, dasatinib was initially approved by the FDA and the European Commission in 2006 as a treatment for adults for all phases of Ph+ CML (chronic, accelerated, or myeloid or lymphoid blast phase) with resistance or intolerance to prior therapy including imatinib and Philadelphia chromosome positive (Ph+) acute lymphoblastic leukaemia (ALL) intolerant or resistant to prior therapy. In the U.S., dasatinib received accelerated FDA approval for this indication. Since then, dasatinib has been approved for this indication in more than 60 countries worldwide.

In 2010, dasatinib 100 mg once daily was approved by the FDA and European Commission for the treatment of adult patients with newly diagnosed Ph+ CML in chronic phase. In the U.S., dasatinib received accelerated FDA approval for this indication. The effectiveness of dasatinib is based on cytogenetic response and major molecular response rates. The first-line trial (known as DASISION) is ongoing and further data will be required to determine long-term outcome. Now, more than 50 countries have approved dasatinib for this indication.

About Chronic Myeloid Leukemia

CML is a slow-growing type of leukaemia in which the body produces an uncontrolled number of abnormal white blood cells.^[3] CML accounts for 15% of all leukaemias.^[4] The incidence is estimated at 1-2 cases per 100,000.^[5]

CML occurs when pieces of two different chromosomes (9 and 22) break off and attach to each other. The new chromosome is called the Philadelphia-positive chromosome, which contains an abnormal gene called BCR-ABL that signals cells to make too many white blood cells. There is no known cause for the genetic change that causes CML.

About Bristol-Myers Squibb

Bristol-Myers Squibb is a global biopharmaceutical company whose mission is to discover, develop and deliver innovative medicines that help patients prevail over serious diseases.

About Otsuka Pharmaceutical Co., Ltd.

Founded in 1964, Otsuka Pharmaceutical Co., Ltd. is a global healthcare company with the corporate philosophy: ‘Otsuka-people creating new products for better health worldwide.’ Otsuka researches, develops, manufactures and markets innovative and original products, with a focus on pharmaceutical products for the treatment of diseases and consumer products for the maintenance of everyday health.

Otsuka Pharmaceutical Co., Ltd. is a wholly owned subsidiary of Otsuka Holdings Co., Ltd., the holding company for the Otsuka Group. The Otsuka Group has business operations in 23 countries and regions around the world.

Visit Otsuka Pharmaceutical Co., Ltd. at http://www.otsuka.co.jp/en.

References: 1. Rea, D., et al. Six-year follow-up of patients with imatinib-resistant or imatinib-intolerant chronic-phase chronic myeloid leukemia (CP-CML) receiving dasatinib. To be presented at: 17th Congress of the European Hematology Association (EHA); June 14-17, 2012; Amsterdam, The Netherlands. 2. Shah, N., et al. Six-year follow-up of patients with imatinib-resistant or -intolerant chronic-phase chronic myeloid leukemia (CML-CP) receiving dasatinib. Oral Presentation at: 2012 American Society of Clinical Oncology Annual Meeting. 3.Macmillan Cancer Support. Leukaemia Overview.   Available at: http://www.macmillan.org.uk/Cancerinformation/Cancertypes/Leukaemia/Leukaemiaoverview.aspx. Last accessed April 2012. 4. National Comprehensive Cancer Network (NCCN). Chronic Myelogenous Leukemia – Clinical Practice Guidelines in Oncology – v.1.2007. 5. Baccarani, M. and Dreyling, M. Annals of Oncology. 2010;21:165-167.

UK job codes: 729UK12NP019 / OPUK/0612/SPC/2016, date of preparation June 2012

SOURCE  Bristol-Myers Squibb & Otsuka Pharmaceutical Europe Ltd.